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The small intestine is part of the body’s digestive system, which also includes the esophagus, stomach, and large intestine. The digestive system removes and processes nutrients (vitamins, minerals, carbohydrates, fats, proteins, and water) from foods and helps pass waste material out of the body. The small intestine is a long tube that connects the stomach to the large intestine. It folds many times to fit inside the abdomen. EnlargeThe small intestine connects the stomach and the colon. It includes the duodenum, jejunum, and ileum. The types of cancer found in the small intestine are adenocarcinoma, sarcoma, carcinoid tumors, gastrointestinal stromal tumor, and lymphoma. This summary discusses adenocarcinoma and leiomyosarcoma (a type of sarcoma). Adenocarcinoma starts in glandular cells in the lining of the small intestine and is the most common type of small intestine cancer. Most of these tumors occur in the part of the small intestine near the stomach. They may grow and block the intestine. Leiomyosarcoma starts in the smooth muscle cells of the small intestine. Most of these tumors occur in the part of the small intestine near the large intestine. For more information on small intestine cancer, see the following: Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn't mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for small intestine cancer include the following: These and other signs and symptoms may be caused by small intestine cancer or by other conditions. Check with your doctor if you have any of the following: Procedures that make pictures of the small intestine and the area around it help diagnose small intestine cancer and show how far the cancer has spread. The process used to find out if cancer cells have spread within and around the small intestine is called staging. In order to plan treatment, it is important to know the type of small intestine cancer and whether the tumor can be removed by surgery. Tests and procedures to detect, diagnose, and stage small intestine cancer are usually done at the same time. The following tests and procedures may be used: The prognosis and treatment options depend on the following: Staging is used to find out how far the cancer has spread, but treatment decisions are not based on stage. See the General Information section for a description of tests and procedures used to detect, diagnose, and stage small intestine cancer. Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if small intestine cancer spreads to the liver, the cancer cells in the liver are actually small intestine cancer cells. The disease is metastatic small intestine cancer, not liver cancer. Treatment depends on whether the tumor can be removed by surgery and if the cancer is being treated as a primary tumor or is metastatic cancer. The cancer may come back in the small intestine or in other parts of the body. Different types of treatments are available for patients with small intestine cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment of small intestine cancer. One of the following types of surgery may be done: After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type of the cancer being treated. External radiation therapy is used to treat small intestine cancer. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on the type and stage of the cancer being treated. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Radiosensitizers are drugs that make tumor cells more sensitive to radiation therapy. Combining radiation therapy with radiosensitizers may kill more tumor cells. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. When possible, treatment of small intestine adenocarcinoma will be surgery to remove the tumor and some of the normal tissue around it. Treatment of small intestine adenocarcinoma that cannot be removed by surgery may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. When possible, treatment of small intestine leiomyosarcoma will be surgery to remove the tumor and some of the normal tissue around it. Treatment of small intestine leiomyosarcoma that cannot be removed by surgery may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent small intestine cancer that has spread to other parts of the body is usually a clinical trial of new anticancer drugs or immunotherapy. Treatment of locally recurrent small intestine cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about small intestine cancer, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of small intestine cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Small Intestine Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/small-intestine/patient/small-intestine-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389461] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Small Intestine Cancer Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is (are) Small Intestine Cancer ?

Key Points - Small intestine cancer is a rare disease in which malignant (cancer) cells form in the tissues of the small intestine. - There are five types of small intestine cancer. - Diet and health history can affect the risk of developing small intestine cancer. - Signs and symptoms of small intestine cancer include unexplained weight loss and abdominal pain. - Tests that examine the small intestine are used to detect (find), diagnose, and stage small intestine cancer. - Certain factors affect prognosis (chance of recovery) and treatment options. Small intestine cancer is a rare disease in which malignant (cancer) cells form in the tissues of the small intestine. The small intestine is part of the bodys digestive system, which also includes the esophagus, stomach, and large intestine. The digestive system removes and processes nutrients (vitamins, minerals, carbohydrates, fats, proteins, and water) from foods and helps pass waste material out of the body. The small intestine is a long tube that connects the stomach to the large intestine. It folds many times to fit inside the abdomen. There are five types of small intestine cancer. The types of cancer found in the small intestine are adenocarcinoma, sarcoma, carcinoid tumors, gastrointestinal stromal tumor, and lymphoma. This summary discusses adenocarcinoma and leiomyosarcoma (a type of sarcoma). Adenocarcinoma starts in glandular cells in the lining of the small intestine and is the most common type of small intestine cancer. Most of these tumors occur in the part of the small intestine near the stomach. They may grow and block the intestine. Leiomyosarcoma starts in the smooth muscle cells of the small intestine. Most of these tumors occur in the part of the small intestine near the large intestine. See the following PDQ summaries for more information on small intestine cancer: - Adult Soft Tissue Sarcoma Treatment - Childhood Soft Tissue Sarcoma Treatment - Adult Non-Hodgkin Lymphoma Treatment - Childhood Non-Hodgkin Lymphoma Treatment - Gastrointestinal Carcinoid Tumors Treatment - Gastrointestinal Stromal Tumors Treatment

The small intestine is part of the body’s digestive system, which also includes the esophagus, stomach, and large intestine. The digestive system removes and processes nutrients (vitamins, minerals, carbohydrates, fats, proteins, and water) from foods and helps pass waste material out of the body. The small intestine is a long tube that connects the stomach to the large intestine. It folds many times to fit inside the abdomen. EnlargeThe small intestine connects the stomach and the colon. It includes the duodenum, jejunum, and ileum. The types of cancer found in the small intestine are adenocarcinoma, sarcoma, carcinoid tumors, gastrointestinal stromal tumor, and lymphoma. This summary discusses adenocarcinoma and leiomyosarcoma (a type of sarcoma). Adenocarcinoma starts in glandular cells in the lining of the small intestine and is the most common type of small intestine cancer. Most of these tumors occur in the part of the small intestine near the stomach. They may grow and block the intestine. Leiomyosarcoma starts in the smooth muscle cells of the small intestine. Most of these tumors occur in the part of the small intestine near the large intestine. For more information on small intestine cancer, see the following: Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn't mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for small intestine cancer include the following: These and other signs and symptoms may be caused by small intestine cancer or by other conditions. Check with your doctor if you have any of the following: Procedures that make pictures of the small intestine and the area around it help diagnose small intestine cancer and show how far the cancer has spread. The process used to find out if cancer cells have spread within and around the small intestine is called staging. In order to plan treatment, it is important to know the type of small intestine cancer and whether the tumor can be removed by surgery. Tests and procedures to detect, diagnose, and stage small intestine cancer are usually done at the same time. The following tests and procedures may be used: The prognosis and treatment options depend on the following: Staging is used to find out how far the cancer has spread, but treatment decisions are not based on stage. See the General Information section for a description of tests and procedures used to detect, diagnose, and stage small intestine cancer. Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if small intestine cancer spreads to the liver, the cancer cells in the liver are actually small intestine cancer cells. The disease is metastatic small intestine cancer, not liver cancer. Treatment depends on whether the tumor can be removed by surgery and if the cancer is being treated as a primary tumor or is metastatic cancer. The cancer may come back in the small intestine or in other parts of the body. Different types of treatments are available for patients with small intestine cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment of small intestine cancer. One of the following types of surgery may be done: After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type of the cancer being treated. External radiation therapy is used to treat small intestine cancer. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on the type and stage of the cancer being treated. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Radiosensitizers are drugs that make tumor cells more sensitive to radiation therapy. Combining radiation therapy with radiosensitizers may kill more tumor cells. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. When possible, treatment of small intestine adenocarcinoma will be surgery to remove the tumor and some of the normal tissue around it. Treatment of small intestine adenocarcinoma that cannot be removed by surgery may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. When possible, treatment of small intestine leiomyosarcoma will be surgery to remove the tumor and some of the normal tissue around it. Treatment of small intestine leiomyosarcoma that cannot be removed by surgery may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent small intestine cancer that has spread to other parts of the body is usually a clinical trial of new anticancer drugs or immunotherapy. Treatment of locally recurrent small intestine cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about small intestine cancer, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of small intestine cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Small Intestine Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/small-intestine/patient/small-intestine-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389461] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Small Intestine Cancer Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

Who is at risk for Small Intestine Cancer? ?

Diet and health history can affect the risk of developing small intestine cancer. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn&apos;t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for small intestine cancer include the following: - Eating a high-fat diet. - Having Crohn disease. - Having celiac disease. - Having familial adenomatous polyposis (FAP).

The small intestine is part of the body’s digestive system, which also includes the esophagus, stomach, and large intestine. The digestive system removes and processes nutrients (vitamins, minerals, carbohydrates, fats, proteins, and water) from foods and helps pass waste material out of the body. The small intestine is a long tube that connects the stomach to the large intestine. It folds many times to fit inside the abdomen. EnlargeThe small intestine connects the stomach and the colon. It includes the duodenum, jejunum, and ileum. The types of cancer found in the small intestine are adenocarcinoma, sarcoma, carcinoid tumors, gastrointestinal stromal tumor, and lymphoma. This summary discusses adenocarcinoma and leiomyosarcoma (a type of sarcoma). Adenocarcinoma starts in glandular cells in the lining of the small intestine and is the most common type of small intestine cancer. Most of these tumors occur in the part of the small intestine near the stomach. They may grow and block the intestine. Leiomyosarcoma starts in the smooth muscle cells of the small intestine. Most of these tumors occur in the part of the small intestine near the large intestine. For more information on small intestine cancer, see the following: Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn't mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for small intestine cancer include the following: These and other signs and symptoms may be caused by small intestine cancer or by other conditions. Check with your doctor if you have any of the following: Procedures that make pictures of the small intestine and the area around it help diagnose small intestine cancer and show how far the cancer has spread. The process used to find out if cancer cells have spread within and around the small intestine is called staging. In order to plan treatment, it is important to know the type of small intestine cancer and whether the tumor can be removed by surgery. Tests and procedures to detect, diagnose, and stage small intestine cancer are usually done at the same time. The following tests and procedures may be used: The prognosis and treatment options depend on the following: Staging is used to find out how far the cancer has spread, but treatment decisions are not based on stage. See the General Information section for a description of tests and procedures used to detect, diagnose, and stage small intestine cancer. Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if small intestine cancer spreads to the liver, the cancer cells in the liver are actually small intestine cancer cells. The disease is metastatic small intestine cancer, not liver cancer. Treatment depends on whether the tumor can be removed by surgery and if the cancer is being treated as a primary tumor or is metastatic cancer. The cancer may come back in the small intestine or in other parts of the body. Different types of treatments are available for patients with small intestine cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment of small intestine cancer. One of the following types of surgery may be done: After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type of the cancer being treated. External radiation therapy is used to treat small intestine cancer. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on the type and stage of the cancer being treated. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Radiosensitizers are drugs that make tumor cells more sensitive to radiation therapy. Combining radiation therapy with radiosensitizers may kill more tumor cells. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. When possible, treatment of small intestine adenocarcinoma will be surgery to remove the tumor and some of the normal tissue around it. Treatment of small intestine adenocarcinoma that cannot be removed by surgery may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. When possible, treatment of small intestine leiomyosarcoma will be surgery to remove the tumor and some of the normal tissue around it. Treatment of small intestine leiomyosarcoma that cannot be removed by surgery may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent small intestine cancer that has spread to other parts of the body is usually a clinical trial of new anticancer drugs or immunotherapy. Treatment of locally recurrent small intestine cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about small intestine cancer, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of small intestine cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Small Intestine Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/small-intestine/patient/small-intestine-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389461] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Small Intestine Cancer Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the symptoms of Small Intestine Cancer ?

Signs and symptoms of small intestine cancer include unexplained weight loss and abdominal pain. These and other signs and symptoms may be caused by small intestine cancer or by other conditions. Check with your doctor if you have any of the following: - Pain or cramps in the middle of the abdomen. - Weight loss with no known reason. - A lump in the abdomen. - Blood in the stool.

The small intestine is part of the body’s digestive system, which also includes the esophagus, stomach, and large intestine. The digestive system removes and processes nutrients (vitamins, minerals, carbohydrates, fats, proteins, and water) from foods and helps pass waste material out of the body. The small intestine is a long tube that connects the stomach to the large intestine. It folds many times to fit inside the abdomen. EnlargeThe small intestine connects the stomach and the colon. It includes the duodenum, jejunum, and ileum. The types of cancer found in the small intestine are adenocarcinoma, sarcoma, carcinoid tumors, gastrointestinal stromal tumor, and lymphoma. This summary discusses adenocarcinoma and leiomyosarcoma (a type of sarcoma). Adenocarcinoma starts in glandular cells in the lining of the small intestine and is the most common type of small intestine cancer. Most of these tumors occur in the part of the small intestine near the stomach. They may grow and block the intestine. Leiomyosarcoma starts in the smooth muscle cells of the small intestine. Most of these tumors occur in the part of the small intestine near the large intestine. For more information on small intestine cancer, see the following: Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn't mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for small intestine cancer include the following: These and other signs and symptoms may be caused by small intestine cancer or by other conditions. Check with your doctor if you have any of the following: Procedures that make pictures of the small intestine and the area around it help diagnose small intestine cancer and show how far the cancer has spread. The process used to find out if cancer cells have spread within and around the small intestine is called staging. In order to plan treatment, it is important to know the type of small intestine cancer and whether the tumor can be removed by surgery. Tests and procedures to detect, diagnose, and stage small intestine cancer are usually done at the same time. The following tests and procedures may be used: The prognosis and treatment options depend on the following: Staging is used to find out how far the cancer has spread, but treatment decisions are not based on stage. See the General Information section for a description of tests and procedures used to detect, diagnose, and stage small intestine cancer. Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if small intestine cancer spreads to the liver, the cancer cells in the liver are actually small intestine cancer cells. The disease is metastatic small intestine cancer, not liver cancer. Treatment depends on whether the tumor can be removed by surgery and if the cancer is being treated as a primary tumor or is metastatic cancer. The cancer may come back in the small intestine or in other parts of the body. Different types of treatments are available for patients with small intestine cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment of small intestine cancer. One of the following types of surgery may be done: After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type of the cancer being treated. External radiation therapy is used to treat small intestine cancer. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on the type and stage of the cancer being treated. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Radiosensitizers are drugs that make tumor cells more sensitive to radiation therapy. Combining radiation therapy with radiosensitizers may kill more tumor cells. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. When possible, treatment of small intestine adenocarcinoma will be surgery to remove the tumor and some of the normal tissue around it. Treatment of small intestine adenocarcinoma that cannot be removed by surgery may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. When possible, treatment of small intestine leiomyosarcoma will be surgery to remove the tumor and some of the normal tissue around it. Treatment of small intestine leiomyosarcoma that cannot be removed by surgery may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent small intestine cancer that has spread to other parts of the body is usually a clinical trial of new anticancer drugs or immunotherapy. Treatment of locally recurrent small intestine cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about small intestine cancer, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of small intestine cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Small Intestine Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/small-intestine/patient/small-intestine-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389461] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Small Intestine Cancer Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

How to diagnose Small Intestine Cancer ?

Tests that examine the small intestine are used to detect (find), diagnose, and stage small intestine cancer.Procedures that make pictures of the small intestine and the area around it help diagnose small intestine cancer and show how far the cancer has spread. The process used to find out if cancer cells have spread within and around the small intestine is called staging. In order to plan treatment, it is important to know the type of small intestine cancer and whether the tumor can be removed by surgery. Tests and procedures to detect, diagnose, and stage small intestine cancer are usually done at the same time. The following tests and procedures may be used: - Physical exam and history : An exam of the body to check general signs of health, including checking for signs of disease, such as lumps or anything else that seems unusual. A history of the patients health habits and past illnesses and treatments will also be taken. - Blood chemistry studies : A procedure in which a blood sample is checked to measure the amounts of certain substances released into the blood by organs and tissues in the body. An unusual (higher or lower than normal) amount of a substance can be a sign of disease. - Liver function tests : A procedure in which a blood sample is checked to measure the amounts of certain substances released into the blood by the liver. A higher than normal amount of a substance can be a sign of liver disease that may be caused by small intestine cancer. - Endoscopy : A procedure to look at organs and tissues inside the body to check for abnormal areas. There are different types of endoscopy: - Upper endoscopy : A procedure to look at the inside of the esophagus, stomach, and duodenum (first part of the small intestine, near the stomach). An endoscope is inserted through the mouth and into the esophagus, stomach, and duodenum. An endoscope is a thin, tube-like instrument with a light and a lens for viewing. It may also have a tool to remove tissue samples, which are checked under a microscope for signs of cancer. - Capsule endoscopy : A procedure to look at the inside of the small intestine. A capsule that is about the size of a large pill and contains a light and a tiny wireless camera is swallowed by the patient. The capsule travels through the digestive tract, including the small intestine, and sends many pictures of the inside of the digestive tract to a recorder that is worn around the waist or over the shoulder. The pictures are sent from the recorder to a computer and viewed by the doctor who checks for signs of cancer. The capsule passes out of the body during a bowel movement. - Double balloon endoscopy : A procedure to look at the inside of the small intestine. A special instrument made up of two tubes (one inside the other) is inserted through the mouth or rectum and into the small intestine. The inside tube (an endoscope with a light and lens for viewing) is moved through part of the small intestine and a balloon at the end of it is inflated to keep the endoscope in place. Next, the outer tube is moved through the small intestine to reach the end of the endoscope, and a balloon at the end of the outer tube is inflated to keep it in place. Then, the balloon at the end of the endoscope is deflated and the endoscope is moved through the next part of the small intestine. These steps are repeated many times as the tubes move through the small intestine. The doctor is able to see the inside of the small intestine through the endoscope and use a tool to remove samples of abnormal tissue. The tissue samples are checked under a microscope for signs of cancer. This procedure may be done if the results of a capsule endoscopy are abnormal. This procedure is also called double balloon enteroscopy. - Laparotomy : A surgical procedure in which an incision (cut) is made in the wall of the abdomen to check the inside of the abdomen for signs of disease. The size of the incision depends on the reason the laparotomy is being done. Sometimes organs or lymph nodes are removed or tissue samples are taken and checked under a microscope for signs of disease. - Biopsy : The removal of cells or tissues so they can be viewed under a microscope to check for signs of cancer. This may be done during an endoscopy or laparotomy. The sample is checked by a pathologist to see if it contains cancer cells. - Upper GI series with small bowel follow-through: A series of x-rays of the esophagus, stomach, and small bowel. The patient drinks a liquid that contains barium (a silver-white metallic compound). The liquid coats the esophagus, stomach, and small bowel. X-rays are taken at different times as the barium travels through the upper GI tract and small bowel. - CT scan (CAT scan): A procedure that makes a series of detailed pictures of areas inside the body, taken from different angles. The pictures are made by a computer linked to an x-ray machine. A dye may be injected into a vein or swallowed to help the organs or tissues show up more clearly. This procedure is also called computed tomography, computerized tomography, or computerized axial tomography. - MRI (magnetic resonance imaging): A procedure that uses a magnet, radio waves, and a computer to make a series of detailed pictures of areas inside the body. This procedure is also called nuclear magnetic resonance imaging (NMRI).

The small intestine is part of the body’s digestive system, which also includes the esophagus, stomach, and large intestine. The digestive system removes and processes nutrients (vitamins, minerals, carbohydrates, fats, proteins, and water) from foods and helps pass waste material out of the body. The small intestine is a long tube that connects the stomach to the large intestine. It folds many times to fit inside the abdomen. EnlargeThe small intestine connects the stomach and the colon. It includes the duodenum, jejunum, and ileum. The types of cancer found in the small intestine are adenocarcinoma, sarcoma, carcinoid tumors, gastrointestinal stromal tumor, and lymphoma. This summary discusses adenocarcinoma and leiomyosarcoma (a type of sarcoma). Adenocarcinoma starts in glandular cells in the lining of the small intestine and is the most common type of small intestine cancer. Most of these tumors occur in the part of the small intestine near the stomach. They may grow and block the intestine. Leiomyosarcoma starts in the smooth muscle cells of the small intestine. Most of these tumors occur in the part of the small intestine near the large intestine. For more information on small intestine cancer, see the following: Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn't mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for small intestine cancer include the following: These and other signs and symptoms may be caused by small intestine cancer or by other conditions. Check with your doctor if you have any of the following: Procedures that make pictures of the small intestine and the area around it help diagnose small intestine cancer and show how far the cancer has spread. The process used to find out if cancer cells have spread within and around the small intestine is called staging. In order to plan treatment, it is important to know the type of small intestine cancer and whether the tumor can be removed by surgery. Tests and procedures to detect, diagnose, and stage small intestine cancer are usually done at the same time. The following tests and procedures may be used: The prognosis and treatment options depend on the following: Staging is used to find out how far the cancer has spread, but treatment decisions are not based on stage. See the General Information section for a description of tests and procedures used to detect, diagnose, and stage small intestine cancer. Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if small intestine cancer spreads to the liver, the cancer cells in the liver are actually small intestine cancer cells. The disease is metastatic small intestine cancer, not liver cancer. Treatment depends on whether the tumor can be removed by surgery and if the cancer is being treated as a primary tumor or is metastatic cancer. The cancer may come back in the small intestine or in other parts of the body. Different types of treatments are available for patients with small intestine cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment of small intestine cancer. One of the following types of surgery may be done: After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type of the cancer being treated. External radiation therapy is used to treat small intestine cancer. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on the type and stage of the cancer being treated. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Radiosensitizers are drugs that make tumor cells more sensitive to radiation therapy. Combining radiation therapy with radiosensitizers may kill more tumor cells. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. When possible, treatment of small intestine adenocarcinoma will be surgery to remove the tumor and some of the normal tissue around it. Treatment of small intestine adenocarcinoma that cannot be removed by surgery may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. When possible, treatment of small intestine leiomyosarcoma will be surgery to remove the tumor and some of the normal tissue around it. Treatment of small intestine leiomyosarcoma that cannot be removed by surgery may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent small intestine cancer that has spread to other parts of the body is usually a clinical trial of new anticancer drugs or immunotherapy. Treatment of locally recurrent small intestine cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about small intestine cancer, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of small intestine cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Small Intestine Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/small-intestine/patient/small-intestine-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389461] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Small Intestine Cancer Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is the outlook for Small Intestine Cancer ?

Certain factors affect prognosis (chance of recovery) and treatment options.The prognosis (chance of recovery) and treatment options depend on the following: - The type of small intestine cancer. - Whether the cancer is in the inner lining of the small intestine only or has spread into or beyond the wall of the small intestine. - Whether the cancer has spread to other places in the body, such as the lymph nodes, liver, or peritoneum (tissue that lines the wall of the abdomen and covers most of the organs in the abdomen). - Whether the cancer can be completely removed by surgery. - Whether the cancer is newly diagnosed or has recurred.

The small intestine is part of the body’s digestive system, which also includes the esophagus, stomach, and large intestine. The digestive system removes and processes nutrients (vitamins, minerals, carbohydrates, fats, proteins, and water) from foods and helps pass waste material out of the body. The small intestine is a long tube that connects the stomach to the large intestine. It folds many times to fit inside the abdomen. EnlargeThe small intestine connects the stomach and the colon. It includes the duodenum, jejunum, and ileum. The types of cancer found in the small intestine are adenocarcinoma, sarcoma, carcinoid tumors, gastrointestinal stromal tumor, and lymphoma. This summary discusses adenocarcinoma and leiomyosarcoma (a type of sarcoma). Adenocarcinoma starts in glandular cells in the lining of the small intestine and is the most common type of small intestine cancer. Most of these tumors occur in the part of the small intestine near the stomach. They may grow and block the intestine. Leiomyosarcoma starts in the smooth muscle cells of the small intestine. Most of these tumors occur in the part of the small intestine near the large intestine. For more information on small intestine cancer, see the following: Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn't mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for small intestine cancer include the following: These and other signs and symptoms may be caused by small intestine cancer or by other conditions. Check with your doctor if you have any of the following: Procedures that make pictures of the small intestine and the area around it help diagnose small intestine cancer and show how far the cancer has spread. The process used to find out if cancer cells have spread within and around the small intestine is called staging. In order to plan treatment, it is important to know the type of small intestine cancer and whether the tumor can be removed by surgery. Tests and procedures to detect, diagnose, and stage small intestine cancer are usually done at the same time. The following tests and procedures may be used: The prognosis and treatment options depend on the following: Staging is used to find out how far the cancer has spread, but treatment decisions are not based on stage. See the General Information section for a description of tests and procedures used to detect, diagnose, and stage small intestine cancer. Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if small intestine cancer spreads to the liver, the cancer cells in the liver are actually small intestine cancer cells. The disease is metastatic small intestine cancer, not liver cancer. Treatment depends on whether the tumor can be removed by surgery and if the cancer is being treated as a primary tumor or is metastatic cancer. The cancer may come back in the small intestine or in other parts of the body. Different types of treatments are available for patients with small intestine cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment of small intestine cancer. One of the following types of surgery may be done: After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type of the cancer being treated. External radiation therapy is used to treat small intestine cancer. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on the type and stage of the cancer being treated. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Radiosensitizers are drugs that make tumor cells more sensitive to radiation therapy. Combining radiation therapy with radiosensitizers may kill more tumor cells. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. When possible, treatment of small intestine adenocarcinoma will be surgery to remove the tumor and some of the normal tissue around it. Treatment of small intestine adenocarcinoma that cannot be removed by surgery may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. When possible, treatment of small intestine leiomyosarcoma will be surgery to remove the tumor and some of the normal tissue around it. Treatment of small intestine leiomyosarcoma that cannot be removed by surgery may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent small intestine cancer that has spread to other parts of the body is usually a clinical trial of new anticancer drugs or immunotherapy. Treatment of locally recurrent small intestine cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about small intestine cancer, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of small intestine cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Small Intestine Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/small-intestine/patient/small-intestine-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389461] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Small Intestine Cancer Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the stages of Small Intestine Cancer ?

Key Points - Tests and procedures to stage small intestine cancer are usually done at the same time as diagnosis. - There are three ways that cancer spreads in the body. - Cancer may spread from where it began to other parts of the body. - Small intestine cancer is grouped according to whether or not the tumor can be completely removed by surgery. Tests and procedures to stage small intestine cancer are usually done at the same time as diagnosis. Staging is used to find out how far the cancer has spread, but treatment decisions are not based on stage. See the General Information section for a description of tests and procedures used to detect, diagnose, and stage small intestine cancer. There are three ways that cancer spreads in the body. Cancer can spread through tissue, the lymph system, and the blood: - Tissue. The cancer spreads from where it began by growing into nearby areas. - Lymph system. The cancer spreads from where it began by getting into the lymph system. The cancer travels through the lymph vessels to other parts of the body. - Blood. The cancer spreads from where it began by getting into the blood. The cancer travels through the blood vessels to other parts of the body. Cancer may spread from where it began to other parts of the body. When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. - Lymph system. The cancer gets into the lymph system, travels through the lymph vessels, and forms a tumor (metastatic tumor) in another part of the body. - Blood. The cancer gets into the blood, travels through the blood vessels, and forms a tumor (metastatic tumor) in another part of the body. The metastatic tumor is the same type of cancer as the primary tumor. For example, if small intestine cancer spreads to the liver, the cancer cells in the liver are actually small intestine cancer cells. The disease is metastatic small intestine cancer, not liver cancer. Small intestine cancer is grouped according to whether or not the tumor can be completely removed by surgery. Treatment depends on whether the tumor can be removed by surgery and if the cancer is being treated as a primary tumor or is metastatic cancer.

The small intestine is part of the body’s digestive system, which also includes the esophagus, stomach, and large intestine. The digestive system removes and processes nutrients (vitamins, minerals, carbohydrates, fats, proteins, and water) from foods and helps pass waste material out of the body. The small intestine is a long tube that connects the stomach to the large intestine. It folds many times to fit inside the abdomen. EnlargeThe small intestine connects the stomach and the colon. It includes the duodenum, jejunum, and ileum. The types of cancer found in the small intestine are adenocarcinoma, sarcoma, carcinoid tumors, gastrointestinal stromal tumor, and lymphoma. This summary discusses adenocarcinoma and leiomyosarcoma (a type of sarcoma). Adenocarcinoma starts in glandular cells in the lining of the small intestine and is the most common type of small intestine cancer. Most of these tumors occur in the part of the small intestine near the stomach. They may grow and block the intestine. Leiomyosarcoma starts in the smooth muscle cells of the small intestine. Most of these tumors occur in the part of the small intestine near the large intestine. For more information on small intestine cancer, see the following: Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn't mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for small intestine cancer include the following: These and other signs and symptoms may be caused by small intestine cancer or by other conditions. Check with your doctor if you have any of the following: Procedures that make pictures of the small intestine and the area around it help diagnose small intestine cancer and show how far the cancer has spread. The process used to find out if cancer cells have spread within and around the small intestine is called staging. In order to plan treatment, it is important to know the type of small intestine cancer and whether the tumor can be removed by surgery. Tests and procedures to detect, diagnose, and stage small intestine cancer are usually done at the same time. The following tests and procedures may be used: The prognosis and treatment options depend on the following: Staging is used to find out how far the cancer has spread, but treatment decisions are not based on stage. See the General Information section for a description of tests and procedures used to detect, diagnose, and stage small intestine cancer. Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if small intestine cancer spreads to the liver, the cancer cells in the liver are actually small intestine cancer cells. The disease is metastatic small intestine cancer, not liver cancer. Treatment depends on whether the tumor can be removed by surgery and if the cancer is being treated as a primary tumor or is metastatic cancer. The cancer may come back in the small intestine or in other parts of the body. Different types of treatments are available for patients with small intestine cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment of small intestine cancer. One of the following types of surgery may be done: After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type of the cancer being treated. External radiation therapy is used to treat small intestine cancer. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on the type and stage of the cancer being treated. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Radiosensitizers are drugs that make tumor cells more sensitive to radiation therapy. Combining radiation therapy with radiosensitizers may kill more tumor cells. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. When possible, treatment of small intestine adenocarcinoma will be surgery to remove the tumor and some of the normal tissue around it. Treatment of small intestine adenocarcinoma that cannot be removed by surgery may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. When possible, treatment of small intestine leiomyosarcoma will be surgery to remove the tumor and some of the normal tissue around it. Treatment of small intestine leiomyosarcoma that cannot be removed by surgery may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent small intestine cancer that has spread to other parts of the body is usually a clinical trial of new anticancer drugs or immunotherapy. Treatment of locally recurrent small intestine cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about small intestine cancer, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of small intestine cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Small Intestine Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/small-intestine/patient/small-intestine-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389461] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Small Intestine Cancer Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the treatments for Small Intestine Cancer ?

Key Points - There are different types of treatment for patients with small intestine cancer. - Three types of standard treatment are used: - Surgery - Radiation therapy - Chemotherapy - New types of treatment are being tested in clinical trials. - Biologic therapy - Radiation therapy with radiosensitizers - Patients may want to think about taking part in a clinical trial. - Patients can enter clinical trials before, during, or after starting their cancer treatment. - Follow-up tests may be needed. There are different types of treatment for patients with small intestine cancer. Different types of treatments are available for patients with small intestine cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Three types of standard treatment are used: Surgery Surgery is the most common treatment of small intestine cancer. One of the following types of surgery may be done: - Resection: Surgery to remove part or all of an organ that contains cancer. The resection may include the small intestine and nearby organs (if the cancer has spread). The doctor may remove the section of the small intestine that contains cancer and perform an anastomosis (joining the cut ends of the intestine together). The doctor will usually remove lymph nodes near the small intestine and examine them under a microscope to see whether they contain cancer. - Bypass: Surgery to allow food in the small intestine to go around (bypass) a tumor that is blocking the intestine but cannot be removed. Even if the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: - External radiation therapy uses a machine outside the body to send radiation toward the cancer. - Internal radiation therapy uses a radioactive substance sealed in needles, seeds, wires, or catheters that are placed directly into or near the cancer. The way the radiation therapy is given depends on the type of the cancer being treated. External radiation therapy is used to treat small intestine cancer. Chemotherapy Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on the type and stage of the cancer being treated. New types of treatment are being tested in clinical trials. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Biologic therapy Biologic therapy is a treatment that uses the patient&apos;s immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body&apos;s natural defenses against cancer. This type of cancer treatment is also called biotherapy or immunotherapy. Radiation therapy with radiosensitizers Radiosensitizers are drugs that make tumor cells more sensitive to radiation therapy. Combining radiation therapy with radiosensitizers may kill more tumor cells. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials. Follow-up tests may be needed. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. Treatment Options for Small Intestine Cancer Small Intestine Adenocarcinoma When possible, treatment of small intestine adenocarcinoma will be surgery to remove the tumor and some of the normal tissue around it. Treatment of small intestine adenocarcinoma that cannot be removed by surgery may include the following: - Surgery to bypass the tumor. - Radiation therapy as palliative therapy to relieve symptoms and improve the patient&apos;s quality of life. - A clinical trial of radiation therapy with radiosensitizers, with or without chemotherapy. - A clinical trial of new anticancer drugs. - A clinical trial of biologic therapy. Check the list of NCI-supported cancer clinical trials that are now accepting patients with small intestine adenocarcinoma. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Small Intestine Leiomyosarcoma When possible, treatment of small intestine leiomyosarcoma will be surgery to remove the tumor and some of the normal tissue around it. Treatment of small intestine leiomyosarcoma that cannot be removed by surgery may include the following: - Surgery (to bypass the tumor) and radiation therapy. - Surgery, radiation therapy, or chemotherapy as palliative therapy to relieve symptoms and improve the patient&apos;s quality of life. - A clinical trial of new anticancer drugs. - A clinical trial of biologic therapy. Check the list of NCI-supported cancer clinical trials that are now accepting patients with small intestine leiomyosarcoma. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Recurrent Small Intestine Cancer Treatment of recurrent small intestine cancer that has spread to other parts of the body is usually a clinical trial of new anticancer drugs or biologic therapy. Treatment of locally recurrent small intestine cancer may include the following: - Surgery. - Radiation therapy or chemotherapy as palliative therapy to relieve symptoms and improve the patient&apos;s quality of life. - A clinical trial of radiation therapy with radiosensitizers, with or without chemotherapy. Check the list of NCI-supported cancer clinical trials that are now accepting patients with recurrent small intestine cancer. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website.

Renal cell cancer (also called kidney cancer or renal cell adenocarcinoma) is a disease in which malignant (cancer) cells are found in the lining of tubules (very small tubes) in the kidney. There are 2 kidneys, one on each side of the backbone, above the waist. Tiny tubules in the kidneys filter and clean the blood. They take out waste products and make urine. The urine passes from each kidney through a long tube called a ureter into the bladder. The bladder holds the urine until it passes through the urethra and leaves the body. Cancer that starts in the ureters or the renal pelvis (the part of the kidney that collects urine and drains it to the ureters) is different from renal cell cancer. For more information, see the PDQ summary about Transitional Cell Cancer of the Renal Pelvis and Ureter Treatment. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn't mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for renal cell cancer include the following: These and other signs and symptoms may be caused by renal cell cancer or by other conditions. There may be no signs or symptoms in the early stages. Signs and symptoms may appear as the tumor grows. Check with your doctor if you have any of the following: The following tests and procedures may be used: The prognosis and treatment options depend on the following: The process used to find out if cancer has spread within the kidney or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests and procedures may be used in the staging process: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if renal cell cancer spreads to the bone, the cancer cells in the bone are actually cancerous renal cells. The disease is metastatic renal cell cancer, not bone cancer. In stage I, the tumor is 7 centimeters or smaller and is found in the kidney only. In stage II, the tumor is larger than 7 centimeters and is found in the kidney only. In stage III, one of the following is found: In stage IV, one of the following is found: The cancer may come back in the kidney or in other parts of the body. Different types of treatments are available for patients with renal cell cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery to remove part or all of the kidney is often used to treat renal cell cancer. The following types of surgery may be used: A person can live with part of 1 working kidney, but if both kidneys are removed or not working, the person will need dialysis (a procedure to clean the blood using a machine outside of the body) or a kidney transplant (replacement with a healthy donated kidney). A kidney transplant may be done when the disease is in the kidney only and a donated kidney can be found. If the patient has to wait for a donated kidney, other treatment is given as needed. When surgery to remove the cancer is not possible, a treatment called arterial embolization may be used to shrink the tumor. A small incision is made and a catheter (thin tube) is inserted into the main blood vessel that flows to the kidney. Small pieces of a special gelatin sponge are injected through the catheter into the blood vessel. The sponges block the blood flow to the kidney and prevent the cancer cells from getting oxygen and other substances they need to grow. After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. External radiation therapy is used to treat renal cell cancer, and may also be used as palliative therapy to relieve symptoms and improve quality of life. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). For more information, see Drugs Approved for Kidney (Renal Cell) Cancer. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. The following types of immunotherapy are being used in the treatment of renal cell cancer: There are two types of immune checkpoint inhibitor therapy: For more information, see Drugs Approved for Kidney (Renal Cell) Cancer. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapy with antiangiogenic agents are used to treat advanced renal cell cancer. Antiangiogenic agents keep blood vessels from forming in a tumor, causing the tumor to starve and stop growing or to shrink. Monoclonal antibodies and kinase inhibitors are two types of antiangiogenic agents used to treat renal cell cancer. Vascular endothelial growth factor (VEGF) inhibitors and mTOR inhibitors are kinase inhibitors used to treat renal cell cancer. For more information, see Drugs Approved for Kidney (Renal Cell) Cancer. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage I renal cell cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage II renal cell cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage III renal cell cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. First-line therapy may include the following: Second-line therapy may include the following: Third-line and fourth-line therapies may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about renal cell cancer, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of renal cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Renal Cell Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/kidney/patient/kidney-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389448] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Renal Cell Cancer Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is (are) Renal Cell Cancer ?

Key Points - Renal cell cancer is a disease in which malignant (cancer) cells form in tubules of the kidney. - Smoking and misuse of certain pain medicines can affect the risk of renal cell cancer. - Signs of renal cell cancer include blood in the urine and a lump in the abdomen. - Tests that examine the abdomen and kidneys are used to detect (find) and diagnose renal cell cancer. - Certain factors affect prognosis (chance of recovery) and treatment options. Renal cell cancer is a disease in which malignant (cancer) cells form in tubules of the kidney. Renal cell cancer (also called kidney cancer or renal adenocarcinoma) is a disease in which malignant (cancer) cells are found in the lining of tubules (very small tubes) in the kidney. There are 2 kidneys, one on each side of the backbone, above the waist. Tiny tubules in the kidneys filter and clean the blood. They take out waste products and make urine. The urine passes from each kidney through a long tube called a ureter into the bladder. The bladder holds the urine until it passes through the urethra and leaves the body. Cancer that starts in the ureters or the renal pelvis (the part of the kidney that collects urine and drains it to the ureters) is different from renal cell cancer. (See the PDQ summary about Transitional Cell Cancer of the Renal Pelvis and Ureter Treatment for more information).

Renal cell cancer (also called kidney cancer or renal cell adenocarcinoma) is a disease in which malignant (cancer) cells are found in the lining of tubules (very small tubes) in the kidney. There are 2 kidneys, one on each side of the backbone, above the waist. Tiny tubules in the kidneys filter and clean the blood. They take out waste products and make urine. The urine passes from each kidney through a long tube called a ureter into the bladder. The bladder holds the urine until it passes through the urethra and leaves the body. Cancer that starts in the ureters or the renal pelvis (the part of the kidney that collects urine and drains it to the ureters) is different from renal cell cancer. For more information, see the PDQ summary about Transitional Cell Cancer of the Renal Pelvis and Ureter Treatment. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn't mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for renal cell cancer include the following: These and other signs and symptoms may be caused by renal cell cancer or by other conditions. There may be no signs or symptoms in the early stages. Signs and symptoms may appear as the tumor grows. Check with your doctor if you have any of the following: The following tests and procedures may be used: The prognosis and treatment options depend on the following: The process used to find out if cancer has spread within the kidney or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests and procedures may be used in the staging process: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if renal cell cancer spreads to the bone, the cancer cells in the bone are actually cancerous renal cells. The disease is metastatic renal cell cancer, not bone cancer. In stage I, the tumor is 7 centimeters or smaller and is found in the kidney only. In stage II, the tumor is larger than 7 centimeters and is found in the kidney only. In stage III, one of the following is found: In stage IV, one of the following is found: The cancer may come back in the kidney or in other parts of the body. Different types of treatments are available for patients with renal cell cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery to remove part or all of the kidney is often used to treat renal cell cancer. The following types of surgery may be used: A person can live with part of 1 working kidney, but if both kidneys are removed or not working, the person will need dialysis (a procedure to clean the blood using a machine outside of the body) or a kidney transplant (replacement with a healthy donated kidney). A kidney transplant may be done when the disease is in the kidney only and a donated kidney can be found. If the patient has to wait for a donated kidney, other treatment is given as needed. When surgery to remove the cancer is not possible, a treatment called arterial embolization may be used to shrink the tumor. A small incision is made and a catheter (thin tube) is inserted into the main blood vessel that flows to the kidney. Small pieces of a special gelatin sponge are injected through the catheter into the blood vessel. The sponges block the blood flow to the kidney and prevent the cancer cells from getting oxygen and other substances they need to grow. After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. External radiation therapy is used to treat renal cell cancer, and may also be used as palliative therapy to relieve symptoms and improve quality of life. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). For more information, see Drugs Approved for Kidney (Renal Cell) Cancer. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. The following types of immunotherapy are being used in the treatment of renal cell cancer: There are two types of immune checkpoint inhibitor therapy: For more information, see Drugs Approved for Kidney (Renal Cell) Cancer. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapy with antiangiogenic agents are used to treat advanced renal cell cancer. Antiangiogenic agents keep blood vessels from forming in a tumor, causing the tumor to starve and stop growing or to shrink. Monoclonal antibodies and kinase inhibitors are two types of antiangiogenic agents used to treat renal cell cancer. Vascular endothelial growth factor (VEGF) inhibitors and mTOR inhibitors are kinase inhibitors used to treat renal cell cancer. For more information, see Drugs Approved for Kidney (Renal Cell) Cancer. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage I renal cell cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage II renal cell cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage III renal cell cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. First-line therapy may include the following: Second-line therapy may include the following: Third-line and fourth-line therapies may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about renal cell cancer, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of renal cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Renal Cell Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/kidney/patient/kidney-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389448] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Renal Cell Cancer Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the stages of Renal Cell Cancer ?

Key Points - After renal cell cancer has been diagnosed, tests are done to find out if cancer cells have spread within the kidney or to other parts of the body. - There are three ways that cancer spreads in the body. - Cancer may spread from where it began to other parts of the body. - The following stages are used for renal cell cancer: - Stage I - Stage II - Stage III - Stage IV After renal cell cancer has been diagnosed, tests are done to find out if cancer cells have spread within the kidney or to other parts of the body. The process used to find out if cancer has spread within the kidney or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests and procedures may be used in the staging process: - CT scan (CAT scan): A procedure that makes a series of detailed pictures of areas inside the body, taken from different angles. The pictures are made by a computer linked to an x-ray machine. A dye may be injected into a vein or swallowed to help the organs or tissues show up more clearly. This procedure is also called computed tomography, computerized tomography, or computerized axial tomography. - MRI (magnetic resonance imaging): A procedure that uses a magnet, radio waves, and a computer to make a series of detailed pictures of areas inside the body. This procedure is also called nuclear magnetic resonance imaging (NMRI). - Chest x-ray : An x-ray of the organs and bones inside the chest. An x-ray is a type of energy beam that can go through the body and onto film, making a picture of areas inside the body. There are three ways that cancer spreads in the body. Cancer can spread through tissue, the lymph system, and the blood: - Tissue. The cancer spreads from where it began by growing into nearby areas. - Lymph system. The cancer spreads from where it began by getting into the lymph system. The cancer travels through the lymph vessels to other parts of the body. - Blood. The cancer spreads from where it began by getting into the blood. The cancer travels through the blood vessels to other parts of the body. Cancer may spread from where it began to other parts of the body. When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. - Lymph system. The cancer gets into the lymph system, travels through the lymph vessels, and forms a tumor (metastatic tumor) in another part of the body. - Blood. The cancer gets into the blood, travels through the blood vessels, and forms a tumor (metastatic tumor) in another part of the body. The metastatic tumor is the same type of cancer as the primary tumor. For example, if renal cell cancer spreads to the bone, the cancer cells in the bone are actually cancerous renal cells. The disease is metastatic renal cell cancer, not bone cancer. The following stages are used for renal cell cancer: Stage I In stage I, the tumor is 7 centimeters or smaller and is found only in the kidney. Stage II In stage II, the tumor is larger than 7 centimeters and is found only in the kidney. Stage III In stage III: - the tumor is any size and cancer is found only in the kidney and in 1 or more nearby lymph nodes; or - cancer is found in the main blood vessels of the kidney or in the layer of fatty tissue around the kidney. Cancer may be found in 1 or more nearby lymph nodes. Stage IV In stage IV, cancer has spread: - beyond the layer of fatty tissue around the kidney and may be found in the adrenal gland above the kidney with cancer, or in nearby lymph nodes; or - to other organs, such as the lungs, liver, bones, or brain, and may have spread to lymph nodes.

Renal cell cancer (also called kidney cancer or renal cell adenocarcinoma) is a disease in which malignant (cancer) cells are found in the lining of tubules (very small tubes) in the kidney. There are 2 kidneys, one on each side of the backbone, above the waist. Tiny tubules in the kidneys filter and clean the blood. They take out waste products and make urine. The urine passes from each kidney through a long tube called a ureter into the bladder. The bladder holds the urine until it passes through the urethra and leaves the body. Cancer that starts in the ureters or the renal pelvis (the part of the kidney that collects urine and drains it to the ureters) is different from renal cell cancer. For more information, see the PDQ summary about Transitional Cell Cancer of the Renal Pelvis and Ureter Treatment. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn't mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for renal cell cancer include the following: These and other signs and symptoms may be caused by renal cell cancer or by other conditions. There may be no signs or symptoms in the early stages. Signs and symptoms may appear as the tumor grows. Check with your doctor if you have any of the following: The following tests and procedures may be used: The prognosis and treatment options depend on the following: The process used to find out if cancer has spread within the kidney or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests and procedures may be used in the staging process: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if renal cell cancer spreads to the bone, the cancer cells in the bone are actually cancerous renal cells. The disease is metastatic renal cell cancer, not bone cancer. In stage I, the tumor is 7 centimeters or smaller and is found in the kidney only. In stage II, the tumor is larger than 7 centimeters and is found in the kidney only. In stage III, one of the following is found: In stage IV, one of the following is found: The cancer may come back in the kidney or in other parts of the body. Different types of treatments are available for patients with renal cell cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery to remove part or all of the kidney is often used to treat renal cell cancer. The following types of surgery may be used: A person can live with part of 1 working kidney, but if both kidneys are removed or not working, the person will need dialysis (a procedure to clean the blood using a machine outside of the body) or a kidney transplant (replacement with a healthy donated kidney). A kidney transplant may be done when the disease is in the kidney only and a donated kidney can be found. If the patient has to wait for a donated kidney, other treatment is given as needed. When surgery to remove the cancer is not possible, a treatment called arterial embolization may be used to shrink the tumor. A small incision is made and a catheter (thin tube) is inserted into the main blood vessel that flows to the kidney. Small pieces of a special gelatin sponge are injected through the catheter into the blood vessel. The sponges block the blood flow to the kidney and prevent the cancer cells from getting oxygen and other substances they need to grow. After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. External radiation therapy is used to treat renal cell cancer, and may also be used as palliative therapy to relieve symptoms and improve quality of life. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). For more information, see Drugs Approved for Kidney (Renal Cell) Cancer. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. The following types of immunotherapy are being used in the treatment of renal cell cancer: There are two types of immune checkpoint inhibitor therapy: For more information, see Drugs Approved for Kidney (Renal Cell) Cancer. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapy with antiangiogenic agents are used to treat advanced renal cell cancer. Antiangiogenic agents keep blood vessels from forming in a tumor, causing the tumor to starve and stop growing or to shrink. Monoclonal antibodies and kinase inhibitors are two types of antiangiogenic agents used to treat renal cell cancer. Vascular endothelial growth factor (VEGF) inhibitors and mTOR inhibitors are kinase inhibitors used to treat renal cell cancer. For more information, see Drugs Approved for Kidney (Renal Cell) Cancer. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage I renal cell cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage II renal cell cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage III renal cell cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. First-line therapy may include the following: Second-line therapy may include the following: Third-line and fourth-line therapies may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about renal cell cancer, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of renal cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Renal Cell Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/kidney/patient/kidney-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389448] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. 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In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Renal Cell Cancer Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the treatments for Renal Cell Cancer ?

Key Points - There are different types of treatment for patients with renal cell cancer. - Five types of standard treatment are used: - Surgery - Radiation therapy - Chemotherapy - Biologic therapy - Targeted therapy - New types of treatment are being tested in clinical trials. - Patients may want to think about taking part in a clinical trial. - Patients can enter clinical trials before, during, or after starting their cancer treatment. - Follow-up tests may be needed. There are different types of treatment for patients with renal cell cancer. Different types of treatments are available for patients with renal cell cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Five types of standard treatment are used: Surgery Surgery to remove part or all of the kidney is often used to treat renal cell cancer. The following types of surgery may be used: - Partial nephrectomy: A surgical procedure to remove the cancer within the kidney and some of the tissue around it. A partial nephrectomy may be done to prevent loss of kidney function when the other kidney is damaged or has already been removed. - Simple nephrectomy: A surgical procedure to remove the kidney only. - Radical nephrectomy: A surgical procedure to remove the kidney, the adrenal gland, surrounding tissue, and, usually, nearby lymph nodes. A person can live with part of 1 working kidney, but if both kidneys are removed or not working, the person will need dialysis (a procedure to clean the blood using a machine outside of the body) or a kidney transplant (replacement with a healthy donated kidney). A kidney transplant may be done when the disease is in the kidney only and a donated kidney can be found. If the patient has to wait for a donated kidney, other treatment is given as needed. When surgery to remove the cancer is not possible, a treatment called arterial embolization may be used to shrink the tumor. A small incision is made and a catheter (thin tube) is inserted into the main blood vessel that flows to the kidney. Small pieces of a special gelatin sponge are injected through the catheter into the blood vessel. The sponges block the blood flow to the kidney and prevent the cancer cells from getting oxygen and other substances they need to grow. Even if the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: - External radiation therapy uses a machine outside the body to send radiation toward the cancer. - Internal radiation therapy uses a radioactive substance sealed in needles, seeds, wires, or catheters that are placed directly into or near the cancer. The way the radiation therapy is given depends on the type and stage of the cancer being treated. External radiation therapy is used to treat renal cell cancer, and may also be used as palliative therapy to relieve symptoms and improve quality of life. Chemotherapy Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on the type and stage of the cancer being treated. See Drugs Approved for Kidney (Renal Cell) Cancer for more information. Biologic therapy Biologic therapy is a treatment that uses the patient&apos;s immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body&apos;s natural defenses against cancer. This type of cancer treatment is also called biotherapy or immunotherapy. The following types of biologic therapy are being used or studied in the treatment of renal cell cancer: - Nivolumab: Nivolumab is a monoclonal antibody that boosts the bodys immune response against renal cell cancer cells. - Interferon: Interferon affects the division of cancer cells and can slow tumor growth. - Interleukin-2 (IL-2): IL-2 boosts the growth and activity of many immune cells, especially lymphocytes (a type of white blood cell). Lymphocytes can attack and kill cancer cells. See Drugs Approved for Kidney (Renal Cell) Cancer for more information. Targeted therapy Targeted therapy uses drugs or other substances to identify and attack specific cancer cells without harming normal cells. Targeted therapy with antiangiogenic agents are used to treat advanced renal cell cancer. Antiangiogenic agents keep blood vessels from forming in a tumor, causing the tumor to starve and stop growing or to shrink. Monoclonal antibodies and kinase inhibitors are two types of antiangiogenic agents used to treat renal cell cancer. Monoclonal antibody therapy uses antibodies made in the laboratory, from a single type of immune system cell. These antibodies can identify substances on cancer cells or normal substances that may help cancer cells grow. The antibodies attach to the substances and kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. Monoclonal antibodies used to treat renal cell cancer attach to and block substances that cause new blood vessels to form in tumors. Kinase inhibitors stop cells from dividing and may prevent the growth of new blood vessels that tumors need to grow. An mTOR inhibitor is a type of kinase inhibitor. Everolimus and temsirolimus are mTOR inhibitors used to treat advanced renal cell cancer. See Drugs Approved for Kidney (Renal Cell) Cancer for more information. New types of treatment are being tested in clinical trials. Information about clinical trials is available from the NCI website. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials. Follow-up tests may be needed. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. Treatment Options for Renal Cell Cancer Stage I Renal Cell Cancer Treatment of stage I renal cell cancer may include the following: - Surgery (radical nephrectomy, simple nephrectomy, or partial nephrectomy). - Radiation therapy as palliative therapy to relieve symptoms in patients who cannot have surgery. - Arterial embolization as palliative therapy. - A clinical trial of a new treatment. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage I renal cell cancer. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Stage II Renal Cell Cancer Treatment of stage II renal cell cancer may include the following: - Surgery (radical nephrectomy or partial nephrectomy). - Surgery (nephrectomy), before or after radiation therapy. - Radiation therapy as palliative therapy to relieve symptoms in patients who cannot have surgery. - Arterial embolization as palliative therapy. - A clinical trial of a new treatment. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage II renal cell cancer. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Stage III Renal Cell Cancer Treatment of stage III renal cell cancer may include the following: - Surgery (radical nephrectomy). Blood vessels of the kidney and some lymph nodes may also be removed. - Arterial embolization followed by surgery (radical nephrectomy). - Radiation therapy as palliative therapy to relieve symptoms and improve the quality of life. - Arterial embolization as palliative therapy. - Surgery (nephrectomy) as palliative therapy. - Radiation therapy before or after surgery (radical nephrectomy). - A clinical trial of biologic therapy following surgery. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage III renal cell cancer. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Stage IV and Recurrent Renal Cell Cancer Treatment of stage IV and recurrent renal cell cancer may include the following: - Surgery (radical nephrectomy). - Surgery (nephrectomy) to reduce the size of the tumor. - Targeted therapy. - Biologic therapy. - Radiation therapy as palliative therapy to relieve symptoms and improve the quality of life. - A clinical trial of a new treatment. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage IV renal cell cancer and recurrent renal cell cancer. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website.

Renal cell cancer (also called kidney cancer or renal cell adenocarcinoma) is a disease in which malignant (cancer) cells are found in the lining of tubules (very small tubes) in the kidney. There are 2 kidneys, one on each side of the backbone, above the waist. Tiny tubules in the kidneys filter and clean the blood. They take out waste products and make urine. The urine passes from each kidney through a long tube called a ureter into the bladder. The bladder holds the urine until it passes through the urethra and leaves the body. Cancer that starts in the ureters or the renal pelvis (the part of the kidney that collects urine and drains it to the ureters) is different from renal cell cancer. For more information, see the PDQ summary about Transitional Cell Cancer of the Renal Pelvis and Ureter Treatment. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn't mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for renal cell cancer include the following: These and other signs and symptoms may be caused by renal cell cancer or by other conditions. There may be no signs or symptoms in the early stages. Signs and symptoms may appear as the tumor grows. Check with your doctor if you have any of the following: The following tests and procedures may be used: The prognosis and treatment options depend on the following: The process used to find out if cancer has spread within the kidney or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests and procedures may be used in the staging process: Cancer can spread through tissue, the lymph system, and the blood: When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if renal cell cancer spreads to the bone, the cancer cells in the bone are actually cancerous renal cells. The disease is metastatic renal cell cancer, not bone cancer. In stage I, the tumor is 7 centimeters or smaller and is found in the kidney only. In stage II, the tumor is larger than 7 centimeters and is found in the kidney only. In stage III, one of the following is found: In stage IV, one of the following is found: The cancer may come back in the kidney or in other parts of the body. Different types of treatments are available for patients with renal cell cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery to remove part or all of the kidney is often used to treat renal cell cancer. The following types of surgery may be used: A person can live with part of 1 working kidney, but if both kidneys are removed or not working, the person will need dialysis (a procedure to clean the blood using a machine outside of the body) or a kidney transplant (replacement with a healthy donated kidney). A kidney transplant may be done when the disease is in the kidney only and a donated kidney can be found. If the patient has to wait for a donated kidney, other treatment is given as needed. When surgery to remove the cancer is not possible, a treatment called arterial embolization may be used to shrink the tumor. A small incision is made and a catheter (thin tube) is inserted into the main blood vessel that flows to the kidney. Small pieces of a special gelatin sponge are injected through the catheter into the blood vessel. The sponges block the blood flow to the kidney and prevent the cancer cells from getting oxygen and other substances they need to grow. After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. External radiation therapy is used to treat renal cell cancer, and may also be used as palliative therapy to relieve symptoms and improve quality of life. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). For more information, see Drugs Approved for Kidney (Renal Cell) Cancer. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. The following types of immunotherapy are being used in the treatment of renal cell cancer: There are two types of immune checkpoint inhibitor therapy: For more information, see Drugs Approved for Kidney (Renal Cell) Cancer. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapy with antiangiogenic agents are used to treat advanced renal cell cancer. Antiangiogenic agents keep blood vessels from forming in a tumor, causing the tumor to starve and stop growing or to shrink. Monoclonal antibodies and kinase inhibitors are two types of antiangiogenic agents used to treat renal cell cancer. Vascular endothelial growth factor (VEGF) inhibitors and mTOR inhibitors are kinase inhibitors used to treat renal cell cancer. For more information, see Drugs Approved for Kidney (Renal Cell) Cancer. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage I renal cell cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage II renal cell cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage III renal cell cancer may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. First-line therapy may include the following: Second-line therapy may include the following: Third-line and fourth-line therapies may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about renal cell cancer, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of renal cancer. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Renal Cell Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/kidney/patient/kidney-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389448] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Renal Cell Cancer Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

what research (or clinical trials) is being done for Renal Cell Cancer ?

New types of treatment are being tested in clinical trials. Information about clinical trials is available from the NCI website. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials.

Lymphoma is a disease in which malignant (cancer) cells form in the lymph system. The lymph system is part of the immune system and is made up of the lymph, lymph vessels, lymph nodes, spleen, thymus, tonsils, and bone marrow. Lymphocytes (carried in the lymph) travel in and out of the central nervous system (CNS). It is thought that some of these lymphocytes become malignant and cause lymphoma to form in the CNS. Primary CNS lymphoma can start in the brain, spinal cord, or meninges (the layers that form the outer covering of the brain). Because the eye is so close to the brain, primary CNS lymphoma can also start in the eye (called ocular lymphoma).EnlargeAnatomy of the lymph system, showing the lymph vessels and lymph organs including lymph nodes, tonsils, thymus, spleen, and bone marrow. Lymph (clear fluid) and lymphocytes travel through the lymph vessels and into the lymph nodes where the lymphocytes destroy harmful substances. The lymph enters the blood through a large vein near the heart. Anything that increases your chance of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Primary CNS lymphoma may occur in patients who have acquired immunodeficiency syndrome (AIDS) or other disorders of the immune system or who have had an organ transplant. For more information about lymphoma in patients with AIDS, see the PDQ summary on AIDS-Related Lymphoma Treatment. These and other signs and symptoms may be caused by primary CNS lymphoma or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: The following tests may be done on the samples of tissue that are removed: The prognosis depends on the following: Treatment options depend on the following: Treatment of primary CNS lymphoma works best when the tumor has not spread outside the cerebrum (the largest part of the brain) and the patient is younger than 60 years, able to carry out most daily activities, and does not have AIDS or other diseases that weaken the immune system. Primary CNS lymphoma usually does not spread beyond the central nervous system or the eye. The process used to find out if cancer has spread is called staging. There is no standard system for staging primary CNS lymphoma. The following tests and procedures may be used to help plan treatment: Primary CNS lymphoma often recurs in the brain, spinal cord, or the eye. Different types of treatment are available for patients with primary CNS lymphoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is not used to treat primary CNS lymphoma. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Because primary CNS lymphoma spreads throughout the brain, external radiation therapy is given to the whole brain. This is called whole-brain radiation therapy. High-dose radiation therapy to the brain can damage healthy tissue and cause disorders that can affect thinking, learning, problem solving, reading, writing, speech, and memory. Clinical trials have tested the use of chemotherapy alone or before radiation therapy to reduce the damage to healthy brain tissue that occurs with the use of radiation therapy. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on where the tumor is in the CNS or eye. Primary CNS lymphoma may be treated with systemic chemotherapy, intrathecal chemotherapy, and/or intraventricular chemotherapy, in which anticancer drugs are placed into the ventricles (fluid-filled cavities) of the brain. If primary CNS lymphoma is found in the eye, anticancer drugs are injected directly into the vitreous humor (jelly-like substance) inside the eye. Steroids are hormones made naturally in the body. They can also be made in a laboratory and used as drugs. Glucocorticoids are steroid drugs that have an anticancer effect in lymphomas. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed primary central nervous system (CNS) lymphoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed primary intraocular lymphoma may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent primary central nervous system (CNS) lymphoma may include the following: For more information from the National Cancer Institute about primary central nervous system (CNS) lymphoma, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of primary CNS lymphoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Primary CNS Lymphoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/lymphoma/patient/primary-cns-lymphoma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389274] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Primary CNS Lymphoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is (are) Primary CNS Lymphoma ?

Key Points - Primary central nervous system (CNS) lymphoma is a disease in which malignant (cancer) cells form in the lymph tissue of the brain and/or spinal cord. - Having a weakened immune system may increase the risk of developing primary CNS lymphoma. - Tests that examine the eyes, brain, and spinal cord are used to detect (find) and diagnose primary CNS lymphoma. - Certain factors affect prognosis (chance of recovery) and treatment options. Primary central nervous system (CNS) lymphoma is a disease in which malignant (cancer) cells form in the lymph tissue of the brain and/or spinal cord. Lymphoma is a disease in which malignant (cancer) cells form in the lymph system. The lymph system is part of the immune system and is made up of the lymph, lymph vessels, lymph nodes, spleen, thymus, tonsils, and bone marrow. Lymphocytes (carried in the lymph) travel in and out of the central nervous system (CNS). It is thought that some of these lymphocytes become malignant and cause lymphoma to form in the CNS. Primary CNS lymphoma can start in the brain, spinal cord, or meninges (the layers that form the outer covering of the brain). Because the eye is so close to the brain, primary CNS lymphoma can also start in the eye (called ocular lymphoma).

Lymphoma is a disease in which malignant (cancer) cells form in the lymph system. The lymph system is part of the immune system and is made up of the lymph, lymph vessels, lymph nodes, spleen, thymus, tonsils, and bone marrow. Lymphocytes (carried in the lymph) travel in and out of the central nervous system (CNS). It is thought that some of these lymphocytes become malignant and cause lymphoma to form in the CNS. Primary CNS lymphoma can start in the brain, spinal cord, or meninges (the layers that form the outer covering of the brain). Because the eye is so close to the brain, primary CNS lymphoma can also start in the eye (called ocular lymphoma).EnlargeAnatomy of the lymph system, showing the lymph vessels and lymph organs including lymph nodes, tonsils, thymus, spleen, and bone marrow. Lymph (clear fluid) and lymphocytes travel through the lymph vessels and into the lymph nodes where the lymphocytes destroy harmful substances. The lymph enters the blood through a large vein near the heart. Anything that increases your chance of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Primary CNS lymphoma may occur in patients who have acquired immunodeficiency syndrome (AIDS) or other disorders of the immune system or who have had an organ transplant. For more information about lymphoma in patients with AIDS, see the PDQ summary on AIDS-Related Lymphoma Treatment. These and other signs and symptoms may be caused by primary CNS lymphoma or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: The following tests may be done on the samples of tissue that are removed: The prognosis depends on the following: Treatment options depend on the following: Treatment of primary CNS lymphoma works best when the tumor has not spread outside the cerebrum (the largest part of the brain) and the patient is younger than 60 years, able to carry out most daily activities, and does not have AIDS or other diseases that weaken the immune system. Primary CNS lymphoma usually does not spread beyond the central nervous system or the eye. The process used to find out if cancer has spread is called staging. There is no standard system for staging primary CNS lymphoma. The following tests and procedures may be used to help plan treatment: Primary CNS lymphoma often recurs in the brain, spinal cord, or the eye. Different types of treatment are available for patients with primary CNS lymphoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is not used to treat primary CNS lymphoma. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Because primary CNS lymphoma spreads throughout the brain, external radiation therapy is given to the whole brain. This is called whole-brain radiation therapy. High-dose radiation therapy to the brain can damage healthy tissue and cause disorders that can affect thinking, learning, problem solving, reading, writing, speech, and memory. Clinical trials have tested the use of chemotherapy alone or before radiation therapy to reduce the damage to healthy brain tissue that occurs with the use of radiation therapy. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on where the tumor is in the CNS or eye. Primary CNS lymphoma may be treated with systemic chemotherapy, intrathecal chemotherapy, and/or intraventricular chemotherapy, in which anticancer drugs are placed into the ventricles (fluid-filled cavities) of the brain. If primary CNS lymphoma is found in the eye, anticancer drugs are injected directly into the vitreous humor (jelly-like substance) inside the eye. Steroids are hormones made naturally in the body. They can also be made in a laboratory and used as drugs. Glucocorticoids are steroid drugs that have an anticancer effect in lymphomas. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed primary central nervous system (CNS) lymphoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed primary intraocular lymphoma may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent primary central nervous system (CNS) lymphoma may include the following: For more information from the National Cancer Institute about primary central nervous system (CNS) lymphoma, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of primary CNS lymphoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Primary CNS Lymphoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/lymphoma/patient/primary-cns-lymphoma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389274] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Primary CNS Lymphoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

Who is at risk for Primary CNS Lymphoma? ?

Having a weakened immune system may increase the risk of developing primary CNS lymphoma. Anything that increases your chance of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesnt mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Primary CNS lymphoma may occur in patients who have acquired immunodeficiency syndrome (AIDS) or other disorders of the immune system or who have had a kidney transplant. For more information about lymphoma in patients with AIDS, see the PDQ summary on AIDS-Related Lymphoma Treatment.

Lymphoma is a disease in which malignant (cancer) cells form in the lymph system. The lymph system is part of the immune system and is made up of the lymph, lymph vessels, lymph nodes, spleen, thymus, tonsils, and bone marrow. Lymphocytes (carried in the lymph) travel in and out of the central nervous system (CNS). It is thought that some of these lymphocytes become malignant and cause lymphoma to form in the CNS. Primary CNS lymphoma can start in the brain, spinal cord, or meninges (the layers that form the outer covering of the brain). Because the eye is so close to the brain, primary CNS lymphoma can also start in the eye (called ocular lymphoma).EnlargeAnatomy of the lymph system, showing the lymph vessels and lymph organs including lymph nodes, tonsils, thymus, spleen, and bone marrow. Lymph (clear fluid) and lymphocytes travel through the lymph vessels and into the lymph nodes where the lymphocytes destroy harmful substances. The lymph enters the blood through a large vein near the heart. Anything that increases your chance of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Primary CNS lymphoma may occur in patients who have acquired immunodeficiency syndrome (AIDS) or other disorders of the immune system or who have had an organ transplant. For more information about lymphoma in patients with AIDS, see the PDQ summary on AIDS-Related Lymphoma Treatment. These and other signs and symptoms may be caused by primary CNS lymphoma or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: The following tests may be done on the samples of tissue that are removed: The prognosis depends on the following: Treatment options depend on the following: Treatment of primary CNS lymphoma works best when the tumor has not spread outside the cerebrum (the largest part of the brain) and the patient is younger than 60 years, able to carry out most daily activities, and does not have AIDS or other diseases that weaken the immune system. Primary CNS lymphoma usually does not spread beyond the central nervous system or the eye. The process used to find out if cancer has spread is called staging. There is no standard system for staging primary CNS lymphoma. The following tests and procedures may be used to help plan treatment: Primary CNS lymphoma often recurs in the brain, spinal cord, or the eye. Different types of treatment are available for patients with primary CNS lymphoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is not used to treat primary CNS lymphoma. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Because primary CNS lymphoma spreads throughout the brain, external radiation therapy is given to the whole brain. This is called whole-brain radiation therapy. High-dose radiation therapy to the brain can damage healthy tissue and cause disorders that can affect thinking, learning, problem solving, reading, writing, speech, and memory. Clinical trials have tested the use of chemotherapy alone or before radiation therapy to reduce the damage to healthy brain tissue that occurs with the use of radiation therapy. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on where the tumor is in the CNS or eye. Primary CNS lymphoma may be treated with systemic chemotherapy, intrathecal chemotherapy, and/or intraventricular chemotherapy, in which anticancer drugs are placed into the ventricles (fluid-filled cavities) of the brain. If primary CNS lymphoma is found in the eye, anticancer drugs are injected directly into the vitreous humor (jelly-like substance) inside the eye. Steroids are hormones made naturally in the body. They can also be made in a laboratory and used as drugs. Glucocorticoids are steroid drugs that have an anticancer effect in lymphomas. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed primary central nervous system (CNS) lymphoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed primary intraocular lymphoma may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent primary central nervous system (CNS) lymphoma may include the following: For more information from the National Cancer Institute about primary central nervous system (CNS) lymphoma, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of primary CNS lymphoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Primary CNS Lymphoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/lymphoma/patient/primary-cns-lymphoma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389274] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Primary CNS Lymphoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

How to diagnose Primary CNS Lymphoma ?

Tests that examine the eyes, brain, and spinal cord are used to detect (find) and diagnose primary CNS lymphoma. The following tests and procedures may be used: - Physical exam and history : An exam of the body to check general signs of health, including checking for signs of disease, such as lumps or anything else that seems unusual. A history of the patients health habits and past illnesses and treatments will also be taken. - Neurological exam : A series of questions and tests to check the brain, spinal cord, and nerve function. The exam checks a persons mental status, coordination, ability to walk normally, and how well the muscles, senses, and reflexes work. This may also be called a neuro exam or a neurologic exam. - Slit-lamp eye exam : An exam that uses a special microscope with a bright, narrow slit of light to check the outside and inside of the eye. - MRI (magnetic resonance imaging): A procedure that uses a magnet, radio waves, and a computer to make a series of detailed pictures of areas inside the brain and spinal cord. A substance called gadolinium is injected into the patient through a vein. The gadolinium collects around the cancer cells so they show up brighter in the picture. This procedure is also called nuclear magnetic resonance imaging (NMRI). - PET scan (positron emission tomography scan): A procedure to find malignant tumor cells in the body. A small amount of radioactive glucose (sugar) is injected into a vein. The PET scanner rotates around the body and makes a picture of where glucose is being used in the body. Malignant tumor cells show up brighter in the picture because they are more active and take up more glucose than normal cells do. - Lumbar puncture : A procedure used to collect cerebrospinal fluid (CSF) from the spinal column. This is done by placing a needle between two bones in the spine and into the CSF around the spinal cord and removing a sample of the fluid. The sample of CSF is checked under a microscope for signs of tumor cells. The sample may also be checked for the amounts of protein and glucose. A higher than normal amount of protein or lower than normal amount of glucose may be a sign of a tumor. This procedure is also called an LP or spinal tap. - Stereotactic biopsy : A biopsy procedure that uses a computer and a 3-dimensional (3-D) scanning device to find a tumor site and guide the removal of tissue so it can be viewed under a microscope to check for signs of cancer. The following tests may be done on the samples of tissue that are removed: - Flow cytometry : A laboratory test that measures the number of cells in a sample, the percentage of live cells in a sample, and certain characteristics of cells, such as size, shape, and the presence of tumor markers on the cell surface. The cells are stained with a light-sensitive dye, placed in a fluid, and passed in a stream before a laser or other type of light. The measurements are based on how the light-sensitive dye reacts to the light. - Immunohistochemistry : A test that uses antibodies to check for certain antigens in a sample of tissue. The antibody is usually linked to a radioactive substance or a dye that causes the tissue to light up under a microscope. This type of test may be used to tell the difference between different types of cancer. - Cytogenetic analysis: A laboratory test in which cells in a sample of tissue are viewed under a microscope to look for certain changes in the chromosomes. Other tests, such as fluorescence in situ hybridization (FISH), may also be done to look for certain changes in the chromosomes. - Complete blood count (CBC) with differential : A procedure in which a sample of blood is drawn and checked for the following: - The number of red blood cells and platelets. - The number and type of white blood cells. - The amount of hemoglobin (the protein that carries oxygen) in the red blood cells. - The portion of the blood sample made up of red blood cells. - Blood chemistry studies : A procedure in which a blood sample is checked to measure the amounts of certain substances released into the blood by organs and tissues in the body. An unusual (higher or lower than normal) amount of a substance can be a sign of disease.

Lymphoma is a disease in which malignant (cancer) cells form in the lymph system. The lymph system is part of the immune system and is made up of the lymph, lymph vessels, lymph nodes, spleen, thymus, tonsils, and bone marrow. Lymphocytes (carried in the lymph) travel in and out of the central nervous system (CNS). It is thought that some of these lymphocytes become malignant and cause lymphoma to form in the CNS. Primary CNS lymphoma can start in the brain, spinal cord, or meninges (the layers that form the outer covering of the brain). Because the eye is so close to the brain, primary CNS lymphoma can also start in the eye (called ocular lymphoma).EnlargeAnatomy of the lymph system, showing the lymph vessels and lymph organs including lymph nodes, tonsils, thymus, spleen, and bone marrow. Lymph (clear fluid) and lymphocytes travel through the lymph vessels and into the lymph nodes where the lymphocytes destroy harmful substances. The lymph enters the blood through a large vein near the heart. Anything that increases your chance of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Primary CNS lymphoma may occur in patients who have acquired immunodeficiency syndrome (AIDS) or other disorders of the immune system or who have had an organ transplant. For more information about lymphoma in patients with AIDS, see the PDQ summary on AIDS-Related Lymphoma Treatment. These and other signs and symptoms may be caused by primary CNS lymphoma or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: The following tests may be done on the samples of tissue that are removed: The prognosis depends on the following: Treatment options depend on the following: Treatment of primary CNS lymphoma works best when the tumor has not spread outside the cerebrum (the largest part of the brain) and the patient is younger than 60 years, able to carry out most daily activities, and does not have AIDS or other diseases that weaken the immune system. Primary CNS lymphoma usually does not spread beyond the central nervous system or the eye. The process used to find out if cancer has spread is called staging. There is no standard system for staging primary CNS lymphoma. The following tests and procedures may be used to help plan treatment: Primary CNS lymphoma often recurs in the brain, spinal cord, or the eye. Different types of treatment are available for patients with primary CNS lymphoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is not used to treat primary CNS lymphoma. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Because primary CNS lymphoma spreads throughout the brain, external radiation therapy is given to the whole brain. This is called whole-brain radiation therapy. High-dose radiation therapy to the brain can damage healthy tissue and cause disorders that can affect thinking, learning, problem solving, reading, writing, speech, and memory. Clinical trials have tested the use of chemotherapy alone or before radiation therapy to reduce the damage to healthy brain tissue that occurs with the use of radiation therapy. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on where the tumor is in the CNS or eye. Primary CNS lymphoma may be treated with systemic chemotherapy, intrathecal chemotherapy, and/or intraventricular chemotherapy, in which anticancer drugs are placed into the ventricles (fluid-filled cavities) of the brain. If primary CNS lymphoma is found in the eye, anticancer drugs are injected directly into the vitreous humor (jelly-like substance) inside the eye. Steroids are hormones made naturally in the body. They can also be made in a laboratory and used as drugs. Glucocorticoids are steroid drugs that have an anticancer effect in lymphomas. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed primary central nervous system (CNS) lymphoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed primary intraocular lymphoma may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent primary central nervous system (CNS) lymphoma may include the following: For more information from the National Cancer Institute about primary central nervous system (CNS) lymphoma, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of primary CNS lymphoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Primary CNS Lymphoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/lymphoma/patient/primary-cns-lymphoma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389274] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Primary CNS Lymphoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is the outlook for Primary CNS Lymphoma ?

Certain factors affect prognosis (chance of recovery) and treatment options. The prognosis (chance of recovery) depends on the following: - The patient&apos;s age and general health. - The level of certain substances in the blood and cerebrospinal fluid (CSF). - Where the tumor is in the central nervous system, eye, or both. - Whether the patient has AIDS. Treatment options depend on the following: - The stage of the cancer. - Where the tumor is in the central nervous system. - The patient&apos;s age and general health. - Whether the cancer has just been diagnosed or has recurred (come back). Treatment of primary CNS lymphoma works best when the tumor has not spread outside the cerebrum (the largest part of the brain) and the patient is younger than 60 years, able to carry out most daily activities, and does not have AIDS or other diseases that weaken the immune system.

Lymphoma is a disease in which malignant (cancer) cells form in the lymph system. The lymph system is part of the immune system and is made up of the lymph, lymph vessels, lymph nodes, spleen, thymus, tonsils, and bone marrow. Lymphocytes (carried in the lymph) travel in and out of the central nervous system (CNS). It is thought that some of these lymphocytes become malignant and cause lymphoma to form in the CNS. Primary CNS lymphoma can start in the brain, spinal cord, or meninges (the layers that form the outer covering of the brain). Because the eye is so close to the brain, primary CNS lymphoma can also start in the eye (called ocular lymphoma).EnlargeAnatomy of the lymph system, showing the lymph vessels and lymph organs including lymph nodes, tonsils, thymus, spleen, and bone marrow. Lymph (clear fluid) and lymphocytes travel through the lymph vessels and into the lymph nodes where the lymphocytes destroy harmful substances. The lymph enters the blood through a large vein near the heart. Anything that increases your chance of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Primary CNS lymphoma may occur in patients who have acquired immunodeficiency syndrome (AIDS) or other disorders of the immune system or who have had an organ transplant. For more information about lymphoma in patients with AIDS, see the PDQ summary on AIDS-Related Lymphoma Treatment. These and other signs and symptoms may be caused by primary CNS lymphoma or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: The following tests may be done on the samples of tissue that are removed: The prognosis depends on the following: Treatment options depend on the following: Treatment of primary CNS lymphoma works best when the tumor has not spread outside the cerebrum (the largest part of the brain) and the patient is younger than 60 years, able to carry out most daily activities, and does not have AIDS or other diseases that weaken the immune system. Primary CNS lymphoma usually does not spread beyond the central nervous system or the eye. The process used to find out if cancer has spread is called staging. There is no standard system for staging primary CNS lymphoma. The following tests and procedures may be used to help plan treatment: Primary CNS lymphoma often recurs in the brain, spinal cord, or the eye. Different types of treatment are available for patients with primary CNS lymphoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is not used to treat primary CNS lymphoma. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Because primary CNS lymphoma spreads throughout the brain, external radiation therapy is given to the whole brain. This is called whole-brain radiation therapy. High-dose radiation therapy to the brain can damage healthy tissue and cause disorders that can affect thinking, learning, problem solving, reading, writing, speech, and memory. Clinical trials have tested the use of chemotherapy alone or before radiation therapy to reduce the damage to healthy brain tissue that occurs with the use of radiation therapy. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on where the tumor is in the CNS or eye. Primary CNS lymphoma may be treated with systemic chemotherapy, intrathecal chemotherapy, and/or intraventricular chemotherapy, in which anticancer drugs are placed into the ventricles (fluid-filled cavities) of the brain. If primary CNS lymphoma is found in the eye, anticancer drugs are injected directly into the vitreous humor (jelly-like substance) inside the eye. Steroids are hormones made naturally in the body. They can also be made in a laboratory and used as drugs. Glucocorticoids are steroid drugs that have an anticancer effect in lymphomas. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed primary central nervous system (CNS) lymphoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed primary intraocular lymphoma may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent primary central nervous system (CNS) lymphoma may include the following: For more information from the National Cancer Institute about primary central nervous system (CNS) lymphoma, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of primary CNS lymphoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Primary CNS Lymphoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/lymphoma/patient/primary-cns-lymphoma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389274] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Primary CNS Lymphoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

what research (or clinical trials) is being done for Primary CNS Lymphoma ?

New types of treatment are being tested in clinical trials. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. High-dose chemotherapy with stem cell transplant High-dose chemotherapy with stem cell transplant is a method of giving high doses of chemotherapy and replacing blood -forming cells destroyed by the cancer treatment. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the chemotherapy is completed, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body&apos;s blood cells. Targeted therapy Targeted therapy is a type of treatment that uses drugs or other substances to attack cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Monoclonal antibody therapy is one type of targeted therapy being studied in the treatment of primary CNS lymphoma. Monoclonal antibody therapy is a cancer treatment that uses antibodies made in the laboratory from a single type of immune system cell. These antibodies can identify substances on cancer cells or normal substances that may help cancer cells grow. The antibodies attach to the substances and kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. Rituximab is a type of monoclonal antibody used to treat newly diagnosed primary CNS lymphoma in patients who do not have AIDS. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials.

Lymphoma is a disease in which malignant (cancer) cells form in the lymph system. The lymph system is part of the immune system and is made up of the lymph, lymph vessels, lymph nodes, spleen, thymus, tonsils, and bone marrow. Lymphocytes (carried in the lymph) travel in and out of the central nervous system (CNS). It is thought that some of these lymphocytes become malignant and cause lymphoma to form in the CNS. Primary CNS lymphoma can start in the brain, spinal cord, or meninges (the layers that form the outer covering of the brain). Because the eye is so close to the brain, primary CNS lymphoma can also start in the eye (called ocular lymphoma).EnlargeAnatomy of the lymph system, showing the lymph vessels and lymph organs including lymph nodes, tonsils, thymus, spleen, and bone marrow. Lymph (clear fluid) and lymphocytes travel through the lymph vessels and into the lymph nodes where the lymphocytes destroy harmful substances. The lymph enters the blood through a large vein near the heart. Anything that increases your chance of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Primary CNS lymphoma may occur in patients who have acquired immunodeficiency syndrome (AIDS) or other disorders of the immune system or who have had an organ transplant. For more information about lymphoma in patients with AIDS, see the PDQ summary on AIDS-Related Lymphoma Treatment. These and other signs and symptoms may be caused by primary CNS lymphoma or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: The following tests may be done on the samples of tissue that are removed: The prognosis depends on the following: Treatment options depend on the following: Treatment of primary CNS lymphoma works best when the tumor has not spread outside the cerebrum (the largest part of the brain) and the patient is younger than 60 years, able to carry out most daily activities, and does not have AIDS or other diseases that weaken the immune system. Primary CNS lymphoma usually does not spread beyond the central nervous system or the eye. The process used to find out if cancer has spread is called staging. There is no standard system for staging primary CNS lymphoma. The following tests and procedures may be used to help plan treatment: Primary CNS lymphoma often recurs in the brain, spinal cord, or the eye. Different types of treatment are available for patients with primary CNS lymphoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is not used to treat primary CNS lymphoma. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Because primary CNS lymphoma spreads throughout the brain, external radiation therapy is given to the whole brain. This is called whole-brain radiation therapy. High-dose radiation therapy to the brain can damage healthy tissue and cause disorders that can affect thinking, learning, problem solving, reading, writing, speech, and memory. Clinical trials have tested the use of chemotherapy alone or before radiation therapy to reduce the damage to healthy brain tissue that occurs with the use of radiation therapy. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on where the tumor is in the CNS or eye. Primary CNS lymphoma may be treated with systemic chemotherapy, intrathecal chemotherapy, and/or intraventricular chemotherapy, in which anticancer drugs are placed into the ventricles (fluid-filled cavities) of the brain. If primary CNS lymphoma is found in the eye, anticancer drugs are injected directly into the vitreous humor (jelly-like substance) inside the eye. Steroids are hormones made naturally in the body. They can also be made in a laboratory and used as drugs. Glucocorticoids are steroid drugs that have an anticancer effect in lymphomas. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed primary central nervous system (CNS) lymphoma may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed primary intraocular lymphoma may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent primary central nervous system (CNS) lymphoma may include the following: For more information from the National Cancer Institute about primary central nervous system (CNS) lymphoma, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of primary CNS lymphoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Primary CNS Lymphoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/lymphoma/patient/primary-cns-lymphoma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389274] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Primary CNS Lymphoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the treatments for Primary CNS Lymphoma ?

Key Points - There are different types of treatment for patients with primary CNS lymphoma. - Three standard treatments are used: - Radiation therapy - Chemotherapy - Steroid therapy - New types of treatment are being tested in clinical trials. - High-dose chemotherapy with stem cell transplant - Targeted therapy - Patients may want to think about taking part in a clinical trial. - Patients can enter clinical trials before, during, or after starting their cancer treatment. - Follow-up tests may be needed. There are different types of treatment for patients with primary CNS lymphoma. Different types of treatment are available for patients with primary central nervous system (CNS) lymphoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is not used to treat primary CNS lymphoma. Three standard treatments are used: Radiation therapy Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: - External radiation therapy uses a machine outside the body to send radiation toward the cancer. Because primary CNS lymphoma spreads throughout the brain, external radiation therapy is given to the whole brain. This is called whole brain radiation therapy. - Internal radiation therapy uses a radioactive substance sealed in needles, seeds, wires, or catheters that are placed directly into or near the cancer. The way the radiation therapy is given depends on whether the patient has primary CNS lymphoma and AIDS. External radiation therapy is used to treat primary CNS lymphoma. High-dose radiation therapy to the brain can damage healthy tissue and cause disorders that can affect thinking, learning, problem solving, speech, reading, writing, and memory. Clinical trials have tested the use of chemotherapy alone or before radiation therapy to reduce the damage to healthy brain tissue that occurs with the use of radiation therapy. Chemotherapy Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on where the tumor is in the CNS or eye. Primary CNS lymphoma may be treated with systemic chemotherapy, intrathecal chemotherapy and/or intraventricular chemotherapy, in which anticancer drugs are placed into the ventricles (fluid -filled cavities) of the brain. If primary CNS lymphoma is found in the eye, anticancer drugs are injected directly into the vitreous humor (jelly-like substance) inside the eye. A network of blood vessels and tissue, called the blood-brain barrier, protects the brain from harmful substances. This barrier can also keep anticancer drugs from reaching the brain. In order to treat CNS lymphoma, certain drugs may be used to make openings between cells in the blood-brain barrier. This is called blood-brain barrier disruption. Anticancer drugs infused into the bloodstream may then reach the brain. Steroid therapy Steroids are hormones made naturally in the body. They can also be made in a laboratory and used as drugs. Glucocorticoids are steroid drugs that have an anticancer effect in lymphomas. New types of treatment are being tested in clinical trials. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. High-dose chemotherapy with stem cell transplant High-dose chemotherapy with stem cell transplant is a method of giving high doses of chemotherapy and replacing blood -forming cells destroyed by the cancer treatment. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the chemotherapy is completed, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body&apos;s blood cells. Targeted therapy Targeted therapy is a type of treatment that uses drugs or other substances to attack cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Monoclonal antibody therapy is one type of targeted therapy being studied in the treatment of primary CNS lymphoma. Monoclonal antibody therapy is a cancer treatment that uses antibodies made in the laboratory from a single type of immune system cell. These antibodies can identify substances on cancer cells or normal substances that may help cancer cells grow. The antibodies attach to the substances and kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. Rituximab is a type of monoclonal antibody used to treat newly diagnosed primary CNS lymphoma in patients who do not have AIDS. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials. Follow-up tests may be needed. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. Treatment Options for Primary CNS Lymphoma Primary CNS Lymphoma Not Related to AIDS Treatment of primary central nervous system (CNS) lymphoma in patients who do not have AIDS may include the following: - Whole brain radiation therapy. - Chemotherapy. - Chemotherapy followed by radiation therapy. - Chemotherapy and targeted therapy (rituximab) followed by high-dose chemotherapy and stem cell transplant. - A clinical trial of high-dose chemotherapy with stem cell transplant. - A clinical trial of high-dose chemotherapy and targeted therapy (rituximab), with or without stem cell transplant or whole brain radiation therapy. Primary CNS Lymphoma Related to AIDS Treatment of primary central nervous system (CNS) lymphoma in patients who do have AIDS may include the following: - Whole brain radiation therapy. - Chemotherapy followed by radiation therapy. Treatment of primary CNS lymphoma is different in patients with AIDS because the treatment side effects may be more severe. (See the PDQ summary on AIDS-Related Lymphoma Treatment for more information). Primary Intraocular Lymphoma Treatment of primary intraocular lymphoma may include the following: - Chemotherapy (intraocular or systemic). - Whole brain radiation therapy. Recurrent Primary CNS Lymphoma Treatment of recurrent primary central nervous system (CNS) lymphoma may include the following: - Chemotherapy. - Radiation therapy (if not received in earlier treatment). - A clinical trial of a new drug or treatment schedule.

This page describes the different types of treatment for bile duct cancer (cholangiocarcinoma). Which of these treatments a patient receives will depend on whether the cancer can be completely removed with surgery (resectable) or not (unresectable). To learn more, see treatment of resectable and treatment of unresectable bile duct cancer. The following types of surgery are used to treat bile duct cancer: After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. It is not yet known whether chemotherapy or radiation therapy given after surgery helps keep the cancer from coming back. The following types of palliative surgery may be done to relieve symptoms caused by a blocked bile duct and improve quality of life: Radiation therapy uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. These are the main ways radiation might be given to treat bile duct cancer: It is not known whether external radiation therapy helps in the treatment of resectable bile duct cancer. In unresectable, metastatic, or recurrent bile duct cancer, new ways to improve the effect of external radiation therapy on cancer cells are being studied: External and internal radiation therapy are used to treat bile duct cancer and may also be used as palliative therapy to relieve symptoms and improve quality of life. To learn more about radiation therapy and its side effects, see Radiation Therapy to Treat Cancer and Radiation Therapy Side Effects. Chemotherapy uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. There are two main types of chemotherapy used to treat bile duct cancer. Systemic chemotherapy is used to treat unresectable, metastatic, or recurrent bile duct cancer. The following chemotherapy drugs may be used: In unresectable, metastatic, or recurrent bile duct cancer, intra-arterial embolization is being studied. It is a procedure in which the blood supply to a tumor is blocked after anticancer drugs are given in blood vessels near the tumor. Sometimes, the anticancer drugs are attached to small beads that are injected into an artery that feeds the tumor. The beads block blood flow to the tumor as they release the drug. This allows a higher amount of drug to reach the tumor for a longer period of time, which may kill more cancer cells. It is not known whether systemic chemotherapy helps in the treatment of resectable bile duct cancer.  To learn about the different ways chemotherapy works against cancer, what to expect when receiving chemotherapy, and how to manage chemotherapy side effects, see Chemotherapy to Treat Cancer. In a liver transplant, the entire liver is removed and replaced with a healthy donated liver. A liver transplant may be done in patients with perihilar bile duct cancer. If the patient has to wait for a donated liver, other treatment is given as needed.  Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. The following targeted therapies are being studied in patients with bile duct cancer that is locally advanced and cannot be removed by surgery or has spread to other parts of the body: To learn more about how targeted therapy works against cancer, what to expect when having targeted therapy, and targeted therapy side effects, see Targeted Therapy to Treat Cancer. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. Immune checkpoint inhibitor therapy is a type of immunotherapy. The following immune checkpoint inhibitor may be used to treat bile duct cancer: To learn more about how immunotherapy works against cancer and immunotherapy side effects, see Immunotherapy to Treat Cancer and Immunotherapy Side Effects. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. For some patients, taking part in a clinical trial may be an option. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. To learn more about clinical trials, see Clinical Trials Information for Patients and Caregivers. If the cancer has not spread and is in a place where surgery can be safely done, the tumor and some of the tissue around it will be removed. This lowers the chance of the cancer coming back. Chemotherapy with or without radiation therapy may be given after surgery. Treatment of resectable intrahepatic bile duct cancer may include the following: Treatment of resectable perihilar bile duct cancer may include the following: Treatment of resectable distal bile duct cancer may include the following: Adjuvant therapy for resectable bile duct cancer may include the following:  Most people with bile duct cancer cannot have their cancer completely removed with surgery. This may be the case if the cancer has spread too far, the cancer is in a place that is too difficult to completely remove with surgery, or the patient is not healthy enough for surgery. Treatment of unresectable bile duct cancer (including metastatic or recurrent disease) may include the following: If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Bile Duct Cancer Treatment was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is (are) Bile Duct Cancer (Cholangiocarcinoma) ?

Key Points - Bile duct cancer is a rare disease in which malignant (cancer) cells form in the bile ducts. - Having colitis or certain liver diseases can increase the risk of bile duct cancer. - Signs of bile duct cancer include jaundice and pain in the abdomen. - Tests that examine the bile ducts and nearby organs are used to detect (find), diagnose, and stage bile duct cancer. - Different procedures may be used to obtain a sample of tissue and diagnose bile duct cancer. - Certain factors affect prognosis (chance of recovery) and treatment options. Bile duct cancer is a rare disease in which malignant (cancer) cells form in the bile ducts. A network of tubes, called ducts, connects the liver, gallbladder, and small intestine. This network begins in the liver where many small ducts collect bile (a fluid made by the liver to break down fats during digestion). The small ducts come together to form the right and left hepatic ducts, which lead out of the liver. The two ducts join outside the liver and form the common hepatic duct. The cystic duct connects the gallbladder to the common hepatic duct. Bile from the liver passes through the hepatic ducts, common hepatic duct, and cystic duct and is stored in the gallbladder. When food is being digested, bile stored in the gallbladder is released and passes through the cystic duct to the common bile duct and into the small intestine. Bile duct cancer is also called cholangiocarcinoma. There are two types of bile duct cancer: - Intrahepatic bile duct cancer : This type of cancer forms in the bile ducts inside the liver. Only a small number of bile duct cancers are intrahepatic. Intrahepatic bile duct cancers are also called intrahepatic cholangiocarcinomas. - Extrahepatic bile duct cancer : The extrahepatic bile duct is made up of the hilum region and the distal region. Cancer can form in either region: - Perihilar bile duct cancer: This type of cancer is found in the hilum region, the area where the right and left bile ducts exit the liver and join to form the common hepatic duct. Perihilar bile duct cancer is also called a Klatskin tumor or perihilar cholangiocarcinoma. - Distal extrahepatic bile duct cancer: This type of cancer is found in the distal region. The distal region is made up of the common bile duct which passes through the pancreas and ends in the small intestine. Distal extrahepatic bile duct cancer is also called extrahepatic cholangiocarcinoma.

This page describes the different types of treatment for bile duct cancer (cholangiocarcinoma). Which of these treatments a patient receives will depend on whether the cancer can be completely removed with surgery (resectable) or not (unresectable). To learn more, see treatment of resectable and treatment of unresectable bile duct cancer. The following types of surgery are used to treat bile duct cancer: After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. It is not yet known whether chemotherapy or radiation therapy given after surgery helps keep the cancer from coming back. The following types of palliative surgery may be done to relieve symptoms caused by a blocked bile duct and improve quality of life: Radiation therapy uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. These are the main ways radiation might be given to treat bile duct cancer: It is not known whether external radiation therapy helps in the treatment of resectable bile duct cancer. In unresectable, metastatic, or recurrent bile duct cancer, new ways to improve the effect of external radiation therapy on cancer cells are being studied: External and internal radiation therapy are used to treat bile duct cancer and may also be used as palliative therapy to relieve symptoms and improve quality of life. To learn more about radiation therapy and its side effects, see Radiation Therapy to Treat Cancer and Radiation Therapy Side Effects. Chemotherapy uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. There are two main types of chemotherapy used to treat bile duct cancer. Systemic chemotherapy is used to treat unresectable, metastatic, or recurrent bile duct cancer. The following chemotherapy drugs may be used: In unresectable, metastatic, or recurrent bile duct cancer, intra-arterial embolization is being studied. It is a procedure in which the blood supply to a tumor is blocked after anticancer drugs are given in blood vessels near the tumor. Sometimes, the anticancer drugs are attached to small beads that are injected into an artery that feeds the tumor. The beads block blood flow to the tumor as they release the drug. This allows a higher amount of drug to reach the tumor for a longer period of time, which may kill more cancer cells. It is not known whether systemic chemotherapy helps in the treatment of resectable bile duct cancer.  To learn about the different ways chemotherapy works against cancer, what to expect when receiving chemotherapy, and how to manage chemotherapy side effects, see Chemotherapy to Treat Cancer. In a liver transplant, the entire liver is removed and replaced with a healthy donated liver. A liver transplant may be done in patients with perihilar bile duct cancer. If the patient has to wait for a donated liver, other treatment is given as needed.  Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. The following targeted therapies are being studied in patients with bile duct cancer that is locally advanced and cannot be removed by surgery or has spread to other parts of the body: To learn more about how targeted therapy works against cancer, what to expect when having targeted therapy, and targeted therapy side effects, see Targeted Therapy to Treat Cancer. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. Immune checkpoint inhibitor therapy is a type of immunotherapy. The following immune checkpoint inhibitor may be used to treat bile duct cancer: To learn more about how immunotherapy works against cancer and immunotherapy side effects, see Immunotherapy to Treat Cancer and Immunotherapy Side Effects. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. For some patients, taking part in a clinical trial may be an option. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. To learn more about clinical trials, see Clinical Trials Information for Patients and Caregivers. If the cancer has not spread and is in a place where surgery can be safely done, the tumor and some of the tissue around it will be removed. This lowers the chance of the cancer coming back. Chemotherapy with or without radiation therapy may be given after surgery. Treatment of resectable intrahepatic bile duct cancer may include the following: Treatment of resectable perihilar bile duct cancer may include the following: Treatment of resectable distal bile duct cancer may include the following: Adjuvant therapy for resectable bile duct cancer may include the following:  Most people with bile duct cancer cannot have their cancer completely removed with surgery. This may be the case if the cancer has spread too far, the cancer is in a place that is too difficult to completely remove with surgery, or the patient is not healthy enough for surgery. Treatment of unresectable bile duct cancer (including metastatic or recurrent disease) may include the following: If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Bile Duct Cancer Treatment was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

Who is at risk for Bile Duct Cancer (Cholangiocarcinoma)? ?

Having colitis or certain liver diseases can increase the risk of bile duct cancer. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn&apos;t mean that you will not get cancer. People who think they may be at risk should discuss this with their doctor. Risk factors for bile duct cancer include the following conditions: - Primary sclerosing cholangitis (a progressive disease in which the bile ducts become blocked by inflammation and scarring). - Chronic ulcerative colitis. - Cysts in the bile ducts (cysts block the flow of bile and can cause swollen bile ducts, inflammation, and infection). - Infection with a Chinese liver fluke parasite.

This page describes the different types of treatment for bile duct cancer (cholangiocarcinoma). Which of these treatments a patient receives will depend on whether the cancer can be completely removed with surgery (resectable) or not (unresectable). To learn more, see treatment of resectable and treatment of unresectable bile duct cancer. The following types of surgery are used to treat bile duct cancer: After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. It is not yet known whether chemotherapy or radiation therapy given after surgery helps keep the cancer from coming back. The following types of palliative surgery may be done to relieve symptoms caused by a blocked bile duct and improve quality of life: Radiation therapy uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. These are the main ways radiation might be given to treat bile duct cancer: It is not known whether external radiation therapy helps in the treatment of resectable bile duct cancer. In unresectable, metastatic, or recurrent bile duct cancer, new ways to improve the effect of external radiation therapy on cancer cells are being studied: External and internal radiation therapy are used to treat bile duct cancer and may also be used as palliative therapy to relieve symptoms and improve quality of life. To learn more about radiation therapy and its side effects, see Radiation Therapy to Treat Cancer and Radiation Therapy Side Effects. Chemotherapy uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. There are two main types of chemotherapy used to treat bile duct cancer. Systemic chemotherapy is used to treat unresectable, metastatic, or recurrent bile duct cancer. The following chemotherapy drugs may be used: In unresectable, metastatic, or recurrent bile duct cancer, intra-arterial embolization is being studied. It is a procedure in which the blood supply to a tumor is blocked after anticancer drugs are given in blood vessels near the tumor. Sometimes, the anticancer drugs are attached to small beads that are injected into an artery that feeds the tumor. The beads block blood flow to the tumor as they release the drug. This allows a higher amount of drug to reach the tumor for a longer period of time, which may kill more cancer cells. It is not known whether systemic chemotherapy helps in the treatment of resectable bile duct cancer.  To learn about the different ways chemotherapy works against cancer, what to expect when receiving chemotherapy, and how to manage chemotherapy side effects, see Chemotherapy to Treat Cancer. In a liver transplant, the entire liver is removed and replaced with a healthy donated liver. A liver transplant may be done in patients with perihilar bile duct cancer. If the patient has to wait for a donated liver, other treatment is given as needed.  Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. The following targeted therapies are being studied in patients with bile duct cancer that is locally advanced and cannot be removed by surgery or has spread to other parts of the body: To learn more about how targeted therapy works against cancer, what to expect when having targeted therapy, and targeted therapy side effects, see Targeted Therapy to Treat Cancer. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. Immune checkpoint inhibitor therapy is a type of immunotherapy. The following immune checkpoint inhibitor may be used to treat bile duct cancer: To learn more about how immunotherapy works against cancer and immunotherapy side effects, see Immunotherapy to Treat Cancer and Immunotherapy Side Effects. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. For some patients, taking part in a clinical trial may be an option. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. To learn more about clinical trials, see Clinical Trials Information for Patients and Caregivers. If the cancer has not spread and is in a place where surgery can be safely done, the tumor and some of the tissue around it will be removed. This lowers the chance of the cancer coming back. Chemotherapy with or without radiation therapy may be given after surgery. Treatment of resectable intrahepatic bile duct cancer may include the following: Treatment of resectable perihilar bile duct cancer may include the following: Treatment of resectable distal bile duct cancer may include the following: Adjuvant therapy for resectable bile duct cancer may include the following:  Most people with bile duct cancer cannot have their cancer completely removed with surgery. This may be the case if the cancer has spread too far, the cancer is in a place that is too difficult to completely remove with surgery, or the patient is not healthy enough for surgery. Treatment of unresectable bile duct cancer (including metastatic or recurrent disease) may include the following: If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Bile Duct Cancer Treatment was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the symptoms of Bile Duct Cancer (Cholangiocarcinoma) ?

Signs of bile duct cancer include jaundice and pain in the abdomen. These and other signs and symptoms may be caused by bile duct cancer or by other conditions. Check with your doctor if you have any of the following: - Jaundice (yellowing of the skin or whites of the eyes). - Dark urine. - Clay colored stool. - Pain in the abdomen. - Fever. - Itchy skin. - Nausea and vomiting. - Weight loss for an unknown reason.

This page describes the different types of treatment for bile duct cancer (cholangiocarcinoma). Which of these treatments a patient receives will depend on whether the cancer can be completely removed with surgery (resectable) or not (unresectable). To learn more, see treatment of resectable and treatment of unresectable bile duct cancer. The following types of surgery are used to treat bile duct cancer: After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. It is not yet known whether chemotherapy or radiation therapy given after surgery helps keep the cancer from coming back. The following types of palliative surgery may be done to relieve symptoms caused by a blocked bile duct and improve quality of life: Radiation therapy uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. These are the main ways radiation might be given to treat bile duct cancer: It is not known whether external radiation therapy helps in the treatment of resectable bile duct cancer. In unresectable, metastatic, or recurrent bile duct cancer, new ways to improve the effect of external radiation therapy on cancer cells are being studied: External and internal radiation therapy are used to treat bile duct cancer and may also be used as palliative therapy to relieve symptoms and improve quality of life. To learn more about radiation therapy and its side effects, see Radiation Therapy to Treat Cancer and Radiation Therapy Side Effects. Chemotherapy uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. There are two main types of chemotherapy used to treat bile duct cancer. Systemic chemotherapy is used to treat unresectable, metastatic, or recurrent bile duct cancer. The following chemotherapy drugs may be used: In unresectable, metastatic, or recurrent bile duct cancer, intra-arterial embolization is being studied. It is a procedure in which the blood supply to a tumor is blocked after anticancer drugs are given in blood vessels near the tumor. Sometimes, the anticancer drugs are attached to small beads that are injected into an artery that feeds the tumor. The beads block blood flow to the tumor as they release the drug. This allows a higher amount of drug to reach the tumor for a longer period of time, which may kill more cancer cells. It is not known whether systemic chemotherapy helps in the treatment of resectable bile duct cancer.  To learn about the different ways chemotherapy works against cancer, what to expect when receiving chemotherapy, and how to manage chemotherapy side effects, see Chemotherapy to Treat Cancer. In a liver transplant, the entire liver is removed and replaced with a healthy donated liver. A liver transplant may be done in patients with perihilar bile duct cancer. If the patient has to wait for a donated liver, other treatment is given as needed.  Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. The following targeted therapies are being studied in patients with bile duct cancer that is locally advanced and cannot be removed by surgery or has spread to other parts of the body: To learn more about how targeted therapy works against cancer, what to expect when having targeted therapy, and targeted therapy side effects, see Targeted Therapy to Treat Cancer. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. Immune checkpoint inhibitor therapy is a type of immunotherapy. The following immune checkpoint inhibitor may be used to treat bile duct cancer: To learn more about how immunotherapy works against cancer and immunotherapy side effects, see Immunotherapy to Treat Cancer and Immunotherapy Side Effects. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. For some patients, taking part in a clinical trial may be an option. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. To learn more about clinical trials, see Clinical Trials Information for Patients and Caregivers. If the cancer has not spread and is in a place where surgery can be safely done, the tumor and some of the tissue around it will be removed. This lowers the chance of the cancer coming back. Chemotherapy with or without radiation therapy may be given after surgery. Treatment of resectable intrahepatic bile duct cancer may include the following: Treatment of resectable perihilar bile duct cancer may include the following: Treatment of resectable distal bile duct cancer may include the following: Adjuvant therapy for resectable bile duct cancer may include the following:  Most people with bile duct cancer cannot have their cancer completely removed with surgery. This may be the case if the cancer has spread too far, the cancer is in a place that is too difficult to completely remove with surgery, or the patient is not healthy enough for surgery. Treatment of unresectable bile duct cancer (including metastatic or recurrent disease) may include the following: If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Bile Duct Cancer Treatment was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

How to diagnose Bile Duct Cancer (Cholangiocarcinoma) ?

Tests that examine the bile ducts and nearby organs are used to detect (find), diagnose, and stage bile duct cancer. Procedures that make pictures of the bile ducts and the nearby area help diagnose bile duct cancer and show how far the cancer has spread. The process used to find out if cancer cells have spread within and around the bile ducts or to distant parts of the body is called staging. In order to plan treatment, it is important to know if the bile duct cancer can be removed by surgery. Tests and procedures to detect, diagnose, and stage bile duct cancer are usually done at the same time. The following tests and procedures may be used: - Physical exam and history : An exam of the body to check general signs of health, including checking for signs of disease, such as lumps or anything else that seems unusual. A history of the patients health habits and past illnesses and treatments will also be taken. - Liver function tests : A procedure in which a blood sample is checked to measure the amounts of bilirubin and alkaline phosphatase released into the blood by the liver. A higher than normal amount of these substances can be a sign of liver disease that may be caused by bile duct cancer. - Laboratory tests : Medical procedures that test samples of tissue, blood, urine, or other substances in the body. These tests help to diagnose disease, plan and check treatment, or monitor the disease over time. - Carcinoembryonic antigen (CEA) and CA 19-9 tumor marker test : A procedure in which a sample of blood, urine, or tissue is checked to measure the amounts of certain substances made by organs, tissues, or tumor cells in the body. Certain substances are linked to specific types of cancer when found in increased levels in the body. These are called tumor markers. Higher than normal levels of carcinoembryonic antigen (CEA) and CA 19-9 may mean there is bile duct cancer. - Ultrasound exam : A procedure in which high-energy sound waves (ultrasound) are bounced off internal tissues or organs, such as the abdomen, and make echoes. The echoes form a picture of body tissues called a sonogram. The picture can be printed to be looked at later. - CT scan (CAT scan): A procedure that makes a series of detailed pictures of areas inside the body, such as the abdomen, taken from different angles. The pictures are made by a computer linked to an x-ray machine. A dye may be injected into a vein or swallowed to help the organs or tissues show up more clearly. This procedure is also called computed tomography, computerized tomography, or computerized axial tomography. - MRI (magnetic resonance imaging): A procedure that uses a magnet, radio waves, and a computer to make a series of detailed pictures of areas inside the body. This procedure is also called nuclear magnetic resonance imaging (NMRI). - MRCP (magnetic resonance cholangiopancreatography): A procedure that uses a magnet, radio waves, and a computer to make a series of detailed pictures of areas inside the body such as the liver, bile ducts, gallbladder, pancreas, and pancreatic duct. Different procedures may be used to obtain a sample of tissue and diagnose bile duct cancer. Cells and tissues are removed during a biopsy so they can be viewed under a microscope by a pathologist to check for signs of cancer. Different procedures may be used to obtain the sample of cells and tissue. The type of procedure used depends on whether the patient is well enough to have surgery. Types of biopsy procedures include the following: - Laparoscopy : A surgical procedure to look at the organs inside the abdomen, such as the bile ducts and liver, to check for signs of cancer. Small incisions (cuts) are made in the wall of the abdomen and a laparoscope (a thin, lighted tube) is inserted into one of the incisions. Other instruments may be inserted through the same or other incisions to perform procedures such as taking tissue samples to be checked for signs of cancer. - Percutaneous transhepatic cholangiography (PTC): A procedure used to x-ray the liver and bile ducts. A thin needle is inserted through the skin below the ribs and into the liver. Dye is injected into the liver or bile ducts and an x-ray is taken. A sample of tissue is removed and checked for signs of cancer. If the bile duct is blocked, a thin, flexible tube called a stent may be left in the liver to drain bile into the small intestine or a collection bag outside the body. This procedure may be used when a patient cannot have surgery. - Endoscopic retrograde cholangiopancreatography (ERCP): A procedure used to x-ray the ducts (tubes) that carry bile from the liver to the gallbladder and from the gallbladder to the small intestine. Sometimes bile duct cancer causes these ducts to narrow and block or slow the flow of bile, causing jaundice. An endoscope is passed through the mouth and stomach and into the small intestine. Dye is injected through the endoscope (thin, tube-like instrument with a light and a lens for viewing) into the bile ducts and an x-ray is taken. A sample of tissue is removed and checked for signs of cancer. If the bile duct is blocked, a thin tube may be inserted into the duct to unblock it. This tube (or stent) may be left in place to keep the duct open. This procedure may be used when a patient cannot have surgery.

This page describes the different types of treatment for bile duct cancer (cholangiocarcinoma). Which of these treatments a patient receives will depend on whether the cancer can be completely removed with surgery (resectable) or not (unresectable). To learn more, see treatment of resectable and treatment of unresectable bile duct cancer. The following types of surgery are used to treat bile duct cancer: After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. It is not yet known whether chemotherapy or radiation therapy given after surgery helps keep the cancer from coming back. The following types of palliative surgery may be done to relieve symptoms caused by a blocked bile duct and improve quality of life: Radiation therapy uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. These are the main ways radiation might be given to treat bile duct cancer: It is not known whether external radiation therapy helps in the treatment of resectable bile duct cancer. In unresectable, metastatic, or recurrent bile duct cancer, new ways to improve the effect of external radiation therapy on cancer cells are being studied: External and internal radiation therapy are used to treat bile duct cancer and may also be used as palliative therapy to relieve symptoms and improve quality of life. To learn more about radiation therapy and its side effects, see Radiation Therapy to Treat Cancer and Radiation Therapy Side Effects. Chemotherapy uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. There are two main types of chemotherapy used to treat bile duct cancer. Systemic chemotherapy is used to treat unresectable, metastatic, or recurrent bile duct cancer. The following chemotherapy drugs may be used: In unresectable, metastatic, or recurrent bile duct cancer, intra-arterial embolization is being studied. It is a procedure in which the blood supply to a tumor is blocked after anticancer drugs are given in blood vessels near the tumor. Sometimes, the anticancer drugs are attached to small beads that are injected into an artery that feeds the tumor. The beads block blood flow to the tumor as they release the drug. This allows a higher amount of drug to reach the tumor for a longer period of time, which may kill more cancer cells. It is not known whether systemic chemotherapy helps in the treatment of resectable bile duct cancer.  To learn about the different ways chemotherapy works against cancer, what to expect when receiving chemotherapy, and how to manage chemotherapy side effects, see Chemotherapy to Treat Cancer. In a liver transplant, the entire liver is removed and replaced with a healthy donated liver. A liver transplant may be done in patients with perihilar bile duct cancer. If the patient has to wait for a donated liver, other treatment is given as needed.  Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. The following targeted therapies are being studied in patients with bile duct cancer that is locally advanced and cannot be removed by surgery or has spread to other parts of the body: To learn more about how targeted therapy works against cancer, what to expect when having targeted therapy, and targeted therapy side effects, see Targeted Therapy to Treat Cancer. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. Immune checkpoint inhibitor therapy is a type of immunotherapy. The following immune checkpoint inhibitor may be used to treat bile duct cancer: To learn more about how immunotherapy works against cancer and immunotherapy side effects, see Immunotherapy to Treat Cancer and Immunotherapy Side Effects. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. For some patients, taking part in a clinical trial may be an option. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. To learn more about clinical trials, see Clinical Trials Information for Patients and Caregivers. If the cancer has not spread and is in a place where surgery can be safely done, the tumor and some of the tissue around it will be removed. This lowers the chance of the cancer coming back. Chemotherapy with or without radiation therapy may be given after surgery. Treatment of resectable intrahepatic bile duct cancer may include the following: Treatment of resectable perihilar bile duct cancer may include the following: Treatment of resectable distal bile duct cancer may include the following: Adjuvant therapy for resectable bile duct cancer may include the following:  Most people with bile duct cancer cannot have their cancer completely removed with surgery. This may be the case if the cancer has spread too far, the cancer is in a place that is too difficult to completely remove with surgery, or the patient is not healthy enough for surgery. Treatment of unresectable bile duct cancer (including metastatic or recurrent disease) may include the following: If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Bile Duct Cancer Treatment was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is the outlook for Bile Duct Cancer (Cholangiocarcinoma) ?

Certain factors affect prognosis (chance of recovery) and treatment options. The prognosis (chance of recovery) and treatment options depend on the following: - Whether the cancer is in the upper or lower part of the bile duct system. - The stage of the cancer (whether it affects only the bile ducts or has spread to the liver, lymph nodes, or other places in the body). - Whether the cancer has spread to nearby nerves or veins. - Whether the cancer can be completely removed by surgery. - Whether the patient has other conditions, such as primary sclerosing cholangitis. - Whether the level of CA 19-9 is higher than normal. - Whether the cancer has just been diagnosed or has recurred (come back). Treatment options may also depend on the symptoms caused by the cancer. Bile duct cancer is usually found after it has spread and can rarely be completely removed by surgery. Palliative therapy may relieve symptoms and improve the patient&apos;s quality of life.

This page describes the different types of treatment for bile duct cancer (cholangiocarcinoma). Which of these treatments a patient receives will depend on whether the cancer can be completely removed with surgery (resectable) or not (unresectable). To learn more, see treatment of resectable and treatment of unresectable bile duct cancer. The following types of surgery are used to treat bile duct cancer: After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. It is not yet known whether chemotherapy or radiation therapy given after surgery helps keep the cancer from coming back. The following types of palliative surgery may be done to relieve symptoms caused by a blocked bile duct and improve quality of life: Radiation therapy uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. These are the main ways radiation might be given to treat bile duct cancer: It is not known whether external radiation therapy helps in the treatment of resectable bile duct cancer. In unresectable, metastatic, or recurrent bile duct cancer, new ways to improve the effect of external radiation therapy on cancer cells are being studied: External and internal radiation therapy are used to treat bile duct cancer and may also be used as palliative therapy to relieve symptoms and improve quality of life. To learn more about radiation therapy and its side effects, see Radiation Therapy to Treat Cancer and Radiation Therapy Side Effects. Chemotherapy uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. There are two main types of chemotherapy used to treat bile duct cancer. Systemic chemotherapy is used to treat unresectable, metastatic, or recurrent bile duct cancer. The following chemotherapy drugs may be used: In unresectable, metastatic, or recurrent bile duct cancer, intra-arterial embolization is being studied. It is a procedure in which the blood supply to a tumor is blocked after anticancer drugs are given in blood vessels near the tumor. Sometimes, the anticancer drugs are attached to small beads that are injected into an artery that feeds the tumor. The beads block blood flow to the tumor as they release the drug. This allows a higher amount of drug to reach the tumor for a longer period of time, which may kill more cancer cells. It is not known whether systemic chemotherapy helps in the treatment of resectable bile duct cancer.  To learn about the different ways chemotherapy works against cancer, what to expect when receiving chemotherapy, and how to manage chemotherapy side effects, see Chemotherapy to Treat Cancer. In a liver transplant, the entire liver is removed and replaced with a healthy donated liver. A liver transplant may be done in patients with perihilar bile duct cancer. If the patient has to wait for a donated liver, other treatment is given as needed.  Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. The following targeted therapies are being studied in patients with bile duct cancer that is locally advanced and cannot be removed by surgery or has spread to other parts of the body: To learn more about how targeted therapy works against cancer, what to expect when having targeted therapy, and targeted therapy side effects, see Targeted Therapy to Treat Cancer. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. Immune checkpoint inhibitor therapy is a type of immunotherapy. The following immune checkpoint inhibitor may be used to treat bile duct cancer: To learn more about how immunotherapy works against cancer and immunotherapy side effects, see Immunotherapy to Treat Cancer and Immunotherapy Side Effects. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. For some patients, taking part in a clinical trial may be an option. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. To learn more about clinical trials, see Clinical Trials Information for Patients and Caregivers. If the cancer has not spread and is in a place where surgery can be safely done, the tumor and some of the tissue around it will be removed. This lowers the chance of the cancer coming back. Chemotherapy with or without radiation therapy may be given after surgery. Treatment of resectable intrahepatic bile duct cancer may include the following: Treatment of resectable perihilar bile duct cancer may include the following: Treatment of resectable distal bile duct cancer may include the following: Adjuvant therapy for resectable bile duct cancer may include the following:  Most people with bile duct cancer cannot have their cancer completely removed with surgery. This may be the case if the cancer has spread too far, the cancer is in a place that is too difficult to completely remove with surgery, or the patient is not healthy enough for surgery. Treatment of unresectable bile duct cancer (including metastatic or recurrent disease) may include the following: If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Bile Duct Cancer Treatment was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

what research (or clinical trials) is being done for Bile Duct Cancer (Cholangiocarcinoma) ?

New types of treatment are being tested in clinical trials. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Liver transplant In a liver transplant, the entire liver is removed and replaced with a healthy donated liver. A liver transplant may be done in patients with perihilar bile duct cancer. If the patient has to wait for a donated liver, other treatment is given as needed. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials.

This page describes the different types of treatment for bile duct cancer (cholangiocarcinoma). Which of these treatments a patient receives will depend on whether the cancer can be completely removed with surgery (resectable) or not (unresectable). To learn more, see treatment of resectable and treatment of unresectable bile duct cancer. The following types of surgery are used to treat bile duct cancer: After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. It is not yet known whether chemotherapy or radiation therapy given after surgery helps keep the cancer from coming back. The following types of palliative surgery may be done to relieve symptoms caused by a blocked bile duct and improve quality of life: Radiation therapy uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. These are the main ways radiation might be given to treat bile duct cancer: It is not known whether external radiation therapy helps in the treatment of resectable bile duct cancer. In unresectable, metastatic, or recurrent bile duct cancer, new ways to improve the effect of external radiation therapy on cancer cells are being studied: External and internal radiation therapy are used to treat bile duct cancer and may also be used as palliative therapy to relieve symptoms and improve quality of life. To learn more about radiation therapy and its side effects, see Radiation Therapy to Treat Cancer and Radiation Therapy Side Effects. Chemotherapy uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. There are two main types of chemotherapy used to treat bile duct cancer. Systemic chemotherapy is used to treat unresectable, metastatic, or recurrent bile duct cancer. The following chemotherapy drugs may be used: In unresectable, metastatic, or recurrent bile duct cancer, intra-arterial embolization is being studied. It is a procedure in which the blood supply to a tumor is blocked after anticancer drugs are given in blood vessels near the tumor. Sometimes, the anticancer drugs are attached to small beads that are injected into an artery that feeds the tumor. The beads block blood flow to the tumor as they release the drug. This allows a higher amount of drug to reach the tumor for a longer period of time, which may kill more cancer cells. It is not known whether systemic chemotherapy helps in the treatment of resectable bile duct cancer.  To learn about the different ways chemotherapy works against cancer, what to expect when receiving chemotherapy, and how to manage chemotherapy side effects, see Chemotherapy to Treat Cancer. In a liver transplant, the entire liver is removed and replaced with a healthy donated liver. A liver transplant may be done in patients with perihilar bile duct cancer. If the patient has to wait for a donated liver, other treatment is given as needed.  Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. The following targeted therapies are being studied in patients with bile duct cancer that is locally advanced and cannot be removed by surgery or has spread to other parts of the body: To learn more about how targeted therapy works against cancer, what to expect when having targeted therapy, and targeted therapy side effects, see Targeted Therapy to Treat Cancer. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. Immune checkpoint inhibitor therapy is a type of immunotherapy. The following immune checkpoint inhibitor may be used to treat bile duct cancer: To learn more about how immunotherapy works against cancer and immunotherapy side effects, see Immunotherapy to Treat Cancer and Immunotherapy Side Effects. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. For some patients, taking part in a clinical trial may be an option. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. To learn more about clinical trials, see Clinical Trials Information for Patients and Caregivers. If the cancer has not spread and is in a place where surgery can be safely done, the tumor and some of the tissue around it will be removed. This lowers the chance of the cancer coming back. Chemotherapy with or without radiation therapy may be given after surgery. Treatment of resectable intrahepatic bile duct cancer may include the following: Treatment of resectable perihilar bile duct cancer may include the following: Treatment of resectable distal bile duct cancer may include the following: Adjuvant therapy for resectable bile duct cancer may include the following:  Most people with bile duct cancer cannot have their cancer completely removed with surgery. This may be the case if the cancer has spread too far, the cancer is in a place that is too difficult to completely remove with surgery, or the patient is not healthy enough for surgery. Treatment of unresectable bile duct cancer (including metastatic or recurrent disease) may include the following: If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Bile Duct Cancer Treatment was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the stages of Bile Duct Cancer (Cholangiocarcinoma) ?

Key Points - The results of diagnostic and staging tests are used to find out if cancer cells have spread. - There are three ways that cancer spreads in the body. - Cancer may spread from where it began to other parts of the body. - Stages are used to describe the different types of bile duct cancer. - Intrahepatic bile duct cancer - Perihilar bile duct cancer - Distal extrahepatic bile duct cancer - The following groups are used to plan treatment: - Resectable (localized) bile duct cancer - Unresectable, metastatic, or recurrent bile duct cancer The results of diagnostic and staging tests are used to find out if cancer cells have spread. The process used to find out if cancer has spread to other parts of the body is called staging. For bile duct cancer, the information gathered from tests and procedures is used to plan treatment, including whether the tumor can be removed by surgery. There are three ways that cancer spreads in the body. Cancer can spread through tissue, the lymph system, and the blood: - Tissue. The cancer spreads from where it began by growing into nearby areas. - Lymph system. The cancer spreads from where it began by getting into the lymph system. The cancer travels through the lymph vessels to other parts of the body. - Blood. The cancer spreads from where it began by getting into the blood. The cancer travels through the blood vessels to other parts of the body. Cancer may spread from where it began to other parts of the body. When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. - Lymph system. The cancer gets into the lymph system, travels through the lymph vessels, and forms a tumor (metastatic tumor) in another part of the body. - Blood. The cancer gets into the blood, travels through the blood vessels, and forms a tumor (metastatic tumor) in another part of the body. The metastatic tumor is the same type of cancer as the primary tumor. For example, if bile duct cancer spreads to the liver, the cancer cells in the liver are actually bile duct cancer cells. The disease is metastatic bile duct cancer, not liver cancer. Stages are used to describe the different types of bile duct cancer. Intrahepatic bile duct cancer - Stage 0: Abnormal cells are found in the innermost layer of tissue lining the intrahepatic bile duct. These abnormal cells may become cancer and spread into nearby normal tissue. Stage 0 is also called carcinoma in situ. - Stage I: There is one tumor that has spread into the intrahepatic bile duct and it has not spread into any blood vessels. - Stage II: There is one tumor that has spread through the wall of the bile duct and into a blood vessel, or there are multiple tumors that may have spread into a blood vessel. - Stage III: The tumor has spread through the tissue that lines the abdominal wall or has spread to organs or tissues near the liver such as the duodenum, colon, and stomach. - Stage IV: Stage IV is divided into stage IVA and stage IVB. - Stage IVA: The cancer has spread along the outside of the intrahepatic bile ducts or the cancer has spread to nearby lymph nodes. - Stage IVB: The cancer has spread to organs in other parts of the body. Perihilar bile duct cancer - Stage 0: Abnormal cells are found in the innermost layer of tissue lining the perihilar bile duct. These abnormal cells may become cancer and spread into nearby normal tissue. Stage 0 is also called carcinoma in situ. - Stage I: Cancer has formed in the innermost layer of the wall of the perihilar bile duct and has spread into the muscle layer or fibrous tissue layer of the wall. - Stage II: Cancer has spread through the wall of the perihilar bile duct to nearby fatty tissue or to the liver. - Stage III: Stage III is divided into stage IIIA and stage IIIB. - Stage IIIA: Cancer has spread to branches on one side of the hepatic artery or of the portal vein. - Stage IIIB: Cancer has spread to nearby lymph nodes. Cancer may have spread into the wall of the perihilar bile duct or through the wall to nearby fatty tissue, the liver, or to branches on one side of the hepatic artery or of the portal vein. - Stage IV: Stage IV is divided into stage IVA and stage IVB. - Stage IVA: Cancer has spread to one or more of the following: - the main part of the portal vein and/or common hepatic artery; - the branches of the portal vein and/or common hepatic artery on both sides; - the right hepatic duct and the left branch of the hepatic artery or of the portal vein; - the left hepatic duct and the right branch of the hepatic artery or of the portal vein. Cancer may have spread to nearby lymph nodes. - Stage IVB: Cancer has spread to lymph nodes in more distant parts of the abdomen, or to organs in other parts of the body. Distal extrahepatic bile duct cancer - Stage 0: Abnormal cells are found in the innermost layer of tissue lining the distal extrahepatic bile duct. These abnormal cells may become cancer and spread into nearby normal tissue. Stage 0 is also called carcinoma in situ. - Stage I: Stage I is divided into stage IA and stage IB. - Stage IA: Cancer has formed and is found in the distal extrahepatic bile duct wall only. - Stage IB: Cancer has formed and has spread through the wall of the distal extrahepatic bile duct but has not spread to nearby organs. - Stage II: Stage II is divided into stage IIA and stage IIB. - Stage IIA: Cancer has spread from the distal extrahepatic bile duct to the gallbladder, pancreas, duodenum, or other nearby organs. - Stage IIB: Cancer has spread from the distal extrahepatic bile duct to nearby lymph nodes. Cancer may have spread through the wall of the duct or to nearby organs. - Stage III: Cancer has spread to the large vessels that carry blood to the organs in the abdomen. Cancer may have spread to nearby lymph nodes. - Stage IV: Cancer has spread to organs in distant parts of the body. The following groups are used to plan treatment: Resectable (localized) bile duct cancer The cancer is in an area, such as the lower part of the common bile duct or perihilar area, where it can be removed completely by surgery. Unresectable, metastatic, or recurrent bile duct cancer Unresectable cancer cannot be removed completely by surgery. Most patients with bile duct cancer cannot have their cancer completely removed by surgery. Metastasis is the spread of cancer from the primary site (place where it started) to other places in the body. Metastatic bile duct cancer may have spread to the liver, other parts of the abdominal cavity, or to distant parts of the body. Recurrent bile duct cancer is cancer that has recurred (come back) after it has been treated. The cancer may come back in the bile ducts, liver, or gallbladder. Less often, it may come back in distant parts of the body.

This page describes the different types of treatment for bile duct cancer (cholangiocarcinoma). Which of these treatments a patient receives will depend on whether the cancer can be completely removed with surgery (resectable) or not (unresectable). To learn more, see treatment of resectable and treatment of unresectable bile duct cancer. The following types of surgery are used to treat bile duct cancer: After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. It is not yet known whether chemotherapy or radiation therapy given after surgery helps keep the cancer from coming back. The following types of palliative surgery may be done to relieve symptoms caused by a blocked bile duct and improve quality of life: Radiation therapy uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. These are the main ways radiation might be given to treat bile duct cancer: It is not known whether external radiation therapy helps in the treatment of resectable bile duct cancer. In unresectable, metastatic, or recurrent bile duct cancer, new ways to improve the effect of external radiation therapy on cancer cells are being studied: External and internal radiation therapy are used to treat bile duct cancer and may also be used as palliative therapy to relieve symptoms and improve quality of life. To learn more about radiation therapy and its side effects, see Radiation Therapy to Treat Cancer and Radiation Therapy Side Effects. Chemotherapy uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. There are two main types of chemotherapy used to treat bile duct cancer. Systemic chemotherapy is used to treat unresectable, metastatic, or recurrent bile duct cancer. The following chemotherapy drugs may be used: In unresectable, metastatic, or recurrent bile duct cancer, intra-arterial embolization is being studied. It is a procedure in which the blood supply to a tumor is blocked after anticancer drugs are given in blood vessels near the tumor. Sometimes, the anticancer drugs are attached to small beads that are injected into an artery that feeds the tumor. The beads block blood flow to the tumor as they release the drug. This allows a higher amount of drug to reach the tumor for a longer period of time, which may kill more cancer cells. It is not known whether systemic chemotherapy helps in the treatment of resectable bile duct cancer.  To learn about the different ways chemotherapy works against cancer, what to expect when receiving chemotherapy, and how to manage chemotherapy side effects, see Chemotherapy to Treat Cancer. In a liver transplant, the entire liver is removed and replaced with a healthy donated liver. A liver transplant may be done in patients with perihilar bile duct cancer. If the patient has to wait for a donated liver, other treatment is given as needed.  Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. The following targeted therapies are being studied in patients with bile duct cancer that is locally advanced and cannot be removed by surgery or has spread to other parts of the body: To learn more about how targeted therapy works against cancer, what to expect when having targeted therapy, and targeted therapy side effects, see Targeted Therapy to Treat Cancer. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. Immune checkpoint inhibitor therapy is a type of immunotherapy. The following immune checkpoint inhibitor may be used to treat bile duct cancer: To learn more about how immunotherapy works against cancer and immunotherapy side effects, see Immunotherapy to Treat Cancer and Immunotherapy Side Effects. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. For some patients, taking part in a clinical trial may be an option. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. To learn more about clinical trials, see Clinical Trials Information for Patients and Caregivers. If the cancer has not spread and is in a place where surgery can be safely done, the tumor and some of the tissue around it will be removed. This lowers the chance of the cancer coming back. Chemotherapy with or without radiation therapy may be given after surgery. Treatment of resectable intrahepatic bile duct cancer may include the following: Treatment of resectable perihilar bile duct cancer may include the following: Treatment of resectable distal bile duct cancer may include the following: Adjuvant therapy for resectable bile duct cancer may include the following:  Most people with bile duct cancer cannot have their cancer completely removed with surgery. This may be the case if the cancer has spread too far, the cancer is in a place that is too difficult to completely remove with surgery, or the patient is not healthy enough for surgery. Treatment of unresectable bile duct cancer (including metastatic or recurrent disease) may include the following: If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Bile Duct Cancer Treatment was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the treatments for Bile Duct Cancer (Cholangiocarcinoma) ?

Key Points - There are different types of treatment for patients with bile duct cancer. - Three types of standard treatment are used: - Surgery - Radiation therapy - Chemotherapy - New types of treatment are being tested in clinical trials. - Liver transplant - Patients may want to think about taking part in a clinical trial. - Patients can enter clinical trials before, during, or after starting their cancer treatment. - Follow-up tests may be needed. There are different types of treatment for patients with bile duct cancer. Different types of treatments are available for patients with bile duct cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Three types of standard treatment are used: Surgery The following types of surgery are used to treat bile duct cancer: - Removal of the bile duct: A surgical procedure to remove part of the bile duct if the tumor is small and in the bile duct only. Lymph nodes are removed and tissue from the lymph nodes is viewed under a microscope to see if there is cancer. - Partial hepatectomy: A surgical procedure in which the part of the liver where cancer is found is removed. The part removed may be a wedge of tissue, an entire lobe, or a larger part of the liver, along with some normal tissue around it. - Whipple procedure: A surgical procedure in which the head of the pancreas, the gallbladder, part of the stomach, part of the small intestine, and the bile duct are removed. Enough of the pancreas is left to make digestive juices and insulin. Even if the doctor removes all the cancer that can be seen at the time of the operation, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. It is not yet known whether chemotherapy or radiation therapy given after surgery helps keep the cancer from coming back. The following types of palliative surgery may be done to relieve symptoms caused by a blocked bile duct and improve quality of life: - Biliary bypass: A surgical procedure in which the part of the bile duct before the blockage is connected with part of the bile duct that is past the blockage or to the small intestine. This allows bile to flow to the gallbladder or small intestine. - Stent placement: A surgical procedure in which a stent (a thin, flexible tube or metal tube) is placed in the bile duct to open it and allow bile to flow into the small intestine or through a catheter that goes to a collection bag outside of the body. - Percutaneous transhepatic biliary drainage: A procedure used to x-ray the liver and bile ducts. A thin needle is inserted through the skin below the ribs and into the liver. Dye is injected into the liver or bile ducts and an x-ray is taken. If the bile duct is blocked, a thin, flexible tube called a stent may be left in the liver to drain bile into the small intestine or a collection bag outside the body. Radiation therapy Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: - External radiation therapy uses a machine outside the body to send radiation toward the cancer. - Internal radiation therapy uses a radioactive substance sealed in needles, seeds, wires, or catheters that are placed directly into or near the cancer. External and internal radiation therapy are used to treat bile duct cancer. It is not yet known whether external radiation therapy helps in the treatment of resectable bile duct cancer. In unresectable, metastatic, or recurrent bile duct cancer, new ways to improve the effect of external radiation therapy on cancer cells are being studied: - Hyperthermia therapy: A treatment in which body tissue is exposed to high temperatures to make cancer cells more sensitive to the effects of radiation therapy and certain anticancer drugs. - Radiosensitizers: Drugs that make cancer cells more sensitive to radiation therapy. Combining radiation therapy with radiosensitizers may kill more cancer cells. Chemotherapy Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Systemic chemotherapy is used to treat unresectable, metastatic, or recurrent bile duct cancer. It is not yet known whether systemic chemotherapy helps in the treatment of resectable bile duct cancer. In unresectable, metastatic, or recurrent bile duct cancer, intra-arterial embolization is being studied. It is a procedure in which the blood supply to a tumor is blocked after anticancer drugs are given in blood vessels near the tumor. Sometimes, the anticancer drugs are attached to small beads that are injected into an artery that feeds the tumor. The beads block blood flow to the tumor as they release the drug. This allows a higher amount of drug to reach the tumor for a longer period of time, which may kill more cancer cells. New types of treatment are being tested in clinical trials. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Liver transplant In a liver transplant, the entire liver is removed and replaced with a healthy donated liver. A liver transplant may be done in patients with perihilar bile duct cancer. If the patient has to wait for a donated liver, other treatment is given as needed. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials. Follow-up tests may be needed. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. Treatment Options for Bile Duct Cancer Intrahepatic Bile Duct Cancer Resectable Intrahepatic Bile Duct Cancer Treatment of resectable intrahepatic bile duct cancer may include: - Surgery to remove the cancer, which may include partial hepatectomy. Embolization may be done before surgery. - Surgery followed by chemotherapy and/or radiation therapy. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage I intrahepatic bile duct cancer and stage II intrahepatic bile duct cancer. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Unresectable, Recurrent, or Metastatic Intrahepatic Bile Duct Cancer Treatment of unresectable, recurrent, or metastatic intrahepatic bile duct cancer may include the following: - Stent placement as palliative treatment to relieve symptoms and improve quality of life. - External or internal radiation therapy as palliative treatment to relieve symptoms and improve the quality of life. - Chemotherapy. - A clinical trial of external radiation therapy combined with hyperthermia therapy, radiosensitizer drugs, or chemotherapy. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage III intrahepatic bile duct cancer, stage IV intrahepatic bile duct cancer and recurrent intrahepatic bile duct cancer. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Perihilar Bile Duct Cancer Resectable Perihilar Bile Duct Cancer Treatment of resectable perihilar bile duct cancer may include the following: - Surgery to remove the cancer, which may include partial hepatectomy. - Stent placement or percutaneous transhepatic biliary drainage as palliative therapy, to relieve jaundice and other symptoms and improve the quality of life. - Surgery followed by radiation therapy and/or chemotherapy. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage I perihilar bile duct cancer and stage II perihilar bile duct cancer. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Unresectable, Recurrent, or Metastatic Perihilar Bile Duct Cancer Treatment of unresectable, recurrent, or metastatic perihilar bile duct cancer may include the following: - Stent placement or biliary bypass as palliative treatment to relieve symptoms and improve the quality of life. - External or internal radiation therapy as palliative treatment to relieve symptoms and improve the quality of life. - Chemotherapy. - A clinical trial of external radiation therapy combined with hyperthermia therapy, radiosensitizer drugs, or chemotherapy. - A clinical trial of chemotherapy and radiation therapy followed by a liver transplant. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage III perihilar bile duct cancer, stage IV perihilar bile duct cancer and recurrent perihilar bile duct cancer. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Distal Extrahepatic Bile Duct Cancer Resectable Distal Extrahepatic Bile Duct Cancer Treatment of resectable distal extrahepatic bile duct cancer may include the following: - Surgery to remove the cancer, which may include a Whipple procedure. - Stent placement or percutaneous transhepatic biliary drainage as palliative therapy, to relieve jaundice and other symptoms and improve the quality of life. - Surgery followed by radiation therapy and/or chemotherapy. Check the list of NCI-supported cancer clinical trials that are now accepting patients with localized extrahepatic bile duct cancer. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Unresectable, Recurrent, or Metastatic Distal Extrahepatic Bile Duct Cancer Treatment of unresectable, recurrent, or metastatic distal extrahepatic bile duct cancer may include the following: - Stent placement or biliary bypass as palliative treatment to relieve symptoms and improve the quality of life. - External or internal radiation therapy as palliative treatment to relieve symptoms and improve the quality of life. - Chemotherapy. - A clinical trial of external radiation therapy combined with hyperthermia therapy, radiosensitizer drugs, or chemotherapy. Check the list of NCI-supported cancer clinical trials that are now accepting patients with unresectable extrahepatic bile duct cancer, recurrent extrahepatic bile duct cancer and metastatic extrahepatic bile duct cancer. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website.

Langerhans cell histiocytosis (LCH) is a rare disease that begins in LCH cells. LCH cells are a type of dendritic cell that normally helps the body fight infection. Sometimes mutations (changes) develop in genes that control how dendritic cells function. These include mutations of the BRAF, MAP2K1, RAS, and ARAF genes. These mutations may cause too many LCH cells to grow and build up in certain parts of the body, where they can damage tissue or form lesions. Doctors who treat cancer also treat LCH, and sometimes they use cancer therapies to treat this disease. For more information, see the sections on the treatment of LCH in children and the treatment of LCH in adults. Anything that increases a person's risk of getting a disease is called a risk factor. Not every child with one or more of these risk factors will develop LCH, and it will develop in some children who don’t have any known risk factors. Talk with your doctor if you think you may be at risk. Risk factors for LCH may include the following: These and other signs and symptoms may be caused by LCH or by other conditions. Check with your doctor if you or your child have any of the following: Signs or symptoms of LCH that affects the bone may include: Children with LCH lesions in bones around the ears or eyes have a high risk of diabetes insipidus and other central nervous system diseases. LCH in infants may affect the skin only. In some cases, skin-only LCH may get worse over weeks or months and become a form called high-risk multisystem LCH. In infants, signs or symptoms of LCH that affects the skin may include: In children and adults, signs or symptoms of LCH that affects the skin and nails may include: Signs or symptoms of LCH that affects the mouth may include: Signs or symptoms of LCH that affects the lymph nodes or thymus may include: Signs or symptoms of LCH that affects the pituitary gland may include: Signs or symptoms of LCH that affects the thyroid may include: Signs or symptoms of LCH that affects the eye may include: Signs or symptoms of LCH that affects the CNS (brain and spinal cord) may include: These signs and symptoms may be caused by lesions in the CNS or by CNS neurodegenerative syndrome. Signs or symptoms of LCH that affects the liver or spleen may include: Signs or symptoms of LCH that affects the lung may include: Signs or symptoms of LCH that affects the bone marrow may include: In addition to asking about your health history and doing a physical exam, your doctor may perform the following tests and procedures to diagnose LCH or conditions caused by LCH: The following test may be done on the tissue that was removed: LCH in organs such as the skin, bones, lymph nodes, or pituitary gland usually gets better with treatment and is called "low-risk." LCH in the spleen, liver, or bone marrow is harder to treat and is called "high-risk." Children with LCH in high-risk organs and the gastrointestinal tract have a greater risk of not responding to treatment than patients with high-risk LCH and no disease in the gastrointestinal tract. High-risk LCH is usually seen in children younger than 2 years. The prognosis and treatment options depend on the following: In infants up to 1 year of age, LCH may go away without treatment. The process used to find out if cancer has spread to other parts of the body is called staging. There is no standard staging system for LCH. LCH is described as single-system disease or multisystem disease, depending on how many body systems are affected: LCH may affect low-risk organs or high-risk organs: Progressive LCH describes LCH that continues to grow, spread, or get worse. Progressive disease may be a sign that the LCH has become refractory to treatment. Refractory LCH describes LCH that does not respond to initial treatment. Recurrent or reactivated LCH describes LCH that has come back after it has been treated. Many patients with LCH get better with treatment. However, when treatment stops, new lesions may appear or old lesions may come back. This is called reactivation (recurrence) and may occur within 1 year after stopping treatment. Patients with multisystem disease are more likely to have a reactivation. Common sites of reactivation are bone, ears, or skin. Diabetes insipidus also may develop. Less common sites of reactivation include lymph nodes, bone marrow, spleen, liver, or lung. Some patients may have more than one reactivation. Different types of treatments are available for patients with LCH. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Whenever possible, patients should take part in a clinical trial in order to receive new types of treatment for LCH. Some clinical trials are open only to patients who have not started treatment. Clinical trials are taking place in many parts of the country. Information about ongoing clinical trials is available from the NCI website. Choosing the most appropriate treatment is a decision that ideally involves the patient, family, and health care team. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric healthcare providers who are experts in treating children with LCH and who specialize in certain areas of medicine. These may include the following specialists: Although it is unknown whether LCH is a type of cancer, some of the treatments used for cancer are effective at treating LCH. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Chemotherapy that is taken by mouth or injected into a vein or muscle enters the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Chemotherapy may also be applied to the skin in a cream or lotion (topical chemotherapy). Chemotherapy may be given by injection, by mouth, or applied to the skin to treat LCH. Surgery may be used to remove LCH lesions and a small amount of nearby healthy tissue. Curettage is a type of surgery that uses a curette (a sharp, spoon-shaped tool) to scrape LCH cells from bone. When there is severe liver or lung damage, the entire organ may be removed and replaced with a healthy liver or lung from a donor. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Ultraviolet B (UVB) radiation therapy may be given using a special lamp that directs radiation toward LCH skin lesions. Photodynamic therapy is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. For LCH, laser light is aimed at the skin and the drug becomes active and kills the cancer cells. Photodynamic therapy causes little damage to healthy tissue. Patients who have photodynamic therapy should not spend too much time in the sun. In one type of photodynamic therapy, called psoralen and ultraviolet A (PUVA) therapy, the patient receives a drug called psoralen and then ultraviolet A radiation is directed to the skin. Immunotherapy is a treatment that uses the patient’s immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body’s natural defenses against cancer. Thalidomide is a type of immunotherapy used to treat LCH. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. There are different types of targeted therapy: Other drugs used to treat LCH include the following: Chemotherapy is given to kill cancer cells. Healthy cells, including blood-forming cells, are destroyed by the LCH treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These stem cells grow into (and restore) the body's blood cells. Observation is closely monitoring a patient's condition without giving any treatment until signs or symptoms appear or change. Information about clinical trials is available from the NCI website. For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. For more information, see Late Effects of Treatment for Childhood Cancer. Many patients with multisystem LCH have late effects caused by treatment or by the disease itself. These patients often have long-term health problems that affect their quality of life. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. LCH patients should be monitored for many years because of the risk of reactivation (recurrence). Some of the tests that were done to diagnose LCH may be repeated. This is to see how well the treatment is working and if there are any new lesions. These tests may include: Other tests that may be needed include: The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood Langerhans cell histiocytosis (LCH) skin lesions may include: When severe rashes, pain, ulceration, or bleeding occur, treatment may include the following: Treatment of newly diagnosed childhood LCH bone lesions in the front, sides, or back of the skull, or in any other single bone may include: Treatment of newly diagnosed childhood LCH lesions in bones around the ears or eyes is done to lower the risk of diabetes insipidus and other long-term problems. Treatment may include: Treatment of newly diagnosed childhood LCH lesions of the spine or thigh bone may include: Treatment of two or more bone lesions may include: Treatment of two or more bone lesions combined with skin lesions, lymph node lesions, or diabetes insipidus may include: Treatment of newly diagnosed childhood LCH central nervous system (CNS) lesions may include: Treatment of newly diagnosed LCH CNS neurodegenerative syndrome may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood LCH multisystem disease lesions in the spleen, liver, or bone marrow and another organ or site may include: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of progressive, refractory, or recurrent low-risk LCH may include: Treatment of progressive, refractory, or recurrent high-risk multisystem LCH may include: Treatments being studied for progressive, refractory, or recurrent childhood LCH include the following: Langerhans cell histiocytosis (LCH) in adults is a lot like LCH in children and can form in the same organs and systems as it does in children. These include the endocrine and central nervous systems, liver, spleen, bone marrow, and gastrointestinal tract. In adults, LCH is most commonly found in the lung as a single-system disease. In some patients, other organs may be involved, including bone, skin, hypothalamus, or pituitary gland. LCH in the lung occurs more often in young adults who smoke. As in children, the signs and symptoms of LCH depend on where it is found in the body. See the General Information section for the signs and symptoms of LCH. Tests that examine the organs and body systems where LCH may occur are used to detect (find) and diagnose LCH. See the General Information section for tests and procedures used to diagnose LCH. In adults, there is not a lot of information about what treatment works best. Sometimes, information comes only from reports of the diagnosis, treatment, and follow-up of one adult or a small group of adults who were given the same type of treatment. Adult patients with LCH have higher rates of other cancers than do adults of the same age without LCH. These cancers may be found before, at the same time, or after an LCH diagnosis, and occur more in patients who smoke. For information about the treatments listed below, see the Treatment Option Overview section. Treatment for LCH of the lung in adults may include: Sometimes LCH of the lung will go away or not get worse even if it's not treated. Treatment for LCH that affects only the bone in adults may include: Treatment for LCH that affects only the skin in adults may include: Treatment for LCH that affects the skin and other body systems in adults may include: Treatment of single-system and multisystem disease in adults that does not affect the lung, bone, or skin may include: For more about LCH trials for adults, see the Histiocyte Society website. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more from the National Cancer Institute about Langerhans cell histiocytosis treatment, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood and adult Langerhans cell histiocytosis. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. 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What is (are) Langerhans Cell Histiocytosis ?

Key Points - Langerhans cell histiocytosis is a type of cancer that can damage tissue or cause lesions to form in one or more places in the body. - Family history or having a parent who was exposed to certain chemicals may increase the risk of LCH. - The signs and symptoms of LCH depend on where it is in the body. - Skin and nails - Mouth - Bone - Lymph nodes and thymus - Endocrine system - Central nervous system (CNS) - Liver and spleen - Lung - Bone marrow - Tests that examine the organs and body systems where LCH may occur are used to detect (find) and diagnose LCH. - Certain factors affect prognosis (chance of recovery) and treatment options. Langerhans cell histiocytosis is a type of cancer that can damage tissue or cause lesions to form in one or more places in the body. Langerhans cell histiocytosis (LCH) is a rare cancer that begins in LCH cells (a type of dendritic cell which fights infection). Sometimes there are mutations (changes) in LCH cells as they form. These include mutations of the BRAF gene. These changes may make the LCH cells grow and multiply quickly. This causes LCH cells to build up in certain parts of the body, where they can damage tissue or form lesions. LCH is not a disease of the Langerhans cells that normally occur in the skin. LCH may occur at any age, but is most common in young children. Treatment of LCH in children is different from treatment of LCH in adults. The treatments for LCH in children and adults are described in separate sections of this summary. Check the list of NCI-supported cancer clinical trials that are now accepting patients with childhood Langerhans cell histiocytosis. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your child&apos;s doctor about clinical trials that may be right for your child. General information about clinical trials is available from the NCI website.

Langerhans cell histiocytosis (LCH) is a rare disease that begins in LCH cells. LCH cells are a type of dendritic cell that normally helps the body fight infection. Sometimes mutations (changes) develop in genes that control how dendritic cells function. These include mutations of the BRAF, MAP2K1, RAS, and ARAF genes. These mutations may cause too many LCH cells to grow and build up in certain parts of the body, where they can damage tissue or form lesions. Doctors who treat cancer also treat LCH, and sometimes they use cancer therapies to treat this disease. For more information, see the sections on the treatment of LCH in children and the treatment of LCH in adults. Anything that increases a person's risk of getting a disease is called a risk factor. Not every child with one or more of these risk factors will develop LCH, and it will develop in some children who don’t have any known risk factors. Talk with your doctor if you think you may be at risk. Risk factors for LCH may include the following: These and other signs and symptoms may be caused by LCH or by other conditions. Check with your doctor if you or your child have any of the following: Signs or symptoms of LCH that affects the bone may include: Children with LCH lesions in bones around the ears or eyes have a high risk of diabetes insipidus and other central nervous system diseases. LCH in infants may affect the skin only. In some cases, skin-only LCH may get worse over weeks or months and become a form called high-risk multisystem LCH. In infants, signs or symptoms of LCH that affects the skin may include: In children and adults, signs or symptoms of LCH that affects the skin and nails may include: Signs or symptoms of LCH that affects the mouth may include: Signs or symptoms of LCH that affects the lymph nodes or thymus may include: Signs or symptoms of LCH that affects the pituitary gland may include: Signs or symptoms of LCH that affects the thyroid may include: Signs or symptoms of LCH that affects the eye may include: Signs or symptoms of LCH that affects the CNS (brain and spinal cord) may include: These signs and symptoms may be caused by lesions in the CNS or by CNS neurodegenerative syndrome. Signs or symptoms of LCH that affects the liver or spleen may include: Signs or symptoms of LCH that affects the lung may include: Signs or symptoms of LCH that affects the bone marrow may include: In addition to asking about your health history and doing a physical exam, your doctor may perform the following tests and procedures to diagnose LCH or conditions caused by LCH: The following test may be done on the tissue that was removed: LCH in organs such as the skin, bones, lymph nodes, or pituitary gland usually gets better with treatment and is called "low-risk." LCH in the spleen, liver, or bone marrow is harder to treat and is called "high-risk." Children with LCH in high-risk organs and the gastrointestinal tract have a greater risk of not responding to treatment than patients with high-risk LCH and no disease in the gastrointestinal tract. High-risk LCH is usually seen in children younger than 2 years. The prognosis and treatment options depend on the following: In infants up to 1 year of age, LCH may go away without treatment. The process used to find out if cancer has spread to other parts of the body is called staging. There is no standard staging system for LCH. LCH is described as single-system disease or multisystem disease, depending on how many body systems are affected: LCH may affect low-risk organs or high-risk organs: Progressive LCH describes LCH that continues to grow, spread, or get worse. Progressive disease may be a sign that the LCH has become refractory to treatment. Refractory LCH describes LCH that does not respond to initial treatment. Recurrent or reactivated LCH describes LCH that has come back after it has been treated. Many patients with LCH get better with treatment. However, when treatment stops, new lesions may appear or old lesions may come back. This is called reactivation (recurrence) and may occur within 1 year after stopping treatment. Patients with multisystem disease are more likely to have a reactivation. Common sites of reactivation are bone, ears, or skin. Diabetes insipidus also may develop. Less common sites of reactivation include lymph nodes, bone marrow, spleen, liver, or lung. Some patients may have more than one reactivation. Different types of treatments are available for patients with LCH. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Whenever possible, patients should take part in a clinical trial in order to receive new types of treatment for LCH. Some clinical trials are open only to patients who have not started treatment. Clinical trials are taking place in many parts of the country. Information about ongoing clinical trials is available from the NCI website. Choosing the most appropriate treatment is a decision that ideally involves the patient, family, and health care team. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric healthcare providers who are experts in treating children with LCH and who specialize in certain areas of medicine. These may include the following specialists: Although it is unknown whether LCH is a type of cancer, some of the treatments used for cancer are effective at treating LCH. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Chemotherapy that is taken by mouth or injected into a vein or muscle enters the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Chemotherapy may also be applied to the skin in a cream or lotion (topical chemotherapy). Chemotherapy may be given by injection, by mouth, or applied to the skin to treat LCH. Surgery may be used to remove LCH lesions and a small amount of nearby healthy tissue. Curettage is a type of surgery that uses a curette (a sharp, spoon-shaped tool) to scrape LCH cells from bone. When there is severe liver or lung damage, the entire organ may be removed and replaced with a healthy liver or lung from a donor. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Ultraviolet B (UVB) radiation therapy may be given using a special lamp that directs radiation toward LCH skin lesions. Photodynamic therapy is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. For LCH, laser light is aimed at the skin and the drug becomes active and kills the cancer cells. Photodynamic therapy causes little damage to healthy tissue. Patients who have photodynamic therapy should not spend too much time in the sun. In one type of photodynamic therapy, called psoralen and ultraviolet A (PUVA) therapy, the patient receives a drug called psoralen and then ultraviolet A radiation is directed to the skin. Immunotherapy is a treatment that uses the patient’s immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body’s natural defenses against cancer. Thalidomide is a type of immunotherapy used to treat LCH. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. There are different types of targeted therapy: Other drugs used to treat LCH include the following: Chemotherapy is given to kill cancer cells. Healthy cells, including blood-forming cells, are destroyed by the LCH treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These stem cells grow into (and restore) the body's blood cells. Observation is closely monitoring a patient's condition without giving any treatment until signs or symptoms appear or change. Information about clinical trials is available from the NCI website. For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. For more information, see Late Effects of Treatment for Childhood Cancer. Many patients with multisystem LCH have late effects caused by treatment or by the disease itself. These patients often have long-term health problems that affect their quality of life. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. LCH patients should be monitored for many years because of the risk of reactivation (recurrence). Some of the tests that were done to diagnose LCH may be repeated. This is to see how well the treatment is working and if there are any new lesions. These tests may include: Other tests that may be needed include: The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood Langerhans cell histiocytosis (LCH) skin lesions may include: When severe rashes, pain, ulceration, or bleeding occur, treatment may include the following: Treatment of newly diagnosed childhood LCH bone lesions in the front, sides, or back of the skull, or in any other single bone may include: Treatment of newly diagnosed childhood LCH lesions in bones around the ears or eyes is done to lower the risk of diabetes insipidus and other long-term problems. Treatment may include: Treatment of newly diagnosed childhood LCH lesions of the spine or thigh bone may include: Treatment of two or more bone lesions may include: Treatment of two or more bone lesions combined with skin lesions, lymph node lesions, or diabetes insipidus may include: Treatment of newly diagnosed childhood LCH central nervous system (CNS) lesions may include: Treatment of newly diagnosed LCH CNS neurodegenerative syndrome may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood LCH multisystem disease lesions in the spleen, liver, or bone marrow and another organ or site may include: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of progressive, refractory, or recurrent low-risk LCH may include: Treatment of progressive, refractory, or recurrent high-risk multisystem LCH may include: Treatments being studied for progressive, refractory, or recurrent childhood LCH include the following: Langerhans cell histiocytosis (LCH) in adults is a lot like LCH in children and can form in the same organs and systems as it does in children. These include the endocrine and central nervous systems, liver, spleen, bone marrow, and gastrointestinal tract. In adults, LCH is most commonly found in the lung as a single-system disease. In some patients, other organs may be involved, including bone, skin, hypothalamus, or pituitary gland. LCH in the lung occurs more often in young adults who smoke. As in children, the signs and symptoms of LCH depend on where it is found in the body. See the General Information section for the signs and symptoms of LCH. Tests that examine the organs and body systems where LCH may occur are used to detect (find) and diagnose LCH. See the General Information section for tests and procedures used to diagnose LCH. In adults, there is not a lot of information about what treatment works best. Sometimes, information comes only from reports of the diagnosis, treatment, and follow-up of one adult or a small group of adults who were given the same type of treatment. Adult patients with LCH have higher rates of other cancers than do adults of the same age without LCH. These cancers may be found before, at the same time, or after an LCH diagnosis, and occur more in patients who smoke. For information about the treatments listed below, see the Treatment Option Overview section. Treatment for LCH of the lung in adults may include: Sometimes LCH of the lung will go away or not get worse even if it's not treated. Treatment for LCH that affects only the bone in adults may include: Treatment for LCH that affects only the skin in adults may include: Treatment for LCH that affects the skin and other body systems in adults may include: Treatment of single-system and multisystem disease in adults that does not affect the lung, bone, or skin may include: For more about LCH trials for adults, see the Histiocyte Society website. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more from the National Cancer Institute about Langerhans cell histiocytosis treatment, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood and adult Langerhans cell histiocytosis. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Langerhans Cell Histiocytosis Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/langerhans/patient/langerhans-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389196] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Langerhans Cell Histiocytosis Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

Who is at risk for Langerhans Cell Histiocytosis? ?

Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn&apos;t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for LCH include the following: - Having a parent who was exposed to certain chemicals such as benzene. - Having a parent who was exposed to metal, granite, or wood dust in the workplace. - A family history of cancer, including LCH. - Having infections as a newborn. - Having a personal history or family history of thyroid disease. - Smoking, especially in young adults. - Being Hispanic.

Langerhans cell histiocytosis (LCH) is a rare disease that begins in LCH cells. LCH cells are a type of dendritic cell that normally helps the body fight infection. Sometimes mutations (changes) develop in genes that control how dendritic cells function. These include mutations of the BRAF, MAP2K1, RAS, and ARAF genes. These mutations may cause too many LCH cells to grow and build up in certain parts of the body, where they can damage tissue or form lesions. Doctors who treat cancer also treat LCH, and sometimes they use cancer therapies to treat this disease. For more information, see the sections on the treatment of LCH in children and the treatment of LCH in adults. Anything that increases a person's risk of getting a disease is called a risk factor. Not every child with one or more of these risk factors will develop LCH, and it will develop in some children who don’t have any known risk factors. Talk with your doctor if you think you may be at risk. Risk factors for LCH may include the following: These and other signs and symptoms may be caused by LCH or by other conditions. Check with your doctor if you or your child have any of the following: Signs or symptoms of LCH that affects the bone may include: Children with LCH lesions in bones around the ears or eyes have a high risk of diabetes insipidus and other central nervous system diseases. LCH in infants may affect the skin only. In some cases, skin-only LCH may get worse over weeks or months and become a form called high-risk multisystem LCH. In infants, signs or symptoms of LCH that affects the skin may include: In children and adults, signs or symptoms of LCH that affects the skin and nails may include: Signs or symptoms of LCH that affects the mouth may include: Signs or symptoms of LCH that affects the lymph nodes or thymus may include: Signs or symptoms of LCH that affects the pituitary gland may include: Signs or symptoms of LCH that affects the thyroid may include: Signs or symptoms of LCH that affects the eye may include: Signs or symptoms of LCH that affects the CNS (brain and spinal cord) may include: These signs and symptoms may be caused by lesions in the CNS or by CNS neurodegenerative syndrome. Signs or symptoms of LCH that affects the liver or spleen may include: Signs or symptoms of LCH that affects the lung may include: Signs or symptoms of LCH that affects the bone marrow may include: In addition to asking about your health history and doing a physical exam, your doctor may perform the following tests and procedures to diagnose LCH or conditions caused by LCH: The following test may be done on the tissue that was removed: LCH in organs such as the skin, bones, lymph nodes, or pituitary gland usually gets better with treatment and is called "low-risk." LCH in the spleen, liver, or bone marrow is harder to treat and is called "high-risk." Children with LCH in high-risk organs and the gastrointestinal tract have a greater risk of not responding to treatment than patients with high-risk LCH and no disease in the gastrointestinal tract. High-risk LCH is usually seen in children younger than 2 years. The prognosis and treatment options depend on the following: In infants up to 1 year of age, LCH may go away without treatment. The process used to find out if cancer has spread to other parts of the body is called staging. There is no standard staging system for LCH. LCH is described as single-system disease or multisystem disease, depending on how many body systems are affected: LCH may affect low-risk organs or high-risk organs: Progressive LCH describes LCH that continues to grow, spread, or get worse. Progressive disease may be a sign that the LCH has become refractory to treatment. Refractory LCH describes LCH that does not respond to initial treatment. Recurrent or reactivated LCH describes LCH that has come back after it has been treated. Many patients with LCH get better with treatment. However, when treatment stops, new lesions may appear or old lesions may come back. This is called reactivation (recurrence) and may occur within 1 year after stopping treatment. Patients with multisystem disease are more likely to have a reactivation. Common sites of reactivation are bone, ears, or skin. Diabetes insipidus also may develop. Less common sites of reactivation include lymph nodes, bone marrow, spleen, liver, or lung. Some patients may have more than one reactivation. Different types of treatments are available for patients with LCH. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Whenever possible, patients should take part in a clinical trial in order to receive new types of treatment for LCH. Some clinical trials are open only to patients who have not started treatment. Clinical trials are taking place in many parts of the country. Information about ongoing clinical trials is available from the NCI website. Choosing the most appropriate treatment is a decision that ideally involves the patient, family, and health care team. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric healthcare providers who are experts in treating children with LCH and who specialize in certain areas of medicine. These may include the following specialists: Although it is unknown whether LCH is a type of cancer, some of the treatments used for cancer are effective at treating LCH. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Chemotherapy that is taken by mouth or injected into a vein or muscle enters the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Chemotherapy may also be applied to the skin in a cream or lotion (topical chemotherapy). Chemotherapy may be given by injection, by mouth, or applied to the skin to treat LCH. Surgery may be used to remove LCH lesions and a small amount of nearby healthy tissue. Curettage is a type of surgery that uses a curette (a sharp, spoon-shaped tool) to scrape LCH cells from bone. When there is severe liver or lung damage, the entire organ may be removed and replaced with a healthy liver or lung from a donor. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Ultraviolet B (UVB) radiation therapy may be given using a special lamp that directs radiation toward LCH skin lesions. Photodynamic therapy is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. For LCH, laser light is aimed at the skin and the drug becomes active and kills the cancer cells. Photodynamic therapy causes little damage to healthy tissue. Patients who have photodynamic therapy should not spend too much time in the sun. In one type of photodynamic therapy, called psoralen and ultraviolet A (PUVA) therapy, the patient receives a drug called psoralen and then ultraviolet A radiation is directed to the skin. Immunotherapy is a treatment that uses the patient’s immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body’s natural defenses against cancer. Thalidomide is a type of immunotherapy used to treat LCH. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. There are different types of targeted therapy: Other drugs used to treat LCH include the following: Chemotherapy is given to kill cancer cells. Healthy cells, including blood-forming cells, are destroyed by the LCH treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These stem cells grow into (and restore) the body's blood cells. Observation is closely monitoring a patient's condition without giving any treatment until signs or symptoms appear or change. Information about clinical trials is available from the NCI website. For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. For more information, see Late Effects of Treatment for Childhood Cancer. Many patients with multisystem LCH have late effects caused by treatment or by the disease itself. These patients often have long-term health problems that affect their quality of life. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. LCH patients should be monitored for many years because of the risk of reactivation (recurrence). Some of the tests that were done to diagnose LCH may be repeated. This is to see how well the treatment is working and if there are any new lesions. These tests may include: Other tests that may be needed include: The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood Langerhans cell histiocytosis (LCH) skin lesions may include: When severe rashes, pain, ulceration, or bleeding occur, treatment may include the following: Treatment of newly diagnosed childhood LCH bone lesions in the front, sides, or back of the skull, or in any other single bone may include: Treatment of newly diagnosed childhood LCH lesions in bones around the ears or eyes is done to lower the risk of diabetes insipidus and other long-term problems. Treatment may include: Treatment of newly diagnosed childhood LCH lesions of the spine or thigh bone may include: Treatment of two or more bone lesions may include: Treatment of two or more bone lesions combined with skin lesions, lymph node lesions, or diabetes insipidus may include: Treatment of newly diagnosed childhood LCH central nervous system (CNS) lesions may include: Treatment of newly diagnosed LCH CNS neurodegenerative syndrome may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood LCH multisystem disease lesions in the spleen, liver, or bone marrow and another organ or site may include: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of progressive, refractory, or recurrent low-risk LCH may include: Treatment of progressive, refractory, or recurrent high-risk multisystem LCH may include: Treatments being studied for progressive, refractory, or recurrent childhood LCH include the following: Langerhans cell histiocytosis (LCH) in adults is a lot like LCH in children and can form in the same organs and systems as it does in children. These include the endocrine and central nervous systems, liver, spleen, bone marrow, and gastrointestinal tract. In adults, LCH is most commonly found in the lung as a single-system disease. In some patients, other organs may be involved, including bone, skin, hypothalamus, or pituitary gland. LCH in the lung occurs more often in young adults who smoke. As in children, the signs and symptoms of LCH depend on where it is found in the body. See the General Information section for the signs and symptoms of LCH. Tests that examine the organs and body systems where LCH may occur are used to detect (find) and diagnose LCH. See the General Information section for tests and procedures used to diagnose LCH. In adults, there is not a lot of information about what treatment works best. Sometimes, information comes only from reports of the diagnosis, treatment, and follow-up of one adult or a small group of adults who were given the same type of treatment. Adult patients with LCH have higher rates of other cancers than do adults of the same age without LCH. These cancers may be found before, at the same time, or after an LCH diagnosis, and occur more in patients who smoke. For information about the treatments listed below, see the Treatment Option Overview section. Treatment for LCH of the lung in adults may include: Sometimes LCH of the lung will go away or not get worse even if it's not treated. Treatment for LCH that affects only the bone in adults may include: Treatment for LCH that affects only the skin in adults may include: Treatment for LCH that affects the skin and other body systems in adults may include: Treatment of single-system and multisystem disease in adults that does not affect the lung, bone, or skin may include: For more about LCH trials for adults, see the Histiocyte Society website. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more from the National Cancer Institute about Langerhans cell histiocytosis treatment, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood and adult Langerhans cell histiocytosis. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Langerhans Cell Histiocytosis Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/langerhans/patient/langerhans-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389196] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Langerhans Cell Histiocytosis Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the symptoms of Langerhans Cell Histiocytosis ?

These and other signs and symptoms may be caused by LCH or by other conditions. Check with your doctor if you or your child have any of the following: Skin and nails LCH in infants may affect the skin only. In some cases, skin-only LCH may get worse over weeks or months and become a form called high-risk multisystem LCH. In infants, signs or symptoms of LCH that affects the skin may include: - Flaking of the scalp that may look like cradle cap. - Raised, brown or purple skin rash anywhere on the body. In children and adults, signs or symptoms of LCH that affects the skin and nails may include: - Flaking of the scalp that may look like dandruff. - Raised, red or brown, crusted rash in the groin area, abdomen, back, or chest, that may be itchy. - Bumps or ulcers on the scalp. - Ulcers behind the ears, under the breasts, or in the groin area. - Fingernails that fall off or have discolored grooves that run the length of the nail. Mouth Signs or symptoms of LCH that affects the mouth may include: - Swollen gums. - Sores on the roof of the mouth, inside the cheeks, or on the tongue or lips. - Teeth that become uneven. - Tooth loss. Bone Signs or symptoms of LCH that affects the bone may include: - Swelling or a lump over a bone, such as the skull, ribs, spine, thigh bone, upper arm bone, elbow, eye socket, or bones around the ear. - Pain where there is swelling or a lump over a bone. Children with LCH lesions in bones around the ears or eyes have a high risk for diabetes insipidus and other central nervous system disease. Lymph nodes and thymus Signs or symptoms of LCH that affects the lymph nodes or thymus may include: - Swollen lymph nodes. - Trouble breathing. - Superior vena cava syndrome. This can cause coughing, trouble breathing, and swelling of the face, neck, and upper arms. Endocrine system Signs or symptoms of LCH that affects the pituitary gland may include: - Diabetes insipidus. This can cause a strong thirst and frequent urination. - Slow growth. - Early or late puberty. - Being very overweight. Signs or symptoms of LCH that affects the thyroid may include: - Swollen thyroid gland. - Hypothyroidism. This can cause tiredness, lack of energy, being sensitive to cold, constipation, dry skin, thinning hair, memory problems, trouble concentrating, and depression. In infants, this can also cause a loss of appetite and choking on food. In children and adolescents, this can also cause behavior problems, weight gain, slow growth, and late puberty. - Trouble breathing. Central nervous system (CNS) Signs or symptoms of LCH that affects the CNS (brain and spinal cord) may include: - Loss of balance, uncoordinated body movements, and trouble walking. - Trouble speaking. - Trouble seeing. - Headaches. - Changes in behavior or personality. - Memory problems. These signs and symptoms may be caused by lesions in the CNS or by CNS neurodegenerative syndrome. Liver and spleen Signs or symptoms of LCH that affects the liver or spleen may include: - Swelling in the abdomen caused by a buildup of extra fluid. - Trouble breathing. - Yellowing of the skin and whites of the eyes. - Itching. - Easy bruising or bleeding. - Feeling very tired. Lung Signs or symptoms of LCH that affects the lung may include: - Collapsed lung. This condition can cause chest pain or tightness, trouble breathing, feeling tired, and a bluish color to the skin. - Trouble breathing, especially in adults who smoke. - Dry cough. - Chest pain. Bone marrow Signs or symptoms of LCH that affects the bone marrow may include: - Easy bruising or bleeding. - Fever. - Frequent infections.

Langerhans cell histiocytosis (LCH) is a rare disease that begins in LCH cells. LCH cells are a type of dendritic cell that normally helps the body fight infection. Sometimes mutations (changes) develop in genes that control how dendritic cells function. These include mutations of the BRAF, MAP2K1, RAS, and ARAF genes. These mutations may cause too many LCH cells to grow and build up in certain parts of the body, where they can damage tissue or form lesions. Doctors who treat cancer also treat LCH, and sometimes they use cancer therapies to treat this disease. For more information, see the sections on the treatment of LCH in children and the treatment of LCH in adults. Anything that increases a person's risk of getting a disease is called a risk factor. Not every child with one or more of these risk factors will develop LCH, and it will develop in some children who don’t have any known risk factors. Talk with your doctor if you think you may be at risk. Risk factors for LCH may include the following: These and other signs and symptoms may be caused by LCH or by other conditions. Check with your doctor if you or your child have any of the following: Signs or symptoms of LCH that affects the bone may include: Children with LCH lesions in bones around the ears or eyes have a high risk of diabetes insipidus and other central nervous system diseases. LCH in infants may affect the skin only. In some cases, skin-only LCH may get worse over weeks or months and become a form called high-risk multisystem LCH. In infants, signs or symptoms of LCH that affects the skin may include: In children and adults, signs or symptoms of LCH that affects the skin and nails may include: Signs or symptoms of LCH that affects the mouth may include: Signs or symptoms of LCH that affects the lymph nodes or thymus may include: Signs or symptoms of LCH that affects the pituitary gland may include: Signs or symptoms of LCH that affects the thyroid may include: Signs or symptoms of LCH that affects the eye may include: Signs or symptoms of LCH that affects the CNS (brain and spinal cord) may include: These signs and symptoms may be caused by lesions in the CNS or by CNS neurodegenerative syndrome. Signs or symptoms of LCH that affects the liver or spleen may include: Signs or symptoms of LCH that affects the lung may include: Signs or symptoms of LCH that affects the bone marrow may include: In addition to asking about your health history and doing a physical exam, your doctor may perform the following tests and procedures to diagnose LCH or conditions caused by LCH: The following test may be done on the tissue that was removed: LCH in organs such as the skin, bones, lymph nodes, or pituitary gland usually gets better with treatment and is called "low-risk." LCH in the spleen, liver, or bone marrow is harder to treat and is called "high-risk." Children with LCH in high-risk organs and the gastrointestinal tract have a greater risk of not responding to treatment than patients with high-risk LCH and no disease in the gastrointestinal tract. High-risk LCH is usually seen in children younger than 2 years. The prognosis and treatment options depend on the following: In infants up to 1 year of age, LCH may go away without treatment. The process used to find out if cancer has spread to other parts of the body is called staging. There is no standard staging system for LCH. LCH is described as single-system disease or multisystem disease, depending on how many body systems are affected: LCH may affect low-risk organs or high-risk organs: Progressive LCH describes LCH that continues to grow, spread, or get worse. Progressive disease may be a sign that the LCH has become refractory to treatment. Refractory LCH describes LCH that does not respond to initial treatment. Recurrent or reactivated LCH describes LCH that has come back after it has been treated. Many patients with LCH get better with treatment. However, when treatment stops, new lesions may appear or old lesions may come back. This is called reactivation (recurrence) and may occur within 1 year after stopping treatment. Patients with multisystem disease are more likely to have a reactivation. Common sites of reactivation are bone, ears, or skin. Diabetes insipidus also may develop. Less common sites of reactivation include lymph nodes, bone marrow, spleen, liver, or lung. Some patients may have more than one reactivation. Different types of treatments are available for patients with LCH. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Whenever possible, patients should take part in a clinical trial in order to receive new types of treatment for LCH. Some clinical trials are open only to patients who have not started treatment. Clinical trials are taking place in many parts of the country. Information about ongoing clinical trials is available from the NCI website. Choosing the most appropriate treatment is a decision that ideally involves the patient, family, and health care team. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric healthcare providers who are experts in treating children with LCH and who specialize in certain areas of medicine. These may include the following specialists: Although it is unknown whether LCH is a type of cancer, some of the treatments used for cancer are effective at treating LCH. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Chemotherapy that is taken by mouth or injected into a vein or muscle enters the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Chemotherapy may also be applied to the skin in a cream or lotion (topical chemotherapy). Chemotherapy may be given by injection, by mouth, or applied to the skin to treat LCH. Surgery may be used to remove LCH lesions and a small amount of nearby healthy tissue. Curettage is a type of surgery that uses a curette (a sharp, spoon-shaped tool) to scrape LCH cells from bone. When there is severe liver or lung damage, the entire organ may be removed and replaced with a healthy liver or lung from a donor. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Ultraviolet B (UVB) radiation therapy may be given using a special lamp that directs radiation toward LCH skin lesions. Photodynamic therapy is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. For LCH, laser light is aimed at the skin and the drug becomes active and kills the cancer cells. Photodynamic therapy causes little damage to healthy tissue. Patients who have photodynamic therapy should not spend too much time in the sun. In one type of photodynamic therapy, called psoralen and ultraviolet A (PUVA) therapy, the patient receives a drug called psoralen and then ultraviolet A radiation is directed to the skin. Immunotherapy is a treatment that uses the patient’s immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body’s natural defenses against cancer. Thalidomide is a type of immunotherapy used to treat LCH. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. There are different types of targeted therapy: Other drugs used to treat LCH include the following: Chemotherapy is given to kill cancer cells. Healthy cells, including blood-forming cells, are destroyed by the LCH treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These stem cells grow into (and restore) the body's blood cells. Observation is closely monitoring a patient's condition without giving any treatment until signs or symptoms appear or change. Information about clinical trials is available from the NCI website. For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. For more information, see Late Effects of Treatment for Childhood Cancer. Many patients with multisystem LCH have late effects caused by treatment or by the disease itself. These patients often have long-term health problems that affect their quality of life. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. LCH patients should be monitored for many years because of the risk of reactivation (recurrence). Some of the tests that were done to diagnose LCH may be repeated. This is to see how well the treatment is working and if there are any new lesions. These tests may include: Other tests that may be needed include: The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood Langerhans cell histiocytosis (LCH) skin lesions may include: When severe rashes, pain, ulceration, or bleeding occur, treatment may include the following: Treatment of newly diagnosed childhood LCH bone lesions in the front, sides, or back of the skull, or in any other single bone may include: Treatment of newly diagnosed childhood LCH lesions in bones around the ears or eyes is done to lower the risk of diabetes insipidus and other long-term problems. Treatment may include: Treatment of newly diagnosed childhood LCH lesions of the spine or thigh bone may include: Treatment of two or more bone lesions may include: Treatment of two or more bone lesions combined with skin lesions, lymph node lesions, or diabetes insipidus may include: Treatment of newly diagnosed childhood LCH central nervous system (CNS) lesions may include: Treatment of newly diagnosed LCH CNS neurodegenerative syndrome may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood LCH multisystem disease lesions in the spleen, liver, or bone marrow and another organ or site may include: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of progressive, refractory, or recurrent low-risk LCH may include: Treatment of progressive, refractory, or recurrent high-risk multisystem LCH may include: Treatments being studied for progressive, refractory, or recurrent childhood LCH include the following: Langerhans cell histiocytosis (LCH) in adults is a lot like LCH in children and can form in the same organs and systems as it does in children. These include the endocrine and central nervous systems, liver, spleen, bone marrow, and gastrointestinal tract. In adults, LCH is most commonly found in the lung as a single-system disease. In some patients, other organs may be involved, including bone, skin, hypothalamus, or pituitary gland. LCH in the lung occurs more often in young adults who smoke. As in children, the signs and symptoms of LCH depend on where it is found in the body. See the General Information section for the signs and symptoms of LCH. Tests that examine the organs and body systems where LCH may occur are used to detect (find) and diagnose LCH. See the General Information section for tests and procedures used to diagnose LCH. In adults, there is not a lot of information about what treatment works best. Sometimes, information comes only from reports of the diagnosis, treatment, and follow-up of one adult or a small group of adults who were given the same type of treatment. Adult patients with LCH have higher rates of other cancers than do adults of the same age without LCH. These cancers may be found before, at the same time, or after an LCH diagnosis, and occur more in patients who smoke. For information about the treatments listed below, see the Treatment Option Overview section. Treatment for LCH of the lung in adults may include: Sometimes LCH of the lung will go away or not get worse even if it's not treated. Treatment for LCH that affects only the bone in adults may include: Treatment for LCH that affects only the skin in adults may include: Treatment for LCH that affects the skin and other body systems in adults may include: Treatment of single-system and multisystem disease in adults that does not affect the lung, bone, or skin may include: For more about LCH trials for adults, see the Histiocyte Society website. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more from the National Cancer Institute about Langerhans cell histiocytosis treatment, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood and adult Langerhans cell histiocytosis. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. 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How to diagnose Langerhans Cell Histiocytosis ?

The following tests and procedures may be used to detect (find) and diagnose LCH or conditions caused by LCH: - Physical exam and history : An exam of the body to check general signs of health, including checking for signs of disease, such as lumps or anything else that seems unusual. A history of the patient&apos;s health habits and past illnesses and treatments will also be taken. - Neurological exam : A series of questions and tests to check the brain, spinal cord, and nerve function. The exam checks a person&apos;s mental status, coordination, and ability to walk normally, and how well the muscles, senses, and reflexes work. This may also be called a neuro exam or a neurologic exam. - Complete blood count (CBC) with differential : A procedure in which a sample of blood is drawn and checked for the following: - The amount of hemoglobin (the protein that carries oxygen) in the red blood cells. - The portion of the blood sample made up of red blood cells. - The number and type of white blood cells. - The number of red blood cells and platelets. - Blood chemistry studies : A procedure in which a blood sample is checked to measure the amounts of certain substances released into the body by organs and tissues in the body. An unusual (higher or lower than normal) amount of a substance can be a sign of disease. - Liver function test : A blood test to measure the blood levels of certain substances released by the liver. A high or low level of these substances can be a sign of disease in the liver. - BRAF gene testing : A laboratory test in which a sample of blood or tissue is tested for mutations of the BRAF gene. - Urinalysis : A test to check the color of urine and its contents, such as sugar, protein, red blood cells, and white blood cells. - Water deprivation test : A test to check how much urine is made and whether it becomes concentrated when little or no water is given. This test is used to diagnose diabetes insipidus, which may be caused by LCH. - Bone marrow aspiration and biopsy : The removal of bone marrow and a small piece of bone by inserting a hollow needle into the hipbone. A pathologist views the bone marrow and bone under a microscope to look for signs of LCH. The following tests may be done on the tissue that was removed: - Immunohistochemistry : A test that uses antibodies to check for certain antigens in a sample of tissue. The antibody is usually linked to a radioactive substance or a dye that causes the tissue to light up under a microscope. This type of test may be used to tell the difference between different types of cancer. - Flow cytometry : A laboratory test that measures the number of cells in a sample, how many cells are live, and the size of the cells. It also shows the shapes of the cells and whether there are tumor markers on the surface of the cells. The cells are stained with a light-sensitive dye, placed in a fluid, and passed in a stream before a laser or other type of light. The measurements are based on how the light-sensitive dye reacts to the light. - Bone scan : A procedure to check if there are rapidly dividing cells in the bone. A very small amount of radioactive material is injected into a vein and travels through the bloodstream. The radioactive material collects in the bones with cancer and is detected by a scanner. - X-ray : An x-ray of the organs and bones inside the body. An x-ray is a type of energy beam that can go through the body and onto film, making a picture of areas inside the body. Sometimes a skeletal survey is done. This is a procedure to x-ray all of the bones in the body. - CT scan (CAT scan): A procedure that makes a series of detailed pictures of areas inside the body, taken from different angles. The pictures are made by a computer linked to an x-ray machine. A dye may be injected into a vein or swallowed to help the organs or tissues show up more clearly. This procedure is also called computed tomography, computerized tomography, or computerized axial tomography. - MRI (magnetic resonance imaging): A procedure that uses a magnet, radio waves, and a computer to make a series of detailed pictures of areas inside the body. A substance called gadolinium may be injected into a vein. The gadolinium collects around the LCH cells so that they show up brighter in the picture. This procedure is also called nuclear magnetic resonance imaging (NMRI). - PET scan (positron emission tomography scan): A procedure to find tumor cells in the body. A small amount of radioactive glucose (sugar) is injected into a vein. The PET scanner rotates around the body and makes a picture of where glucose is being used in the body. Tumor cells show up brighter in the picture because they are more active and take up more glucose than normal cells do. - Ultrasound exam: A procedure in which high-energy sound waves (ultrasound) are bounced off internal tissues or organs and make echoes. The echoes form a picture of body tissues called a sonogram. The picture can be printed to be looked at later. - Bronchoscopy : A procedure to look inside the trachea and large airways in the lung for abnormal areas. A bronchoscope is inserted through the nose or mouth into the trachea and lungs. A bronchoscope is a thin, tube-like instrument with a light and a lens for viewing. It may also have a tool to remove tissue samples, which are checked under a microscope for signs of cancer. - Endoscopy : A procedure to look at organs and tissues inside the body to check for abnormal areas in the gastrointestinal tract or lungs. An endoscope is inserted through an incision (cut) in the skin or opening in the body, such as the mouth. An endoscope is a thin, tube-like instrument with a light and a lens for viewing. It may also have a tool to remove tissue or lymph node samples, which are checked under a microscope for signs of disease. - Biopsy : The removal of cells or tissues so they can be viewed under a microscope by a pathologist to check for LCH cells. To diagnose LCH, a biopsy of bone lesions, skin, lymph nodes, or the liver may be done.

Langerhans cell histiocytosis (LCH) is a rare disease that begins in LCH cells. LCH cells are a type of dendritic cell that normally helps the body fight infection. Sometimes mutations (changes) develop in genes that control how dendritic cells function. These include mutations of the BRAF, MAP2K1, RAS, and ARAF genes. These mutations may cause too many LCH cells to grow and build up in certain parts of the body, where they can damage tissue or form lesions. Doctors who treat cancer also treat LCH, and sometimes they use cancer therapies to treat this disease. For more information, see the sections on the treatment of LCH in children and the treatment of LCH in adults. Anything that increases a person's risk of getting a disease is called a risk factor. Not every child with one or more of these risk factors will develop LCH, and it will develop in some children who don’t have any known risk factors. Talk with your doctor if you think you may be at risk. Risk factors for LCH may include the following: These and other signs and symptoms may be caused by LCH or by other conditions. Check with your doctor if you or your child have any of the following: Signs or symptoms of LCH that affects the bone may include: Children with LCH lesions in bones around the ears or eyes have a high risk of diabetes insipidus and other central nervous system diseases. LCH in infants may affect the skin only. In some cases, skin-only LCH may get worse over weeks or months and become a form called high-risk multisystem LCH. In infants, signs or symptoms of LCH that affects the skin may include: In children and adults, signs or symptoms of LCH that affects the skin and nails may include: Signs or symptoms of LCH that affects the mouth may include: Signs or symptoms of LCH that affects the lymph nodes or thymus may include: Signs or symptoms of LCH that affects the pituitary gland may include: Signs or symptoms of LCH that affects the thyroid may include: Signs or symptoms of LCH that affects the eye may include: Signs or symptoms of LCH that affects the CNS (brain and spinal cord) may include: These signs and symptoms may be caused by lesions in the CNS or by CNS neurodegenerative syndrome. Signs or symptoms of LCH that affects the liver or spleen may include: Signs or symptoms of LCH that affects the lung may include: Signs or symptoms of LCH that affects the bone marrow may include: In addition to asking about your health history and doing a physical exam, your doctor may perform the following tests and procedures to diagnose LCH or conditions caused by LCH: The following test may be done on the tissue that was removed: LCH in organs such as the skin, bones, lymph nodes, or pituitary gland usually gets better with treatment and is called "low-risk." LCH in the spleen, liver, or bone marrow is harder to treat and is called "high-risk." Children with LCH in high-risk organs and the gastrointestinal tract have a greater risk of not responding to treatment than patients with high-risk LCH and no disease in the gastrointestinal tract. High-risk LCH is usually seen in children younger than 2 years. The prognosis and treatment options depend on the following: In infants up to 1 year of age, LCH may go away without treatment. The process used to find out if cancer has spread to other parts of the body is called staging. There is no standard staging system for LCH. LCH is described as single-system disease or multisystem disease, depending on how many body systems are affected: LCH may affect low-risk organs or high-risk organs: Progressive LCH describes LCH that continues to grow, spread, or get worse. Progressive disease may be a sign that the LCH has become refractory to treatment. Refractory LCH describes LCH that does not respond to initial treatment. Recurrent or reactivated LCH describes LCH that has come back after it has been treated. Many patients with LCH get better with treatment. However, when treatment stops, new lesions may appear or old lesions may come back. This is called reactivation (recurrence) and may occur within 1 year after stopping treatment. Patients with multisystem disease are more likely to have a reactivation. Common sites of reactivation are bone, ears, or skin. Diabetes insipidus also may develop. Less common sites of reactivation include lymph nodes, bone marrow, spleen, liver, or lung. Some patients may have more than one reactivation. Different types of treatments are available for patients with LCH. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Whenever possible, patients should take part in a clinical trial in order to receive new types of treatment for LCH. Some clinical trials are open only to patients who have not started treatment. Clinical trials are taking place in many parts of the country. Information about ongoing clinical trials is available from the NCI website. Choosing the most appropriate treatment is a decision that ideally involves the patient, family, and health care team. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric healthcare providers who are experts in treating children with LCH and who specialize in certain areas of medicine. These may include the following specialists: Although it is unknown whether LCH is a type of cancer, some of the treatments used for cancer are effective at treating LCH. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Chemotherapy that is taken by mouth or injected into a vein or muscle enters the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Chemotherapy may also be applied to the skin in a cream or lotion (topical chemotherapy). Chemotherapy may be given by injection, by mouth, or applied to the skin to treat LCH. Surgery may be used to remove LCH lesions and a small amount of nearby healthy tissue. Curettage is a type of surgery that uses a curette (a sharp, spoon-shaped tool) to scrape LCH cells from bone. When there is severe liver or lung damage, the entire organ may be removed and replaced with a healthy liver or lung from a donor. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Ultraviolet B (UVB) radiation therapy may be given using a special lamp that directs radiation toward LCH skin lesions. Photodynamic therapy is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. For LCH, laser light is aimed at the skin and the drug becomes active and kills the cancer cells. Photodynamic therapy causes little damage to healthy tissue. Patients who have photodynamic therapy should not spend too much time in the sun. In one type of photodynamic therapy, called psoralen and ultraviolet A (PUVA) therapy, the patient receives a drug called psoralen and then ultraviolet A radiation is directed to the skin. Immunotherapy is a treatment that uses the patient’s immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body’s natural defenses against cancer. Thalidomide is a type of immunotherapy used to treat LCH. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. There are different types of targeted therapy: Other drugs used to treat LCH include the following: Chemotherapy is given to kill cancer cells. Healthy cells, including blood-forming cells, are destroyed by the LCH treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These stem cells grow into (and restore) the body's blood cells. Observation is closely monitoring a patient's condition without giving any treatment until signs or symptoms appear or change. Information about clinical trials is available from the NCI website. For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. For more information, see Late Effects of Treatment for Childhood Cancer. Many patients with multisystem LCH have late effects caused by treatment or by the disease itself. These patients often have long-term health problems that affect their quality of life. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. LCH patients should be monitored for many years because of the risk of reactivation (recurrence). Some of the tests that were done to diagnose LCH may be repeated. This is to see how well the treatment is working and if there are any new lesions. These tests may include: Other tests that may be needed include: The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood Langerhans cell histiocytosis (LCH) skin lesions may include: When severe rashes, pain, ulceration, or bleeding occur, treatment may include the following: Treatment of newly diagnosed childhood LCH bone lesions in the front, sides, or back of the skull, or in any other single bone may include: Treatment of newly diagnosed childhood LCH lesions in bones around the ears or eyes is done to lower the risk of diabetes insipidus and other long-term problems. Treatment may include: Treatment of newly diagnosed childhood LCH lesions of the spine or thigh bone may include: Treatment of two or more bone lesions may include: Treatment of two or more bone lesions combined with skin lesions, lymph node lesions, or diabetes insipidus may include: Treatment of newly diagnosed childhood LCH central nervous system (CNS) lesions may include: Treatment of newly diagnosed LCH CNS neurodegenerative syndrome may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood LCH multisystem disease lesions in the spleen, liver, or bone marrow and another organ or site may include: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of progressive, refractory, or recurrent low-risk LCH may include: Treatment of progressive, refractory, or recurrent high-risk multisystem LCH may include: Treatments being studied for progressive, refractory, or recurrent childhood LCH include the following: Langerhans cell histiocytosis (LCH) in adults is a lot like LCH in children and can form in the same organs and systems as it does in children. These include the endocrine and central nervous systems, liver, spleen, bone marrow, and gastrointestinal tract. In adults, LCH is most commonly found in the lung as a single-system disease. In some patients, other organs may be involved, including bone, skin, hypothalamus, or pituitary gland. LCH in the lung occurs more often in young adults who smoke. As in children, the signs and symptoms of LCH depend on where it is found in the body. See the General Information section for the signs and symptoms of LCH. Tests that examine the organs and body systems where LCH may occur are used to detect (find) and diagnose LCH. See the General Information section for tests and procedures used to diagnose LCH. In adults, there is not a lot of information about what treatment works best. Sometimes, information comes only from reports of the diagnosis, treatment, and follow-up of one adult or a small group of adults who were given the same type of treatment. Adult patients with LCH have higher rates of other cancers than do adults of the same age without LCH. These cancers may be found before, at the same time, or after an LCH diagnosis, and occur more in patients who smoke. For information about the treatments listed below, see the Treatment Option Overview section. Treatment for LCH of the lung in adults may include: Sometimes LCH of the lung will go away or not get worse even if it's not treated. Treatment for LCH that affects only the bone in adults may include: Treatment for LCH that affects only the skin in adults may include: Treatment for LCH that affects the skin and other body systems in adults may include: Treatment of single-system and multisystem disease in adults that does not affect the lung, bone, or skin may include: For more about LCH trials for adults, see the Histiocyte Society website. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more from the National Cancer Institute about Langerhans cell histiocytosis treatment, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood and adult Langerhans cell histiocytosis. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Langerhans Cell Histiocytosis Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/langerhans/patient/langerhans-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389196] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Langerhans Cell Histiocytosis Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is the outlook for Langerhans Cell Histiocytosis ?

LCH in organs such as the skin, bones, lymph nodes, or pituitary gland usually gets better with treatment and is called &quot;low- risk&quot;. LCH in the spleen, liver, or bone marrow is harder to treat and is called &quot;high-risk&quot;. The prognosis (chance of recovery) and treatment options depend on the following: - Whether there are mutations of the BRAF gene. - How old the patient is when diagnosed with LCH. - How many organs or body systems the cancer affects. - Whether the cancer is found in the liver, spleen, bone marrow, or certain bones in the skull. - How quickly the cancer responds to initial treatment. - Whether the cancer has just been diagnosed or has come back (recurred). In infants up to one year of age, LCH may go away without treatment.

Langerhans cell histiocytosis (LCH) is a rare disease that begins in LCH cells. LCH cells are a type of dendritic cell that normally helps the body fight infection. Sometimes mutations (changes) develop in genes that control how dendritic cells function. These include mutations of the BRAF, MAP2K1, RAS, and ARAF genes. These mutations may cause too many LCH cells to grow and build up in certain parts of the body, where they can damage tissue or form lesions. Doctors who treat cancer also treat LCH, and sometimes they use cancer therapies to treat this disease. For more information, see the sections on the treatment of LCH in children and the treatment of LCH in adults. Anything that increases a person's risk of getting a disease is called a risk factor. Not every child with one or more of these risk factors will develop LCH, and it will develop in some children who don’t have any known risk factors. Talk with your doctor if you think you may be at risk. Risk factors for LCH may include the following: These and other signs and symptoms may be caused by LCH or by other conditions. Check with your doctor if you or your child have any of the following: Signs or symptoms of LCH that affects the bone may include: Children with LCH lesions in bones around the ears or eyes have a high risk of diabetes insipidus and other central nervous system diseases. LCH in infants may affect the skin only. In some cases, skin-only LCH may get worse over weeks or months and become a form called high-risk multisystem LCH. In infants, signs or symptoms of LCH that affects the skin may include: In children and adults, signs or symptoms of LCH that affects the skin and nails may include: Signs or symptoms of LCH that affects the mouth may include: Signs or symptoms of LCH that affects the lymph nodes or thymus may include: Signs or symptoms of LCH that affects the pituitary gland may include: Signs or symptoms of LCH that affects the thyroid may include: Signs or symptoms of LCH that affects the eye may include: Signs or symptoms of LCH that affects the CNS (brain and spinal cord) may include: These signs and symptoms may be caused by lesions in the CNS or by CNS neurodegenerative syndrome. Signs or symptoms of LCH that affects the liver or spleen may include: Signs or symptoms of LCH that affects the lung may include: Signs or symptoms of LCH that affects the bone marrow may include: In addition to asking about your health history and doing a physical exam, your doctor may perform the following tests and procedures to diagnose LCH or conditions caused by LCH: The following test may be done on the tissue that was removed: LCH in organs such as the skin, bones, lymph nodes, or pituitary gland usually gets better with treatment and is called "low-risk." LCH in the spleen, liver, or bone marrow is harder to treat and is called "high-risk." Children with LCH in high-risk organs and the gastrointestinal tract have a greater risk of not responding to treatment than patients with high-risk LCH and no disease in the gastrointestinal tract. High-risk LCH is usually seen in children younger than 2 years. The prognosis and treatment options depend on the following: In infants up to 1 year of age, LCH may go away without treatment. The process used to find out if cancer has spread to other parts of the body is called staging. There is no standard staging system for LCH. LCH is described as single-system disease or multisystem disease, depending on how many body systems are affected: LCH may affect low-risk organs or high-risk organs: Progressive LCH describes LCH that continues to grow, spread, or get worse. Progressive disease may be a sign that the LCH has become refractory to treatment. Refractory LCH describes LCH that does not respond to initial treatment. Recurrent or reactivated LCH describes LCH that has come back after it has been treated. Many patients with LCH get better with treatment. However, when treatment stops, new lesions may appear or old lesions may come back. This is called reactivation (recurrence) and may occur within 1 year after stopping treatment. Patients with multisystem disease are more likely to have a reactivation. Common sites of reactivation are bone, ears, or skin. Diabetes insipidus also may develop. Less common sites of reactivation include lymph nodes, bone marrow, spleen, liver, or lung. Some patients may have more than one reactivation. Different types of treatments are available for patients with LCH. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Whenever possible, patients should take part in a clinical trial in order to receive new types of treatment for LCH. Some clinical trials are open only to patients who have not started treatment. Clinical trials are taking place in many parts of the country. Information about ongoing clinical trials is available from the NCI website. Choosing the most appropriate treatment is a decision that ideally involves the patient, family, and health care team. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric healthcare providers who are experts in treating children with LCH and who specialize in certain areas of medicine. These may include the following specialists: Although it is unknown whether LCH is a type of cancer, some of the treatments used for cancer are effective at treating LCH. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Chemotherapy that is taken by mouth or injected into a vein or muscle enters the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Chemotherapy may also be applied to the skin in a cream or lotion (topical chemotherapy). Chemotherapy may be given by injection, by mouth, or applied to the skin to treat LCH. Surgery may be used to remove LCH lesions and a small amount of nearby healthy tissue. Curettage is a type of surgery that uses a curette (a sharp, spoon-shaped tool) to scrape LCH cells from bone. When there is severe liver or lung damage, the entire organ may be removed and replaced with a healthy liver or lung from a donor. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Ultraviolet B (UVB) radiation therapy may be given using a special lamp that directs radiation toward LCH skin lesions. Photodynamic therapy is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. For LCH, laser light is aimed at the skin and the drug becomes active and kills the cancer cells. Photodynamic therapy causes little damage to healthy tissue. Patients who have photodynamic therapy should not spend too much time in the sun. In one type of photodynamic therapy, called psoralen and ultraviolet A (PUVA) therapy, the patient receives a drug called psoralen and then ultraviolet A radiation is directed to the skin. Immunotherapy is a treatment that uses the patient’s immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body’s natural defenses against cancer. Thalidomide is a type of immunotherapy used to treat LCH. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. There are different types of targeted therapy: Other drugs used to treat LCH include the following: Chemotherapy is given to kill cancer cells. Healthy cells, including blood-forming cells, are destroyed by the LCH treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These stem cells grow into (and restore) the body's blood cells. Observation is closely monitoring a patient's condition without giving any treatment until signs or symptoms appear or change. Information about clinical trials is available from the NCI website. For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. For more information, see Late Effects of Treatment for Childhood Cancer. Many patients with multisystem LCH have late effects caused by treatment or by the disease itself. These patients often have long-term health problems that affect their quality of life. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. LCH patients should be monitored for many years because of the risk of reactivation (recurrence). Some of the tests that were done to diagnose LCH may be repeated. This is to see how well the treatment is working and if there are any new lesions. These tests may include: Other tests that may be needed include: The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood Langerhans cell histiocytosis (LCH) skin lesions may include: When severe rashes, pain, ulceration, or bleeding occur, treatment may include the following: Treatment of newly diagnosed childhood LCH bone lesions in the front, sides, or back of the skull, or in any other single bone may include: Treatment of newly diagnosed childhood LCH lesions in bones around the ears or eyes is done to lower the risk of diabetes insipidus and other long-term problems. Treatment may include: Treatment of newly diagnosed childhood LCH lesions of the spine or thigh bone may include: Treatment of two or more bone lesions may include: Treatment of two or more bone lesions combined with skin lesions, lymph node lesions, or diabetes insipidus may include: Treatment of newly diagnosed childhood LCH central nervous system (CNS) lesions may include: Treatment of newly diagnosed LCH CNS neurodegenerative syndrome may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood LCH multisystem disease lesions in the spleen, liver, or bone marrow and another organ or site may include: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of progressive, refractory, or recurrent low-risk LCH may include: Treatment of progressive, refractory, or recurrent high-risk multisystem LCH may include: Treatments being studied for progressive, refractory, or recurrent childhood LCH include the following: Langerhans cell histiocytosis (LCH) in adults is a lot like LCH in children and can form in the same organs and systems as it does in children. These include the endocrine and central nervous systems, liver, spleen, bone marrow, and gastrointestinal tract. In adults, LCH is most commonly found in the lung as a single-system disease. In some patients, other organs may be involved, including bone, skin, hypothalamus, or pituitary gland. LCH in the lung occurs more often in young adults who smoke. As in children, the signs and symptoms of LCH depend on where it is found in the body. See the General Information section for the signs and symptoms of LCH. Tests that examine the organs and body systems where LCH may occur are used to detect (find) and diagnose LCH. See the General Information section for tests and procedures used to diagnose LCH. In adults, there is not a lot of information about what treatment works best. Sometimes, information comes only from reports of the diagnosis, treatment, and follow-up of one adult or a small group of adults who were given the same type of treatment. Adult patients with LCH have higher rates of other cancers than do adults of the same age without LCH. These cancers may be found before, at the same time, or after an LCH diagnosis, and occur more in patients who smoke. For information about the treatments listed below, see the Treatment Option Overview section. Treatment for LCH of the lung in adults may include: Sometimes LCH of the lung will go away or not get worse even if it's not treated. Treatment for LCH that affects only the bone in adults may include: Treatment for LCH that affects only the skin in adults may include: Treatment for LCH that affects the skin and other body systems in adults may include: Treatment of single-system and multisystem disease in adults that does not affect the lung, bone, or skin may include: For more about LCH trials for adults, see the Histiocyte Society website. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more from the National Cancer Institute about Langerhans cell histiocytosis treatment, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood and adult Langerhans cell histiocytosis. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Langerhans Cell Histiocytosis Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/langerhans/patient/langerhans-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389196] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Langerhans Cell Histiocytosis Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the stages of Langerhans Cell Histiocytosis ?

Key Points - There is no staging system for Langerhans cell histiocytosis (LCH). - Treatment of LCH is based on where LCH cells are found in the body and how many body systems are affected. There is no staging system for Langerhans cell histiocytosis (LCH). The extent or spread of cancer is usually described as stages. There is no staging system for LCH. Treatment of LCH is based on where LCH cells are found in the body and how many body systems are affected. LCH is described as single-system disease or multisystem disease, depending on how many body systems are affected: - Single-system LCH: LCH is found in one part of an organ or body system (unifocal) or in more than one part of that organ or body system (multifocal). Bone is the most common single place for LCH to be found. - Multisystem LCH: LCH occurs in two or more organs or body systems or may be spread throughout the body. Multisystem LCH is less common than single-system LCH. LCH may affect low-risk organs or high-risk organs: - Low-risk organs include the skin, bone, lungs, lymph nodes, gastrointestinal tract, pituitary gland, and central nervous system (CNS). - High-risk organs include the liver, spleen, and bone marrow.

Langerhans cell histiocytosis (LCH) is a rare disease that begins in LCH cells. LCH cells are a type of dendritic cell that normally helps the body fight infection. Sometimes mutations (changes) develop in genes that control how dendritic cells function. These include mutations of the BRAF, MAP2K1, RAS, and ARAF genes. These mutations may cause too many LCH cells to grow and build up in certain parts of the body, where they can damage tissue or form lesions. Doctors who treat cancer also treat LCH, and sometimes they use cancer therapies to treat this disease. For more information, see the sections on the treatment of LCH in children and the treatment of LCH in adults. Anything that increases a person's risk of getting a disease is called a risk factor. Not every child with one or more of these risk factors will develop LCH, and it will develop in some children who don’t have any known risk factors. Talk with your doctor if you think you may be at risk. Risk factors for LCH may include the following: These and other signs and symptoms may be caused by LCH or by other conditions. Check with your doctor if you or your child have any of the following: Signs or symptoms of LCH that affects the bone may include: Children with LCH lesions in bones around the ears or eyes have a high risk of diabetes insipidus and other central nervous system diseases. LCH in infants may affect the skin only. In some cases, skin-only LCH may get worse over weeks or months and become a form called high-risk multisystem LCH. In infants, signs or symptoms of LCH that affects the skin may include: In children and adults, signs or symptoms of LCH that affects the skin and nails may include: Signs or symptoms of LCH that affects the mouth may include: Signs or symptoms of LCH that affects the lymph nodes or thymus may include: Signs or symptoms of LCH that affects the pituitary gland may include: Signs or symptoms of LCH that affects the thyroid may include: Signs or symptoms of LCH that affects the eye may include: Signs or symptoms of LCH that affects the CNS (brain and spinal cord) may include: These signs and symptoms may be caused by lesions in the CNS or by CNS neurodegenerative syndrome. Signs or symptoms of LCH that affects the liver or spleen may include: Signs or symptoms of LCH that affects the lung may include: Signs or symptoms of LCH that affects the bone marrow may include: In addition to asking about your health history and doing a physical exam, your doctor may perform the following tests and procedures to diagnose LCH or conditions caused by LCH: The following test may be done on the tissue that was removed: LCH in organs such as the skin, bones, lymph nodes, or pituitary gland usually gets better with treatment and is called "low-risk." LCH in the spleen, liver, or bone marrow is harder to treat and is called "high-risk." Children with LCH in high-risk organs and the gastrointestinal tract have a greater risk of not responding to treatment than patients with high-risk LCH and no disease in the gastrointestinal tract. High-risk LCH is usually seen in children younger than 2 years. The prognosis and treatment options depend on the following: In infants up to 1 year of age, LCH may go away without treatment. The process used to find out if cancer has spread to other parts of the body is called staging. There is no standard staging system for LCH. LCH is described as single-system disease or multisystem disease, depending on how many body systems are affected: LCH may affect low-risk organs or high-risk organs: Progressive LCH describes LCH that continues to grow, spread, or get worse. Progressive disease may be a sign that the LCH has become refractory to treatment. Refractory LCH describes LCH that does not respond to initial treatment. Recurrent or reactivated LCH describes LCH that has come back after it has been treated. Many patients with LCH get better with treatment. However, when treatment stops, new lesions may appear or old lesions may come back. This is called reactivation (recurrence) and may occur within 1 year after stopping treatment. Patients with multisystem disease are more likely to have a reactivation. Common sites of reactivation are bone, ears, or skin. Diabetes insipidus also may develop. Less common sites of reactivation include lymph nodes, bone marrow, spleen, liver, or lung. Some patients may have more than one reactivation. Different types of treatments are available for patients with LCH. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Whenever possible, patients should take part in a clinical trial in order to receive new types of treatment for LCH. Some clinical trials are open only to patients who have not started treatment. Clinical trials are taking place in many parts of the country. Information about ongoing clinical trials is available from the NCI website. Choosing the most appropriate treatment is a decision that ideally involves the patient, family, and health care team. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric healthcare providers who are experts in treating children with LCH and who specialize in certain areas of medicine. These may include the following specialists: Although it is unknown whether LCH is a type of cancer, some of the treatments used for cancer are effective at treating LCH. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Chemotherapy that is taken by mouth or injected into a vein or muscle enters the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Chemotherapy may also be applied to the skin in a cream or lotion (topical chemotherapy). Chemotherapy may be given by injection, by mouth, or applied to the skin to treat LCH. Surgery may be used to remove LCH lesions and a small amount of nearby healthy tissue. Curettage is a type of surgery that uses a curette (a sharp, spoon-shaped tool) to scrape LCH cells from bone. When there is severe liver or lung damage, the entire organ may be removed and replaced with a healthy liver or lung from a donor. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Ultraviolet B (UVB) radiation therapy may be given using a special lamp that directs radiation toward LCH skin lesions. Photodynamic therapy is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. For LCH, laser light is aimed at the skin and the drug becomes active and kills the cancer cells. Photodynamic therapy causes little damage to healthy tissue. Patients who have photodynamic therapy should not spend too much time in the sun. In one type of photodynamic therapy, called psoralen and ultraviolet A (PUVA) therapy, the patient receives a drug called psoralen and then ultraviolet A radiation is directed to the skin. Immunotherapy is a treatment that uses the patient’s immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body’s natural defenses against cancer. Thalidomide is a type of immunotherapy used to treat LCH. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. There are different types of targeted therapy: Other drugs used to treat LCH include the following: Chemotherapy is given to kill cancer cells. Healthy cells, including blood-forming cells, are destroyed by the LCH treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These stem cells grow into (and restore) the body's blood cells. Observation is closely monitoring a patient's condition without giving any treatment until signs or symptoms appear or change. Information about clinical trials is available from the NCI website. For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. For more information, see Late Effects of Treatment for Childhood Cancer. Many patients with multisystem LCH have late effects caused by treatment or by the disease itself. These patients often have long-term health problems that affect their quality of life. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. LCH patients should be monitored for many years because of the risk of reactivation (recurrence). Some of the tests that were done to diagnose LCH may be repeated. This is to see how well the treatment is working and if there are any new lesions. These tests may include: Other tests that may be needed include: The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood Langerhans cell histiocytosis (LCH) skin lesions may include: When severe rashes, pain, ulceration, or bleeding occur, treatment may include the following: Treatment of newly diagnosed childhood LCH bone lesions in the front, sides, or back of the skull, or in any other single bone may include: Treatment of newly diagnosed childhood LCH lesions in bones around the ears or eyes is done to lower the risk of diabetes insipidus and other long-term problems. Treatment may include: Treatment of newly diagnosed childhood LCH lesions of the spine or thigh bone may include: Treatment of two or more bone lesions may include: Treatment of two or more bone lesions combined with skin lesions, lymph node lesions, or diabetes insipidus may include: Treatment of newly diagnosed childhood LCH central nervous system (CNS) lesions may include: Treatment of newly diagnosed LCH CNS neurodegenerative syndrome may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood LCH multisystem disease lesions in the spleen, liver, or bone marrow and another organ or site may include: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of progressive, refractory, or recurrent low-risk LCH may include: Treatment of progressive, refractory, or recurrent high-risk multisystem LCH may include: Treatments being studied for progressive, refractory, or recurrent childhood LCH include the following: Langerhans cell histiocytosis (LCH) in adults is a lot like LCH in children and can form in the same organs and systems as it does in children. These include the endocrine and central nervous systems, liver, spleen, bone marrow, and gastrointestinal tract. In adults, LCH is most commonly found in the lung as a single-system disease. In some patients, other organs may be involved, including bone, skin, hypothalamus, or pituitary gland. LCH in the lung occurs more often in young adults who smoke. As in children, the signs and symptoms of LCH depend on where it is found in the body. See the General Information section for the signs and symptoms of LCH. Tests that examine the organs and body systems where LCH may occur are used to detect (find) and diagnose LCH. See the General Information section for tests and procedures used to diagnose LCH. In adults, there is not a lot of information about what treatment works best. Sometimes, information comes only from reports of the diagnosis, treatment, and follow-up of one adult or a small group of adults who were given the same type of treatment. Adult patients with LCH have higher rates of other cancers than do adults of the same age without LCH. These cancers may be found before, at the same time, or after an LCH diagnosis, and occur more in patients who smoke. For information about the treatments listed below, see the Treatment Option Overview section. Treatment for LCH of the lung in adults may include: Sometimes LCH of the lung will go away or not get worse even if it's not treated. Treatment for LCH that affects only the bone in adults may include: Treatment for LCH that affects only the skin in adults may include: Treatment for LCH that affects the skin and other body systems in adults may include: Treatment of single-system and multisystem disease in adults that does not affect the lung, bone, or skin may include: For more about LCH trials for adults, see the Histiocyte Society website. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more from the National Cancer Institute about Langerhans cell histiocytosis treatment, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood and adult Langerhans cell histiocytosis. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. 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What are the treatments for Langerhans Cell Histiocytosis ?

Key Points - There are different types of treatment for patients with Langerhans cell histiocytosis (LCH). - Children with LCH should have their treatment planned by a team of health care providers who are experts in treating childhood cancer. - Some cancer treatments cause side effects months or years after treatment for childhood cancer has ended. - Nine types of standard treatment are used: - Chemotherapy - Surgery - Radiation therapy - Photodynamic therapy - Biologic therapy - Targeted therapy - Other drug therapy - Stem cell transplant - Observation - New types of treatment are being tested in clinical trials. - Patients may want to think about taking part in a clinical trial. - Patients can enter clinical trials before, during, or after starting their treatment. - When treatment of LCH stops, new lesions may appear or old lesions may come back. - Follow-up tests may be needed. There are different types of treatment for patients with Langerhans cell histiocytosis (LCH). Different types of treatments are available for patients with LCH. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Whenever possible, patients should take part in a clinical trial in order to receive new types of treatment for LCH. Some clinical trials are open only to patients who have not started treatment. Clinical trials are taking place in many parts of the country. Information about ongoing clinical trials is available from the NCI website. Choosing the most appropriate treatment is a decision that ideally involves the patient, family, and health care team. Children with LCH should have their treatment planned by a team of health care providers who are experts in treating childhood cancer. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric healthcare providers who are experts in treating children with LCH and who specialize in certain areas of medicine. These may include the following specialists: - Pediatrician. - Primary care physician. - Pediatric surgeon. - Pediatric hematologist. - Radiation oncologist. - Neurologist. - Endocrinologist. - Pediatric nurse specialist. - Rehabilitation specialist. - Psychologist. - Social worker. Some cancer treatments cause side effects months or years after treatment for childhood cancer has ended. Side effects from cancer treatment that begin during or after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: - Slow growth and development. - Hearing loss. - Bone, tooth, liver, and lung problems. - Changes in mood, feeling, learning, thinking, or memory. - Second cancers, such as leukemia, retinoblastoma, Ewing sarcoma, brain or liver cancer. Some late effects may be treated or controlled. It is important to talk with your child&apos;s doctors about the effects cancer treatment can have on your child. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information.) Many patients with multisystem LCH have late effects caused by treatment or by the disease itself. These patients often have long-term health problems that affect their quality of life. Nine types of standard treatment are used: Chemotherapy Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly onto the skin or into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Chemotherapy agents given by injection or by mouth are used to treat LCH. Chemotherapy agents include vinblastine, cytarabine, cladribine, and methotrexate. Nitrogen mustard is a drug that is put directly on the skin to treat small LCH lesions. Surgery Surgery may be used to remove LCH lesions and a small amount of nearby healthy tissue. Curettage is a type of surgery that uses a curette (a sharp, spoon-shaped tool) to scrape LCH cells from bone. When there is severe liver or lung damage, the entire organ may be removed and replaced with a healthy liver or lung from a donor. Radiation therapy Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the cancer. In LCH, a special lamp may be used to send ultraviolet B (UVB) radiation toward LCH skin lesions. Photodynamic therapy Photodynamic therapy is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. For LCH, laser light is aimed at the skin and the drug becomes active and kills the cancer cells. Photodynamic therapy causes little damage to healthy tissue. Patients who have photodynamic therapy should not spend too much time in the sun. In one type of photodynamic therapy, called psoralen and ultraviolet A (PUVA) therapy, the patient receives a drug called psoralen and then ultraviolet A radiation is directed to the skin. Biologic therapy Biologic therapy is a treatment that uses the patients immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the bodys natural defenses against cancer. This type of cancer treatment is also called biotherapy or immunotherapy. Interferon is a type of biologic therapy used to treat LCH of the skin. Immunomodulators are also a type of biologic therapy. Thalidomide is an immunomodulator used to treat LCH. Targeted therapy Targeted therapy is a type of treatment that uses drugs or other substances to find and attack LCH cells without harming normal cells. Imatinib mesylate is a type of targeted therapy called a tyrosine kinase inhibitor. It stops blood stem cells from turning into dendritic cells that may become cancer cells. Other types of kinase inhibitors that affect cells with mutations (changes) in the BRAF gene, such as dabrafenib and vemurafenib, are being studied in clinical trials for LCH. A family of genes, called ras genes, may cause cancer when they are mutated. Ras genes make proteins that are involved in cell signaling pathways, cell growth, and cell death. Ras pathway inhibitors are a type of targeted therapy being studied in clinical trials. They block the actions of a mutated ras gene or its protein and may stop the growth of cancer. Other drug therapy Other drugs used to treat LCH include the following: - Steroid therapy, such as prednisone, is used to treat LCH lesions. - Bisphosphonate therapy (such as pamidronate, zoledronate, or alendronate) is used to treat LCH lesions of the bone and to lessen bone pain. - Nonsteroidal anti-inflammatory drugs (NSAIDs) are drugs (such as aspirin and ibuprofen) that are commonly used to decrease fever, swelling, pain, and redness. Sometimes an NSAID called indomethacin is used to treat LCH. - Retinoids, such as isotretinoin, are drugs related to vitamin A that can slow the growth of LCH cells in the skin. The retinoids are taken by mouth. Stem cell transplant Stem cell transplant is a method of giving chemotherapy and replacing blood-forming cells destroyed by the LCH treatment. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the chemotherapy is completed, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body&apos;s blood cells. Observation Observation is closely monitoring a patient&apos;s condition without giving any treatment until signs or symptoms appear or change. New types of treatment are being tested in clinical trials. Information about clinical trials is available from the NCI website. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options for Childhood LCH and the Treatment Options for Adult LCH sections for links to current treatment clinical trials. These have been retrieved from NCI&apos;s clinical trials database. When treatment of LCH stops, new lesions may appear or old lesions may come back. Many patients with LCH get better with treatment. However, when treatment stops, new lesions may appear or old lesions may come back. This is called reactivation (recurrence) and may occur within one year after stopping treatment. Patients with multisystem disease are more likely to have a reactivation. More common sites of reactivation are bone, ears, or skin. Diabetes insipidus also may develop. Less common sites of reactivation include lymph nodes, bone marrow, spleen, liver, or lung. Some patients may have more than one reactivation over a number of years. Follow-up tests may be needed. Some of the tests that were done to diagnose LCH may be repeated. This is to see how well the treatment is working and if there are any new lesions. These tests may include: - Physical exam. - Neurological exam. - Ultrasound exam. - MRI. - CT scan. - PET scan. Other tests that may be needed include: - Brain stem auditory evoked response (BAER) test: A test that measures the brain&apos;s response to clicking sounds or certain tones. - Pulmonary function test (PFT): A test to see how well the lungs are working. It measures how much air the lungs can hold and how quickly air moves into and out of the lungs. It also measures how much oxygen is used and how much carbon dioxide is given off during breathing. This is also called a lung function test. - Chest x-ray: An x-ray of the organs and bones inside the chest. An x-ray is a type of energy beam that can go through the body and onto film, making a picture of areas inside the body. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. Treatment Options for LCH in Children Treatment of Low-Risk Disease in Children Skin Lesions Treatment of childhood Langerhans cell histiocytosis (LCH) skin lesions may include the following: - Observation. When severe rashes, pain, ulceration, or bleeding occur, treatment may include the following: - Steroid therapy. - Chemotherapy, given by mouth. - Nitrogen mustard applied to the skin. - Photodynamic therapy with psoralen and ultraviolet A (PUVA) therapy. - UVB radiation therapy. Lesions in Bones or Other Low-Risk Organs Treatment of childhood LCH bone lesions in the front, sides, or back of the skull, or in any other single bone may include the following: - Surgery (curettage) with or without steroid therapy. - Low-dose radiation therapy for lesions that affect nearby organs. Treatment of childhood LCH lesions in bones around the ears or eyes is done to lower the risk of diabetes insipidus and other long-term problems. Treatment may include: - Chemotherapy and steroid therapy. - Surgery (curettage). Treatment of childhood LCH lesions of the spine or thigh bone lesions may include: - Observation. - Low-dose radiation therapy. - Chemotherapy, for lesions that spread from the spine into nearby tissue. - Surgery to strengthen the weakened bone by bracing or fusing the bones together. Treatment of two or more bone lesions may include: - Chemotherapy and steroid therapy. Treatment of two or more bone lesions combined with childhood LCH skin lesions, lymph node lesions, or diabetes insipidus may include: - Chemotherapy with or without steroid therapy. - Bisphosphonate therapy. Treatment of High-Risk Disease in Children Treatment of childhood LCH multisystem disease lesions in the spleen, liver, or bone marrow (with or without skin, bone, lymph node, lung, or pituitary gland lesions) may include: - Chemotherapy and steroid therapy. Higher doses of combination chemotherapy and steroid therapy may be given to patients whose tumors do not respond to initial chemotherapy. - A liver transplant for patients with severe liver damage. Treatment of childhood LCH central nervous system (CNS) lesions may include: - Chemotherapy with or without steroid therapy. - Steroid therapy. Treatment of LCH CNS neurodegenerative syndrome may include: - Retinoid therapy. - Chemotherapy. Treatment Options for Recurrent, Refractory, and Progressive Childhood LCH in Children Recurrent LCH is cancer that cannot be detected for some time after treatment and then comes back. Treatment of recurrent childhood LCH in the skin, bone, lymph nodes, gastrointestinal tract, pituitary gland, or central nervous system (low-risk organs) may include: - Chemotherapy with or without steroid therapy. - Bisphosphonate therapy. - Nonsteroidal anti-inflammatory drug (NSAID) therapy with indomethacin. - A clinical trial of a targeted therapy. Refractory LCH is cancer that does not get better with treatment. Treatment of refractory childhood LCH in high-risk organs and in multisystem low-risk organs may include high-dose chemotherapy. Treatment of childhood LCH in multisystem high-risk organs that did not respond to chemotherapy may include stem cell transplant. Progressive LCH is cancer that continues to grow during treatment. Treatment of progressive childhood LCH in patients with multisystem disease may include anticancer drugs that have not been given to the patient before. Treatment Options for LCH in Adults Treatment Options for LCH of the Lung in Adults Treatment for LCH of the lung in adults may include: - Quitting smoking for all patients who smoke. Lung damage will get worse over time in patients who do not quit smoking. In patients who quit smoking, lung damage may get better or it may get worse over time. - Chemotherapy. - Lung transplant for patients with severe lung damage. Sometimes LCH of the lung will go away or not get worse even if it&apos;s not treated. Treatment Options for LCH of the Bone in Adults Treatment for LCH that affects only the bone in adults may include: - Surgery with or without steroid therapy. - Chemotherapy with or without low-dose radiation therapy. - Radiation therapy. - Bisphosphonate therapy, for severe bone pain. Treatment Options for LCH of the Skin in Adults Treatment for LCH that affects only the skin in adults may include: - Surgery. - Steroid or other drug therapy applied or injected into the skin. - Photodynamic therapy with psoralen and ultraviolet A (PUVA) radiation. - UVB radiation therapy. - Chemotherapy or biologic therapy given by mouth, such as methotrexate, thalidomide, or interferon. - Retinoid therapy may be used if the skin lesions do not get better with other treatment. Treatment for LCH that affects the skin and other body systems in adults may include: - Chemotherapy. Treatment Options for Single-System and Multisystem LCH in Adults Treatment of single-system and multisystem disease in adults may include: - Chemotherapy with or without a drug given to weaken the immune system. - Bisphosphonate therapy, for severe bone pain. - A clinical trial of a targeted therapy.

Langerhans cell histiocytosis (LCH) is a rare disease that begins in LCH cells. LCH cells are a type of dendritic cell that normally helps the body fight infection. Sometimes mutations (changes) develop in genes that control how dendritic cells function. These include mutations of the BRAF, MAP2K1, RAS, and ARAF genes. These mutations may cause too many LCH cells to grow and build up in certain parts of the body, where they can damage tissue or form lesions. Doctors who treat cancer also treat LCH, and sometimes they use cancer therapies to treat this disease. For more information, see the sections on the treatment of LCH in children and the treatment of LCH in adults. Anything that increases a person's risk of getting a disease is called a risk factor. Not every child with one or more of these risk factors will develop LCH, and it will develop in some children who don’t have any known risk factors. Talk with your doctor if you think you may be at risk. Risk factors for LCH may include the following: These and other signs and symptoms may be caused by LCH or by other conditions. Check with your doctor if you or your child have any of the following: Signs or symptoms of LCH that affects the bone may include: Children with LCH lesions in bones around the ears or eyes have a high risk of diabetes insipidus and other central nervous system diseases. LCH in infants may affect the skin only. In some cases, skin-only LCH may get worse over weeks or months and become a form called high-risk multisystem LCH. In infants, signs or symptoms of LCH that affects the skin may include: In children and adults, signs or symptoms of LCH that affects the skin and nails may include: Signs or symptoms of LCH that affects the mouth may include: Signs or symptoms of LCH that affects the lymph nodes or thymus may include: Signs or symptoms of LCH that affects the pituitary gland may include: Signs or symptoms of LCH that affects the thyroid may include: Signs or symptoms of LCH that affects the eye may include: Signs or symptoms of LCH that affects the CNS (brain and spinal cord) may include: These signs and symptoms may be caused by lesions in the CNS or by CNS neurodegenerative syndrome. Signs or symptoms of LCH that affects the liver or spleen may include: Signs or symptoms of LCH that affects the lung may include: Signs or symptoms of LCH that affects the bone marrow may include: In addition to asking about your health history and doing a physical exam, your doctor may perform the following tests and procedures to diagnose LCH or conditions caused by LCH: The following test may be done on the tissue that was removed: LCH in organs such as the skin, bones, lymph nodes, or pituitary gland usually gets better with treatment and is called "low-risk." LCH in the spleen, liver, or bone marrow is harder to treat and is called "high-risk." Children with LCH in high-risk organs and the gastrointestinal tract have a greater risk of not responding to treatment than patients with high-risk LCH and no disease in the gastrointestinal tract. High-risk LCH is usually seen in children younger than 2 years. The prognosis and treatment options depend on the following: In infants up to 1 year of age, LCH may go away without treatment. The process used to find out if cancer has spread to other parts of the body is called staging. There is no standard staging system for LCH. LCH is described as single-system disease or multisystem disease, depending on how many body systems are affected: LCH may affect low-risk organs or high-risk organs: Progressive LCH describes LCH that continues to grow, spread, or get worse. Progressive disease may be a sign that the LCH has become refractory to treatment. Refractory LCH describes LCH that does not respond to initial treatment. Recurrent or reactivated LCH describes LCH that has come back after it has been treated. Many patients with LCH get better with treatment. However, when treatment stops, new lesions may appear or old lesions may come back. This is called reactivation (recurrence) and may occur within 1 year after stopping treatment. Patients with multisystem disease are more likely to have a reactivation. Common sites of reactivation are bone, ears, or skin. Diabetes insipidus also may develop. Less common sites of reactivation include lymph nodes, bone marrow, spleen, liver, or lung. Some patients may have more than one reactivation. Different types of treatments are available for patients with LCH. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Whenever possible, patients should take part in a clinical trial in order to receive new types of treatment for LCH. Some clinical trials are open only to patients who have not started treatment. Clinical trials are taking place in many parts of the country. Information about ongoing clinical trials is available from the NCI website. Choosing the most appropriate treatment is a decision that ideally involves the patient, family, and health care team. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric healthcare providers who are experts in treating children with LCH and who specialize in certain areas of medicine. These may include the following specialists: Although it is unknown whether LCH is a type of cancer, some of the treatments used for cancer are effective at treating LCH. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Chemotherapy that is taken by mouth or injected into a vein or muscle enters the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Chemotherapy may also be applied to the skin in a cream or lotion (topical chemotherapy). Chemotherapy may be given by injection, by mouth, or applied to the skin to treat LCH. Surgery may be used to remove LCH lesions and a small amount of nearby healthy tissue. Curettage is a type of surgery that uses a curette (a sharp, spoon-shaped tool) to scrape LCH cells from bone. When there is severe liver or lung damage, the entire organ may be removed and replaced with a healthy liver or lung from a donor. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Ultraviolet B (UVB) radiation therapy may be given using a special lamp that directs radiation toward LCH skin lesions. Photodynamic therapy is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. For LCH, laser light is aimed at the skin and the drug becomes active and kills the cancer cells. Photodynamic therapy causes little damage to healthy tissue. Patients who have photodynamic therapy should not spend too much time in the sun. In one type of photodynamic therapy, called psoralen and ultraviolet A (PUVA) therapy, the patient receives a drug called psoralen and then ultraviolet A radiation is directed to the skin. Immunotherapy is a treatment that uses the patient’s immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body’s natural defenses against cancer. Thalidomide is a type of immunotherapy used to treat LCH. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. There are different types of targeted therapy: Other drugs used to treat LCH include the following: Chemotherapy is given to kill cancer cells. Healthy cells, including blood-forming cells, are destroyed by the LCH treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These stem cells grow into (and restore) the body's blood cells. Observation is closely monitoring a patient's condition without giving any treatment until signs or symptoms appear or change. Information about clinical trials is available from the NCI website. For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. For more information, see Late Effects of Treatment for Childhood Cancer. Many patients with multisystem LCH have late effects caused by treatment or by the disease itself. These patients often have long-term health problems that affect their quality of life. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. LCH patients should be monitored for many years because of the risk of reactivation (recurrence). Some of the tests that were done to diagnose LCH may be repeated. This is to see how well the treatment is working and if there are any new lesions. These tests may include: Other tests that may be needed include: The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood Langerhans cell histiocytosis (LCH) skin lesions may include: When severe rashes, pain, ulceration, or bleeding occur, treatment may include the following: Treatment of newly diagnosed childhood LCH bone lesions in the front, sides, or back of the skull, or in any other single bone may include: Treatment of newly diagnosed childhood LCH lesions in bones around the ears or eyes is done to lower the risk of diabetes insipidus and other long-term problems. Treatment may include: Treatment of newly diagnosed childhood LCH lesions of the spine or thigh bone may include: Treatment of two or more bone lesions may include: Treatment of two or more bone lesions combined with skin lesions, lymph node lesions, or diabetes insipidus may include: Treatment of newly diagnosed childhood LCH central nervous system (CNS) lesions may include: Treatment of newly diagnosed LCH CNS neurodegenerative syndrome may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed childhood LCH multisystem disease lesions in the spleen, liver, or bone marrow and another organ or site may include: For information about the treatments listed below, see the Treatment Option Overview section. Treatment of progressive, refractory, or recurrent low-risk LCH may include: Treatment of progressive, refractory, or recurrent high-risk multisystem LCH may include: Treatments being studied for progressive, refractory, or recurrent childhood LCH include the following: Langerhans cell histiocytosis (LCH) in adults is a lot like LCH in children and can form in the same organs and systems as it does in children. These include the endocrine and central nervous systems, liver, spleen, bone marrow, and gastrointestinal tract. In adults, LCH is most commonly found in the lung as a single-system disease. In some patients, other organs may be involved, including bone, skin, hypothalamus, or pituitary gland. LCH in the lung occurs more often in young adults who smoke. As in children, the signs and symptoms of LCH depend on where it is found in the body. See the General Information section for the signs and symptoms of LCH. Tests that examine the organs and body systems where LCH may occur are used to detect (find) and diagnose LCH. See the General Information section for tests and procedures used to diagnose LCH. In adults, there is not a lot of information about what treatment works best. Sometimes, information comes only from reports of the diagnosis, treatment, and follow-up of one adult or a small group of adults who were given the same type of treatment. Adult patients with LCH have higher rates of other cancers than do adults of the same age without LCH. These cancers may be found before, at the same time, or after an LCH diagnosis, and occur more in patients who smoke. For information about the treatments listed below, see the Treatment Option Overview section. Treatment for LCH of the lung in adults may include: Sometimes LCH of the lung will go away or not get worse even if it's not treated. Treatment for LCH that affects only the bone in adults may include: Treatment for LCH that affects only the skin in adults may include: Treatment for LCH that affects the skin and other body systems in adults may include: Treatment of single-system and multisystem disease in adults that does not affect the lung, bone, or skin may include: For more about LCH trials for adults, see the Histiocyte Society website. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more from the National Cancer Institute about Langerhans cell histiocytosis treatment, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood and adult Langerhans cell histiocytosis. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. 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what research (or clinical trials) is being done for Langerhans Cell Histiocytosis ?

New types of treatment are being tested in clinical trials. Information about clinical trials is available from the NCI website. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options for Childhood LCH and the Treatment Options for Adult LCH sections for links to current treatment clinical trials. These have been retrieved from NCI&apos;s clinical trials database.

These tumors tend to spread through the cerebrospinal fluid (CSF) to other parts of the brain and spinal cord. Medulloblastoma and other CNS embryonal tumors may be malignant (cancer) or benign (not cancer). Most of the tumors in children are malignant. Malignant brain tumors are likely to grow quickly and spread into other parts of the brain. When a tumor grows into or presses on an area of the brain, it may stop that part of the brain from working the way it should. Benign brain tumors grow and press on nearby areas of the brain. They rarely spread to other parts of the brain. Both benign and malignant brain tumors can cause signs or symptoms and need treatment. Although cancer is rare in children, brain tumors are the second most common type of childhood cancer, after leukemia. This summary is about the treatment of primary brain tumors (tumors that begin in the brain). For information about the different types of brain and spinal cord tumors, see the PDQ summary on Childhood Brain and Spinal Cord Tumors Treatment Overview. Brain tumors occur in both children and adults. Treatment for adults may be different from treatment for children. See the PDQ summary on Adult Central Nervous System Tumors Treatment for more information on the treatment of adults. The treatment of metastatic brain tumors, which begin in other parts of the body and spread to the brain, is not discussed in this summary. The different types of CNS embryonal tumors include: Most CNS embryonal tumors are medulloblastomas. Medulloblastomas are fast-growing tumors that form in brain cells in the cerebellum. The cerebellum is at the lower back part of the brain between the cerebrum and the brain stem. The cerebellum controls movement, balance, and posture. Medulloblastomas sometimes spread to the bone, bone marrow, lung, or other parts of the body, but this is rare. Nonmedulloblastoma embryonal tumors are fast-growing tumors that usually form in brain cells in the cerebrum. The cerebrum is at the top of the head and is the largest part of the brain. The cerebrum controls thinking, learning, problem-solving, emotions, speech, reading, writing, and voluntary movement. Nonmedulloblastoma embryonal tumors may also form in the brain stem or spinal cord. These tumors sometimes spread to the bone, bone marrow, lung, or other parts of the body, but this is rare. There are six types of nonmedulloblastoma embryonal tumors: Embryonal tumors with multilayered rosettes (ETMR) are rare tumors that form in the brain and spinal cord. ETMR most commonly occur in young children and are fast-growing tumors. Medulloepitheliomas are fast-growing tumors that usually form in the brain, spinal cord, or nerves just outside the spinal column. They occur most often in infants and young children. CNS neuroblastomas are a very rare type of neuroblastoma that form in the nerve tissue of the cerebrum or the layers of tissue that cover the brain and spinal cord. CNS neuroblastomas may be large and spread to other parts of the brain or spinal cord. CNS ganglioneuroblastomas are rare tumors that form in nerve tissue of the brain and spinal cord. They may form in one area and be fast growing or form in more than one area and be slow growing. Central nervous system embryonal tumor, not otherwise specified (NOS) was previously called primitive neuroectodermal tumors (PNET). These are rare tumors that form in the brain and spinal cord. CNS embryonal tumor, NOS most commonly occurs in young children. CNS embryonal tumor with rhabdoid features is a rare tumor that forms in the brain and spinal cord. These tumors are similar to CNS atypical teratoid/rhabdoid tumors but lack certain gene changes. CNS atypical teratoid/rhabdoid tumor is also a type of embryonal tumor, but it is treated differently than other childhood CNS embryonal tumors. See the PDQ summary on Childhood Central Nervous System Atypical Teratoid/Rhabdoid Tumor Treatment for more information. The pineal gland is a tiny organ in the center of the brain. The gland makes melatonin, a substance that helps control our sleep cycle. Pineoblastomas form in cells of the pineal gland and are usually malignant. Pineoblastomas are fast-growing tumors with cells that look very different from normal pineal gland cells. Pineoblastomas are not a type of CNS embryonal tumor but treatment for them is a lot like treatment for CNS embryonal tumors. Pineoblastoma is linked with inherited changes in the retinoblastoma (RB1) gene. A child with the inherited form of retinoblastoma (cancer that forms in the tissues of the retina) has an increased risk of pineoblastoma. When retinoblastoma forms at the same time as a tumor in or near the pineal gland, it is called trilateral retinoblastoma. MRI (magnetic resonance imaging) testing in children with retinoblastoma may detect pineoblastoma at an early stage when it can be treated successfully. Anything that increases the risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your child’s doctor if you think your child may be at risk. Risk factors for medulloblastoma and other CNS embryonal tumors include having the following inherited diseases: Children with certain gene changes or a family history of cancers linked to changes in the BRCA gene may be considered for genetic testing. Although rare, this is to check whether the child has a cancer predisposition syndrome that places the child at risk for other diseases or types of cancer. In most cases, the cause of medulloblastoma and other CNS embryonal tumors is not known. These and other signs and symptoms may be caused by childhood medulloblastoma and other CNS embryonal tumors or other conditions. Check with your child's doctor if your child has any of the following: Infants and young children with these tumors may be irritable or grow slowly. Also they may not eat well or meet developmental milestones such as sitting, walking, and talking in sentences. The following tests and procedures may be used: If doctors think your child may have a medulloblastoma or other CNS embryonal tumor, a biopsy may be done. For brain tumors, the biopsy is done by removing part of the skull and using a needle to remove a sample of tissue. Sometimes, a computer-guided needle is used to remove the tissue sample. A pathologist views the tissue under a microscope to look for cancer cells. If cancer cells are found, the doctor may remove as much tumor as safely possible during the same surgery. The piece of skull is usually put back in place after the procedure. The following test may be done on the sample of tissue that is removed: The prognosis and treatment options depend on: Staging is the process used to find out how much cancer there is and if cancer has spread. It is important to know the stage in order to plan treatment. There is no standard staging system for childhood medulloblastoma and other central nervous system (CNS) embryonal tumors. Instead, treatment depends on the type of tumor and the child's age (3 years and younger or older than 3 years). Medulloblastomas are called average risk when all of the following are true: Medulloblastomas are called high risk if any of the following are true: In general, cancer is more likely to recur (come back) in patients with a high-risk tumor. Some of the tests used to detect childhood medulloblastoma and other CNS embryonal tumors are repeated after surgery to remove the tumor. (See the General Information section.) This is to find out how much tumor remains after surgery. Other tests and procedures may be done to find out if the cancer has spread: Childhood medulloblastoma and other CNS embryonal tumors most often recur (come back) within 3 years after treatment but may come back many years later. Recurrent childhood medulloblastoma and other CNS embryonal tumors may come back in the same place as the original tumor and/or in a different place in the brain or spinal cord. Medulloblastoma and other CNS embryonal tumors rarely spread to other parts of the body. Different types of treatment are available for children with medulloblastoma and other central nervous system (CNS) embryonal tumors. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating children with brain tumors and who specialize in certain areas of medicine. These may include the following specialists: Signs or symptoms caused by the tumor may begin before the cancer is diagnosed and continue for months or years. It is important to talk with your child's doctors about signs or symptoms caused by the tumor that may continue after treatment. Surgery is used to diagnose and treat childhood medulloblastoma and other CNS embryonal tumors as described in the General Information section of this summary. After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy, radiation therapy, or both after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Certain ways of giving radiation therapy can help keep radiation from damaging nearby healthy tissue. These types of radiation therapy include the following: Because radiation therapy can affect growth and brain development in young children, especially children who are 3 years old or younger, chemotherapy may be given to delay or reduce the need for radiation therapy. Radiation therapy to the brain can also affect growth and development in children older than three. For this reason, clinical trials are studying new ways of giving radiation that may have fewer side effects than standard methods. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Although most cannot, some chemotherapy drugs can cross the blood-brain barrier and reach tumor cells in the brain. When chemotherapy is placed directly into the cerebrospinal fluid, it is called intrathecal chemotherapy. Intraventricular chemotherapy is a treatment in which anticancer drugs are placed into the ventricles (fluid-filled cavities) of the brain. Combination chemotherapy is treatment using more than one anticancer drug. The way the chemotherapy is given depends on the type of cancer being treated. EnlargeIntrathecal chemotherapy. Anticancer drugs are injected into the intrathecal space, which is the space that holds the cerebrospinal fluid (CSF, shown in blue). There are two different ways to do this. One way, shown in the top part of the figure, is to inject the drugs into an Ommaya reservoir (a dome-shaped container that is placed under the scalp during surgery; it holds the drugs as they flow through a small tube into the brain). The other way, shown in the bottom part of the figure, is to inject the drugs directly into the CSF in the lower part of the spinal column, after a small area on the lower back is numbed. High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Signal transduction inhibitors: This treatment blocks signals that are passed from one molecule to another inside a cell. Blocking these signals may kill cancer cells. Vismodegib is a type of signal transduction inhibitor that may be used to treat recurrent medulloblastoma in children who have finished growing. Targeted therapy is being studied for the treatment of childhood medulloblastoma and other CNS embryonal tumors that have recurred (come back). Information about clinical trials is available from the NCI website. For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Children diagnosed with medulloblastoma may have certain problems after surgery or radiation therapy such as changes in the ability to think, learn, and pay attention. Also, cerebellar mutism syndrome may occur after surgery. Signs of this syndrome include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child and the types of symptoms to expect after cancer treatment has ended. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information). For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. (See the General Information section for a list of tests.) Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. This is sometimes called re-staging. Some of the imaging tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the brain tumor has recurred (come back). If the imaging tests show abnormal tissue in the brain, a biopsy may also be done to find out if the tissue is made up of dead tumor cells or if new cancer cells are growing. These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Younger children with medulloblastoma Standard treatment of newly diagnosed medulloblastoma in children aged 3 years and younger includes the following: Other treatments that may be given after surgery include the following: Children older than 3 years with average-risk medulloblastoma Standard treatment of newly diagnosed average-risk medulloblastoma in children older than 3 years includes the following: Children older than 3 years with high-risk medulloblastoma Standard treatment of newly diagnosed high-risk medulloblastoma in children older than 3 years includes the following: For information about the treatments listed below, see the Treatment Option Overview section. Children aged 3 years and younger with nonmedulloblastoma, nonmedulloepithelioma embryonal tumors, embryonal tumors with multilayered rosettes, or medulloepithelioma Standard treatment of newly diagnosed nonmedulloblastoma, nonmedulloepithelioma embryonal tumors, embryonal tumors with multilayered rosettes, or medulloepithelioma in children 3 years or younger includes the following: Other treatments that may be given after surgery include the following: Children older than 3 years with nonmedulloblastoma, nonmedulloepithelioma embryonal tumors Standard treatment of newly diagnosed nonmedulloblastoma, nonmedulloepithelioma embryonal tumors in children older than 3 years includes the following: Children older than 3 years with embryonal tumors with multilayered rosettes or medulloepithelioma Standard treatment of newly diagnosed embryonal tumor with multilayered rosettes (ETMR) or medulloepithelioma includes the following: For information about the treatments listed below, see the Treatment Option Overview section. Children aged 3 years and younger Standard treatment of newly diagnosed pineoblastoma in children aged 3 years and younger includes the following: Children older than 3 years Standard treatment of newly diagnosed pineoblastoma in children older than 3 years includes the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment for recurrent childhood medulloblastoma and other CNS embryonal tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information about childhood medulloblastoma and other central nervous system embryonal tumor, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood medulloblastoma and other central nervous system embryonal tumors. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Medulloblastoma and Other Central Nervous System Embryonal Tumors Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/brain/patient/child-cns-embryonal-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389401] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Childhood Medulloblastoma and Other Central Nervous System Embryonal Tumors Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is (are) Childhood Central Nervous System Embryonal Tumors ?

Key Points - Central nervous system (CNS) embryonal tumors may begin in embryonic (fetal) cells that remain in the brain after birth. - There are different types of CNS embryonal tumors. - Pineoblastomas form in cells of the pineal gland. - Certain genetic conditions increase the risk of childhood CNS embryonal tumors. - Signs and symptoms of childhood CNS embryonal tumors or pineoblastomas depend on the child&apos;s age and where the tumor is. - Tests that examine the brain and spinal cord are used to detect (find) childhood CNS embryonal tumors or pineoblastomas. - A biopsy may be done to be sure of the diagnosis of CNS embryonal tumor or pineoblastoma. - Certain factors affect prognosis (chance of recovery) and treatment options. Central nervous system (CNS) embryonal tumors may begin in embryonic (fetal) cells that remain in the brain after birth. Central nervous system (CNS) embryonal tumors form in embryonic cells that remain in the brain after birth. CNS embryonal tumors tend to spread through the cerebrospinal fluid (CSF) to other parts of the brain and spinal cord. The tumors may be malignant (cancer) or benign (not cancer). Most CNS embryonal tumors in children are malignant. Malignant brain tumors are likely to grow quickly and spread into other parts of the brain. When a tumor grows into or presses on an area of the brain, it may stop that part of the brain from working the way it should. Benign brain tumors grow and press on nearby areas of the brain. They rarely spread to other parts of the brain. Both benign and malignant brain tumors can cause signs or symptoms and need treatment. Although cancer is rare in children, brain tumors are the third most common type of childhood cancer, after leukemia and lymphoma. This summary is about the treatment of primary brain tumors (tumors that begin in the brain). The treatment of metastatic brain tumors, which begin in other parts of the body and spread to the brain, is not discussed in this summary. For information about the different types of brain and spinal cord tumors, see the PDQ summary on Childhood Brain and Spinal Cord Tumors Treatment Overview. Brain tumors occur in both children and adults. Treatment for adults may be different from treatment for children. See the PDQ summary on Adult Central Nervous System Tumors Treatment for more information on the treatment of adults. There are different types of CNS embryonal tumors. The different types of CNS embryonal tumors include: - Medulloblastomas Most CNS embryonal tumors are medulloblastomas. Medulloblastomas are fast-growing tumors that form in brain cells in the cerebellum. The cerebellum is at the lower back part of the brain between the cerebrum and the brain stem. The cerebellum controls movement, balance, and posture. Medulloblastomas sometimes spread to the bone, bone marrow, lung, or other parts of the body, but this is rare. - Nonmedulloblastoma embryonal tumors Nonmedulloblastoma embryonal tumors are fast-growing tumors that usually form in brain cells in the cerebrum. The cerebrum is at the top of the head and is the largest part of the brain. The cerebrum controls thinking, learning, problem-solving, emotions, speech, reading, writing, and voluntary movement. Nonmedulloblastoma embryonal tumors may also form in the brain stem or spinal cord. There are four types of nonmedulloblastoma embryonal tumors: - Embryonal tumors with multilayered rosettes Embryonal tumors with multilayered rosettes (ETMR) are rare tumors that form in the brain and spinal cord. ETMR most commonly occur in young children and are fast-growing tumors. - Medulloepitheliomas Medulloepitheliomas are fast-growing tumors that usually form in the brain, spinal cord or nerves just outside the spinal column. They occur most often in infants and young children. - CNS neuroblastomas CNS neuroblastomas are a very rare type of neuroblastoma that form in the nerve tissue of the cerebrum or the layers of tissue that cover the brain and spinal cord. CNS neuroblastomas may be large and spread to other parts of the brain or spinal cord. - CNS ganglioneuroblastomas CNS ganglioneuroblastomas are rare tumors that form in nerve tissue of the brain and spinal cord. They may form in one area and be fast growing or form in more than one area and be slow growing. Childhood CNS atypical teratoid/rhabdoid tumor is also a type of embryonal tumor, but it is treated differently than other childhood CNS embryonal tumors. See the PDQ summary on Childhood Central Nervous System Atypical Teratoid/Rhabdoid Tumor Treatment for more information. Pineoblastomas form in cells of the pineal gland. The pineal gland is a tiny organ in the center of the brain. The gland makes melatonin, a substance that helps control our sleep cycle. Pineoblastomas form in cells of the pineal gland and are usually malignant. Pineoblastomas are fast-growing tumors with cells that look very different from normal pineal gland cells. Pineoblastomas are not a type of CNS embryonal tumor but treatment for them is a lot like treatment for CNS embryonal tumors. Pineoblastoma is linked with inherited changes in the retinoblastoma (RB1) gene. A child with the inherited form of retinoblastoma (cancer than forms in the tissues of the retina) has an increased risk of pineoblastoma. When retinoblastoma forms at the same time as a tumor in or near the pineal gland, it is called trilateral retinoblastoma. MRI (magnetic resonance imaging) testing in children with retinoblastoma may detect pineoblastoma at an early stage when it can be treated successfully.

These tumors tend to spread through the cerebrospinal fluid (CSF) to other parts of the brain and spinal cord. Medulloblastoma and other CNS embryonal tumors may be malignant (cancer) or benign (not cancer). Most of the tumors in children are malignant. Malignant brain tumors are likely to grow quickly and spread into other parts of the brain. When a tumor grows into or presses on an area of the brain, it may stop that part of the brain from working the way it should. Benign brain tumors grow and press on nearby areas of the brain. They rarely spread to other parts of the brain. Both benign and malignant brain tumors can cause signs or symptoms and need treatment. Although cancer is rare in children, brain tumors are the second most common type of childhood cancer, after leukemia. This summary is about the treatment of primary brain tumors (tumors that begin in the brain). For information about the different types of brain and spinal cord tumors, see the PDQ summary on Childhood Brain and Spinal Cord Tumors Treatment Overview. Brain tumors occur in both children and adults. Treatment for adults may be different from treatment for children. See the PDQ summary on Adult Central Nervous System Tumors Treatment for more information on the treatment of adults. The treatment of metastatic brain tumors, which begin in other parts of the body and spread to the brain, is not discussed in this summary. The different types of CNS embryonal tumors include: Most CNS embryonal tumors are medulloblastomas. Medulloblastomas are fast-growing tumors that form in brain cells in the cerebellum. The cerebellum is at the lower back part of the brain between the cerebrum and the brain stem. The cerebellum controls movement, balance, and posture. Medulloblastomas sometimes spread to the bone, bone marrow, lung, or other parts of the body, but this is rare. Nonmedulloblastoma embryonal tumors are fast-growing tumors that usually form in brain cells in the cerebrum. The cerebrum is at the top of the head and is the largest part of the brain. The cerebrum controls thinking, learning, problem-solving, emotions, speech, reading, writing, and voluntary movement. Nonmedulloblastoma embryonal tumors may also form in the brain stem or spinal cord. These tumors sometimes spread to the bone, bone marrow, lung, or other parts of the body, but this is rare. There are six types of nonmedulloblastoma embryonal tumors: Embryonal tumors with multilayered rosettes (ETMR) are rare tumors that form in the brain and spinal cord. ETMR most commonly occur in young children and are fast-growing tumors. Medulloepitheliomas are fast-growing tumors that usually form in the brain, spinal cord, or nerves just outside the spinal column. They occur most often in infants and young children. CNS neuroblastomas are a very rare type of neuroblastoma that form in the nerve tissue of the cerebrum or the layers of tissue that cover the brain and spinal cord. CNS neuroblastomas may be large and spread to other parts of the brain or spinal cord. CNS ganglioneuroblastomas are rare tumors that form in nerve tissue of the brain and spinal cord. They may form in one area and be fast growing or form in more than one area and be slow growing. Central nervous system embryonal tumor, not otherwise specified (NOS) was previously called primitive neuroectodermal tumors (PNET). These are rare tumors that form in the brain and spinal cord. CNS embryonal tumor, NOS most commonly occurs in young children. CNS embryonal tumor with rhabdoid features is a rare tumor that forms in the brain and spinal cord. These tumors are similar to CNS atypical teratoid/rhabdoid tumors but lack certain gene changes. CNS atypical teratoid/rhabdoid tumor is also a type of embryonal tumor, but it is treated differently than other childhood CNS embryonal tumors. See the PDQ summary on Childhood Central Nervous System Atypical Teratoid/Rhabdoid Tumor Treatment for more information. The pineal gland is a tiny organ in the center of the brain. The gland makes melatonin, a substance that helps control our sleep cycle. Pineoblastomas form in cells of the pineal gland and are usually malignant. Pineoblastomas are fast-growing tumors with cells that look very different from normal pineal gland cells. Pineoblastomas are not a type of CNS embryonal tumor but treatment for them is a lot like treatment for CNS embryonal tumors. Pineoblastoma is linked with inherited changes in the retinoblastoma (RB1) gene. A child with the inherited form of retinoblastoma (cancer that forms in the tissues of the retina) has an increased risk of pineoblastoma. When retinoblastoma forms at the same time as a tumor in or near the pineal gland, it is called trilateral retinoblastoma. MRI (magnetic resonance imaging) testing in children with retinoblastoma may detect pineoblastoma at an early stage when it can be treated successfully. Anything that increases the risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your child’s doctor if you think your child may be at risk. Risk factors for medulloblastoma and other CNS embryonal tumors include having the following inherited diseases: Children with certain gene changes or a family history of cancers linked to changes in the BRCA gene may be considered for genetic testing. Although rare, this is to check whether the child has a cancer predisposition syndrome that places the child at risk for other diseases or types of cancer. In most cases, the cause of medulloblastoma and other CNS embryonal tumors is not known. These and other signs and symptoms may be caused by childhood medulloblastoma and other CNS embryonal tumors or other conditions. Check with your child's doctor if your child has any of the following: Infants and young children with these tumors may be irritable or grow slowly. Also they may not eat well or meet developmental milestones such as sitting, walking, and talking in sentences. The following tests and procedures may be used: If doctors think your child may have a medulloblastoma or other CNS embryonal tumor, a biopsy may be done. For brain tumors, the biopsy is done by removing part of the skull and using a needle to remove a sample of tissue. Sometimes, a computer-guided needle is used to remove the tissue sample. A pathologist views the tissue under a microscope to look for cancer cells. If cancer cells are found, the doctor may remove as much tumor as safely possible during the same surgery. The piece of skull is usually put back in place after the procedure. The following test may be done on the sample of tissue that is removed: The prognosis and treatment options depend on: Staging is the process used to find out how much cancer there is and if cancer has spread. It is important to know the stage in order to plan treatment. There is no standard staging system for childhood medulloblastoma and other central nervous system (CNS) embryonal tumors. Instead, treatment depends on the type of tumor and the child's age (3 years and younger or older than 3 years). Medulloblastomas are called average risk when all of the following are true: Medulloblastomas are called high risk if any of the following are true: In general, cancer is more likely to recur (come back) in patients with a high-risk tumor. Some of the tests used to detect childhood medulloblastoma and other CNS embryonal tumors are repeated after surgery to remove the tumor. (See the General Information section.) This is to find out how much tumor remains after surgery. Other tests and procedures may be done to find out if the cancer has spread: Childhood medulloblastoma and other CNS embryonal tumors most often recur (come back) within 3 years after treatment but may come back many years later. Recurrent childhood medulloblastoma and other CNS embryonal tumors may come back in the same place as the original tumor and/or in a different place in the brain or spinal cord. Medulloblastoma and other CNS embryonal tumors rarely spread to other parts of the body. Different types of treatment are available for children with medulloblastoma and other central nervous system (CNS) embryonal tumors. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating children with brain tumors and who specialize in certain areas of medicine. These may include the following specialists: Signs or symptoms caused by the tumor may begin before the cancer is diagnosed and continue for months or years. It is important to talk with your child's doctors about signs or symptoms caused by the tumor that may continue after treatment. Surgery is used to diagnose and treat childhood medulloblastoma and other CNS embryonal tumors as described in the General Information section of this summary. After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy, radiation therapy, or both after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Certain ways of giving radiation therapy can help keep radiation from damaging nearby healthy tissue. These types of radiation therapy include the following: Because radiation therapy can affect growth and brain development in young children, especially children who are 3 years old or younger, chemotherapy may be given to delay or reduce the need for radiation therapy. Radiation therapy to the brain can also affect growth and development in children older than three. For this reason, clinical trials are studying new ways of giving radiation that may have fewer side effects than standard methods. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Although most cannot, some chemotherapy drugs can cross the blood-brain barrier and reach tumor cells in the brain. When chemotherapy is placed directly into the cerebrospinal fluid, it is called intrathecal chemotherapy. Intraventricular chemotherapy is a treatment in which anticancer drugs are placed into the ventricles (fluid-filled cavities) of the brain. Combination chemotherapy is treatment using more than one anticancer drug. The way the chemotherapy is given depends on the type of cancer being treated. EnlargeIntrathecal chemotherapy. Anticancer drugs are injected into the intrathecal space, which is the space that holds the cerebrospinal fluid (CSF, shown in blue). There are two different ways to do this. One way, shown in the top part of the figure, is to inject the drugs into an Ommaya reservoir (a dome-shaped container that is placed under the scalp during surgery; it holds the drugs as they flow through a small tube into the brain). The other way, shown in the bottom part of the figure, is to inject the drugs directly into the CSF in the lower part of the spinal column, after a small area on the lower back is numbed. High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Signal transduction inhibitors: This treatment blocks signals that are passed from one molecule to another inside a cell. Blocking these signals may kill cancer cells. Vismodegib is a type of signal transduction inhibitor that may be used to treat recurrent medulloblastoma in children who have finished growing. Targeted therapy is being studied for the treatment of childhood medulloblastoma and other CNS embryonal tumors that have recurred (come back). Information about clinical trials is available from the NCI website. For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Children diagnosed with medulloblastoma may have certain problems after surgery or radiation therapy such as changes in the ability to think, learn, and pay attention. Also, cerebellar mutism syndrome may occur after surgery. Signs of this syndrome include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child and the types of symptoms to expect after cancer treatment has ended. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information). For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. (See the General Information section for a list of tests.) Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. This is sometimes called re-staging. Some of the imaging tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the brain tumor has recurred (come back). If the imaging tests show abnormal tissue in the brain, a biopsy may also be done to find out if the tissue is made up of dead tumor cells or if new cancer cells are growing. These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Younger children with medulloblastoma Standard treatment of newly diagnosed medulloblastoma in children aged 3 years and younger includes the following: Other treatments that may be given after surgery include the following: Children older than 3 years with average-risk medulloblastoma Standard treatment of newly diagnosed average-risk medulloblastoma in children older than 3 years includes the following: Children older than 3 years with high-risk medulloblastoma Standard treatment of newly diagnosed high-risk medulloblastoma in children older than 3 years includes the following: For information about the treatments listed below, see the Treatment Option Overview section. Children aged 3 years and younger with nonmedulloblastoma, nonmedulloepithelioma embryonal tumors, embryonal tumors with multilayered rosettes, or medulloepithelioma Standard treatment of newly diagnosed nonmedulloblastoma, nonmedulloepithelioma embryonal tumors, embryonal tumors with multilayered rosettes, or medulloepithelioma in children 3 years or younger includes the following: Other treatments that may be given after surgery include the following: Children older than 3 years with nonmedulloblastoma, nonmedulloepithelioma embryonal tumors Standard treatment of newly diagnosed nonmedulloblastoma, nonmedulloepithelioma embryonal tumors in children older than 3 years includes the following: Children older than 3 years with embryonal tumors with multilayered rosettes or medulloepithelioma Standard treatment of newly diagnosed embryonal tumor with multilayered rosettes (ETMR) or medulloepithelioma includes the following: For information about the treatments listed below, see the Treatment Option Overview section. Children aged 3 years and younger Standard treatment of newly diagnosed pineoblastoma in children aged 3 years and younger includes the following: Children older than 3 years Standard treatment of newly diagnosed pineoblastoma in children older than 3 years includes the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment for recurrent childhood medulloblastoma and other CNS embryonal tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information about childhood medulloblastoma and other central nervous system embryonal tumor, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood medulloblastoma and other central nervous system embryonal tumors. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Medulloblastoma and Other Central Nervous System Embryonal Tumors Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/brain/patient/child-cns-embryonal-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389401] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Childhood Medulloblastoma and Other Central Nervous System Embryonal Tumors Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

Who is at risk for Childhood Central Nervous System Embryonal Tumors? ?

Certain genetic conditions increase the risk of childhood CNS embryonal tumors. Anything that increases the risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesnt mean that you will not get cancer. Talk with your childs doctor if you think your child may be at risk. Risk factors for CNS embryonal tumors include having the following inherited diseases: - Turcot syndrome. - Rubinstein-Taybi syndrome. - Nevoid basal cell carcinoma (Gorlin) syndrome. - Li-Fraumeni syndrome. - Fanconi anemia. In most cases, the cause of CNS embryonal tumors is not known.

These tumors tend to spread through the cerebrospinal fluid (CSF) to other parts of the brain and spinal cord. Medulloblastoma and other CNS embryonal tumors may be malignant (cancer) or benign (not cancer). Most of the tumors in children are malignant. Malignant brain tumors are likely to grow quickly and spread into other parts of the brain. When a tumor grows into or presses on an area of the brain, it may stop that part of the brain from working the way it should. Benign brain tumors grow and press on nearby areas of the brain. They rarely spread to other parts of the brain. Both benign and malignant brain tumors can cause signs or symptoms and need treatment. Although cancer is rare in children, brain tumors are the second most common type of childhood cancer, after leukemia. This summary is about the treatment of primary brain tumors (tumors that begin in the brain). For information about the different types of brain and spinal cord tumors, see the PDQ summary on Childhood Brain and Spinal Cord Tumors Treatment Overview. Brain tumors occur in both children and adults. Treatment for adults may be different from treatment for children. See the PDQ summary on Adult Central Nervous System Tumors Treatment for more information on the treatment of adults. The treatment of metastatic brain tumors, which begin in other parts of the body and spread to the brain, is not discussed in this summary. The different types of CNS embryonal tumors include: Most CNS embryonal tumors are medulloblastomas. Medulloblastomas are fast-growing tumors that form in brain cells in the cerebellum. The cerebellum is at the lower back part of the brain between the cerebrum and the brain stem. The cerebellum controls movement, balance, and posture. Medulloblastomas sometimes spread to the bone, bone marrow, lung, or other parts of the body, but this is rare. Nonmedulloblastoma embryonal tumors are fast-growing tumors that usually form in brain cells in the cerebrum. The cerebrum is at the top of the head and is the largest part of the brain. The cerebrum controls thinking, learning, problem-solving, emotions, speech, reading, writing, and voluntary movement. Nonmedulloblastoma embryonal tumors may also form in the brain stem or spinal cord. These tumors sometimes spread to the bone, bone marrow, lung, or other parts of the body, but this is rare. There are six types of nonmedulloblastoma embryonal tumors: Embryonal tumors with multilayered rosettes (ETMR) are rare tumors that form in the brain and spinal cord. ETMR most commonly occur in young children and are fast-growing tumors. Medulloepitheliomas are fast-growing tumors that usually form in the brain, spinal cord, or nerves just outside the spinal column. They occur most often in infants and young children. CNS neuroblastomas are a very rare type of neuroblastoma that form in the nerve tissue of the cerebrum or the layers of tissue that cover the brain and spinal cord. CNS neuroblastomas may be large and spread to other parts of the brain or spinal cord. CNS ganglioneuroblastomas are rare tumors that form in nerve tissue of the brain and spinal cord. They may form in one area and be fast growing or form in more than one area and be slow growing. Central nervous system embryonal tumor, not otherwise specified (NOS) was previously called primitive neuroectodermal tumors (PNET). These are rare tumors that form in the brain and spinal cord. CNS embryonal tumor, NOS most commonly occurs in young children. CNS embryonal tumor with rhabdoid features is a rare tumor that forms in the brain and spinal cord. These tumors are similar to CNS atypical teratoid/rhabdoid tumors but lack certain gene changes. CNS atypical teratoid/rhabdoid tumor is also a type of embryonal tumor, but it is treated differently than other childhood CNS embryonal tumors. See the PDQ summary on Childhood Central Nervous System Atypical Teratoid/Rhabdoid Tumor Treatment for more information. The pineal gland is a tiny organ in the center of the brain. The gland makes melatonin, a substance that helps control our sleep cycle. Pineoblastomas form in cells of the pineal gland and are usually malignant. Pineoblastomas are fast-growing tumors with cells that look very different from normal pineal gland cells. Pineoblastomas are not a type of CNS embryonal tumor but treatment for them is a lot like treatment for CNS embryonal tumors. Pineoblastoma is linked with inherited changes in the retinoblastoma (RB1) gene. A child with the inherited form of retinoblastoma (cancer that forms in the tissues of the retina) has an increased risk of pineoblastoma. When retinoblastoma forms at the same time as a tumor in or near the pineal gland, it is called trilateral retinoblastoma. MRI (magnetic resonance imaging) testing in children with retinoblastoma may detect pineoblastoma at an early stage when it can be treated successfully. Anything that increases the risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your child’s doctor if you think your child may be at risk. Risk factors for medulloblastoma and other CNS embryonal tumors include having the following inherited diseases: Children with certain gene changes or a family history of cancers linked to changes in the BRCA gene may be considered for genetic testing. Although rare, this is to check whether the child has a cancer predisposition syndrome that places the child at risk for other diseases or types of cancer. In most cases, the cause of medulloblastoma and other CNS embryonal tumors is not known. These and other signs and symptoms may be caused by childhood medulloblastoma and other CNS embryonal tumors or other conditions. Check with your child's doctor if your child has any of the following: Infants and young children with these tumors may be irritable or grow slowly. Also they may not eat well or meet developmental milestones such as sitting, walking, and talking in sentences. The following tests and procedures may be used: If doctors think your child may have a medulloblastoma or other CNS embryonal tumor, a biopsy may be done. For brain tumors, the biopsy is done by removing part of the skull and using a needle to remove a sample of tissue. Sometimes, a computer-guided needle is used to remove the tissue sample. A pathologist views the tissue under a microscope to look for cancer cells. If cancer cells are found, the doctor may remove as much tumor as safely possible during the same surgery. The piece of skull is usually put back in place after the procedure. The following test may be done on the sample of tissue that is removed: The prognosis and treatment options depend on: Staging is the process used to find out how much cancer there is and if cancer has spread. It is important to know the stage in order to plan treatment. There is no standard staging system for childhood medulloblastoma and other central nervous system (CNS) embryonal tumors. Instead, treatment depends on the type of tumor and the child's age (3 years and younger or older than 3 years). Medulloblastomas are called average risk when all of the following are true: Medulloblastomas are called high risk if any of the following are true: In general, cancer is more likely to recur (come back) in patients with a high-risk tumor. Some of the tests used to detect childhood medulloblastoma and other CNS embryonal tumors are repeated after surgery to remove the tumor. (See the General Information section.) This is to find out how much tumor remains after surgery. Other tests and procedures may be done to find out if the cancer has spread: Childhood medulloblastoma and other CNS embryonal tumors most often recur (come back) within 3 years after treatment but may come back many years later. Recurrent childhood medulloblastoma and other CNS embryonal tumors may come back in the same place as the original tumor and/or in a different place in the brain or spinal cord. Medulloblastoma and other CNS embryonal tumors rarely spread to other parts of the body. Different types of treatment are available for children with medulloblastoma and other central nervous system (CNS) embryonal tumors. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating children with brain tumors and who specialize in certain areas of medicine. These may include the following specialists: Signs or symptoms caused by the tumor may begin before the cancer is diagnosed and continue for months or years. It is important to talk with your child's doctors about signs or symptoms caused by the tumor that may continue after treatment. Surgery is used to diagnose and treat childhood medulloblastoma and other CNS embryonal tumors as described in the General Information section of this summary. After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy, radiation therapy, or both after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Certain ways of giving radiation therapy can help keep radiation from damaging nearby healthy tissue. These types of radiation therapy include the following: Because radiation therapy can affect growth and brain development in young children, especially children who are 3 years old or younger, chemotherapy may be given to delay or reduce the need for radiation therapy. Radiation therapy to the brain can also affect growth and development in children older than three. For this reason, clinical trials are studying new ways of giving radiation that may have fewer side effects than standard methods. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Although most cannot, some chemotherapy drugs can cross the blood-brain barrier and reach tumor cells in the brain. When chemotherapy is placed directly into the cerebrospinal fluid, it is called intrathecal chemotherapy. Intraventricular chemotherapy is a treatment in which anticancer drugs are placed into the ventricles (fluid-filled cavities) of the brain. Combination chemotherapy is treatment using more than one anticancer drug. The way the chemotherapy is given depends on the type of cancer being treated. EnlargeIntrathecal chemotherapy. Anticancer drugs are injected into the intrathecal space, which is the space that holds the cerebrospinal fluid (CSF, shown in blue). There are two different ways to do this. One way, shown in the top part of the figure, is to inject the drugs into an Ommaya reservoir (a dome-shaped container that is placed under the scalp during surgery; it holds the drugs as they flow through a small tube into the brain). The other way, shown in the bottom part of the figure, is to inject the drugs directly into the CSF in the lower part of the spinal column, after a small area on the lower back is numbed. High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Signal transduction inhibitors: This treatment blocks signals that are passed from one molecule to another inside a cell. Blocking these signals may kill cancer cells. Vismodegib is a type of signal transduction inhibitor that may be used to treat recurrent medulloblastoma in children who have finished growing. Targeted therapy is being studied for the treatment of childhood medulloblastoma and other CNS embryonal tumors that have recurred (come back). Information about clinical trials is available from the NCI website. For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Children diagnosed with medulloblastoma may have certain problems after surgery or radiation therapy such as changes in the ability to think, learn, and pay attention. Also, cerebellar mutism syndrome may occur after surgery. Signs of this syndrome include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child and the types of symptoms to expect after cancer treatment has ended. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information). For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. (See the General Information section for a list of tests.) Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. This is sometimes called re-staging. Some of the imaging tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the brain tumor has recurred (come back). If the imaging tests show abnormal tissue in the brain, a biopsy may also be done to find out if the tissue is made up of dead tumor cells or if new cancer cells are growing. These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Younger children with medulloblastoma Standard treatment of newly diagnosed medulloblastoma in children aged 3 years and younger includes the following: Other treatments that may be given after surgery include the following: Children older than 3 years with average-risk medulloblastoma Standard treatment of newly diagnosed average-risk medulloblastoma in children older than 3 years includes the following: Children older than 3 years with high-risk medulloblastoma Standard treatment of newly diagnosed high-risk medulloblastoma in children older than 3 years includes the following: For information about the treatments listed below, see the Treatment Option Overview section. Children aged 3 years and younger with nonmedulloblastoma, nonmedulloepithelioma embryonal tumors, embryonal tumors with multilayered rosettes, or medulloepithelioma Standard treatment of newly diagnosed nonmedulloblastoma, nonmedulloepithelioma embryonal tumors, embryonal tumors with multilayered rosettes, or medulloepithelioma in children 3 years or younger includes the following: Other treatments that may be given after surgery include the following: Children older than 3 years with nonmedulloblastoma, nonmedulloepithelioma embryonal tumors Standard treatment of newly diagnosed nonmedulloblastoma, nonmedulloepithelioma embryonal tumors in children older than 3 years includes the following: Children older than 3 years with embryonal tumors with multilayered rosettes or medulloepithelioma Standard treatment of newly diagnosed embryonal tumor with multilayered rosettes (ETMR) or medulloepithelioma includes the following: For information about the treatments listed below, see the Treatment Option Overview section. Children aged 3 years and younger Standard treatment of newly diagnosed pineoblastoma in children aged 3 years and younger includes the following: Children older than 3 years Standard treatment of newly diagnosed pineoblastoma in children older than 3 years includes the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment for recurrent childhood medulloblastoma and other CNS embryonal tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information about childhood medulloblastoma and other central nervous system embryonal tumor, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood medulloblastoma and other central nervous system embryonal tumors. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. 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In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Childhood Medulloblastoma and Other Central Nervous System Embryonal Tumors Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the symptoms of Childhood Central Nervous System Embryonal Tumors ?

Signs and symptoms of childhood CNS embryonal tumors or pineoblastomas depend on the child&apos;s age and where the tumor is. These and other signs and symptoms may be caused by childhood CNS embryonal tumors, pineoblastomas, or other conditions. Check with your child&apos;s doctor if your child has any of the following: - Loss of balance, trouble walking, worsening handwriting, or slow speech. - Lack of coordination. - Headache, especially in the morning, or headache that goes away after vomiting. - Double vision or other eye problems. - Nausea and vomiting. - General weakness or weakness on one side of the face. - Unusual sleepiness or change in energy level. - Seizures. Infants and young children with these tumors may be irritable or grow slowly. Also they may not eat well or meet developmental milestones such as sitting, walking, and talking in sentences.

These tumors tend to spread through the cerebrospinal fluid (CSF) to other parts of the brain and spinal cord. Medulloblastoma and other CNS embryonal tumors may be malignant (cancer) or benign (not cancer). Most of the tumors in children are malignant. Malignant brain tumors are likely to grow quickly and spread into other parts of the brain. When a tumor grows into or presses on an area of the brain, it may stop that part of the brain from working the way it should. Benign brain tumors grow and press on nearby areas of the brain. They rarely spread to other parts of the brain. Both benign and malignant brain tumors can cause signs or symptoms and need treatment. Although cancer is rare in children, brain tumors are the second most common type of childhood cancer, after leukemia. This summary is about the treatment of primary brain tumors (tumors that begin in the brain). For information about the different types of brain and spinal cord tumors, see the PDQ summary on Childhood Brain and Spinal Cord Tumors Treatment Overview. Brain tumors occur in both children and adults. Treatment for adults may be different from treatment for children. See the PDQ summary on Adult Central Nervous System Tumors Treatment for more information on the treatment of adults. The treatment of metastatic brain tumors, which begin in other parts of the body and spread to the brain, is not discussed in this summary. The different types of CNS embryonal tumors include: Most CNS embryonal tumors are medulloblastomas. Medulloblastomas are fast-growing tumors that form in brain cells in the cerebellum. The cerebellum is at the lower back part of the brain between the cerebrum and the brain stem. The cerebellum controls movement, balance, and posture. Medulloblastomas sometimes spread to the bone, bone marrow, lung, or other parts of the body, but this is rare. Nonmedulloblastoma embryonal tumors are fast-growing tumors that usually form in brain cells in the cerebrum. The cerebrum is at the top of the head and is the largest part of the brain. The cerebrum controls thinking, learning, problem-solving, emotions, speech, reading, writing, and voluntary movement. Nonmedulloblastoma embryonal tumors may also form in the brain stem or spinal cord. These tumors sometimes spread to the bone, bone marrow, lung, or other parts of the body, but this is rare. There are six types of nonmedulloblastoma embryonal tumors: Embryonal tumors with multilayered rosettes (ETMR) are rare tumors that form in the brain and spinal cord. ETMR most commonly occur in young children and are fast-growing tumors. Medulloepitheliomas are fast-growing tumors that usually form in the brain, spinal cord, or nerves just outside the spinal column. They occur most often in infants and young children. CNS neuroblastomas are a very rare type of neuroblastoma that form in the nerve tissue of the cerebrum or the layers of tissue that cover the brain and spinal cord. CNS neuroblastomas may be large and spread to other parts of the brain or spinal cord. CNS ganglioneuroblastomas are rare tumors that form in nerve tissue of the brain and spinal cord. They may form in one area and be fast growing or form in more than one area and be slow growing. Central nervous system embryonal tumor, not otherwise specified (NOS) was previously called primitive neuroectodermal tumors (PNET). These are rare tumors that form in the brain and spinal cord. CNS embryonal tumor, NOS most commonly occurs in young children. CNS embryonal tumor with rhabdoid features is a rare tumor that forms in the brain and spinal cord. These tumors are similar to CNS atypical teratoid/rhabdoid tumors but lack certain gene changes. CNS atypical teratoid/rhabdoid tumor is also a type of embryonal tumor, but it is treated differently than other childhood CNS embryonal tumors. See the PDQ summary on Childhood Central Nervous System Atypical Teratoid/Rhabdoid Tumor Treatment for more information. The pineal gland is a tiny organ in the center of the brain. The gland makes melatonin, a substance that helps control our sleep cycle. Pineoblastomas form in cells of the pineal gland and are usually malignant. Pineoblastomas are fast-growing tumors with cells that look very different from normal pineal gland cells. Pineoblastomas are not a type of CNS embryonal tumor but treatment for them is a lot like treatment for CNS embryonal tumors. Pineoblastoma is linked with inherited changes in the retinoblastoma (RB1) gene. A child with the inherited form of retinoblastoma (cancer that forms in the tissues of the retina) has an increased risk of pineoblastoma. When retinoblastoma forms at the same time as a tumor in or near the pineal gland, it is called trilateral retinoblastoma. MRI (magnetic resonance imaging) testing in children with retinoblastoma may detect pineoblastoma at an early stage when it can be treated successfully. Anything that increases the risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your child’s doctor if you think your child may be at risk. Risk factors for medulloblastoma and other CNS embryonal tumors include having the following inherited diseases: Children with certain gene changes or a family history of cancers linked to changes in the BRCA gene may be considered for genetic testing. Although rare, this is to check whether the child has a cancer predisposition syndrome that places the child at risk for other diseases or types of cancer. In most cases, the cause of medulloblastoma and other CNS embryonal tumors is not known. These and other signs and symptoms may be caused by childhood medulloblastoma and other CNS embryonal tumors or other conditions. Check with your child's doctor if your child has any of the following: Infants and young children with these tumors may be irritable or grow slowly. Also they may not eat well or meet developmental milestones such as sitting, walking, and talking in sentences. The following tests and procedures may be used: If doctors think your child may have a medulloblastoma or other CNS embryonal tumor, a biopsy may be done. For brain tumors, the biopsy is done by removing part of the skull and using a needle to remove a sample of tissue. Sometimes, a computer-guided needle is used to remove the tissue sample. A pathologist views the tissue under a microscope to look for cancer cells. If cancer cells are found, the doctor may remove as much tumor as safely possible during the same surgery. The piece of skull is usually put back in place after the procedure. The following test may be done on the sample of tissue that is removed: The prognosis and treatment options depend on: Staging is the process used to find out how much cancer there is and if cancer has spread. It is important to know the stage in order to plan treatment. There is no standard staging system for childhood medulloblastoma and other central nervous system (CNS) embryonal tumors. Instead, treatment depends on the type of tumor and the child's age (3 years and younger or older than 3 years). Medulloblastomas are called average risk when all of the following are true: Medulloblastomas are called high risk if any of the following are true: In general, cancer is more likely to recur (come back) in patients with a high-risk tumor. Some of the tests used to detect childhood medulloblastoma and other CNS embryonal tumors are repeated after surgery to remove the tumor. (See the General Information section.) This is to find out how much tumor remains after surgery. Other tests and procedures may be done to find out if the cancer has spread: Childhood medulloblastoma and other CNS embryonal tumors most often recur (come back) within 3 years after treatment but may come back many years later. Recurrent childhood medulloblastoma and other CNS embryonal tumors may come back in the same place as the original tumor and/or in a different place in the brain or spinal cord. Medulloblastoma and other CNS embryonal tumors rarely spread to other parts of the body. Different types of treatment are available for children with medulloblastoma and other central nervous system (CNS) embryonal tumors. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating children with brain tumors and who specialize in certain areas of medicine. These may include the following specialists: Signs or symptoms caused by the tumor may begin before the cancer is diagnosed and continue for months or years. It is important to talk with your child's doctors about signs or symptoms caused by the tumor that may continue after treatment. Surgery is used to diagnose and treat childhood medulloblastoma and other CNS embryonal tumors as described in the General Information section of this summary. After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy, radiation therapy, or both after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Certain ways of giving radiation therapy can help keep radiation from damaging nearby healthy tissue. These types of radiation therapy include the following: Because radiation therapy can affect growth and brain development in young children, especially children who are 3 years old or younger, chemotherapy may be given to delay or reduce the need for radiation therapy. Radiation therapy to the brain can also affect growth and development in children older than three. For this reason, clinical trials are studying new ways of giving radiation that may have fewer side effects than standard methods. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Although most cannot, some chemotherapy drugs can cross the blood-brain barrier and reach tumor cells in the brain. When chemotherapy is placed directly into the cerebrospinal fluid, it is called intrathecal chemotherapy. Intraventricular chemotherapy is a treatment in which anticancer drugs are placed into the ventricles (fluid-filled cavities) of the brain. Combination chemotherapy is treatment using more than one anticancer drug. The way the chemotherapy is given depends on the type of cancer being treated. EnlargeIntrathecal chemotherapy. Anticancer drugs are injected into the intrathecal space, which is the space that holds the cerebrospinal fluid (CSF, shown in blue). There are two different ways to do this. One way, shown in the top part of the figure, is to inject the drugs into an Ommaya reservoir (a dome-shaped container that is placed under the scalp during surgery; it holds the drugs as they flow through a small tube into the brain). The other way, shown in the bottom part of the figure, is to inject the drugs directly into the CSF in the lower part of the spinal column, after a small area on the lower back is numbed. High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Signal transduction inhibitors: This treatment blocks signals that are passed from one molecule to another inside a cell. Blocking these signals may kill cancer cells. Vismodegib is a type of signal transduction inhibitor that may be used to treat recurrent medulloblastoma in children who have finished growing. Targeted therapy is being studied for the treatment of childhood medulloblastoma and other CNS embryonal tumors that have recurred (come back). Information about clinical trials is available from the NCI website. For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Children diagnosed with medulloblastoma may have certain problems after surgery or radiation therapy such as changes in the ability to think, learn, and pay attention. Also, cerebellar mutism syndrome may occur after surgery. Signs of this syndrome include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child and the types of symptoms to expect after cancer treatment has ended. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information). For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. (See the General Information section for a list of tests.) Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. This is sometimes called re-staging. Some of the imaging tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the brain tumor has recurred (come back). If the imaging tests show abnormal tissue in the brain, a biopsy may also be done to find out if the tissue is made up of dead tumor cells or if new cancer cells are growing. These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Younger children with medulloblastoma Standard treatment of newly diagnosed medulloblastoma in children aged 3 years and younger includes the following: Other treatments that may be given after surgery include the following: Children older than 3 years with average-risk medulloblastoma Standard treatment of newly diagnosed average-risk medulloblastoma in children older than 3 years includes the following: Children older than 3 years with high-risk medulloblastoma Standard treatment of newly diagnosed high-risk medulloblastoma in children older than 3 years includes the following: For information about the treatments listed below, see the Treatment Option Overview section. Children aged 3 years and younger with nonmedulloblastoma, nonmedulloepithelioma embryonal tumors, embryonal tumors with multilayered rosettes, or medulloepithelioma Standard treatment of newly diagnosed nonmedulloblastoma, nonmedulloepithelioma embryonal tumors, embryonal tumors with multilayered rosettes, or medulloepithelioma in children 3 years or younger includes the following: Other treatments that may be given after surgery include the following: Children older than 3 years with nonmedulloblastoma, nonmedulloepithelioma embryonal tumors Standard treatment of newly diagnosed nonmedulloblastoma, nonmedulloepithelioma embryonal tumors in children older than 3 years includes the following: Children older than 3 years with embryonal tumors with multilayered rosettes or medulloepithelioma Standard treatment of newly diagnosed embryonal tumor with multilayered rosettes (ETMR) or medulloepithelioma includes the following: For information about the treatments listed below, see the Treatment Option Overview section. Children aged 3 years and younger Standard treatment of newly diagnosed pineoblastoma in children aged 3 years and younger includes the following: Children older than 3 years Standard treatment of newly diagnosed pineoblastoma in children older than 3 years includes the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment for recurrent childhood medulloblastoma and other CNS embryonal tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information about childhood medulloblastoma and other central nervous system embryonal tumor, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood medulloblastoma and other central nervous system embryonal tumors. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Medulloblastoma and Other Central Nervous System Embryonal Tumors Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/brain/patient/child-cns-embryonal-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389401] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Childhood Medulloblastoma and Other Central Nervous System Embryonal Tumors Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

How to diagnose Childhood Central Nervous System Embryonal Tumors ?

Tests that examine the brain and spinal cord are used to detect (find) childhood CNS embryonal tumors or pineoblastomas. The following tests and procedures may be used: - Physical exam and history : An exam of the body to check general signs of health, including checking for signs of disease, such as lumps or anything else that seems unusual. A history of the patients health habits and past illnesses and treatments will also be taken. - Neurological exam : A series of questions and tests to check the brain, spinal cord, and nerve function. The exam checks a patient&apos;s mental status, coordination, and ability to walk normally, and how well the muscles, senses, and reflexes work. This may also be called a neuro exam or a neurologic exam. - MRI (magnetic resonance imaging) of the brain and spinal cord with gadolinium : A procedure that uses a magnet, radio waves, and a computer to make a series of detailed pictures of areas inside the brain and spinal cord. A substance called gadolinium is injected into a vein. The gadolinium collects around the cancer cells so they show up brighter in the picture. This procedure is also called nuclear magnetic resonance imaging (NMRI). Sometimes magnetic resonance spectroscopy (MRS) is done during the MRI scan to look at the chemicals in brain tissue. - Lumbar puncture : A procedure used to collect cerebrospinal fluid (CSF) from the spinal column. This is done by placing a needle between two bones in the spine and into the CSF around the spinal cord and removing a sample of the fluid. The sample of CSF is checked under a microscope for signs of tumor cells. The sample may also be checked for the amounts of protein and glucose. A higher than normal amount of protein or lower than normal amount of glucose may be a sign of a tumor. This procedure is also called an LP or spinal tap. A biopsy may be done to be sure of the diagnosis of CNS embryonal tumor or pineoblastoma. If doctors think your child may have a CNS embryonal tumor or pineoblastoma, a biopsy may be done. For brain tumors, the biopsy is done by removing part of the skull and using a needle to remove a sample of tissue. Sometimes, a computer-guided needle is used to remove the tissue sample. A pathologist views the tissue under a microscope to look for cancer cells. If cancer cells are found, the doctor may remove as much tumor as safely possible during the same surgery. The piece of skull is usually put back in place after the procedure. The following test may be done on the sample of tissue that is removed: - Immunohistochemistry : A test that uses antibodies to check for certain antigens in a sample of tissue. The antibody is usually linked to a radioactive substance or a dye that causes the tissue to light up under a microscope. This type of test may be used to tell the difference between different types of brain tumors.

These tumors tend to spread through the cerebrospinal fluid (CSF) to other parts of the brain and spinal cord. Medulloblastoma and other CNS embryonal tumors may be malignant (cancer) or benign (not cancer). Most of the tumors in children are malignant. Malignant brain tumors are likely to grow quickly and spread into other parts of the brain. When a tumor grows into or presses on an area of the brain, it may stop that part of the brain from working the way it should. Benign brain tumors grow and press on nearby areas of the brain. They rarely spread to other parts of the brain. Both benign and malignant brain tumors can cause signs or symptoms and need treatment. Although cancer is rare in children, brain tumors are the second most common type of childhood cancer, after leukemia. This summary is about the treatment of primary brain tumors (tumors that begin in the brain). For information about the different types of brain and spinal cord tumors, see the PDQ summary on Childhood Brain and Spinal Cord Tumors Treatment Overview. Brain tumors occur in both children and adults. Treatment for adults may be different from treatment for children. See the PDQ summary on Adult Central Nervous System Tumors Treatment for more information on the treatment of adults. The treatment of metastatic brain tumors, which begin in other parts of the body and spread to the brain, is not discussed in this summary. The different types of CNS embryonal tumors include: Most CNS embryonal tumors are medulloblastomas. Medulloblastomas are fast-growing tumors that form in brain cells in the cerebellum. The cerebellum is at the lower back part of the brain between the cerebrum and the brain stem. The cerebellum controls movement, balance, and posture. Medulloblastomas sometimes spread to the bone, bone marrow, lung, or other parts of the body, but this is rare. Nonmedulloblastoma embryonal tumors are fast-growing tumors that usually form in brain cells in the cerebrum. The cerebrum is at the top of the head and is the largest part of the brain. The cerebrum controls thinking, learning, problem-solving, emotions, speech, reading, writing, and voluntary movement. Nonmedulloblastoma embryonal tumors may also form in the brain stem or spinal cord. These tumors sometimes spread to the bone, bone marrow, lung, or other parts of the body, but this is rare. There are six types of nonmedulloblastoma embryonal tumors: Embryonal tumors with multilayered rosettes (ETMR) are rare tumors that form in the brain and spinal cord. ETMR most commonly occur in young children and are fast-growing tumors. Medulloepitheliomas are fast-growing tumors that usually form in the brain, spinal cord, or nerves just outside the spinal column. They occur most often in infants and young children. CNS neuroblastomas are a very rare type of neuroblastoma that form in the nerve tissue of the cerebrum or the layers of tissue that cover the brain and spinal cord. CNS neuroblastomas may be large and spread to other parts of the brain or spinal cord. CNS ganglioneuroblastomas are rare tumors that form in nerve tissue of the brain and spinal cord. They may form in one area and be fast growing or form in more than one area and be slow growing. Central nervous system embryonal tumor, not otherwise specified (NOS) was previously called primitive neuroectodermal tumors (PNET). These are rare tumors that form in the brain and spinal cord. CNS embryonal tumor, NOS most commonly occurs in young children. CNS embryonal tumor with rhabdoid features is a rare tumor that forms in the brain and spinal cord. These tumors are similar to CNS atypical teratoid/rhabdoid tumors but lack certain gene changes. CNS atypical teratoid/rhabdoid tumor is also a type of embryonal tumor, but it is treated differently than other childhood CNS embryonal tumors. See the PDQ summary on Childhood Central Nervous System Atypical Teratoid/Rhabdoid Tumor Treatment for more information. The pineal gland is a tiny organ in the center of the brain. The gland makes melatonin, a substance that helps control our sleep cycle. Pineoblastomas form in cells of the pineal gland and are usually malignant. Pineoblastomas are fast-growing tumors with cells that look very different from normal pineal gland cells. Pineoblastomas are not a type of CNS embryonal tumor but treatment for them is a lot like treatment for CNS embryonal tumors. Pineoblastoma is linked with inherited changes in the retinoblastoma (RB1) gene. A child with the inherited form of retinoblastoma (cancer that forms in the tissues of the retina) has an increased risk of pineoblastoma. When retinoblastoma forms at the same time as a tumor in or near the pineal gland, it is called trilateral retinoblastoma. MRI (magnetic resonance imaging) testing in children with retinoblastoma may detect pineoblastoma at an early stage when it can be treated successfully. Anything that increases the risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your child’s doctor if you think your child may be at risk. Risk factors for medulloblastoma and other CNS embryonal tumors include having the following inherited diseases: Children with certain gene changes or a family history of cancers linked to changes in the BRCA gene may be considered for genetic testing. Although rare, this is to check whether the child has a cancer predisposition syndrome that places the child at risk for other diseases or types of cancer. In most cases, the cause of medulloblastoma and other CNS embryonal tumors is not known. These and other signs and symptoms may be caused by childhood medulloblastoma and other CNS embryonal tumors or other conditions. Check with your child's doctor if your child has any of the following: Infants and young children with these tumors may be irritable or grow slowly. Also they may not eat well or meet developmental milestones such as sitting, walking, and talking in sentences. The following tests and procedures may be used: If doctors think your child may have a medulloblastoma or other CNS embryonal tumor, a biopsy may be done. For brain tumors, the biopsy is done by removing part of the skull and using a needle to remove a sample of tissue. Sometimes, a computer-guided needle is used to remove the tissue sample. A pathologist views the tissue under a microscope to look for cancer cells. If cancer cells are found, the doctor may remove as much tumor as safely possible during the same surgery. The piece of skull is usually put back in place after the procedure. The following test may be done on the sample of tissue that is removed: The prognosis and treatment options depend on: Staging is the process used to find out how much cancer there is and if cancer has spread. It is important to know the stage in order to plan treatment. There is no standard staging system for childhood medulloblastoma and other central nervous system (CNS) embryonal tumors. Instead, treatment depends on the type of tumor and the child's age (3 years and younger or older than 3 years). Medulloblastomas are called average risk when all of the following are true: Medulloblastomas are called high risk if any of the following are true: In general, cancer is more likely to recur (come back) in patients with a high-risk tumor. Some of the tests used to detect childhood medulloblastoma and other CNS embryonal tumors are repeated after surgery to remove the tumor. (See the General Information section.) This is to find out how much tumor remains after surgery. Other tests and procedures may be done to find out if the cancer has spread: Childhood medulloblastoma and other CNS embryonal tumors most often recur (come back) within 3 years after treatment but may come back many years later. Recurrent childhood medulloblastoma and other CNS embryonal tumors may come back in the same place as the original tumor and/or in a different place in the brain or spinal cord. Medulloblastoma and other CNS embryonal tumors rarely spread to other parts of the body. Different types of treatment are available for children with medulloblastoma and other central nervous system (CNS) embryonal tumors. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating children with brain tumors and who specialize in certain areas of medicine. These may include the following specialists: Signs or symptoms caused by the tumor may begin before the cancer is diagnosed and continue for months or years. It is important to talk with your child's doctors about signs or symptoms caused by the tumor that may continue after treatment. Surgery is used to diagnose and treat childhood medulloblastoma and other CNS embryonal tumors as described in the General Information section of this summary. After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy, radiation therapy, or both after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Certain ways of giving radiation therapy can help keep radiation from damaging nearby healthy tissue. These types of radiation therapy include the following: Because radiation therapy can affect growth and brain development in young children, especially children who are 3 years old or younger, chemotherapy may be given to delay or reduce the need for radiation therapy. Radiation therapy to the brain can also affect growth and development in children older than three. For this reason, clinical trials are studying new ways of giving radiation that may have fewer side effects than standard methods. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Although most cannot, some chemotherapy drugs can cross the blood-brain barrier and reach tumor cells in the brain. When chemotherapy is placed directly into the cerebrospinal fluid, it is called intrathecal chemotherapy. Intraventricular chemotherapy is a treatment in which anticancer drugs are placed into the ventricles (fluid-filled cavities) of the brain. Combination chemotherapy is treatment using more than one anticancer drug. The way the chemotherapy is given depends on the type of cancer being treated. EnlargeIntrathecal chemotherapy. Anticancer drugs are injected into the intrathecal space, which is the space that holds the cerebrospinal fluid (CSF, shown in blue). There are two different ways to do this. One way, shown in the top part of the figure, is to inject the drugs into an Ommaya reservoir (a dome-shaped container that is placed under the scalp during surgery; it holds the drugs as they flow through a small tube into the brain). The other way, shown in the bottom part of the figure, is to inject the drugs directly into the CSF in the lower part of the spinal column, after a small area on the lower back is numbed. High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Signal transduction inhibitors: This treatment blocks signals that are passed from one molecule to another inside a cell. Blocking these signals may kill cancer cells. Vismodegib is a type of signal transduction inhibitor that may be used to treat recurrent medulloblastoma in children who have finished growing. Targeted therapy is being studied for the treatment of childhood medulloblastoma and other CNS embryonal tumors that have recurred (come back). Information about clinical trials is available from the NCI website. For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Children diagnosed with medulloblastoma may have certain problems after surgery or radiation therapy such as changes in the ability to think, learn, and pay attention. Also, cerebellar mutism syndrome may occur after surgery. Signs of this syndrome include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child and the types of symptoms to expect after cancer treatment has ended. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information). For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. (See the General Information section for a list of tests.) Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. This is sometimes called re-staging. Some of the imaging tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the brain tumor has recurred (come back). If the imaging tests show abnormal tissue in the brain, a biopsy may also be done to find out if the tissue is made up of dead tumor cells or if new cancer cells are growing. These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Younger children with medulloblastoma Standard treatment of newly diagnosed medulloblastoma in children aged 3 years and younger includes the following: Other treatments that may be given after surgery include the following: Children older than 3 years with average-risk medulloblastoma Standard treatment of newly diagnosed average-risk medulloblastoma in children older than 3 years includes the following: Children older than 3 years with high-risk medulloblastoma Standard treatment of newly diagnosed high-risk medulloblastoma in children older than 3 years includes the following: For information about the treatments listed below, see the Treatment Option Overview section. Children aged 3 years and younger with nonmedulloblastoma, nonmedulloepithelioma embryonal tumors, embryonal tumors with multilayered rosettes, or medulloepithelioma Standard treatment of newly diagnosed nonmedulloblastoma, nonmedulloepithelioma embryonal tumors, embryonal tumors with multilayered rosettes, or medulloepithelioma in children 3 years or younger includes the following: Other treatments that may be given after surgery include the following: Children older than 3 years with nonmedulloblastoma, nonmedulloepithelioma embryonal tumors Standard treatment of newly diagnosed nonmedulloblastoma, nonmedulloepithelioma embryonal tumors in children older than 3 years includes the following: Children older than 3 years with embryonal tumors with multilayered rosettes or medulloepithelioma Standard treatment of newly diagnosed embryonal tumor with multilayered rosettes (ETMR) or medulloepithelioma includes the following: For information about the treatments listed below, see the Treatment Option Overview section. Children aged 3 years and younger Standard treatment of newly diagnosed pineoblastoma in children aged 3 years and younger includes the following: Children older than 3 years Standard treatment of newly diagnosed pineoblastoma in children older than 3 years includes the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment for recurrent childhood medulloblastoma and other CNS embryonal tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information about childhood medulloblastoma and other central nervous system embryonal tumor, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood medulloblastoma and other central nervous system embryonal tumors. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Medulloblastoma and Other Central Nervous System Embryonal Tumors Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/brain/patient/child-cns-embryonal-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389401] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Childhood Medulloblastoma and Other Central Nervous System Embryonal Tumors Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is the outlook for Childhood Central Nervous System Embryonal Tumors ?

Certain factors affect prognosis (chance of recovery) and treatment options. The prognosis (chance of recovery) and treatment options depend on: - The type of tumor and where it is in the brain. - Whether the cancer has spread within the brain and spinal cord when the tumor is found. - The age of the child when the tumor is found. - How much of the tumor remains after surgery. - Whether there are certain changes in the chromosomes, genes, or brain cells. - Whether the tumor has just been diagnosed or has recurred (come back).

These tumors tend to spread through the cerebrospinal fluid (CSF) to other parts of the brain and spinal cord. Medulloblastoma and other CNS embryonal tumors may be malignant (cancer) or benign (not cancer). Most of the tumors in children are malignant. Malignant brain tumors are likely to grow quickly and spread into other parts of the brain. When a tumor grows into or presses on an area of the brain, it may stop that part of the brain from working the way it should. Benign brain tumors grow and press on nearby areas of the brain. They rarely spread to other parts of the brain. Both benign and malignant brain tumors can cause signs or symptoms and need treatment. Although cancer is rare in children, brain tumors are the second most common type of childhood cancer, after leukemia. This summary is about the treatment of primary brain tumors (tumors that begin in the brain). For information about the different types of brain and spinal cord tumors, see the PDQ summary on Childhood Brain and Spinal Cord Tumors Treatment Overview. Brain tumors occur in both children and adults. Treatment for adults may be different from treatment for children. See the PDQ summary on Adult Central Nervous System Tumors Treatment for more information on the treatment of adults. The treatment of metastatic brain tumors, which begin in other parts of the body and spread to the brain, is not discussed in this summary. The different types of CNS embryonal tumors include: Most CNS embryonal tumors are medulloblastomas. Medulloblastomas are fast-growing tumors that form in brain cells in the cerebellum. The cerebellum is at the lower back part of the brain between the cerebrum and the brain stem. The cerebellum controls movement, balance, and posture. Medulloblastomas sometimes spread to the bone, bone marrow, lung, or other parts of the body, but this is rare. Nonmedulloblastoma embryonal tumors are fast-growing tumors that usually form in brain cells in the cerebrum. The cerebrum is at the top of the head and is the largest part of the brain. The cerebrum controls thinking, learning, problem-solving, emotions, speech, reading, writing, and voluntary movement. Nonmedulloblastoma embryonal tumors may also form in the brain stem or spinal cord. These tumors sometimes spread to the bone, bone marrow, lung, or other parts of the body, but this is rare. There are six types of nonmedulloblastoma embryonal tumors: Embryonal tumors with multilayered rosettes (ETMR) are rare tumors that form in the brain and spinal cord. ETMR most commonly occur in young children and are fast-growing tumors. Medulloepitheliomas are fast-growing tumors that usually form in the brain, spinal cord, or nerves just outside the spinal column. They occur most often in infants and young children. CNS neuroblastomas are a very rare type of neuroblastoma that form in the nerve tissue of the cerebrum or the layers of tissue that cover the brain and spinal cord. CNS neuroblastomas may be large and spread to other parts of the brain or spinal cord. CNS ganglioneuroblastomas are rare tumors that form in nerve tissue of the brain and spinal cord. They may form in one area and be fast growing or form in more than one area and be slow growing. Central nervous system embryonal tumor, not otherwise specified (NOS) was previously called primitive neuroectodermal tumors (PNET). These are rare tumors that form in the brain and spinal cord. CNS embryonal tumor, NOS most commonly occurs in young children. CNS embryonal tumor with rhabdoid features is a rare tumor that forms in the brain and spinal cord. These tumors are similar to CNS atypical teratoid/rhabdoid tumors but lack certain gene changes. CNS atypical teratoid/rhabdoid tumor is also a type of embryonal tumor, but it is treated differently than other childhood CNS embryonal tumors. See the PDQ summary on Childhood Central Nervous System Atypical Teratoid/Rhabdoid Tumor Treatment for more information. The pineal gland is a tiny organ in the center of the brain. The gland makes melatonin, a substance that helps control our sleep cycle. Pineoblastomas form in cells of the pineal gland and are usually malignant. Pineoblastomas are fast-growing tumors with cells that look very different from normal pineal gland cells. Pineoblastomas are not a type of CNS embryonal tumor but treatment for them is a lot like treatment for CNS embryonal tumors. Pineoblastoma is linked with inherited changes in the retinoblastoma (RB1) gene. A child with the inherited form of retinoblastoma (cancer that forms in the tissues of the retina) has an increased risk of pineoblastoma. When retinoblastoma forms at the same time as a tumor in or near the pineal gland, it is called trilateral retinoblastoma. MRI (magnetic resonance imaging) testing in children with retinoblastoma may detect pineoblastoma at an early stage when it can be treated successfully. Anything that increases the risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your child’s doctor if you think your child may be at risk. Risk factors for medulloblastoma and other CNS embryonal tumors include having the following inherited diseases: Children with certain gene changes or a family history of cancers linked to changes in the BRCA gene may be considered for genetic testing. Although rare, this is to check whether the child has a cancer predisposition syndrome that places the child at risk for other diseases or types of cancer. In most cases, the cause of medulloblastoma and other CNS embryonal tumors is not known. These and other signs and symptoms may be caused by childhood medulloblastoma and other CNS embryonal tumors or other conditions. Check with your child's doctor if your child has any of the following: Infants and young children with these tumors may be irritable or grow slowly. Also they may not eat well or meet developmental milestones such as sitting, walking, and talking in sentences. The following tests and procedures may be used: If doctors think your child may have a medulloblastoma or other CNS embryonal tumor, a biopsy may be done. For brain tumors, the biopsy is done by removing part of the skull and using a needle to remove a sample of tissue. Sometimes, a computer-guided needle is used to remove the tissue sample. A pathologist views the tissue under a microscope to look for cancer cells. If cancer cells are found, the doctor may remove as much tumor as safely possible during the same surgery. The piece of skull is usually put back in place after the procedure. The following test may be done on the sample of tissue that is removed: The prognosis and treatment options depend on: Staging is the process used to find out how much cancer there is and if cancer has spread. It is important to know the stage in order to plan treatment. There is no standard staging system for childhood medulloblastoma and other central nervous system (CNS) embryonal tumors. Instead, treatment depends on the type of tumor and the child's age (3 years and younger or older than 3 years). Medulloblastomas are called average risk when all of the following are true: Medulloblastomas are called high risk if any of the following are true: In general, cancer is more likely to recur (come back) in patients with a high-risk tumor. Some of the tests used to detect childhood medulloblastoma and other CNS embryonal tumors are repeated after surgery to remove the tumor. (See the General Information section.) This is to find out how much tumor remains after surgery. Other tests and procedures may be done to find out if the cancer has spread: Childhood medulloblastoma and other CNS embryonal tumors most often recur (come back) within 3 years after treatment but may come back many years later. Recurrent childhood medulloblastoma and other CNS embryonal tumors may come back in the same place as the original tumor and/or in a different place in the brain or spinal cord. Medulloblastoma and other CNS embryonal tumors rarely spread to other parts of the body. Different types of treatment are available for children with medulloblastoma and other central nervous system (CNS) embryonal tumors. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating children with brain tumors and who specialize in certain areas of medicine. These may include the following specialists: Signs or symptoms caused by the tumor may begin before the cancer is diagnosed and continue for months or years. It is important to talk with your child's doctors about signs or symptoms caused by the tumor that may continue after treatment. Surgery is used to diagnose and treat childhood medulloblastoma and other CNS embryonal tumors as described in the General Information section of this summary. After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy, radiation therapy, or both after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Certain ways of giving radiation therapy can help keep radiation from damaging nearby healthy tissue. These types of radiation therapy include the following: Because radiation therapy can affect growth and brain development in young children, especially children who are 3 years old or younger, chemotherapy may be given to delay or reduce the need for radiation therapy. Radiation therapy to the brain can also affect growth and development in children older than three. For this reason, clinical trials are studying new ways of giving radiation that may have fewer side effects than standard methods. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Although most cannot, some chemotherapy drugs can cross the blood-brain barrier and reach tumor cells in the brain. When chemotherapy is placed directly into the cerebrospinal fluid, it is called intrathecal chemotherapy. Intraventricular chemotherapy is a treatment in which anticancer drugs are placed into the ventricles (fluid-filled cavities) of the brain. Combination chemotherapy is treatment using more than one anticancer drug. The way the chemotherapy is given depends on the type of cancer being treated. EnlargeIntrathecal chemotherapy. Anticancer drugs are injected into the intrathecal space, which is the space that holds the cerebrospinal fluid (CSF, shown in blue). There are two different ways to do this. One way, shown in the top part of the figure, is to inject the drugs into an Ommaya reservoir (a dome-shaped container that is placed under the scalp during surgery; it holds the drugs as they flow through a small tube into the brain). The other way, shown in the bottom part of the figure, is to inject the drugs directly into the CSF in the lower part of the spinal column, after a small area on the lower back is numbed. High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Signal transduction inhibitors: This treatment blocks signals that are passed from one molecule to another inside a cell. Blocking these signals may kill cancer cells. Vismodegib is a type of signal transduction inhibitor that may be used to treat recurrent medulloblastoma in children who have finished growing. Targeted therapy is being studied for the treatment of childhood medulloblastoma and other CNS embryonal tumors that have recurred (come back). Information about clinical trials is available from the NCI website. For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Children diagnosed with medulloblastoma may have certain problems after surgery or radiation therapy such as changes in the ability to think, learn, and pay attention. Also, cerebellar mutism syndrome may occur after surgery. Signs of this syndrome include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child and the types of symptoms to expect after cancer treatment has ended. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information). For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. (See the General Information section for a list of tests.) Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. This is sometimes called re-staging. Some of the imaging tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the brain tumor has recurred (come back). If the imaging tests show abnormal tissue in the brain, a biopsy may also be done to find out if the tissue is made up of dead tumor cells or if new cancer cells are growing. These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Younger children with medulloblastoma Standard treatment of newly diagnosed medulloblastoma in children aged 3 years and younger includes the following: Other treatments that may be given after surgery include the following: Children older than 3 years with average-risk medulloblastoma Standard treatment of newly diagnosed average-risk medulloblastoma in children older than 3 years includes the following: Children older than 3 years with high-risk medulloblastoma Standard treatment of newly diagnosed high-risk medulloblastoma in children older than 3 years includes the following: For information about the treatments listed below, see the Treatment Option Overview section. Children aged 3 years and younger with nonmedulloblastoma, nonmedulloepithelioma embryonal tumors, embryonal tumors with multilayered rosettes, or medulloepithelioma Standard treatment of newly diagnosed nonmedulloblastoma, nonmedulloepithelioma embryonal tumors, embryonal tumors with multilayered rosettes, or medulloepithelioma in children 3 years or younger includes the following: Other treatments that may be given after surgery include the following: Children older than 3 years with nonmedulloblastoma, nonmedulloepithelioma embryonal tumors Standard treatment of newly diagnosed nonmedulloblastoma, nonmedulloepithelioma embryonal tumors in children older than 3 years includes the following: Children older than 3 years with embryonal tumors with multilayered rosettes or medulloepithelioma Standard treatment of newly diagnosed embryonal tumor with multilayered rosettes (ETMR) or medulloepithelioma includes the following: For information about the treatments listed below, see the Treatment Option Overview section. Children aged 3 years and younger Standard treatment of newly diagnosed pineoblastoma in children aged 3 years and younger includes the following: Children older than 3 years Standard treatment of newly diagnosed pineoblastoma in children older than 3 years includes the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment for recurrent childhood medulloblastoma and other CNS embryonal tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information about childhood medulloblastoma and other central nervous system embryonal tumor, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood medulloblastoma and other central nervous system embryonal tumors. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Medulloblastoma and Other Central Nervous System Embryonal Tumors Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/brain/patient/child-cns-embryonal-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389401] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. 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In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Childhood Medulloblastoma and Other Central Nervous System Embryonal Tumors Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

what research (or clinical trials) is being done for Childhood Central Nervous System Embryonal Tumors ?

New types of treatment are being tested in clinical trials. Information about clinical trials is available from the NCI website. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials.

These tumors tend to spread through the cerebrospinal fluid (CSF) to other parts of the brain and spinal cord. Medulloblastoma and other CNS embryonal tumors may be malignant (cancer) or benign (not cancer). Most of the tumors in children are malignant. Malignant brain tumors are likely to grow quickly and spread into other parts of the brain. When a tumor grows into or presses on an area of the brain, it may stop that part of the brain from working the way it should. Benign brain tumors grow and press on nearby areas of the brain. They rarely spread to other parts of the brain. Both benign and malignant brain tumors can cause signs or symptoms and need treatment. Although cancer is rare in children, brain tumors are the second most common type of childhood cancer, after leukemia. This summary is about the treatment of primary brain tumors (tumors that begin in the brain). For information about the different types of brain and spinal cord tumors, see the PDQ summary on Childhood Brain and Spinal Cord Tumors Treatment Overview. Brain tumors occur in both children and adults. Treatment for adults may be different from treatment for children. See the PDQ summary on Adult Central Nervous System Tumors Treatment for more information on the treatment of adults. The treatment of metastatic brain tumors, which begin in other parts of the body and spread to the brain, is not discussed in this summary. The different types of CNS embryonal tumors include: Most CNS embryonal tumors are medulloblastomas. Medulloblastomas are fast-growing tumors that form in brain cells in the cerebellum. The cerebellum is at the lower back part of the brain between the cerebrum and the brain stem. The cerebellum controls movement, balance, and posture. Medulloblastomas sometimes spread to the bone, bone marrow, lung, or other parts of the body, but this is rare. Nonmedulloblastoma embryonal tumors are fast-growing tumors that usually form in brain cells in the cerebrum. The cerebrum is at the top of the head and is the largest part of the brain. The cerebrum controls thinking, learning, problem-solving, emotions, speech, reading, writing, and voluntary movement. Nonmedulloblastoma embryonal tumors may also form in the brain stem or spinal cord. These tumors sometimes spread to the bone, bone marrow, lung, or other parts of the body, but this is rare. There are six types of nonmedulloblastoma embryonal tumors: Embryonal tumors with multilayered rosettes (ETMR) are rare tumors that form in the brain and spinal cord. ETMR most commonly occur in young children and are fast-growing tumors. Medulloepitheliomas are fast-growing tumors that usually form in the brain, spinal cord, or nerves just outside the spinal column. They occur most often in infants and young children. CNS neuroblastomas are a very rare type of neuroblastoma that form in the nerve tissue of the cerebrum or the layers of tissue that cover the brain and spinal cord. CNS neuroblastomas may be large and spread to other parts of the brain or spinal cord. CNS ganglioneuroblastomas are rare tumors that form in nerve tissue of the brain and spinal cord. They may form in one area and be fast growing or form in more than one area and be slow growing. Central nervous system embryonal tumor, not otherwise specified (NOS) was previously called primitive neuroectodermal tumors (PNET). These are rare tumors that form in the brain and spinal cord. CNS embryonal tumor, NOS most commonly occurs in young children. CNS embryonal tumor with rhabdoid features is a rare tumor that forms in the brain and spinal cord. These tumors are similar to CNS atypical teratoid/rhabdoid tumors but lack certain gene changes. CNS atypical teratoid/rhabdoid tumor is also a type of embryonal tumor, but it is treated differently than other childhood CNS embryonal tumors. See the PDQ summary on Childhood Central Nervous System Atypical Teratoid/Rhabdoid Tumor Treatment for more information. The pineal gland is a tiny organ in the center of the brain. The gland makes melatonin, a substance that helps control our sleep cycle. Pineoblastomas form in cells of the pineal gland and are usually malignant. Pineoblastomas are fast-growing tumors with cells that look very different from normal pineal gland cells. Pineoblastomas are not a type of CNS embryonal tumor but treatment for them is a lot like treatment for CNS embryonal tumors. Pineoblastoma is linked with inherited changes in the retinoblastoma (RB1) gene. A child with the inherited form of retinoblastoma (cancer that forms in the tissues of the retina) has an increased risk of pineoblastoma. When retinoblastoma forms at the same time as a tumor in or near the pineal gland, it is called trilateral retinoblastoma. MRI (magnetic resonance imaging) testing in children with retinoblastoma may detect pineoblastoma at an early stage when it can be treated successfully. Anything that increases the risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your child’s doctor if you think your child may be at risk. Risk factors for medulloblastoma and other CNS embryonal tumors include having the following inherited diseases: Children with certain gene changes or a family history of cancers linked to changes in the BRCA gene may be considered for genetic testing. Although rare, this is to check whether the child has a cancer predisposition syndrome that places the child at risk for other diseases or types of cancer. In most cases, the cause of medulloblastoma and other CNS embryonal tumors is not known. These and other signs and symptoms may be caused by childhood medulloblastoma and other CNS embryonal tumors or other conditions. Check with your child's doctor if your child has any of the following: Infants and young children with these tumors may be irritable or grow slowly. Also they may not eat well or meet developmental milestones such as sitting, walking, and talking in sentences. The following tests and procedures may be used: If doctors think your child may have a medulloblastoma or other CNS embryonal tumor, a biopsy may be done. For brain tumors, the biopsy is done by removing part of the skull and using a needle to remove a sample of tissue. Sometimes, a computer-guided needle is used to remove the tissue sample. A pathologist views the tissue under a microscope to look for cancer cells. If cancer cells are found, the doctor may remove as much tumor as safely possible during the same surgery. The piece of skull is usually put back in place after the procedure. The following test may be done on the sample of tissue that is removed: The prognosis and treatment options depend on: Staging is the process used to find out how much cancer there is and if cancer has spread. It is important to know the stage in order to plan treatment. There is no standard staging system for childhood medulloblastoma and other central nervous system (CNS) embryonal tumors. Instead, treatment depends on the type of tumor and the child's age (3 years and younger or older than 3 years). Medulloblastomas are called average risk when all of the following are true: Medulloblastomas are called high risk if any of the following are true: In general, cancer is more likely to recur (come back) in patients with a high-risk tumor. Some of the tests used to detect childhood medulloblastoma and other CNS embryonal tumors are repeated after surgery to remove the tumor. (See the General Information section.) This is to find out how much tumor remains after surgery. Other tests and procedures may be done to find out if the cancer has spread: Childhood medulloblastoma and other CNS embryonal tumors most often recur (come back) within 3 years after treatment but may come back many years later. Recurrent childhood medulloblastoma and other CNS embryonal tumors may come back in the same place as the original tumor and/or in a different place in the brain or spinal cord. Medulloblastoma and other CNS embryonal tumors rarely spread to other parts of the body. Different types of treatment are available for children with medulloblastoma and other central nervous system (CNS) embryonal tumors. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating children with brain tumors and who specialize in certain areas of medicine. These may include the following specialists: Signs or symptoms caused by the tumor may begin before the cancer is diagnosed and continue for months or years. It is important to talk with your child's doctors about signs or symptoms caused by the tumor that may continue after treatment. Surgery is used to diagnose and treat childhood medulloblastoma and other CNS embryonal tumors as described in the General Information section of this summary. After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy, radiation therapy, or both after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Certain ways of giving radiation therapy can help keep radiation from damaging nearby healthy tissue. These types of radiation therapy include the following: Because radiation therapy can affect growth and brain development in young children, especially children who are 3 years old or younger, chemotherapy may be given to delay or reduce the need for radiation therapy. Radiation therapy to the brain can also affect growth and development in children older than three. For this reason, clinical trials are studying new ways of giving radiation that may have fewer side effects than standard methods. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Although most cannot, some chemotherapy drugs can cross the blood-brain barrier and reach tumor cells in the brain. When chemotherapy is placed directly into the cerebrospinal fluid, it is called intrathecal chemotherapy. Intraventricular chemotherapy is a treatment in which anticancer drugs are placed into the ventricles (fluid-filled cavities) of the brain. Combination chemotherapy is treatment using more than one anticancer drug. The way the chemotherapy is given depends on the type of cancer being treated. EnlargeIntrathecal chemotherapy. Anticancer drugs are injected into the intrathecal space, which is the space that holds the cerebrospinal fluid (CSF, shown in blue). There are two different ways to do this. One way, shown in the top part of the figure, is to inject the drugs into an Ommaya reservoir (a dome-shaped container that is placed under the scalp during surgery; it holds the drugs as they flow through a small tube into the brain). The other way, shown in the bottom part of the figure, is to inject the drugs directly into the CSF in the lower part of the spinal column, after a small area on the lower back is numbed. High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Signal transduction inhibitors: This treatment blocks signals that are passed from one molecule to another inside a cell. Blocking these signals may kill cancer cells. Vismodegib is a type of signal transduction inhibitor that may be used to treat recurrent medulloblastoma in children who have finished growing. Targeted therapy is being studied for the treatment of childhood medulloblastoma and other CNS embryonal tumors that have recurred (come back). Information about clinical trials is available from the NCI website. For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Children diagnosed with medulloblastoma may have certain problems after surgery or radiation therapy such as changes in the ability to think, learn, and pay attention. Also, cerebellar mutism syndrome may occur after surgery. Signs of this syndrome include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child and the types of symptoms to expect after cancer treatment has ended. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information). For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. (See the General Information section for a list of tests.) Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. This is sometimes called re-staging. Some of the imaging tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the brain tumor has recurred (come back). If the imaging tests show abnormal tissue in the brain, a biopsy may also be done to find out if the tissue is made up of dead tumor cells or if new cancer cells are growing. These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Younger children with medulloblastoma Standard treatment of newly diagnosed medulloblastoma in children aged 3 years and younger includes the following: Other treatments that may be given after surgery include the following: Children older than 3 years with average-risk medulloblastoma Standard treatment of newly diagnosed average-risk medulloblastoma in children older than 3 years includes the following: Children older than 3 years with high-risk medulloblastoma Standard treatment of newly diagnosed high-risk medulloblastoma in children older than 3 years includes the following: For information about the treatments listed below, see the Treatment Option Overview section. Children aged 3 years and younger with nonmedulloblastoma, nonmedulloepithelioma embryonal tumors, embryonal tumors with multilayered rosettes, or medulloepithelioma Standard treatment of newly diagnosed nonmedulloblastoma, nonmedulloepithelioma embryonal tumors, embryonal tumors with multilayered rosettes, or medulloepithelioma in children 3 years or younger includes the following: Other treatments that may be given after surgery include the following: Children older than 3 years with nonmedulloblastoma, nonmedulloepithelioma embryonal tumors Standard treatment of newly diagnosed nonmedulloblastoma, nonmedulloepithelioma embryonal tumors in children older than 3 years includes the following: Children older than 3 years with embryonal tumors with multilayered rosettes or medulloepithelioma Standard treatment of newly diagnosed embryonal tumor with multilayered rosettes (ETMR) or medulloepithelioma includes the following: For information about the treatments listed below, see the Treatment Option Overview section. Children aged 3 years and younger Standard treatment of newly diagnosed pineoblastoma in children aged 3 years and younger includes the following: Children older than 3 years Standard treatment of newly diagnosed pineoblastoma in children older than 3 years includes the following: For information about the treatments listed below, see the Treatment Option Overview section. Treatment for recurrent childhood medulloblastoma and other CNS embryonal tumors may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information about childhood medulloblastoma and other central nervous system embryonal tumor, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood medulloblastoma and other central nervous system embryonal tumors. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. 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What are the treatments for Childhood Central Nervous System Embryonal Tumors ?

Key Points - There are different types of treatment for children who have central nervous system (CNS) embryonal tumors. - Children who have CNS embryonal tumors should have their treatment planned by a team of health care providers who are experts in treating brain tumors in children. - Childhood brain tumors may cause signs or symptoms that begin before the cancer is diagnosed and continue for months or years. - Some cancer treatments cause side effects months or years after treatment has ended. - Five types of treatment are used: - Surgery - Radiation therapy - Chemotherapy - High-dose chemotherapy with stem cell rescue - Targeted therapy - New types of treatment are being tested in clinical trials. - Patients may want to think about taking part in a clinical trial. - Patients can enter clinical trials before, during, or after starting their cancer treatment. - Follow-up tests may be needed. There are different types of treatment for children who have central nervous system (CNS) embryonal tumors. Different types of treatment are available for children with central nervous system (CNS) embryonal tumors. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Children who have CNS embryonal tumors should have their treatment planned by a team of health care providers who are experts in treating brain tumors in children. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating children with brain tumors and who specialize in certain areas of medicine. These may include the following specialists: - Pediatrician. - Neurosurgeon. - Neurologist. - Neuropathologist. - Neuroradiologist. - Rehabilitation specialist. - Radiation oncologist. - Psychologist. Childhood brain tumors may cause signs or symptoms that begin before the cancer is diagnosed and continue for months or years. Signs or symptoms caused by the tumor may begin before the cancer is diagnosed and continue for months or years. It is important to talk with your child&apos;s doctors about signs or symptoms caused by the tumor that may continue after treatment. Some cancer treatments cause side effects months or years after treatment has ended. Side effects from cancer treatment that begin during or after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: - Physical problems. - Changes in mood, feelings, thinking, learning, or memory. - Second cancers (new types of cancer). Children diagnosed with medulloblastoma may have certain problems after surgery or radiation therapy such as changes in the ability to think, learn, and pay attention. Also, cerebellar mutism syndrome may occur after surgery. Signs of this syndrome include the following: - Delayed ability to speak. - Trouble swallowing and eating. - Loss of balance, trouble walking, and worsening handwriting. - Loss of muscle tone. - Mood swings and changes in personality. Some late effects may be treated or controlled. It is important to talk with your child&apos;s doctors about the effects cancer treatment can have on your child. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information). Five types of treatment are used: Surgery Surgery is used to diagnose and treat a childhood CNS embryonal tumor as described in the General Information section of this summary. Even if the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy and/or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: - External radiation therapy uses a machine outside the body to send radiation toward the cancer. Certain ways of giving radiation therapy can help keep radiation from damaging nearby healthy tissue. These types of radiation therapy include the following: - Conformal radiation therapy: Conformal radiation therapy is a type of external radiation therapy that uses a computer to make a 3-dimensional (3-D) picture of the tumor and shapes the radiation beams to fit the tumor. This allows a high dose of radiation to reach the tumor and causes less damage to nearby healthy tissue. - Stereotactic radiation therapy: Stereotactic radiation therapy is a type of external radiation therapy. A rigid head frame is attached to the skull to keep the head still during the radiation treatment. A machine aims radiation directly at the tumor, causing less damage to nearby healthy tissue. The total dose of radiation is divided into several smaller doses given over several days. This procedure is also called stereotactic external-beam radiation therapy and stereotaxic radiation therapy. - Internal radiation therapy uses a radioactive substance sealed in needles, seeds, wires, or catheters that are placed directly into or near the cancer. Radiation therapy to the brain can affect growth and development in young children. For this reason, clinical trials are studying new ways of giving radiation that may have fewer side effects than standard methods. The way the radiation therapy is given depends on the type of cancer being treated. External radiation therapy is used to treat childhood CNS embryonal tumors. Because radiation therapy can affect growth and brain development in young children, especially children who are three years old or younger, chemotherapy may be given to delay or reduce the need for radiation therapy. Chemotherapy Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Combination chemotherapy is treatment using more than one anticancer drug. The way the chemotherapy is given depends on the type of cancer being treated. Regular dose anticancer drugs given by mouth or vein to treat central nervous system tumors cannot cross the blood-brain barrier and enter the fluid that surrounds the brain and spinal cord. Instead, an anticancer drug is injected into the fluid-filled space to kill cancer cells that may have spread there. This is called intrathecal or intraventricular chemotherapy. High-dose chemotherapy with stem cell rescue High-dose chemotherapy with stem cell rescue is a way of giving high doses of chemotherapy and replacing blood -forming cells destroyed by the cancer treatment. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the chemotherapy is completed, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the bodys blood cells. Targeted therapy Targeted therapy is a type of treatment that uses drugs or other substances to attack cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Signal transduction inhibitors are a type of targeted therapy used to treat recurrent medulloblastoma. Signal transduction inhibitors block signals that are passed from one molecule to another inside a cell. Blocking these signals may kill cancer cells. Vismodegib is a type of signal transduction inhibitor. New types of treatment are being tested in clinical trials. Information about clinical trials is available from the NCI website. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials. Follow-up tests may be needed. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. (See the General Information section for a list of tests.) Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. This is sometimes called re-staging. Some of the imaging tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child&apos;s condition has changed or if the brain tumor has recurred (come back). If the imaging tests show abnormal tissue in the brain, a biopsy may also be done to find out if the tissue is made up of dead tumor cells or if new cancer cells are growing. These tests are sometimes called follow-up tests or check-ups. Treatment Options for Childhood Central Nervous System Embryonal Tumors and Childhood Pineoblastoma Newly Diagnosed Childhood Medulloblastoma In newly diagnosed childhood medulloblastoma, the tumor itself has not been treated. The child may have received drugs or treatment to relieve signs or symptoms caused by the tumor. Children older than 3 years with average-risk medulloblastoma Standard treatment of average-risk medulloblastoma in children older than 3 years includes the following: - Surgery to remove as much of the tumor as possible. This is followed by radiation therapy to the brain and spinal cord. Chemotherapy is also given during and after radiation therapy. - Surgery to remove the tumor, radiation therapy, and high-dose chemotherapy with stem cell rescue. Children older than 3 years with high-risk medulloblastoma Standard treatment of high-risk medulloblastoma in children older than 3 years includes the following: - Surgery to remove as much of the tumor as possible. This is followed by a larger dose of radiation therapy to the brain and spinal cord than the dose given for average-risk medulloblastoma. Chemotherapy is also given during and after radiation therapy. - Surgery to remove the tumor, radiation therapy, and high-dose chemotherapy with stem cell rescue. - A clinical trial of new combinations of radiation therapy and chemotherapy. Children aged 3 years and younger Standard treatment of medulloblastoma in children aged 3 years and younger is: - Surgery to remove as much of the tumor as possible, followed by chemotherapy. Other treatments that may be given after surgery include the following: - Chemotherapy with or without radiation therapy to the area where the tumor was removed. - High-dose chemotherapy with stem cell rescue. Check the list of NCI-supported cancer clinical trials that are now accepting patients with untreated childhood medulloblastoma. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your child&apos;s doctor about clinical trials that may be right for your child. General information about clinical trials is available from the NCI website. Newly Diagnosed Childhood Nonmedulloblastoma Embryonal Tumors In newly diagnosed childhood nonmedulloblastoma embryonal tumors, the tumor itself has not been treated. The child may have received drugs or treatment to relieve symptoms caused by the tumor. Children older than 3 years Standard treatment of nonmedulloblastoma embryonal tumors in children older than 3 years is: - Surgery to remove as much of the tumor as possible. This is followed by radiation therapy to the brain and spinal cord. Chemotherapy is also given during and after radiation therapy. Children aged 3 years and younger Standard treatment of nonmedulloblastoma embryonal tumors in children aged 3 years and younger is: - Surgery to remove as much of the tumor as possible, followed by chemotherapy. Other treatments that may be given after surgery include the following: - Chemotherapy and radiation therapy to the area where the tumor was removed. - High-dose chemotherapy with stem cell rescue. Check the list of NCI-supported cancer clinical trials that are now accepting patients with untreated childhood nonmedulloblastoma. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your child&apos;s doctor about clinical trials that may be right for your child. General information about clinical trials is available from the NCI website. Newly Diagnosed Childhood Medulloepithelioma In newly diagnosed childhood medulloepithelioma, the tumor itself has not been treated. The child may have received drugs or treatment to relieve symptoms caused by the tumor. Children older than 3 years Standard treatment of medulloepithelioma in children older than 3 years includes the following: - Surgery to remove as much of the tumor as possible. This is followed by radiation therapy to the brain and spinal cord. Chemotherapy is also given during and after radiation therapy. - Surgery to remove the tumor, radiation therapy, and high-dose chemotherapy with stem cell rescue. - A clinical trial of new combinations of radiation therapy and chemotherapy. Children aged 3 years and younger Standard treatment of medulloepithelioma in children aged 3 years and younger includes the following: - Surgery to remove as much of the tumor as possible, followed by chemotherapy. - High-dose chemotherapy with stem cell rescue. - Radiation therapy, when the child is older. - A clinical trial of new combinations and schedules of chemotherapy or new combinations of chemotherapy with stem cell rescue. Treatment of medulloepithelioma in children aged 3 years and younger is often within a clinical trial. Check the list of NCI-supported cancer clinical trials that are now accepting patients with childhood medulloepithelioma. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your child&apos;s doctor about clinical trials that may be right for your child. General information about clinical trials is available from the NCI website. Newly Diagnosed Childhood Pineoblastoma In newly diagnosed childhood pineoblastoma, the tumor itself has not been treated. The child may have received drugs or treatment to relieve symptoms caused by the tumor. Children older than 3 years Standard treatment of pineoblastoma in children older than 3 years includes the following: - Surgery to remove the tumor. The tumor usually cannot be completely removed because of where it is in the brain. Surgery is often followed by radiation therapy to the brain and spinal cord and chemotherapy. - A clinical trial of high-dose chemotherapy after radiation therapy and stem cell rescue. - A clinical trial of chemotherapy during radiation therapy. Children aged 3 years and younger Standard treatment of pineoblastoma in children aged 3 years and younger includes the following: - Biopsy to diagnose pineoblastoma followed by chemotherapy. - If the tumor responds to chemotherapy, radiation therapy is given when the child is older. - High-dose chemotherapy with stem cell rescue. Check the list of NCI-supported cancer clinical trials that are now accepting patients with untreated childhood pineoblastoma. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your child&apos;s doctor about clinical trials that may be right for your child. General information about clinical trials is available from the NCI website. Recurrent Childhood Central Nervous System Embryonal Tumors and Pineoblastomas The treatment of central nervous system (CNS) embryonal tumors and pineoblastoma that recur (come back) depends on: - The type of tumor. - Whether the tumor recurred where it first formed or has spread to other parts of the brain, spinal cord, or body. - The type of treatment given in the past. - How much time has passed since the initial treatment ended. - Whether the patient has signs or symptoms. Treatment for recurrent childhood CNS embryonal tumors and pineoblastomas may include the following: - For children who previously received radiation therapy and chemotherapy, treatment may include repeat radiation at the site where the cancer started and where the tumor has spread. Stereotactic radiation therapy and/or chemotherapy may also be used. - For infants and young children who previously received chemotherapy only and have a local recurrence, treatment may be chemotherapy with radiation therapy to the tumor and the area close to it. Surgery to remove the tumor may also be done. - For patients who previously received radiation therapy, high-dose chemotherapy and stem cell rescue may be used. It is not known whether this treatment improves survival. - Targeted therapy with a signal transduction inhibitor for patients whose cancer has certain changes in the genes. Check the list of NCI-supported cancer clinical trials that are now accepting patients with recurrent childhood central nervous system embryonal tumor. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your child&apos;s doctor about clinical trials that may be right for your child. General information about clinical trials is available from the NCI website.

The immune system helps protect the body from infection and disease. The lymph system is made up of the following: Lymph tissue is also found in other parts of the body such as the stomach, thyroid gland, brain, and skin. Non-Hodgkin lymphoma can begin in B lymphocytes, T lymphocytes, or natural killer cells. There are two general types of lymphomas: Hodgkin lymphoma and non-Hodgkin lymphoma. This summary is about the treatment of childhood non-Hodgkin lymphoma. For information about the treatment of childhood Hodgkin lymphoma, see Childhood Hodgkin Lymphoma Treatment. Treatment of non-Hodgkin lymphoma is different for children and adults. For information about treatment of adults, see the following: The type of lymphoma is determined by how the cells look under a microscope. The three major types of childhood non-Hodgkin lymphoma are: Aggressive mature B-cell non-Hodgkin lymphomas include: Both Burkitt leukemia and Burkitt lymphoma have been linked to infection with the Epstein-Barr virus (EBV), although EBV infection is more likely to occur in patients in Africa than in the United States. Burkitt lymphoma/leukemia is more common in White people. Burkitt lymphoma/leukemia is diagnosed when a sample of tissue is checked and a certain change to the MYC gene is found. Lymphoblastic lymphoma is a type of lymphoma that mainly affects T-cell lymphocytes. It usually forms in the mediastinum (the area between the lungs). This causes trouble breathing, wheezing, trouble swallowing, or swelling of the head and neck. It may spread to lymph nodes, bone, bone marrow, skin, the CNS, abdominal organs, and other areas. Lymphoblastic lymphoma is a lot like acute lymphoblastic leukemia (ALL). Anaplastic large cell lymphoma is a type of lymphoma that mainly affects T-cell lymphocytes. It usually forms in the lymph nodes, skin, or bone, and sometimes forms in the gastrointestinal tract, lung, tissue that covers the lungs, and muscle. Patients with anaplastic large cell lymphoma have a receptor, called CD30, on the surface of their T cells. In many children, anaplastic large cell lymphoma is marked by changes in the ALK gene that makes a protein called anaplastic lymphoma kinase. A pathologist checks for these cell and gene changes to help diagnose anaplastic large cell lymphoma. Some types of childhood non-Hodgkin lymphoma are less common. These include: Anything that increases a person's risk of getting a disease is called a risk factor. Not every child with one or more of these risk factors will develop NHL, and it will develop in some children who don't have any known risk factors. Talk with your child's doctor if you think your child may be at risk. Some of the types of immune system problems that have been linked with a higher risk of childhood NHL include the following: If lymphoma or lymphoproliferative disease is linked to a weakened immune system from certain inherited diseases, HIV infection, a transplant or medicines given after a transplant, the condition is called lymphoproliferative disease associated with immunodeficiency. The different types of lymphoproliferative disease associated with immunodeficiency include: These and other signs may be caused by childhood non-Hodgkin lymphoma or by other conditions. Check with a doctor if your child has any of the following: In addition to asking about your child's personal and family health history and doing a physical exam, your child's doctor may perform the following tests and procedures: Cells and tissues are removed during a biopsy so they can be viewed under a microscope by a pathologist to check for cancer cells. Because treatment depends on the type of non-Hodgkin lymphoma, biopsy samples should be checked by a pathologist who has experience in diagnosing childhood non-Hodgkin lymphoma. One of the following types of biopsies may be done: The procedure used to remove the sample of tissue depends on where the tumor is in the body: If cancer is found, the following tests may be done to study the cancer cells: The prognosis and treatment options depend on: The process used to find out if cancer has spread within the lymph system or to other parts of the body is called staging. The results of tests and procedures used to diagnose non-Hodgkin lymphoma may also be used for staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. For a description of the tests and procedures used to diagnose non-Hodgkin lymphoma, see General Information. Cancer can spread through tissue, the lymph system, and the blood: In stage I childhood non-Hodgkin lymphoma, cancer is found: No cancer is found in the abdomen or mediastinum (area between the lungs). In stage II childhood non-Hodgkin lymphoma, cancer is found: In stage III childhood non-Hodgkin lymphoma, cancer is found: In stage IV childhood non-Hodgkin lymphoma, cancer is found in the bone marrow, brain, or cerebrospinal fluid. Cancer may also be found in other parts of the body. Refractory non-Hodgkin lymphoma is cancer that does not respond to initial treatment. Recurrent non-Hodgkin lymphoma is cancer that has come back after treatment. It may come back in the lymph system or in other parts of the body. Different types of treatment are available for children with non-Hodgkin lymphoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Taking part in a clinical trial should be considered for all children with non-Hodgkin lymphoma. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other health care providers who are experts in treating children with non-Hodgkin lymphoma and who specialize in certain areas of medicine. These may include the following specialists: Surgery may be done to remove as much of the tumor as possible for some types of childhood non-Hodgkin lymphoma. After the doctor removes all the cancer that can be seen at the time of surgery, patients may be given chemotherapy to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas. Combination chemotherapy is treatment using two or more anticancer drugs. Intrathecal chemotherapy may be used to treat childhood non-Hodgkin lymphoma that has spread, or may spread, to the brain. When used to lessen the chance cancer will spread to the brain, it is called CNS prophylaxis. Intrathecal chemotherapy is given in addition to chemotherapy by mouth or vein. Higher than usual doses of chemotherapy may also be used as CNS prophylaxis. The way the chemotherapy is given depends on the type and stage of the cancer being treated. For more information, see Drugs Approved for Non-Hodgkin Lymphoma. Radiation therapy is a cancer treatment that uses high energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. External radiation therapy may be used to treat childhood non-Hodgkin lymphoma that has spread, or may spread, to the brain and spinal cord. External radiation therapy may also be used to treat cutaneous T-cell lymphoma (mycosis fungoides). High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. For more information, see Drugs Approved for Non-Hodgkin Lymphoma. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Types of targeted therapy include the following: Types of monoclonal antibodies include: Other targeted therapies are being studied for the treatment of childhood non-Hodgkin lymphoma that has not responded to treatment or has recurred (come back). For more information, see Drugs Approved for Non-Hodgkin Lymphoma. Retinoids are drugs related to vitamin A. Retinoid therapy with bexarotene is used to treat several types of cutaneous T-cell lymphoma. Steroids are hormones made naturally in the body. They can also be made in a laboratory and used as drugs. Steroid therapy that is applied to the skin is used to treat cutaneous T-cell lymphoma. Dexamethasone and prednisone are steroids used with other drugs to treat certain types of lymphoma. Antibiotics are drugs used to treat infections and cancer caused by bacteria or other microorganisms. Antibiotics have been used to treat mucosa-associated lymphoid tissue (MALT) lymphoma. Treatment with antibiotic therapy has been mostly studied in adults. Phototherapy is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. For skin cancer in the skin, laser light is shined onto the skin and the drug becomes active and kills the cancer cells. Phototherapy is used in the treatment of cutaneous T-cell lymphoma. Watchful waiting is closely monitoring a patient's condition without giving any treatment until signs or symptoms appear or change. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Immunotherapy is a treatment that uses the patient’s immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body’s natural defenses against cancer. This cancer treatment is a type of biological therapy. For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. For more information, see Late Effects of Treatment for Childhood Cancer. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see Treatment Option Overview. Treatment of newly diagnosed Burkitt lymphoma/leukemia may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory Burkitt lymphoma/leukemia may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of newly diagnosed diffuse large B-cell lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory diffuse large B-cell lymphoma may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of newly diagnosed primary mediastinal B-cell lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory primary mediastinal B-cell lymphoma may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Lymphoblastic lymphoma may be classified as the same disease as acute lymphoblastic leukemia (ALL). Treatment of lymphoblastic lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory  lymphoblastic lymphoma may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of anaplastic large cell lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory anaplastic large cell lymphoma may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of lymphoproliferative disease in children and adolescents with weakened immune systems may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of non-Hodgkin lymphoma associated with DNA repair defect syndromes in children may include: For information about the treatments listed below, see Treatment Option Overview. Treatment with highly active antiretroviral therapy or HAART (a combination of antiretroviral drugs) lowers the risk of non-Hodgkin lymphoma in patients infected with the human immunodeficiency virus (HIV). Treatment of HIV-related non-Hodgkin lymphoma (NHL) in children may include: For treatment of recurrent disease, treatment options depend on the type of non-Hodgkin lymphoma. For information about the treatments listed below, see Treatment Option Overview. Treatment of post-transplant lymphoproliferative disease may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of follicular lymphoma in children may include: For children whose cancer has certain changes in the genes, treatment is similar to that given to adults with follicular lymphoma. For information about the treatment of follicular lymphoma in adults, see Adult Non-Hodgkin Lymphoma. For information about the treatments listed below, see Treatment Option Overview. Treatment of marginal zone lymphoma (including mucosa-associated lymphoid tissue [MALT] lymphoma) in children may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of primary CNS lymphoma in children may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of peripheral T-cell lymphoma in children may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of subcutaneous panniculitis-like T-cell lymphoma in children may include: Treatment of cutaneous anaplastic large cell lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. In children, treatment of mycosis fungoides may include: For more information from the National Cancer Institute about childhood non-Hodgkin lymphoma, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood non-Hodgkin lymphoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Non-Hodgkin Lymphoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. 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What is (are) Childhood Non-Hodgkin Lymphoma ?

Key Points - Childhood non-Hodgkin lymphoma is a disease in which malignant (cancer) cells form in the lymph system. - The main types of lymphoma are Hodgkin lymphoma and non-Hodgkin lymphoma. - There are three major types of childhood non-Hodgkin lymphoma. - Mature B-cell non-Hodgkin lymphoma - Lymphoblastic lymphoma - Anaplastic large cell lymphoma - Some types of non-Hodgkin lymphoma are rare in children. - Past treatment for cancer and having a weakened immune system affect the risk of having childhood non-Hodgkin lymphoma. - Signs of childhood non-Hodgkin lymphoma include breathing problems and swollen lymph nodes. - Tests that examine the body and lymph system are used to detect (find) and diagnose childhood non-Hodgkin lymphoma. - A biopsy is done to diagnose childhood non-Hodgkin lymphoma. - Certain factors affect prognosis (chance of recovery) and treatment options. Childhood non-Hodgkin lymphoma is a disease in which malignant (cancer) cells form in the lymph system. Childhood non-Hodgkin lymphoma is a type of cancer that forms in the lymph system, which is part of the body&apos;s immune system. The immune system protects the body from foreign substances, infection, and diseases. The lymph system is made up of the following: - Lymph: Colorless, watery fluid that carries white blood cells called lymphocytes through the lymph system. Lymphocytes protect the body against infections and the growth of tumors. There are three types of lymphocytes: - B lymphocytes that make antibodies to help fight infection. - T lymphocytes that help B lymphocytes make the antibodies that help fight infection. - Natural killer cells that attack cancer cells and viruses. - Lymph vessels: A network of thin tubes that collect lymph from different parts of the body and return it to the bloodstream. - Lymph nodes: Small, bean-shaped structures that filter lymph and store white blood cells that help fight infection and disease. Lymph nodes are located along the network of lymph vessels found throughout the body. Clusters of lymph nodes are found in the neck, underarm, abdomen, pelvis, and groin. - Spleen: An organ that makes lymphocytes, filters the blood, stores blood cells, and destroys old blood cells. The spleen is on the left side of the abdomen near the stomach. - Thymus: An organ in which lymphocytes grow and multiply. The thymus is in the chest behind the breastbone. - Tonsils: Two small masses of lymph tissue at the back of the throat. The tonsils make lymphocytes. - Bone marrow: The soft, spongy tissue in the center of large bones. Bone marrow makes white blood cells, red blood cells, and platelets. Non-Hodgkin lymphoma can begin in B lymphocytes, T lymphocytes, or natural killer cells. Lymphocytes can also be found in the blood and collect in the lymph nodes, spleen, and thymus. Lymph tissue is also found in other parts of the body such as the stomach, thyroid gland, brain, and skin. Non-Hodgkin lymphoma can occur in both adults and children. Treatment for children is different than treatment for adults. See the following PDQ summaries for information about treatment of non-Hodgkin lymphoma in adults: - Adult Non-Hodgkin Lymphoma - Primary CNS Lymphoma Treatment - Mycosis Fungoides and the Sezary Syndrome Treatment The main types of lymphoma are Hodgkin lymphoma and non-Hodgkin lymphoma. Lymphomas are divided into two general types: Hodgkin lymphoma and non-Hodgkin lymphoma. This summary is about the treatment of childhood non-Hodgkin lymphoma. See the PDQ summary on Childhood Hodgkin Lymphoma Treatment for information about childhood Hodgkin lymphoma. There are three major types of childhood non-Hodgkin lymphoma. The type of lymphoma is determined by how the cells look under a microscope. The three major types of childhood non-Hodgkin lymphoma are: Mature B-cell non-Hodgkin lymphoma Mature B-cell non-Hodgkin lymphomas include: - Burkitt and Burkitt-like lymphoma/leukemia: Burkitt lymphoma and Burkitt leukemia are different forms of the same disease. Burkitt lymphoma/leukemia is an aggressive (fast-growing) disorder of B lymphocytes that is most common in children and young adults. It may form in the abdomen, Waldeyer&apos;s ring, testicles, bone, bone marrow, skin, or central nervous system (CNS). Burkitt leukemia may start in the lymph nodes as Burkitt lymphoma and then spread to the blood and bone marrow, or it may start in the blood and bone marrow without forming in the lymph nodes first. Both Burkitt leukemia and Burkitt lymphoma have been linked to infection with the Epstein-Barr virus (EBV), although EBV infection is more likely to occur in patients in Africa than in the United States. Burkitt and Burkitt-like lymphoma/leukemia are diagnosed when a sample of tissue is checked and a certain change to the c-myc gene is found. - Diffuse large B-cell lymphoma: Diffuse large B-cell lymphoma is the most common type of non-Hodgkin lymphoma. It is a type of B-cell non-Hodgkin lymphoma that grows quickly in the lymph nodes. The spleen, liver, bone marrow, or other organs are also often affected. Diffuse large B-cell lymphoma occurs more often in adolescents than in children. - Primary mediastinal B-cell lymphoma: A type of lymphoma that develops from B cells in the mediastinum (the area behind the breastbone). It may spread to nearby organs including the lungs and the sac around the heart. It may also spread to lymph nodes and distant organs including the kidneys. In children and adolescents, primary mediastinal B-cell lymphoma occurs more often in older adolescents. Lymphoblastic lymphoma Lymphoblastic lymphoma is a type of lymphoma that mainly affects T-cell lymphocytes. It usually forms in the mediastinum (the area behind the breastbone). This causes trouble breathing, wheezing, trouble swallowing, or swelling of the head and neck. It may spread to lymph nodes, bone, bone marrow, skin, the CNS, abdominal organs, and other areas. Lymphoblastic lymphoma is a lot like acute lymphoblastic leukemia (ALL). Anaplastic large cell lymphoma Anaplastic large cell lymphoma is a type of lymphoma that mainly affects T-cell lymphocytes. It usually forms in the lymph nodes, skin, or bone, and sometimes forms in the gastrointestinal tract, lung, tissue that covers the lungs, and muscle. Patients with anaplastic large cell lymphoma have a receptor, called CD30, on the surface of their T cells. In many children, anaplastic large cell lymphoma is marked by changes in the ALK gene that makes a protein called anaplastic lymphoma kinase. A pathologist checks for these cell and gene changes to help diagnose anaplastic large cell lymphoma. Some types of non-Hodgkin lymphoma are rare in children. Some types of childhood non-Hodgkin lymphoma are less common. These include: - Pediatric-type follicular lymphoma : In children, follicular lymphoma occurs mainly in males. It is more likely to be found in one area and does not spread to other places in the body. It usually forms in the tonsils and lymph nodes in the neck, but may also form in the testicles, kidney, gastrointestinal tract, and salivary gland. - Marginal zone lymphoma : Marginal zone lymphoma is a type of lymphoma that tends to grow and spread slowly and is usually found at an early stage. It may be found in the lymph nodes or in areas outside the lymph nodes. Marginal zone lymphoma found outside the lymph nodes in children is called mucosa-associated lymphoid tissue (MALT) lymphoma and may be linked to Helicobacter pylori infection of the gastrointestinal tract and Chlamydophila psittaci infection of the conjunctival membrane which lines the eye. - Primary central nervous system (CNS) lymphoma : Primary CNS lymphoma is extremely rare in children. - Peripheral T-cell lymphoma : Peripheral T-cell lymphoma is an aggressive (fast-growing) non-Hodgkin lymphoma that begins in mature T lymphocytes. The T lymphocytes mature in the thymus gland and travel to other parts of the lymph system, such as the lymph nodes, bone marrow, and spleen. - Cutaneous T-cell lymphoma : Cutaneous T-cell lymphoma begins in the skin and can cause the skin to thicken or form a tumor. It is very rare in children, but is more common in adolescents and young adults. There are different types of cutaneous T-cell lymphoma, such as cutaneous anaplastic large cell lymphoma, subcutaneous panniculitis-like T-cell lymphoma, gamma-delta T-cell lymphoma, and mycosis fungoides. Mycosis fungoides rarely occurs in children and adolescents. Past treatment for cancer and having a weakened immune system affect the risk of having childhood non-Hodgkin lymphoma. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesnt mean that you will not get cancer. Talk with your child&apos;s doctor if you think your child may be at risk. Possible risk factors for childhood non-Hodgkin lymphoma include the following: - Past treatment for cancer. - Being infected with the Epstein-Barr virus or human immunodeficiency virus (HIV). - Having a weakened immune system after a transplant or from medicines given after a transplant. - Having certain inherited diseases of the immune system. If lymphoma or lymphoproliferative disease is linked to a weakened immune system from certain inherited diseases, HIV infection, a transplant or medicines given after a transplant, the condition is called lymphoproliferative disease associated with immunodeficiency. The different types of lymphoproliferative disease associated with immunodeficiency include: - Lymphoproliferative disease associated with primary immunodeficiency. - HIV-associated non-Hodgkin lymphoma. - Post-transplant lymphoproliferative disease.

The immune system helps protect the body from infection and disease. The lymph system is made up of the following: Lymph tissue is also found in other parts of the body such as the stomach, thyroid gland, brain, and skin. Non-Hodgkin lymphoma can begin in B lymphocytes, T lymphocytes, or natural killer cells. There are two general types of lymphomas: Hodgkin lymphoma and non-Hodgkin lymphoma. This summary is about the treatment of childhood non-Hodgkin lymphoma. For information about the treatment of childhood Hodgkin lymphoma, see Childhood Hodgkin Lymphoma Treatment. Treatment of non-Hodgkin lymphoma is different for children and adults. For information about treatment of adults, see the following: The type of lymphoma is determined by how the cells look under a microscope. The three major types of childhood non-Hodgkin lymphoma are: Aggressive mature B-cell non-Hodgkin lymphomas include: Both Burkitt leukemia and Burkitt lymphoma have been linked to infection with the Epstein-Barr virus (EBV), although EBV infection is more likely to occur in patients in Africa than in the United States. Burkitt lymphoma/leukemia is more common in White people. Burkitt lymphoma/leukemia is diagnosed when a sample of tissue is checked and a certain change to the MYC gene is found. Lymphoblastic lymphoma is a type of lymphoma that mainly affects T-cell lymphocytes. It usually forms in the mediastinum (the area between the lungs). This causes trouble breathing, wheezing, trouble swallowing, or swelling of the head and neck. It may spread to lymph nodes, bone, bone marrow, skin, the CNS, abdominal organs, and other areas. Lymphoblastic lymphoma is a lot like acute lymphoblastic leukemia (ALL). Anaplastic large cell lymphoma is a type of lymphoma that mainly affects T-cell lymphocytes. It usually forms in the lymph nodes, skin, or bone, and sometimes forms in the gastrointestinal tract, lung, tissue that covers the lungs, and muscle. Patients with anaplastic large cell lymphoma have a receptor, called CD30, on the surface of their T cells. In many children, anaplastic large cell lymphoma is marked by changes in the ALK gene that makes a protein called anaplastic lymphoma kinase. A pathologist checks for these cell and gene changes to help diagnose anaplastic large cell lymphoma. Some types of childhood non-Hodgkin lymphoma are less common. These include: Anything that increases a person's risk of getting a disease is called a risk factor. Not every child with one or more of these risk factors will develop NHL, and it will develop in some children who don't have any known risk factors. Talk with your child's doctor if you think your child may be at risk. Some of the types of immune system problems that have been linked with a higher risk of childhood NHL include the following: If lymphoma or lymphoproliferative disease is linked to a weakened immune system from certain inherited diseases, HIV infection, a transplant or medicines given after a transplant, the condition is called lymphoproliferative disease associated with immunodeficiency. The different types of lymphoproliferative disease associated with immunodeficiency include: These and other signs may be caused by childhood non-Hodgkin lymphoma or by other conditions. Check with a doctor if your child has any of the following: In addition to asking about your child's personal and family health history and doing a physical exam, your child's doctor may perform the following tests and procedures: Cells and tissues are removed during a biopsy so they can be viewed under a microscope by a pathologist to check for cancer cells. Because treatment depends on the type of non-Hodgkin lymphoma, biopsy samples should be checked by a pathologist who has experience in diagnosing childhood non-Hodgkin lymphoma. One of the following types of biopsies may be done: The procedure used to remove the sample of tissue depends on where the tumor is in the body: If cancer is found, the following tests may be done to study the cancer cells: The prognosis and treatment options depend on: The process used to find out if cancer has spread within the lymph system or to other parts of the body is called staging. The results of tests and procedures used to diagnose non-Hodgkin lymphoma may also be used for staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. For a description of the tests and procedures used to diagnose non-Hodgkin lymphoma, see General Information. Cancer can spread through tissue, the lymph system, and the blood: In stage I childhood non-Hodgkin lymphoma, cancer is found: No cancer is found in the abdomen or mediastinum (area between the lungs). In stage II childhood non-Hodgkin lymphoma, cancer is found: In stage III childhood non-Hodgkin lymphoma, cancer is found: In stage IV childhood non-Hodgkin lymphoma, cancer is found in the bone marrow, brain, or cerebrospinal fluid. Cancer may also be found in other parts of the body. Refractory non-Hodgkin lymphoma is cancer that does not respond to initial treatment. Recurrent non-Hodgkin lymphoma is cancer that has come back after treatment. It may come back in the lymph system or in other parts of the body. Different types of treatment are available for children with non-Hodgkin lymphoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Taking part in a clinical trial should be considered for all children with non-Hodgkin lymphoma. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other health care providers who are experts in treating children with non-Hodgkin lymphoma and who specialize in certain areas of medicine. These may include the following specialists: Surgery may be done to remove as much of the tumor as possible for some types of childhood non-Hodgkin lymphoma. After the doctor removes all the cancer that can be seen at the time of surgery, patients may be given chemotherapy to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas. Combination chemotherapy is treatment using two or more anticancer drugs. Intrathecal chemotherapy may be used to treat childhood non-Hodgkin lymphoma that has spread, or may spread, to the brain. When used to lessen the chance cancer will spread to the brain, it is called CNS prophylaxis. Intrathecal chemotherapy is given in addition to chemotherapy by mouth or vein. Higher than usual doses of chemotherapy may also be used as CNS prophylaxis. The way the chemotherapy is given depends on the type and stage of the cancer being treated. For more information, see Drugs Approved for Non-Hodgkin Lymphoma. Radiation therapy is a cancer treatment that uses high energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. External radiation therapy may be used to treat childhood non-Hodgkin lymphoma that has spread, or may spread, to the brain and spinal cord. External radiation therapy may also be used to treat cutaneous T-cell lymphoma (mycosis fungoides). High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. For more information, see Drugs Approved for Non-Hodgkin Lymphoma. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Types of targeted therapy include the following: Types of monoclonal antibodies include: Other targeted therapies are being studied for the treatment of childhood non-Hodgkin lymphoma that has not responded to treatment or has recurred (come back). For more information, see Drugs Approved for Non-Hodgkin Lymphoma. Retinoids are drugs related to vitamin A. Retinoid therapy with bexarotene is used to treat several types of cutaneous T-cell lymphoma. Steroids are hormones made naturally in the body. They can also be made in a laboratory and used as drugs. Steroid therapy that is applied to the skin is used to treat cutaneous T-cell lymphoma. Dexamethasone and prednisone are steroids used with other drugs to treat certain types of lymphoma. Antibiotics are drugs used to treat infections and cancer caused by bacteria or other microorganisms. Antibiotics have been used to treat mucosa-associated lymphoid tissue (MALT) lymphoma. Treatment with antibiotic therapy has been mostly studied in adults. Phototherapy is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. For skin cancer in the skin, laser light is shined onto the skin and the drug becomes active and kills the cancer cells. Phototherapy is used in the treatment of cutaneous T-cell lymphoma. Watchful waiting is closely monitoring a patient's condition without giving any treatment until signs or symptoms appear or change. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Immunotherapy is a treatment that uses the patient’s immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body’s natural defenses against cancer. This cancer treatment is a type of biological therapy. For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. For more information, see Late Effects of Treatment for Childhood Cancer. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see Treatment Option Overview. Treatment of newly diagnosed Burkitt lymphoma/leukemia may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory Burkitt lymphoma/leukemia may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of newly diagnosed diffuse large B-cell lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory diffuse large B-cell lymphoma may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of newly diagnosed primary mediastinal B-cell lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory primary mediastinal B-cell lymphoma may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Lymphoblastic lymphoma may be classified as the same disease as acute lymphoblastic leukemia (ALL). Treatment of lymphoblastic lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory  lymphoblastic lymphoma may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of anaplastic large cell lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory anaplastic large cell lymphoma may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of lymphoproliferative disease in children and adolescents with weakened immune systems may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of non-Hodgkin lymphoma associated with DNA repair defect syndromes in children may include: For information about the treatments listed below, see Treatment Option Overview. Treatment with highly active antiretroviral therapy or HAART (a combination of antiretroviral drugs) lowers the risk of non-Hodgkin lymphoma in patients infected with the human immunodeficiency virus (HIV). Treatment of HIV-related non-Hodgkin lymphoma (NHL) in children may include: For treatment of recurrent disease, treatment options depend on the type of non-Hodgkin lymphoma. For information about the treatments listed below, see Treatment Option Overview. Treatment of post-transplant lymphoproliferative disease may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of follicular lymphoma in children may include: For children whose cancer has certain changes in the genes, treatment is similar to that given to adults with follicular lymphoma. For information about the treatment of follicular lymphoma in adults, see Adult Non-Hodgkin Lymphoma. For information about the treatments listed below, see Treatment Option Overview. Treatment of marginal zone lymphoma (including mucosa-associated lymphoid tissue [MALT] lymphoma) in children may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of primary CNS lymphoma in children may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of peripheral T-cell lymphoma in children may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of subcutaneous panniculitis-like T-cell lymphoma in children may include: Treatment of cutaneous anaplastic large cell lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. In children, treatment of mycosis fungoides may include: For more information from the National Cancer Institute about childhood non-Hodgkin lymphoma, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood non-Hodgkin lymphoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Non-Hodgkin Lymphoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/lymphoma/patient/child-nhl-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389294] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Childhood Non-Hodgkin Lymphoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the symptoms of Childhood Non-Hodgkin Lymphoma ?

Signs of childhood non-Hodgkin lymphoma include breathing problems and swollen lymph nodes. These and other signs may be caused by childhood non-Hodgkin lymphoma or by other conditions. Check with a doctor if your child has any of the following: - Trouble breathing. - Wheezing. - Coughing. - High-pitched breathing sounds. - Swelling of the head, neck, upper body, or arms. - Trouble swallowing. - Painless swelling of the lymph nodes in the neck, underarm, stomach, or groin. - Painless lump or swelling in a testicle. - Fever for no known reason. - Weight loss for no known reason. - Night sweats.

The immune system helps protect the body from infection and disease. The lymph system is made up of the following: Lymph tissue is also found in other parts of the body such as the stomach, thyroid gland, brain, and skin. Non-Hodgkin lymphoma can begin in B lymphocytes, T lymphocytes, or natural killer cells. There are two general types of lymphomas: Hodgkin lymphoma and non-Hodgkin lymphoma. This summary is about the treatment of childhood non-Hodgkin lymphoma. For information about the treatment of childhood Hodgkin lymphoma, see Childhood Hodgkin Lymphoma Treatment. Treatment of non-Hodgkin lymphoma is different for children and adults. For information about treatment of adults, see the following: The type of lymphoma is determined by how the cells look under a microscope. The three major types of childhood non-Hodgkin lymphoma are: Aggressive mature B-cell non-Hodgkin lymphomas include: Both Burkitt leukemia and Burkitt lymphoma have been linked to infection with the Epstein-Barr virus (EBV), although EBV infection is more likely to occur in patients in Africa than in the United States. Burkitt lymphoma/leukemia is more common in White people. Burkitt lymphoma/leukemia is diagnosed when a sample of tissue is checked and a certain change to the MYC gene is found. Lymphoblastic lymphoma is a type of lymphoma that mainly affects T-cell lymphocytes. It usually forms in the mediastinum (the area between the lungs). This causes trouble breathing, wheezing, trouble swallowing, or swelling of the head and neck. It may spread to lymph nodes, bone, bone marrow, skin, the CNS, abdominal organs, and other areas. Lymphoblastic lymphoma is a lot like acute lymphoblastic leukemia (ALL). Anaplastic large cell lymphoma is a type of lymphoma that mainly affects T-cell lymphocytes. It usually forms in the lymph nodes, skin, or bone, and sometimes forms in the gastrointestinal tract, lung, tissue that covers the lungs, and muscle. Patients with anaplastic large cell lymphoma have a receptor, called CD30, on the surface of their T cells. In many children, anaplastic large cell lymphoma is marked by changes in the ALK gene that makes a protein called anaplastic lymphoma kinase. A pathologist checks for these cell and gene changes to help diagnose anaplastic large cell lymphoma. Some types of childhood non-Hodgkin lymphoma are less common. These include: Anything that increases a person's risk of getting a disease is called a risk factor. Not every child with one or more of these risk factors will develop NHL, and it will develop in some children who don't have any known risk factors. Talk with your child's doctor if you think your child may be at risk. Some of the types of immune system problems that have been linked with a higher risk of childhood NHL include the following: If lymphoma or lymphoproliferative disease is linked to a weakened immune system from certain inherited diseases, HIV infection, a transplant or medicines given after a transplant, the condition is called lymphoproliferative disease associated with immunodeficiency. The different types of lymphoproliferative disease associated with immunodeficiency include: These and other signs may be caused by childhood non-Hodgkin lymphoma or by other conditions. Check with a doctor if your child has any of the following: In addition to asking about your child's personal and family health history and doing a physical exam, your child's doctor may perform the following tests and procedures: Cells and tissues are removed during a biopsy so they can be viewed under a microscope by a pathologist to check for cancer cells. Because treatment depends on the type of non-Hodgkin lymphoma, biopsy samples should be checked by a pathologist who has experience in diagnosing childhood non-Hodgkin lymphoma. One of the following types of biopsies may be done: The procedure used to remove the sample of tissue depends on where the tumor is in the body: If cancer is found, the following tests may be done to study the cancer cells: The prognosis and treatment options depend on: The process used to find out if cancer has spread within the lymph system or to other parts of the body is called staging. The results of tests and procedures used to diagnose non-Hodgkin lymphoma may also be used for staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. For a description of the tests and procedures used to diagnose non-Hodgkin lymphoma, see General Information. Cancer can spread through tissue, the lymph system, and the blood: In stage I childhood non-Hodgkin lymphoma, cancer is found: No cancer is found in the abdomen or mediastinum (area between the lungs). In stage II childhood non-Hodgkin lymphoma, cancer is found: In stage III childhood non-Hodgkin lymphoma, cancer is found: In stage IV childhood non-Hodgkin lymphoma, cancer is found in the bone marrow, brain, or cerebrospinal fluid. Cancer may also be found in other parts of the body. Refractory non-Hodgkin lymphoma is cancer that does not respond to initial treatment. Recurrent non-Hodgkin lymphoma is cancer that has come back after treatment. It may come back in the lymph system or in other parts of the body. Different types of treatment are available for children with non-Hodgkin lymphoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Taking part in a clinical trial should be considered for all children with non-Hodgkin lymphoma. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other health care providers who are experts in treating children with non-Hodgkin lymphoma and who specialize in certain areas of medicine. These may include the following specialists: Surgery may be done to remove as much of the tumor as possible for some types of childhood non-Hodgkin lymphoma. After the doctor removes all the cancer that can be seen at the time of surgery, patients may be given chemotherapy to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas. Combination chemotherapy is treatment using two or more anticancer drugs. Intrathecal chemotherapy may be used to treat childhood non-Hodgkin lymphoma that has spread, or may spread, to the brain. When used to lessen the chance cancer will spread to the brain, it is called CNS prophylaxis. Intrathecal chemotherapy is given in addition to chemotherapy by mouth or vein. Higher than usual doses of chemotherapy may also be used as CNS prophylaxis. The way the chemotherapy is given depends on the type and stage of the cancer being treated. For more information, see Drugs Approved for Non-Hodgkin Lymphoma. Radiation therapy is a cancer treatment that uses high energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. External radiation therapy may be used to treat childhood non-Hodgkin lymphoma that has spread, or may spread, to the brain and spinal cord. External radiation therapy may also be used to treat cutaneous T-cell lymphoma (mycosis fungoides). High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. For more information, see Drugs Approved for Non-Hodgkin Lymphoma. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Types of targeted therapy include the following: Types of monoclonal antibodies include: Other targeted therapies are being studied for the treatment of childhood non-Hodgkin lymphoma that has not responded to treatment or has recurred (come back). For more information, see Drugs Approved for Non-Hodgkin Lymphoma. Retinoids are drugs related to vitamin A. Retinoid therapy with bexarotene is used to treat several types of cutaneous T-cell lymphoma. Steroids are hormones made naturally in the body. They can also be made in a laboratory and used as drugs. Steroid therapy that is applied to the skin is used to treat cutaneous T-cell lymphoma. Dexamethasone and prednisone are steroids used with other drugs to treat certain types of lymphoma. Antibiotics are drugs used to treat infections and cancer caused by bacteria or other microorganisms. Antibiotics have been used to treat mucosa-associated lymphoid tissue (MALT) lymphoma. Treatment with antibiotic therapy has been mostly studied in adults. Phototherapy is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. For skin cancer in the skin, laser light is shined onto the skin and the drug becomes active and kills the cancer cells. Phototherapy is used in the treatment of cutaneous T-cell lymphoma. Watchful waiting is closely monitoring a patient's condition without giving any treatment until signs or symptoms appear or change. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Immunotherapy is a treatment that uses the patient’s immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body’s natural defenses against cancer. This cancer treatment is a type of biological therapy. For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. For more information, see Late Effects of Treatment for Childhood Cancer. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see Treatment Option Overview. Treatment of newly diagnosed Burkitt lymphoma/leukemia may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory Burkitt lymphoma/leukemia may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of newly diagnosed diffuse large B-cell lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory diffuse large B-cell lymphoma may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of newly diagnosed primary mediastinal B-cell lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory primary mediastinal B-cell lymphoma may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Lymphoblastic lymphoma may be classified as the same disease as acute lymphoblastic leukemia (ALL). Treatment of lymphoblastic lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory  lymphoblastic lymphoma may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of anaplastic large cell lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory anaplastic large cell lymphoma may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of lymphoproliferative disease in children and adolescents with weakened immune systems may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of non-Hodgkin lymphoma associated with DNA repair defect syndromes in children may include: For information about the treatments listed below, see Treatment Option Overview. Treatment with highly active antiretroviral therapy or HAART (a combination of antiretroviral drugs) lowers the risk of non-Hodgkin lymphoma in patients infected with the human immunodeficiency virus (HIV). Treatment of HIV-related non-Hodgkin lymphoma (NHL) in children may include: For treatment of recurrent disease, treatment options depend on the type of non-Hodgkin lymphoma. For information about the treatments listed below, see Treatment Option Overview. Treatment of post-transplant lymphoproliferative disease may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of follicular lymphoma in children may include: For children whose cancer has certain changes in the genes, treatment is similar to that given to adults with follicular lymphoma. For information about the treatment of follicular lymphoma in adults, see Adult Non-Hodgkin Lymphoma. For information about the treatments listed below, see Treatment Option Overview. Treatment of marginal zone lymphoma (including mucosa-associated lymphoid tissue [MALT] lymphoma) in children may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of primary CNS lymphoma in children may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of peripheral T-cell lymphoma in children may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of subcutaneous panniculitis-like T-cell lymphoma in children may include: Treatment of cutaneous anaplastic large cell lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. In children, treatment of mycosis fungoides may include: For more information from the National Cancer Institute about childhood non-Hodgkin lymphoma, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood non-Hodgkin lymphoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Non-Hodgkin Lymphoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/lymphoma/patient/child-nhl-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389294] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Childhood Non-Hodgkin Lymphoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

How to diagnose Childhood Non-Hodgkin Lymphoma ?

Tests that examine the body and lymph system are used to detect (find) and diagnose childhood non-Hodgkin lymphoma. The following tests and procedures may be used: - Physical exam and history : An exam of the body to check general signs of health, including checking for signs of disease, such as lumps or anything else that seems unusual. A history of the patients health habits and past illnesses and treatments will also be taken. - Blood chemistry studies : A procedure in which a blood sample is checked to measure the amounts of certain substances released into the blood by organs and tissues in the body, including electrolytes, uric acid, blood urea nitrogen (BUN), creatinine, and liver function values. An unusual (higher or lower than normal) amount of a substance can be a sign of disease. - Liver function tests : A procedure in which a blood sample is checked to measure the amounts of certain substances released into the blood by the liver. A higher than normal amount of a substance can be a sign of cancer. - CT scan (CAT scan): A procedure that makes a series of detailed pictures of areas inside the body, taken from different angles. The pictures are made by a computer linked to an x-ray machine. A dye may be injected into a vein or swallowed to help the organs or tissues show up more clearly. This procedure is also called computed tomography, computerized tomography, or computerized axial tomography. - PET scan (positron emission tomography scan): A procedure to find malignant tumor cells in the body. A small amount of radioactive glucose (sugar) is injected into a vein. The PET scanner rotates around the body and makes a picture of where glucose is being used in the body. Malignant tumor cells show up brighter in the picture because they are more active and take up more glucose than normal cells do. Sometimes a PET scan and a CT scan are done at the same time. If there is any cancer, this increases the chance that it will be found. - MRI (magnetic resonance imaging): A procedure that uses a magnet, radio waves, and a computer to make a series of detailed pictures of areas inside the body. This procedure is also called nuclear magnetic resonance imaging (NMRI). - Lumbar puncture : A procedure used to collect cerebrospinal fluid (CSF) from the spinal column. This is done by placing a needle between two bones in the spine and into the CSF around the spinal cord and removing a sample of the fluid. The sample of CSF is checked under a microscope for signs that the cancer has spread to the brain and spinal cord. This procedure is also called an LP or spinal tap. - Chest x-ray : An x-ray of the organs and bones inside the chest. An x-ray is a type of energy beam that can go through the body and onto film, making a picture of areas inside the body. - Ultrasound exam: A procedure in which high-energy sound waves (ultrasound) are bounced off internal tissues or organs and make echoes. The echoes form a picture of body tissues called a sonogram. The picture can be printed to be looked at later. - A biopsy is done to diagnose childhood non-Hodgkin lymphoma: Cells and tissues are removed during a biopsy so they can be viewed under a microscope by a pathologist to check for signs of cancer. Because treatment depends on the type of non-Hodgkin lymphoma, biopsy samples should be checked by a pathologist who has experience in diagnosing childhood non-Hodgkin lymphoma. One of the following types of biopsies may be done: - Excisional biopsy : The removal of an entire lymph node or lump of tissue. - Incisional biopsy : The removal of part of a lump, lymph node, or sample of tissue. - Core biopsy : The removal of tissue or part of a lymph node using a wide needle. - Fine-needle aspiration (FNA) biopsy : The removal of tissue or part of a lymph node using a thin needle. The procedure used to remove the sample of tissue depends on where the tumor is in the body: - Bone marrow aspiration and biopsy : The removal of bone marrow and a small piece of bone by inserting a hollow needle into the hipbone or breastbone. - Mediastinoscopy : A surgical procedure to look at the organs, tissues, and lymph nodes between the lungs for abnormal areas. An incision (cut) is made at the top of the breastbone and a mediastinoscope is inserted into the chest. A mediastinoscope is a thin, tube-like instrument with a light and a lens for viewing. It also has a tool to remove tissue or lymph node samples, which are checked under a microscope for signs of cancer. - Anterior mediastinotomy : A surgical procedure to look at the organs and tissues between the lungs and between the breastbone and heart for abnormal areas. An incision (cut) is made next to the breastbone and a mediastinoscope is inserted into the chest. A mediastinoscope is a thin, tube-like instrument with a light and a lens for viewing. It also has a tool to remove tissue or lymph node samples, which are checked under a microscope for signs of cancer. This is also called the Chamberlain procedure. - Thoracentesis : The removal of fluid from the space between the lining of the chest and the lung, using a needle. A pathologist views the fluid under a microscope to look for cancer cells. If cancer is found, the following tests may be done to study the cancer cells: - Immunohistochemistry : A laboratory test that uses antibodies to check for certain antigens in a sample of tissue. The antibody is usually linked to a radioactive substance or a dye that causes the tissue to light up under a microscope. This type of test may be used to tell the difference between different types of cancer. - Flow cytometry : A laboratory test that measures the number of cells in a sample, the percentage of live cells in a sample, and certain characteristics of cells, such as size, shape, and the presence of tumor markers on the cell surface. The cells are stained with a light-sensitive dye, placed in a fluid, and passed in a stream before a laser or other type of light. The measurements are based on how the light-sensitive dye reacts to the light. - Cytogenetic analysis : A laboratory test in which cells in a sample of tissue are viewed under a microscope to look for certain changes in the chromosomes. - FISH (fluorescence in situ hybridization): A laboratory test used to look at genes or chromosomes in cells and tissues. Pieces of DNA that contain a fluorescent dye are made in the laboratory and added to cells or tissues on a glass slide. When these pieces of DNA attach to certain genes or areas of chromosomes on the slide, they light up when viewed under a microscope with a special light. This type of test is used to find certain gene changes. - Immunophenotyping : A laboratory test used to identify cells, based on the types of antigens or markers on the surface of the cell. This test is used to diagnose specific types of lymphoma by comparing the cancer cells to normal cells of the immune system.

The immune system helps protect the body from infection and disease. The lymph system is made up of the following: Lymph tissue is also found in other parts of the body such as the stomach, thyroid gland, brain, and skin. Non-Hodgkin lymphoma can begin in B lymphocytes, T lymphocytes, or natural killer cells. There are two general types of lymphomas: Hodgkin lymphoma and non-Hodgkin lymphoma. This summary is about the treatment of childhood non-Hodgkin lymphoma. For information about the treatment of childhood Hodgkin lymphoma, see Childhood Hodgkin Lymphoma Treatment. Treatment of non-Hodgkin lymphoma is different for children and adults. For information about treatment of adults, see the following: The type of lymphoma is determined by how the cells look under a microscope. The three major types of childhood non-Hodgkin lymphoma are: Aggressive mature B-cell non-Hodgkin lymphomas include: Both Burkitt leukemia and Burkitt lymphoma have been linked to infection with the Epstein-Barr virus (EBV), although EBV infection is more likely to occur in patients in Africa than in the United States. Burkitt lymphoma/leukemia is more common in White people. Burkitt lymphoma/leukemia is diagnosed when a sample of tissue is checked and a certain change to the MYC gene is found. Lymphoblastic lymphoma is a type of lymphoma that mainly affects T-cell lymphocytes. It usually forms in the mediastinum (the area between the lungs). This causes trouble breathing, wheezing, trouble swallowing, or swelling of the head and neck. It may spread to lymph nodes, bone, bone marrow, skin, the CNS, abdominal organs, and other areas. Lymphoblastic lymphoma is a lot like acute lymphoblastic leukemia (ALL). Anaplastic large cell lymphoma is a type of lymphoma that mainly affects T-cell lymphocytes. It usually forms in the lymph nodes, skin, or bone, and sometimes forms in the gastrointestinal tract, lung, tissue that covers the lungs, and muscle. Patients with anaplastic large cell lymphoma have a receptor, called CD30, on the surface of their T cells. In many children, anaplastic large cell lymphoma is marked by changes in the ALK gene that makes a protein called anaplastic lymphoma kinase. A pathologist checks for these cell and gene changes to help diagnose anaplastic large cell lymphoma. Some types of childhood non-Hodgkin lymphoma are less common. These include: Anything that increases a person's risk of getting a disease is called a risk factor. Not every child with one or more of these risk factors will develop NHL, and it will develop in some children who don't have any known risk factors. Talk with your child's doctor if you think your child may be at risk. Some of the types of immune system problems that have been linked with a higher risk of childhood NHL include the following: If lymphoma or lymphoproliferative disease is linked to a weakened immune system from certain inherited diseases, HIV infection, a transplant or medicines given after a transplant, the condition is called lymphoproliferative disease associated with immunodeficiency. The different types of lymphoproliferative disease associated with immunodeficiency include: These and other signs may be caused by childhood non-Hodgkin lymphoma or by other conditions. Check with a doctor if your child has any of the following: In addition to asking about your child's personal and family health history and doing a physical exam, your child's doctor may perform the following tests and procedures: Cells and tissues are removed during a biopsy so they can be viewed under a microscope by a pathologist to check for cancer cells. Because treatment depends on the type of non-Hodgkin lymphoma, biopsy samples should be checked by a pathologist who has experience in diagnosing childhood non-Hodgkin lymphoma. One of the following types of biopsies may be done: The procedure used to remove the sample of tissue depends on where the tumor is in the body: If cancer is found, the following tests may be done to study the cancer cells: The prognosis and treatment options depend on: The process used to find out if cancer has spread within the lymph system or to other parts of the body is called staging. The results of tests and procedures used to diagnose non-Hodgkin lymphoma may also be used for staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. For a description of the tests and procedures used to diagnose non-Hodgkin lymphoma, see General Information. Cancer can spread through tissue, the lymph system, and the blood: In stage I childhood non-Hodgkin lymphoma, cancer is found: No cancer is found in the abdomen or mediastinum (area between the lungs). In stage II childhood non-Hodgkin lymphoma, cancer is found: In stage III childhood non-Hodgkin lymphoma, cancer is found: In stage IV childhood non-Hodgkin lymphoma, cancer is found in the bone marrow, brain, or cerebrospinal fluid. Cancer may also be found in other parts of the body. Refractory non-Hodgkin lymphoma is cancer that does not respond to initial treatment. Recurrent non-Hodgkin lymphoma is cancer that has come back after treatment. It may come back in the lymph system or in other parts of the body. Different types of treatment are available for children with non-Hodgkin lymphoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Taking part in a clinical trial should be considered for all children with non-Hodgkin lymphoma. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other health care providers who are experts in treating children with non-Hodgkin lymphoma and who specialize in certain areas of medicine. These may include the following specialists: Surgery may be done to remove as much of the tumor as possible for some types of childhood non-Hodgkin lymphoma. After the doctor removes all the cancer that can be seen at the time of surgery, patients may be given chemotherapy to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas. Combination chemotherapy is treatment using two or more anticancer drugs. Intrathecal chemotherapy may be used to treat childhood non-Hodgkin lymphoma that has spread, or may spread, to the brain. When used to lessen the chance cancer will spread to the brain, it is called CNS prophylaxis. Intrathecal chemotherapy is given in addition to chemotherapy by mouth or vein. Higher than usual doses of chemotherapy may also be used as CNS prophylaxis. The way the chemotherapy is given depends on the type and stage of the cancer being treated. For more information, see Drugs Approved for Non-Hodgkin Lymphoma. Radiation therapy is a cancer treatment that uses high energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. External radiation therapy may be used to treat childhood non-Hodgkin lymphoma that has spread, or may spread, to the brain and spinal cord. External radiation therapy may also be used to treat cutaneous T-cell lymphoma (mycosis fungoides). High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. For more information, see Drugs Approved for Non-Hodgkin Lymphoma. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Types of targeted therapy include the following: Types of monoclonal antibodies include: Other targeted therapies are being studied for the treatment of childhood non-Hodgkin lymphoma that has not responded to treatment or has recurred (come back). For more information, see Drugs Approved for Non-Hodgkin Lymphoma. Retinoids are drugs related to vitamin A. Retinoid therapy with bexarotene is used to treat several types of cutaneous T-cell lymphoma. Steroids are hormones made naturally in the body. They can also be made in a laboratory and used as drugs. Steroid therapy that is applied to the skin is used to treat cutaneous T-cell lymphoma. Dexamethasone and prednisone are steroids used with other drugs to treat certain types of lymphoma. Antibiotics are drugs used to treat infections and cancer caused by bacteria or other microorganisms. Antibiotics have been used to treat mucosa-associated lymphoid tissue (MALT) lymphoma. Treatment with antibiotic therapy has been mostly studied in adults. Phototherapy is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. For skin cancer in the skin, laser light is shined onto the skin and the drug becomes active and kills the cancer cells. Phototherapy is used in the treatment of cutaneous T-cell lymphoma. Watchful waiting is closely monitoring a patient's condition without giving any treatment until signs or symptoms appear or change. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Immunotherapy is a treatment that uses the patient’s immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body’s natural defenses against cancer. This cancer treatment is a type of biological therapy. For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. For more information, see Late Effects of Treatment for Childhood Cancer. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see Treatment Option Overview. Treatment of newly diagnosed Burkitt lymphoma/leukemia may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory Burkitt lymphoma/leukemia may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of newly diagnosed diffuse large B-cell lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory diffuse large B-cell lymphoma may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of newly diagnosed primary mediastinal B-cell lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory primary mediastinal B-cell lymphoma may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Lymphoblastic lymphoma may be classified as the same disease as acute lymphoblastic leukemia (ALL). Treatment of lymphoblastic lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory  lymphoblastic lymphoma may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of anaplastic large cell lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory anaplastic large cell lymphoma may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of lymphoproliferative disease in children and adolescents with weakened immune systems may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of non-Hodgkin lymphoma associated with DNA repair defect syndromes in children may include: For information about the treatments listed below, see Treatment Option Overview. Treatment with highly active antiretroviral therapy or HAART (a combination of antiretroviral drugs) lowers the risk of non-Hodgkin lymphoma in patients infected with the human immunodeficiency virus (HIV). Treatment of HIV-related non-Hodgkin lymphoma (NHL) in children may include: For treatment of recurrent disease, treatment options depend on the type of non-Hodgkin lymphoma. For information about the treatments listed below, see Treatment Option Overview. Treatment of post-transplant lymphoproliferative disease may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of follicular lymphoma in children may include: For children whose cancer has certain changes in the genes, treatment is similar to that given to adults with follicular lymphoma. For information about the treatment of follicular lymphoma in adults, see Adult Non-Hodgkin Lymphoma. For information about the treatments listed below, see Treatment Option Overview. Treatment of marginal zone lymphoma (including mucosa-associated lymphoid tissue [MALT] lymphoma) in children may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of primary CNS lymphoma in children may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of peripheral T-cell lymphoma in children may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of subcutaneous panniculitis-like T-cell lymphoma in children may include: Treatment of cutaneous anaplastic large cell lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. In children, treatment of mycosis fungoides may include: For more information from the National Cancer Institute about childhood non-Hodgkin lymphoma, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. 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What is the outlook for Childhood Non-Hodgkin Lymphoma ?

Certain factors affect prognosis (chance of recovery) and treatment options. The prognosis (chance of recovery) and treatment options depend on: - The type of lymphoma. - Where the tumor is in the body when the tumor is diagnosed. - The stage of the cancer. - Whether there are certain changes in the chromosomes. - The type of initial treatment. - Whether the lymphoma responded to initial treatment. - The patients age and general health.

The immune system helps protect the body from infection and disease. The lymph system is made up of the following: Lymph tissue is also found in other parts of the body such as the stomach, thyroid gland, brain, and skin. Non-Hodgkin lymphoma can begin in B lymphocytes, T lymphocytes, or natural killer cells. There are two general types of lymphomas: Hodgkin lymphoma and non-Hodgkin lymphoma. This summary is about the treatment of childhood non-Hodgkin lymphoma. For information about the treatment of childhood Hodgkin lymphoma, see Childhood Hodgkin Lymphoma Treatment. Treatment of non-Hodgkin lymphoma is different for children and adults. For information about treatment of adults, see the following: The type of lymphoma is determined by how the cells look under a microscope. The three major types of childhood non-Hodgkin lymphoma are: Aggressive mature B-cell non-Hodgkin lymphomas include: Both Burkitt leukemia and Burkitt lymphoma have been linked to infection with the Epstein-Barr virus (EBV), although EBV infection is more likely to occur in patients in Africa than in the United States. Burkitt lymphoma/leukemia is more common in White people. Burkitt lymphoma/leukemia is diagnosed when a sample of tissue is checked and a certain change to the MYC gene is found. Lymphoblastic lymphoma is a type of lymphoma that mainly affects T-cell lymphocytes. It usually forms in the mediastinum (the area between the lungs). This causes trouble breathing, wheezing, trouble swallowing, or swelling of the head and neck. It may spread to lymph nodes, bone, bone marrow, skin, the CNS, abdominal organs, and other areas. Lymphoblastic lymphoma is a lot like acute lymphoblastic leukemia (ALL). Anaplastic large cell lymphoma is a type of lymphoma that mainly affects T-cell lymphocytes. It usually forms in the lymph nodes, skin, or bone, and sometimes forms in the gastrointestinal tract, lung, tissue that covers the lungs, and muscle. Patients with anaplastic large cell lymphoma have a receptor, called CD30, on the surface of their T cells. In many children, anaplastic large cell lymphoma is marked by changes in the ALK gene that makes a protein called anaplastic lymphoma kinase. A pathologist checks for these cell and gene changes to help diagnose anaplastic large cell lymphoma. Some types of childhood non-Hodgkin lymphoma are less common. These include: Anything that increases a person's risk of getting a disease is called a risk factor. Not every child with one or more of these risk factors will develop NHL, and it will develop in some children who don't have any known risk factors. Talk with your child's doctor if you think your child may be at risk. Some of the types of immune system problems that have been linked with a higher risk of childhood NHL include the following: If lymphoma or lymphoproliferative disease is linked to a weakened immune system from certain inherited diseases, HIV infection, a transplant or medicines given after a transplant, the condition is called lymphoproliferative disease associated with immunodeficiency. The different types of lymphoproliferative disease associated with immunodeficiency include: These and other signs may be caused by childhood non-Hodgkin lymphoma or by other conditions. Check with a doctor if your child has any of the following: In addition to asking about your child's personal and family health history and doing a physical exam, your child's doctor may perform the following tests and procedures: Cells and tissues are removed during a biopsy so they can be viewed under a microscope by a pathologist to check for cancer cells. Because treatment depends on the type of non-Hodgkin lymphoma, biopsy samples should be checked by a pathologist who has experience in diagnosing childhood non-Hodgkin lymphoma. One of the following types of biopsies may be done: The procedure used to remove the sample of tissue depends on where the tumor is in the body: If cancer is found, the following tests may be done to study the cancer cells: The prognosis and treatment options depend on: The process used to find out if cancer has spread within the lymph system or to other parts of the body is called staging. The results of tests and procedures used to diagnose non-Hodgkin lymphoma may also be used for staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. For a description of the tests and procedures used to diagnose non-Hodgkin lymphoma, see General Information. Cancer can spread through tissue, the lymph system, and the blood: In stage I childhood non-Hodgkin lymphoma, cancer is found: No cancer is found in the abdomen or mediastinum (area between the lungs). In stage II childhood non-Hodgkin lymphoma, cancer is found: In stage III childhood non-Hodgkin lymphoma, cancer is found: In stage IV childhood non-Hodgkin lymphoma, cancer is found in the bone marrow, brain, or cerebrospinal fluid. Cancer may also be found in other parts of the body. Refractory non-Hodgkin lymphoma is cancer that does not respond to initial treatment. Recurrent non-Hodgkin lymphoma is cancer that has come back after treatment. It may come back in the lymph system or in other parts of the body. Different types of treatment are available for children with non-Hodgkin lymphoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Taking part in a clinical trial should be considered for all children with non-Hodgkin lymphoma. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other health care providers who are experts in treating children with non-Hodgkin lymphoma and who specialize in certain areas of medicine. These may include the following specialists: Surgery may be done to remove as much of the tumor as possible for some types of childhood non-Hodgkin lymphoma. After the doctor removes all the cancer that can be seen at the time of surgery, patients may be given chemotherapy to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas. Combination chemotherapy is treatment using two or more anticancer drugs. Intrathecal chemotherapy may be used to treat childhood non-Hodgkin lymphoma that has spread, or may spread, to the brain. When used to lessen the chance cancer will spread to the brain, it is called CNS prophylaxis. Intrathecal chemotherapy is given in addition to chemotherapy by mouth or vein. Higher than usual doses of chemotherapy may also be used as CNS prophylaxis. The way the chemotherapy is given depends on the type and stage of the cancer being treated. For more information, see Drugs Approved for Non-Hodgkin Lymphoma. Radiation therapy is a cancer treatment that uses high energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. External radiation therapy may be used to treat childhood non-Hodgkin lymphoma that has spread, or may spread, to the brain and spinal cord. External radiation therapy may also be used to treat cutaneous T-cell lymphoma (mycosis fungoides). High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. For more information, see Drugs Approved for Non-Hodgkin Lymphoma. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Types of targeted therapy include the following: Types of monoclonal antibodies include: Other targeted therapies are being studied for the treatment of childhood non-Hodgkin lymphoma that has not responded to treatment or has recurred (come back). For more information, see Drugs Approved for Non-Hodgkin Lymphoma. Retinoids are drugs related to vitamin A. Retinoid therapy with bexarotene is used to treat several types of cutaneous T-cell lymphoma. Steroids are hormones made naturally in the body. They can also be made in a laboratory and used as drugs. Steroid therapy that is applied to the skin is used to treat cutaneous T-cell lymphoma. Dexamethasone and prednisone are steroids used with other drugs to treat certain types of lymphoma. Antibiotics are drugs used to treat infections and cancer caused by bacteria or other microorganisms. Antibiotics have been used to treat mucosa-associated lymphoid tissue (MALT) lymphoma. Treatment with antibiotic therapy has been mostly studied in adults. Phototherapy is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. For skin cancer in the skin, laser light is shined onto the skin and the drug becomes active and kills the cancer cells. Phototherapy is used in the treatment of cutaneous T-cell lymphoma. Watchful waiting is closely monitoring a patient's condition without giving any treatment until signs or symptoms appear or change. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Immunotherapy is a treatment that uses the patient’s immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body’s natural defenses against cancer. This cancer treatment is a type of biological therapy. For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. For more information, see Late Effects of Treatment for Childhood Cancer. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see Treatment Option Overview. Treatment of newly diagnosed Burkitt lymphoma/leukemia may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory Burkitt lymphoma/leukemia may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of newly diagnosed diffuse large B-cell lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory diffuse large B-cell lymphoma may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of newly diagnosed primary mediastinal B-cell lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory primary mediastinal B-cell lymphoma may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Lymphoblastic lymphoma may be classified as the same disease as acute lymphoblastic leukemia (ALL). Treatment of lymphoblastic lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory  lymphoblastic lymphoma may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of anaplastic large cell lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory anaplastic large cell lymphoma may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of lymphoproliferative disease in children and adolescents with weakened immune systems may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of non-Hodgkin lymphoma associated with DNA repair defect syndromes in children may include: For information about the treatments listed below, see Treatment Option Overview. Treatment with highly active antiretroviral therapy or HAART (a combination of antiretroviral drugs) lowers the risk of non-Hodgkin lymphoma in patients infected with the human immunodeficiency virus (HIV). Treatment of HIV-related non-Hodgkin lymphoma (NHL) in children may include: For treatment of recurrent disease, treatment options depend on the type of non-Hodgkin lymphoma. For information about the treatments listed below, see Treatment Option Overview. Treatment of post-transplant lymphoproliferative disease may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of follicular lymphoma in children may include: For children whose cancer has certain changes in the genes, treatment is similar to that given to adults with follicular lymphoma. For information about the treatment of follicular lymphoma in adults, see Adult Non-Hodgkin Lymphoma. For information about the treatments listed below, see Treatment Option Overview. Treatment of marginal zone lymphoma (including mucosa-associated lymphoid tissue [MALT] lymphoma) in children may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of primary CNS lymphoma in children may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of peripheral T-cell lymphoma in children may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of subcutaneous panniculitis-like T-cell lymphoma in children may include: Treatment of cutaneous anaplastic large cell lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. In children, treatment of mycosis fungoides may include: For more information from the National Cancer Institute about childhood non-Hodgkin lymphoma, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood non-Hodgkin lymphoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Non-Hodgkin Lymphoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/lymphoma/patient/child-nhl-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389294] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. 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what research (or clinical trials) is being done for Childhood Non-Hodgkin Lymphoma ?

New types of treatment are being tested in clinical trials. Information about clinical trials is available from the NCI website. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials.

The immune system helps protect the body from infection and disease. The lymph system is made up of the following: Lymph tissue is also found in other parts of the body such as the stomach, thyroid gland, brain, and skin. Non-Hodgkin lymphoma can begin in B lymphocytes, T lymphocytes, or natural killer cells. There are two general types of lymphomas: Hodgkin lymphoma and non-Hodgkin lymphoma. This summary is about the treatment of childhood non-Hodgkin lymphoma. For information about the treatment of childhood Hodgkin lymphoma, see Childhood Hodgkin Lymphoma Treatment. Treatment of non-Hodgkin lymphoma is different for children and adults. For information about treatment of adults, see the following: The type of lymphoma is determined by how the cells look under a microscope. The three major types of childhood non-Hodgkin lymphoma are: Aggressive mature B-cell non-Hodgkin lymphomas include: Both Burkitt leukemia and Burkitt lymphoma have been linked to infection with the Epstein-Barr virus (EBV), although EBV infection is more likely to occur in patients in Africa than in the United States. Burkitt lymphoma/leukemia is more common in White people. Burkitt lymphoma/leukemia is diagnosed when a sample of tissue is checked and a certain change to the MYC gene is found. Lymphoblastic lymphoma is a type of lymphoma that mainly affects T-cell lymphocytes. It usually forms in the mediastinum (the area between the lungs). This causes trouble breathing, wheezing, trouble swallowing, or swelling of the head and neck. It may spread to lymph nodes, bone, bone marrow, skin, the CNS, abdominal organs, and other areas. Lymphoblastic lymphoma is a lot like acute lymphoblastic leukemia (ALL). Anaplastic large cell lymphoma is a type of lymphoma that mainly affects T-cell lymphocytes. It usually forms in the lymph nodes, skin, or bone, and sometimes forms in the gastrointestinal tract, lung, tissue that covers the lungs, and muscle. Patients with anaplastic large cell lymphoma have a receptor, called CD30, on the surface of their T cells. In many children, anaplastic large cell lymphoma is marked by changes in the ALK gene that makes a protein called anaplastic lymphoma kinase. A pathologist checks for these cell and gene changes to help diagnose anaplastic large cell lymphoma. Some types of childhood non-Hodgkin lymphoma are less common. These include: Anything that increases a person's risk of getting a disease is called a risk factor. Not every child with one or more of these risk factors will develop NHL, and it will develop in some children who don't have any known risk factors. Talk with your child's doctor if you think your child may be at risk. Some of the types of immune system problems that have been linked with a higher risk of childhood NHL include the following: If lymphoma or lymphoproliferative disease is linked to a weakened immune system from certain inherited diseases, HIV infection, a transplant or medicines given after a transplant, the condition is called lymphoproliferative disease associated with immunodeficiency. The different types of lymphoproliferative disease associated with immunodeficiency include: These and other signs may be caused by childhood non-Hodgkin lymphoma or by other conditions. Check with a doctor if your child has any of the following: In addition to asking about your child's personal and family health history and doing a physical exam, your child's doctor may perform the following tests and procedures: Cells and tissues are removed during a biopsy so they can be viewed under a microscope by a pathologist to check for cancer cells. Because treatment depends on the type of non-Hodgkin lymphoma, biopsy samples should be checked by a pathologist who has experience in diagnosing childhood non-Hodgkin lymphoma. One of the following types of biopsies may be done: The procedure used to remove the sample of tissue depends on where the tumor is in the body: If cancer is found, the following tests may be done to study the cancer cells: The prognosis and treatment options depend on: The process used to find out if cancer has spread within the lymph system or to other parts of the body is called staging. The results of tests and procedures used to diagnose non-Hodgkin lymphoma may also be used for staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. For a description of the tests and procedures used to diagnose non-Hodgkin lymphoma, see General Information. Cancer can spread through tissue, the lymph system, and the blood: In stage I childhood non-Hodgkin lymphoma, cancer is found: No cancer is found in the abdomen or mediastinum (area between the lungs). In stage II childhood non-Hodgkin lymphoma, cancer is found: In stage III childhood non-Hodgkin lymphoma, cancer is found: In stage IV childhood non-Hodgkin lymphoma, cancer is found in the bone marrow, brain, or cerebrospinal fluid. Cancer may also be found in other parts of the body. Refractory non-Hodgkin lymphoma is cancer that does not respond to initial treatment. Recurrent non-Hodgkin lymphoma is cancer that has come back after treatment. It may come back in the lymph system or in other parts of the body. Different types of treatment are available for children with non-Hodgkin lymphoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Taking part in a clinical trial should be considered for all children with non-Hodgkin lymphoma. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other health care providers who are experts in treating children with non-Hodgkin lymphoma and who specialize in certain areas of medicine. These may include the following specialists: Surgery may be done to remove as much of the tumor as possible for some types of childhood non-Hodgkin lymphoma. After the doctor removes all the cancer that can be seen at the time of surgery, patients may be given chemotherapy to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas. Combination chemotherapy is treatment using two or more anticancer drugs. Intrathecal chemotherapy may be used to treat childhood non-Hodgkin lymphoma that has spread, or may spread, to the brain. When used to lessen the chance cancer will spread to the brain, it is called CNS prophylaxis. Intrathecal chemotherapy is given in addition to chemotherapy by mouth or vein. Higher than usual doses of chemotherapy may also be used as CNS prophylaxis. The way the chemotherapy is given depends on the type and stage of the cancer being treated. For more information, see Drugs Approved for Non-Hodgkin Lymphoma. Radiation therapy is a cancer treatment that uses high energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. External radiation therapy may be used to treat childhood non-Hodgkin lymphoma that has spread, or may spread, to the brain and spinal cord. External radiation therapy may also be used to treat cutaneous T-cell lymphoma (mycosis fungoides). High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. For more information, see Drugs Approved for Non-Hodgkin Lymphoma. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Types of targeted therapy include the following: Types of monoclonal antibodies include: Other targeted therapies are being studied for the treatment of childhood non-Hodgkin lymphoma that has not responded to treatment or has recurred (come back). For more information, see Drugs Approved for Non-Hodgkin Lymphoma. Retinoids are drugs related to vitamin A. Retinoid therapy with bexarotene is used to treat several types of cutaneous T-cell lymphoma. Steroids are hormones made naturally in the body. They can also be made in a laboratory and used as drugs. Steroid therapy that is applied to the skin is used to treat cutaneous T-cell lymphoma. Dexamethasone and prednisone are steroids used with other drugs to treat certain types of lymphoma. Antibiotics are drugs used to treat infections and cancer caused by bacteria or other microorganisms. Antibiotics have been used to treat mucosa-associated lymphoid tissue (MALT) lymphoma. Treatment with antibiotic therapy has been mostly studied in adults. Phototherapy is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. For skin cancer in the skin, laser light is shined onto the skin and the drug becomes active and kills the cancer cells. Phototherapy is used in the treatment of cutaneous T-cell lymphoma. Watchful waiting is closely monitoring a patient's condition without giving any treatment until signs or symptoms appear or change. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Immunotherapy is a treatment that uses the patient’s immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body’s natural defenses against cancer. This cancer treatment is a type of biological therapy. For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. For more information, see Late Effects of Treatment for Childhood Cancer. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see Treatment Option Overview. Treatment of newly diagnosed Burkitt lymphoma/leukemia may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory Burkitt lymphoma/leukemia may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of newly diagnosed diffuse large B-cell lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory diffuse large B-cell lymphoma may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of newly diagnosed primary mediastinal B-cell lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory primary mediastinal B-cell lymphoma may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Lymphoblastic lymphoma may be classified as the same disease as acute lymphoblastic leukemia (ALL). Treatment of lymphoblastic lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory  lymphoblastic lymphoma may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of anaplastic large cell lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory anaplastic large cell lymphoma may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of lymphoproliferative disease in children and adolescents with weakened immune systems may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of non-Hodgkin lymphoma associated with DNA repair defect syndromes in children may include: For information about the treatments listed below, see Treatment Option Overview. Treatment with highly active antiretroviral therapy or HAART (a combination of antiretroviral drugs) lowers the risk of non-Hodgkin lymphoma in patients infected with the human immunodeficiency virus (HIV). Treatment of HIV-related non-Hodgkin lymphoma (NHL) in children may include: For treatment of recurrent disease, treatment options depend on the type of non-Hodgkin lymphoma. For information about the treatments listed below, see Treatment Option Overview. Treatment of post-transplant lymphoproliferative disease may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of follicular lymphoma in children may include: For children whose cancer has certain changes in the genes, treatment is similar to that given to adults with follicular lymphoma. For information about the treatment of follicular lymphoma in adults, see Adult Non-Hodgkin Lymphoma. For information about the treatments listed below, see Treatment Option Overview. Treatment of marginal zone lymphoma (including mucosa-associated lymphoid tissue [MALT] lymphoma) in children may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of primary CNS lymphoma in children may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of peripheral T-cell lymphoma in children may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of subcutaneous panniculitis-like T-cell lymphoma in children may include: Treatment of cutaneous anaplastic large cell lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. In children, treatment of mycosis fungoides may include: For more information from the National Cancer Institute about childhood non-Hodgkin lymphoma, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood non-Hodgkin lymphoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Non-Hodgkin Lymphoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/lymphoma/patient/child-nhl-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389294] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Childhood Non-Hodgkin Lymphoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the stages of Childhood Non-Hodgkin Lymphoma ?

Key Points - After childhood non-Hodgkin lymphoma has been diagnosed, tests are done to find out if cancer cells have spread within the lymph system or to other parts of the body. - There are three ways that cancer spreads in the body. - The following stages are used for childhood non-Hodgkin lymphoma: - Stage I - Stage II - Stage III - Stage IV After childhood non-Hodgkin lymphoma has been diagnosed, tests are done to find out if cancer cells have spread within the lymph system or to other parts of the body. The process used to find out if cancer has spread within the lymph system or to other parts of the body is called staging. The results of tests and procedures used to diagnose non-Hodgkin lymphoma may also be used for staging. See the General Information section for a description of these tests and procedures. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following procedure also may be used to determine the stage: - Bone scan : A procedure to check if there are rapidly dividing cells, such as cancer cells, in the bone. A very small amount of radioactive material is injected into a vein and travels through the bloodstream. The radioactive material collects in the bones with cancer and is detected by a scanner. There are three ways that cancer spreads in the body. Cancer can spread through tissue, the lymph system, and the blood: - Tissue. The cancer spreads from where it began by growing into nearby areas. - Lymph system. The cancer spreads from where it began by getting into the lymph system. The cancer travels through the lymph vessels to other parts of the body. - Blood. The cancer spreads from where it began by getting into the blood. The cancer travels through the blood vessels to other parts of the body. The following stages are used for childhood non-Hodgkin lymphoma: Stage I In stage I childhood non-Hodgkin lymphoma, cancer is found: - in one group of lymph nodes; or - in one area outside the lymph nodes. No cancer is found in the abdomen or mediastinum (area between the lungs). Stage II In stage II childhood non-Hodgkin lymphoma, cancer is found: - in one area outside the lymph nodes and in nearby lymph nodes; or - in two or more areas either above or below the diaphragm, and may have spread to nearby lymph nodes; or - to have started in the stomach or intestines and can be completely removed by surgery. Cancer may have spread to certain nearby lymph nodes. Stage III In stage III childhood non-Hodgkin lymphoma, cancer is found: - in at least one area above the diaphragm and in at least one area below the diaphragm; or - to have started in the chest; or - to have started in the abdomen and spread throughout the abdomen; or - in the area around the spine. Stage IV In stage IV childhood non-Hodgkin lymphoma, cancer is found in the bone marrow, brain, or cerebrospinal fluid. Cancer may also be found in other parts of the body.

The immune system helps protect the body from infection and disease. The lymph system is made up of the following: Lymph tissue is also found in other parts of the body such as the stomach, thyroid gland, brain, and skin. Non-Hodgkin lymphoma can begin in B lymphocytes, T lymphocytes, or natural killer cells. There are two general types of lymphomas: Hodgkin lymphoma and non-Hodgkin lymphoma. This summary is about the treatment of childhood non-Hodgkin lymphoma. For information about the treatment of childhood Hodgkin lymphoma, see Childhood Hodgkin Lymphoma Treatment. Treatment of non-Hodgkin lymphoma is different for children and adults. For information about treatment of adults, see the following: The type of lymphoma is determined by how the cells look under a microscope. The three major types of childhood non-Hodgkin lymphoma are: Aggressive mature B-cell non-Hodgkin lymphomas include: Both Burkitt leukemia and Burkitt lymphoma have been linked to infection with the Epstein-Barr virus (EBV), although EBV infection is more likely to occur in patients in Africa than in the United States. Burkitt lymphoma/leukemia is more common in White people. Burkitt lymphoma/leukemia is diagnosed when a sample of tissue is checked and a certain change to the MYC gene is found. Lymphoblastic lymphoma is a type of lymphoma that mainly affects T-cell lymphocytes. It usually forms in the mediastinum (the area between the lungs). This causes trouble breathing, wheezing, trouble swallowing, or swelling of the head and neck. It may spread to lymph nodes, bone, bone marrow, skin, the CNS, abdominal organs, and other areas. Lymphoblastic lymphoma is a lot like acute lymphoblastic leukemia (ALL). Anaplastic large cell lymphoma is a type of lymphoma that mainly affects T-cell lymphocytes. It usually forms in the lymph nodes, skin, or bone, and sometimes forms in the gastrointestinal tract, lung, tissue that covers the lungs, and muscle. Patients with anaplastic large cell lymphoma have a receptor, called CD30, on the surface of their T cells. In many children, anaplastic large cell lymphoma is marked by changes in the ALK gene that makes a protein called anaplastic lymphoma kinase. A pathologist checks for these cell and gene changes to help diagnose anaplastic large cell lymphoma. Some types of childhood non-Hodgkin lymphoma are less common. These include: Anything that increases a person's risk of getting a disease is called a risk factor. Not every child with one or more of these risk factors will develop NHL, and it will develop in some children who don't have any known risk factors. Talk with your child's doctor if you think your child may be at risk. Some of the types of immune system problems that have been linked with a higher risk of childhood NHL include the following: If lymphoma or lymphoproliferative disease is linked to a weakened immune system from certain inherited diseases, HIV infection, a transplant or medicines given after a transplant, the condition is called lymphoproliferative disease associated with immunodeficiency. The different types of lymphoproliferative disease associated with immunodeficiency include: These and other signs may be caused by childhood non-Hodgkin lymphoma or by other conditions. Check with a doctor if your child has any of the following: In addition to asking about your child's personal and family health history and doing a physical exam, your child's doctor may perform the following tests and procedures: Cells and tissues are removed during a biopsy so they can be viewed under a microscope by a pathologist to check for cancer cells. Because treatment depends on the type of non-Hodgkin lymphoma, biopsy samples should be checked by a pathologist who has experience in diagnosing childhood non-Hodgkin lymphoma. One of the following types of biopsies may be done: The procedure used to remove the sample of tissue depends on where the tumor is in the body: If cancer is found, the following tests may be done to study the cancer cells: The prognosis and treatment options depend on: The process used to find out if cancer has spread within the lymph system or to other parts of the body is called staging. The results of tests and procedures used to diagnose non-Hodgkin lymphoma may also be used for staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. For a description of the tests and procedures used to diagnose non-Hodgkin lymphoma, see General Information. Cancer can spread through tissue, the lymph system, and the blood: In stage I childhood non-Hodgkin lymphoma, cancer is found: No cancer is found in the abdomen or mediastinum (area between the lungs). In stage II childhood non-Hodgkin lymphoma, cancer is found: In stage III childhood non-Hodgkin lymphoma, cancer is found: In stage IV childhood non-Hodgkin lymphoma, cancer is found in the bone marrow, brain, or cerebrospinal fluid. Cancer may also be found in other parts of the body. Refractory non-Hodgkin lymphoma is cancer that does not respond to initial treatment. Recurrent non-Hodgkin lymphoma is cancer that has come back after treatment. It may come back in the lymph system or in other parts of the body. Different types of treatment are available for children with non-Hodgkin lymphoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Taking part in a clinical trial should be considered for all children with non-Hodgkin lymphoma. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other health care providers who are experts in treating children with non-Hodgkin lymphoma and who specialize in certain areas of medicine. These may include the following specialists: Surgery may be done to remove as much of the tumor as possible for some types of childhood non-Hodgkin lymphoma. After the doctor removes all the cancer that can be seen at the time of surgery, patients may be given chemotherapy to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas. Combination chemotherapy is treatment using two or more anticancer drugs. Intrathecal chemotherapy may be used to treat childhood non-Hodgkin lymphoma that has spread, or may spread, to the brain. When used to lessen the chance cancer will spread to the brain, it is called CNS prophylaxis. Intrathecal chemotherapy is given in addition to chemotherapy by mouth or vein. Higher than usual doses of chemotherapy may also be used as CNS prophylaxis. The way the chemotherapy is given depends on the type and stage of the cancer being treated. For more information, see Drugs Approved for Non-Hodgkin Lymphoma. Radiation therapy is a cancer treatment that uses high energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. External radiation therapy may be used to treat childhood non-Hodgkin lymphoma that has spread, or may spread, to the brain and spinal cord. External radiation therapy may also be used to treat cutaneous T-cell lymphoma (mycosis fungoides). High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. For more information, see Drugs Approved for Non-Hodgkin Lymphoma. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Types of targeted therapy include the following: Types of monoclonal antibodies include: Other targeted therapies are being studied for the treatment of childhood non-Hodgkin lymphoma that has not responded to treatment or has recurred (come back). For more information, see Drugs Approved for Non-Hodgkin Lymphoma. Retinoids are drugs related to vitamin A. Retinoid therapy with bexarotene is used to treat several types of cutaneous T-cell lymphoma. Steroids are hormones made naturally in the body. They can also be made in a laboratory and used as drugs. Steroid therapy that is applied to the skin is used to treat cutaneous T-cell lymphoma. Dexamethasone and prednisone are steroids used with other drugs to treat certain types of lymphoma. Antibiotics are drugs used to treat infections and cancer caused by bacteria or other microorganisms. Antibiotics have been used to treat mucosa-associated lymphoid tissue (MALT) lymphoma. Treatment with antibiotic therapy has been mostly studied in adults. Phototherapy is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. For skin cancer in the skin, laser light is shined onto the skin and the drug becomes active and kills the cancer cells. Phototherapy is used in the treatment of cutaneous T-cell lymphoma. Watchful waiting is closely monitoring a patient's condition without giving any treatment until signs or symptoms appear or change. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Immunotherapy is a treatment that uses the patient’s immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body’s natural defenses against cancer. This cancer treatment is a type of biological therapy. For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. For more information, see Late Effects of Treatment for Childhood Cancer. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see Treatment Option Overview. Treatment of newly diagnosed Burkitt lymphoma/leukemia may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory Burkitt lymphoma/leukemia may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of newly diagnosed diffuse large B-cell lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory diffuse large B-cell lymphoma may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of newly diagnosed primary mediastinal B-cell lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory primary mediastinal B-cell lymphoma may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Lymphoblastic lymphoma may be classified as the same disease as acute lymphoblastic leukemia (ALL). Treatment of lymphoblastic lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory  lymphoblastic lymphoma may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of anaplastic large cell lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of recurrent or refractory anaplastic large cell lymphoma may include: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see Treatment Option Overview. Treatment of lymphoproliferative disease in children and adolescents with weakened immune systems may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of non-Hodgkin lymphoma associated with DNA repair defect syndromes in children may include: For information about the treatments listed below, see Treatment Option Overview. Treatment with highly active antiretroviral therapy or HAART (a combination of antiretroviral drugs) lowers the risk of non-Hodgkin lymphoma in patients infected with the human immunodeficiency virus (HIV). Treatment of HIV-related non-Hodgkin lymphoma (NHL) in children may include: For treatment of recurrent disease, treatment options depend on the type of non-Hodgkin lymphoma. For information about the treatments listed below, see Treatment Option Overview. Treatment of post-transplant lymphoproliferative disease may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of follicular lymphoma in children may include: For children whose cancer has certain changes in the genes, treatment is similar to that given to adults with follicular lymphoma. For information about the treatment of follicular lymphoma in adults, see Adult Non-Hodgkin Lymphoma. For information about the treatments listed below, see Treatment Option Overview. Treatment of marginal zone lymphoma (including mucosa-associated lymphoid tissue [MALT] lymphoma) in children may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of primary CNS lymphoma in children may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of peripheral T-cell lymphoma in children may include: For information about the treatments listed below, see Treatment Option Overview. Treatment of subcutaneous panniculitis-like T-cell lymphoma in children may include: Treatment of cutaneous anaplastic large cell lymphoma may include: For information about the treatments listed below, see Treatment Option Overview. In children, treatment of mycosis fungoides may include: For more information from the National Cancer Institute about childhood non-Hodgkin lymphoma, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood non-Hodgkin lymphoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Non-Hodgkin Lymphoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/lymphoma/patient/child-nhl-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389294] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Childhood Non-Hodgkin Lymphoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the treatments for Childhood Non-Hodgkin Lymphoma ?

Key Points - There are different types of treatment for children with non-Hodgkin lymphoma. - Children with non-Hodgkin lymphoma should have their treatment planned by a team of doctors who are experts in treating childhood cancer. - Some cancer treatments cause side effects months or years after treatment has ended. - Six types of standard treatment are used: - Chemotherapy - Radiation therapy - High-dose chemotherapy with stem cell transplant - Targeted therapy - Other drug therapy - Phototherapy - New types of treatment are being tested in clinical trials. - Patients may want to think about taking part in a clinical trial. - Patients can enter clinical trials before, during, or after starting their cancer treatment. - Follow-up tests may be needed. There are different types of treatment for children with non-Hodgkin lymphoma. Different types of treatment are available for children with non-Hodgkin lymphoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Taking part in a clinical trial should be considered for all children with non-Hodgkin lymphoma. Some clinical trials are open only to patients who have not started treatment. Children with non-Hodgkin lymphoma should have their treatment planned by a team of doctors who are experts in treating childhood cancer. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other health care providers who are experts in treating children with non-Hodgkin lymphoma and who specialize in certain areas of medicine. These may include the following specialists: - Pediatrician. - Radiation oncologist. - Pediatric hematologist. - Pediatric surgeon. - Pediatric nurse specialist. - Rehabilitation specialist. - Psychologist. - Social worker. Some cancer treatments cause side effects months or years after treatment has ended. Side effects from cancer treatment that begin during or after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following: - Physical problems. - Changes in mood, feelings, thinking, learning, or memory. - Second cancers (new types of cancer). Some late effects may be treated or controlled. It is important to talk with your child&apos;s doctors about the effects cancer treatment can have on your child. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information.) Six types of standard treatment are used: Chemotherapy Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas. Combination chemotherapy is treatment using two or more anticancer drugs. The way the chemotherapy is given depends on the type and stage of the cancer being treated. Intrathecal chemotherapy may be used to treat childhood non-Hodgkin lymphoma that has spread, or may spread, to the brain. When used to lessen the chance cancer will spread to the brain, it is called CNS prophylaxis. Intrathecal chemotherapy is given in addition to chemotherapy by mouth or vein. Higher than usual doses of chemotherapy may also be used as CNS prophylaxis. See Drugs Approved for Non-Hodgkin Lymphoma for more information. Radiation therapy Radiation therapy is a cancer treatment that uses high energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: - External radiation therapy uses a machine outside the body to send radiation toward the cancer. - Internal radiation therapy uses a radioactive substance sealed in needles, seeds, wires, or catheters that are placed directly into or near the cancer. The way the radiation therapy is given depends on the type of non-Hodgkin lymphoma being treated. External radiation therapy may be used to treat childhood non-Hodgkin lymphoma that has spread, or may spread, to the brain and spinal cord. Internal radiation therapy is not used to treat non-Hodgkin lymphoma. High-dose chemotherapy with stem cell transplant This treatment is a way of giving high doses of chemotherapy and then replacing blood -forming cells destroyed by the cancer treatment. Stem cells (immature blood cells) are removed from the bone marrow or blood of the patient or a donor and are frozen and stored. After the chemotherapy is completed, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the bodys blood cells. See Drugs Approved for Non-Hodgkin Lymphoma for more information. Targeted therapy Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells without harming normal cells. Monoclonal antibodies, tyrosine kinase inhibitors, and immunotoxins are three types of targeted therapy being used or studied in the treatment of childhood non-Hodgkin lymphoma. Monoclonal antibody therapy is a cancer treatment that uses antibodies made in the laboratory from a single type of immune system cell. These antibodies can identify substances on cancer cells or normal substances that may help cancer cells grow. The antibodies attach to the substances and kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. - Rituximab is used to treat several types of childhood non-Hodgkin lymphoma. - Brentuximab vedotin is a monoclonal antibody combined with an anticancer drug that is used to treat anaplastic large cell lymphoma. A bispecific monoclonal antibody is made up of two different monoclonal antibodies that bind to two different substances and kills cancer cells. Bispecific monoclonal antibody therapy is used in the treatment of Burkitt and Burkitt-like lymphoma /leukemia and diffuse large B-cell lymphoma. Tyrosine kinase inhibitors (TKIs) block signals that tumors need to grow. Some TKIs also keep tumors from growing by preventing the growth of new blood vessels to the tumors. Other types of kinase inhibitors, such as crizotinib, are being studied for childhood non-Hodgkin lymphoma. Immunotoxins can bind to cancer cells and kill them. Denileukin diftitox is an immunotoxin used to treat cutaneous T-cell lymphoma. See Drugs Approved for Non-Hodgkin Lymphoma for more information. Other drug therapy Retinoids are drugs related to vitamin A. Retinoid therapy with bexarotene is used to treat several types of cutaneous T-cell lymphoma. Steroids are hormones made naturally in the body. They can also be made in a laboratory and used as drugs. Steroid therapy is used to treat cutaneous T-cell lymphoma. Phototherapy Phototherapy is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. For skin cancer in the skin, laser light is shined onto the skin and the drug becomes active and kills the cancer cells. Phototherapy is used in the treatment of cutaneous T-cell lymphoma. New types of treatment are being tested in clinical trials. Information about clinical trials is available from the NCI website. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials. Follow-up tests may be needed. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child&apos;s condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. Treatment Options for Childhood Non-Hodgkin Lymphoma Burkitt and Burkitt-like lymphoma/leukemia Treatment options for newly diagnosed Burkitt and Burkitt-like lymphoma/leukemia Treatment options for newly diagnosed Burkitt and Burkitt-like lymphoma /leukemia may include: - Surgery to remove as much of the tumor as possible, followed by combination chemotherapy. - Combination chemotherapy. - Combination chemotherapy and targeted therapy (rituximab). Treatment options for recurrent Burkitt and Burkitt-like lymphoma/leukemia Treatment options for recurrent Burkitt and Burkitt-like non-Hodgkin lymphoma /leukemia may include: - Combination chemotherapy and targeted therapy (rituximab). - High-dose chemotherapy with stem cell transplant with the patient&apos;s own cells or cells from a donor. - Targeted therapy with a bispecific antibody. Check the list of NCI-supported cancer clinical trials that are now accepting patients with childhood Burkitt lymphoma, stage I childhood small noncleaved cell lymphoma, stage II childhood small noncleaved cell lymphoma, stage III childhood small noncleaved cell lymphoma, stage IV childhood small noncleaved cell lymphoma and recurrent childhood small noncleaved cell lymphoma. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your child&apos;s doctor about clinical trials that may be right for your child. General information about clinical trials is available from the NCI website. Diffuse large B-cell lymphoma Treatment options for newly diagnosed diffuse large B-cell lymphoma Treatment options for newly diagnosed diffuse large B-cell lymphoma may include: - Surgery to remove as much of the tumor as possible, followed by combination chemotherapy. - Combination chemotherapy. - Combination chemotherapy and targeted therapy (rituximab). Treatment options for recurrent diffuse large B-cell lymphoma Treatment options for recurrent diffuse large B-cell lymphoma may include: - Combination chemotherapy and targeted therapy (rituximab). - High-dose chemotherapy with stem cell transplant with the patient&apos;s own cells or cells from a donor. - Targeted therapy with a bispecific antibody. Check the list of NCI-supported cancer clinical trials that are now accepting patients with childhood diffuse large cell lymphoma, stage I childhood large cell lymphoma, stage II childhood large cell lymphoma, stage III childhood large cell lymphoma, stage IV childhood large cell lymphoma and recurrent childhood large cell lymphoma. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your child&apos;s doctor about clinical trials that may be right for your child. General information about clinical trials is available from the NCI website. Primary Mediastinal B-cell Lymphoma Treatment options for primary mediastinal B-cell lymphoma Treatment options for primary mediastinal B-cell lymphoma may include: - Combination chemotherapy and targeted therapy (rituximab). Lymphoblastic Lymphoma Treatment options for newly diagnosed lymphoblastic lymphoma Lymphoblastic lymphoma may be classified as the same disease as acute lymphoblastic leukemia (ALL). Treatment options for lymphoblastic lymphoma may include: - Combination chemotherapy. CNS prophylaxis with radiation therapy or chemotherapy is also given if cancer has spread to the brain and spinal cord. - A clinical trial of chemotherapy with different regimens for CNS prophylaxis. - A clinical trial of combination chemotherapy with or without targeted therapy (bortezomib). Treatment options for recurrent lymphoblastic lymphoma Treatment options for recurrent lymphoblastic lymphoma may include: - Combination chemotherapy. - High-dose chemotherapy with stem cell transplant with cells from a donor. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage I childhood lymphoblastic lymphoma, stage II childhood lymphoblastic lymphoma, stage III childhood lymphoblastic lymphoma, stage IV childhood lymphoblastic lymphoma and recurrent childhood lymphoblastic lymphoma. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your child&apos;s doctor about clinical trials that may be right for your child. General information about clinical trials is available from the NCI website. Anaplastic Large Cell Lymphoma Treatment options for newly diagnosed anaplastic large cell lymphoma Treatment options for anaplastic large cell lymphoma may include: - Surgery followed by combination chemotherapy. - Combination chemotherapy. - Intrathecal and systemic chemotherapy, for patients with cancer in the brain or spinal cord. - A clinical trial of targeted therapy (crizotinib or brentuximab) and combination chemotherapy. Treatment options for recurrent anaplastic large cell lymphoma Treatment options for recurrent anaplastic large cell lymphoma may include: - Chemotherapy with one or more drugs. - Stem cell transplant with the patient&apos;s own cells or cells from a donor. - A clinical trial of targeted therapy (crizotinib) in children with recurrent anaplastic large cell lymphoma and changes in the ALK gene. - A clinical trial of targeted therapy (crizotinib) and combination chemotherapy. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage I childhood anaplastic large cell lymphoma, stage II childhood anaplastic large cell lymphoma, stage III childhood anaplastic large cell lymphoma, stage IV childhood anaplastic large cell lymphoma and recurrent childhood anaplastic large cell lymphoma. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your child&apos;s doctor about clinical trials that may be right for your child. General information about clinical trials is available from the NCI website. Lymphoproliferative Disease Associated With Immunodeficiency in Children Treatment options for lymphoproliferative disease associated with primary immunodeficiency Treatment options for lymphoproliferative disease in children and adolescents with weakened immune systems may include: - Chemotherapy. - Stem cell transplant with cells from a donor. Treatment options for HIV-associated non-Hodgkin lymphoma Treatment with highly active antiretroviral therapy or HAART (a combination of antiretroviral drugs) lowers the risk of non-Hodgkin lymphoma in patients infected with the human immunodeficiency virus (HIV). Treatment options for HIV-related non-Hodgkin lymphoma (NHL) in children may include: - Chemotherapy. For treatment of recurrent disease, treatment options depend on the type of non-Hodgkin lymphoma. Treatment options for post-transplant lymphoproliferative disease Treatment options for post-transplant lymphoproliferative disease may include: - Surgery to remove the tumor. If possible, lower doses of immunosuppressive drugs after a stem cell or organ transplant may be given. - Targeted therapy (rituximab). - Chemotherapy with or without targeted therapy (rituximab). - A clinical trial of immunotherapy using donor lymphocytes or the patient&apos;s own T cells to target Epstein-Barr infection. Rare NHL Occurring in Children Treatment options for pediatric-type follicular lymphoma Treatment options for follicular lymphoma in children may include: - Surgery. - Combination chemotherapy. For children whose cancer has certain changes in the genes, treatment is similar to that given to adults with follicular lymphoma. See the Follicular Lymphoma section in the PDQ summary on Adult Non-Hodgkin Lymphoma for information. Treatment options for marginal zone lymphoma Treatment options for marginal zone lymphoma in children may include: - Surgery. - Radiation therapy. - Antibiotic therapy, for mucosa-associated lymphoid tissue (MALT) lymphoma. Treatment options for primary CNS lymphoma Treatment options for primary CNS lymphoma in children may include: - Chemotherapy. Treatment options for peripheral T-cell lymphoma Treatment options for peripheral T-cell lymphoma in children may include: - Chemotherapy. - Radiation therapy. - Stem cell transplant with the patient&apos;s own cells or cells from a donor. Treatment options for cutaneous T-cell lymphoma Treatment options for subcutaneous panniculitis-like cutaneous T-cell lymphoma in children may include: - Watchful waiting. - High-dose steroids. - Targeted therapy (denileukin diftitox). - Combination chemotherapy. - Retinoid therapy. - Stem cell transplant. Treatment options for cutaneous anaplastic large cell lymphoma may include: - Surgery, radiation therapy, or both. In children, treatment options for mycosis fungoides may include: - Steroids applied to the skin. - Retinoid therapy. - Radiation therapy. - Phototherapy (light therapy using ultraviolet B radiation).

Screening is looking for cancer before a person has any symptoms. This can help find cancer at an early stage. When abnormal tissue or cancer is found early, it may be easier to treat. By the time symptoms appear, cancer may have begun to spread. Scientists are trying to better understand which people are more likely to get certain types of cancer. They also study the things we do and the things around us to see if they cause cancer. This information helps doctors recommend who should be screened for cancer, which screening tests should be used, and how often the tests should be done. It is important to remember that your doctor does not necessarily think you have cancer if he or she suggests a screening test. Screening tests are given when you have no cancer symptoms. Screening tests may be repeated on a regular basis. If a screening test result is abnormal, you may need to have more tests done to find out if you have cancer. These are called diagnostic tests. The prostate is a gland in the male reproductive system located just below the bladder (the organ that collects and empties urine) and in front of the rectum (the lower part of the intestine). It is about the size of a walnut and surrounds part of the urethra (the tube that empties urine from the bladder). The prostate gland produces fluid that makes up part of semen. EnlargeAnatomy of the male reproductive and urinary systems, showing the prostate, testicles, bladder, and other organs. As men age, the prostate may get bigger. A bigger prostate may block the flow of urine from the bladder and cause problems with sexual function. This condition is called benign prostatic hyperplasia (BPH), and although it is not cancer, surgery may be needed to correct it. The symptoms of benign prostatic hyperplasia or of other problems in the prostate may be similar to symptoms of prostate cancer. EnlargeNormal prostate and benign prostatic hyperplasia (BPH). A normal prostate does not block the flow of urine from the bladder. An enlarged prostate presses on the bladder and urethra and blocks the flow of urine. See the following PDQ summaries for more information about prostate cancer: Prostate cancer is found mainly in older men. In the U.S., about one out of every 8 men will be diagnosed with prostate cancer. Most men diagnosed with this disease do not die from it. Prostate cancer causes more deaths in men than any other cancer except lung cancer. Prostate cancer occurs more often in African American men than in White men. African American men with prostate cancer are more likely to die from the disease than White men with prostate cancer. Anything that increases a person's chance of developing a disease is called a risk factor. Anything that decreases your chance of getting a disease is called a protective factor. For information about risk factors and protective factors for prostate cancer, see the PDQ summary on Prostate Cancer Prevention. Scientists study screening tests to find those with the fewest harms and most benefits. Cancer screening trials also are meant to show whether early detection (finding cancer before it causes symptoms) helps a person live longer or decreases a person's chance of dying from the disease. For some types of cancer, the chance of recovery is better if the disease is found and treated at an early stage. Although there are no standard or routine screening tests for prostate cancer, the following tests are being used or studied to screen for it: Digital rectal exam (DRE) is an exam of the rectum. The doctor or nurse inserts a lubricated, gloved finger into the lower part of the rectum to feel the prostate for lumps or anything else that seems unusual.EnlargeDigital rectal exam (DRE). The doctor inserts a gloved, lubricated finger into the rectum and feels the rectum, anus, and prostate (in males) to check for anything abnormal. A prostate-specific antigen (PSA) test is a test that measures the level of PSA in the blood. PSA is a substance made mostly by the prostate that may be found in an increased amount in the blood of men who have prostate cancer. The level of PSA may also be high in men who have an infection or inflammation of the prostate or benign prostatic hyperplasia (BPH; an enlarged, but noncancerous, prostate). A PSA test or a DRE may be able to detect prostate cancer at an early stage, but it is not clear whether early detection and treatment decrease the risk of dying from prostate cancer. Studies are being done to find ways to make PSA testing more accurate for early cancer detection. If a man had a high PSA level and a biopsy of the prostate did not show cancer and the PSA level remains high after the biopsy, a prostate cancer gene 3 (PCA3) RNA test may be done. This test measures the amount of PCA3 RNA in the urine after a DRE. If the PCA3 RNA level is higher than normal, another biopsy may help diagnose prostate cancer. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Decisions about screening tests can be difficult. Not all screening tests are helpful and most have risks. Before having any screening test, you may want to discuss the test with your doctor. It is important to know the risks of the test and whether it has been proven to reduce the risk of dying from cancer. Screening may not improve your health or help you live longer if you have cancer that has already spread to the area outside of the prostate or to other places in your body. Some cancers never cause symptoms or become life-threatening, but if found by a screening test, the cancer may be treated. Finding these cancers is called overdiagnosis. It is not known if treatment of these cancers would help you live longer than if no treatment were given. Treatments for prostate cancer, such as radical prostatectomy and radiation therapy, may have long-term side effects in many men. The most common side effects are erectile dysfunction and urinary incontinence. Some studies of patients with newly diagnosed prostate cancer showed these patients had a higher risk of death from cardiovascular (heart and blood vessel) disease or suicide. The risk was greatest in the first weeks or months after diagnosis. If a PSA test is higher than normal, a biopsy of the prostate may be done. Complications from a biopsy of the prostate may include fever, pain, blood in the urine or semen, and urinary tract infection. Even if a biopsy shows that a patient does not have prostate cancer, he may worry more about developing prostate cancer in the future. Magnetic resonance imaging (MRI)−guided biopsy is being studied in the diagnosis of prostate cancer, either in place of, or in addition to, standard prostate needle biopsy. Screening test results may appear to be normal even though prostate cancer is present. A man who receives a false-negative test result (one that shows there is no cancer when there really is) may delay seeking medical care even if he has symptoms. Screening test results may appear to be abnormal even though no cancer is present. A false-positive test result (one that shows there is cancer when there really isn't) can cause anxiety and is usually followed by more tests, (such as biopsy) which also have risks. Your doctor can advise you about your risk for prostate cancer and your need for screening tests. Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about prostate cancer screening. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Screening and Prevention Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Screening and Prevention Editorial Board. PDQ Prostate Cancer Screening. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/prostate/patient/prostate-screening-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389306] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Prostate Cancer Screening (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is (are) Prostate Cancer ?

Key Points - Prostate cancer is a disease in which malignant (cancer) cells form in the tissues of the prostate. - Prostate cancer is the most common nonskin cancer among men in the United States. - Different factors increase or decrease the risk of developing prostate cancer. Prostate cancer is a disease in which malignant (cancer) cells form in the tissues of the prostate. The prostate is a gland in the male reproductive system located just below the bladder (the organ that collects and empties urine) and in front of the rectum (the lower part of the intestine). It is about the size of a walnut and surrounds part of the urethra (the tube that empties urine from the bladder). The prostate gland produces fluid that makes up part of semen. As men age, the prostate may get bigger. A bigger prostate may block the flow of urine from the bladder and cause problems with sexual function. This condition is called benign prostatic hyperplasia (BPH), and although it is not cancer, surgery may be needed to correct it. The symptoms of benign prostatic hyperplasia or of other problems in the prostate may be similar to symptoms of prostate cancer. See the following PDQ summaries for more information about prostate cancer: - Prostate Cancer Prevention - Prostate Cancer Treatment

Screening is looking for cancer before a person has any symptoms. This can help find cancer at an early stage. When abnormal tissue or cancer is found early, it may be easier to treat. By the time symptoms appear, cancer may have begun to spread. Scientists are trying to better understand which people are more likely to get certain types of cancer. They also study the things we do and the things around us to see if they cause cancer. This information helps doctors recommend who should be screened for cancer, which screening tests should be used, and how often the tests should be done. It is important to remember that your doctor does not necessarily think you have cancer if he or she suggests a screening test. Screening tests are given when you have no cancer symptoms. Screening tests may be repeated on a regular basis. If a screening test result is abnormal, you may need to have more tests done to find out if you have cancer. These are called diagnostic tests. The prostate is a gland in the male reproductive system located just below the bladder (the organ that collects and empties urine) and in front of the rectum (the lower part of the intestine). It is about the size of a walnut and surrounds part of the urethra (the tube that empties urine from the bladder). The prostate gland produces fluid that makes up part of semen. EnlargeAnatomy of the male reproductive and urinary systems, showing the prostate, testicles, bladder, and other organs. As men age, the prostate may get bigger. A bigger prostate may block the flow of urine from the bladder and cause problems with sexual function. This condition is called benign prostatic hyperplasia (BPH), and although it is not cancer, surgery may be needed to correct it. The symptoms of benign prostatic hyperplasia or of other problems in the prostate may be similar to symptoms of prostate cancer. EnlargeNormal prostate and benign prostatic hyperplasia (BPH). A normal prostate does not block the flow of urine from the bladder. An enlarged prostate presses on the bladder and urethra and blocks the flow of urine. See the following PDQ summaries for more information about prostate cancer: Prostate cancer is found mainly in older men. In the U.S., about one out of every 8 men will be diagnosed with prostate cancer. Most men diagnosed with this disease do not die from it. Prostate cancer causes more deaths in men than any other cancer except lung cancer. Prostate cancer occurs more often in African American men than in White men. African American men with prostate cancer are more likely to die from the disease than White men with prostate cancer. Anything that increases a person's chance of developing a disease is called a risk factor. Anything that decreases your chance of getting a disease is called a protective factor. For information about risk factors and protective factors for prostate cancer, see the PDQ summary on Prostate Cancer Prevention. Scientists study screening tests to find those with the fewest harms and most benefits. Cancer screening trials also are meant to show whether early detection (finding cancer before it causes symptoms) helps a person live longer or decreases a person's chance of dying from the disease. For some types of cancer, the chance of recovery is better if the disease is found and treated at an early stage. Although there are no standard or routine screening tests for prostate cancer, the following tests are being used or studied to screen for it: Digital rectal exam (DRE) is an exam of the rectum. The doctor or nurse inserts a lubricated, gloved finger into the lower part of the rectum to feel the prostate for lumps or anything else that seems unusual.EnlargeDigital rectal exam (DRE). The doctor inserts a gloved, lubricated finger into the rectum and feels the rectum, anus, and prostate (in males) to check for anything abnormal. A prostate-specific antigen (PSA) test is a test that measures the level of PSA in the blood. PSA is a substance made mostly by the prostate that may be found in an increased amount in the blood of men who have prostate cancer. The level of PSA may also be high in men who have an infection or inflammation of the prostate or benign prostatic hyperplasia (BPH; an enlarged, but noncancerous, prostate). A PSA test or a DRE may be able to detect prostate cancer at an early stage, but it is not clear whether early detection and treatment decrease the risk of dying from prostate cancer. Studies are being done to find ways to make PSA testing more accurate for early cancer detection. If a man had a high PSA level and a biopsy of the prostate did not show cancer and the PSA level remains high after the biopsy, a prostate cancer gene 3 (PCA3) RNA test may be done. This test measures the amount of PCA3 RNA in the urine after a DRE. If the PCA3 RNA level is higher than normal, another biopsy may help diagnose prostate cancer. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Decisions about screening tests can be difficult. Not all screening tests are helpful and most have risks. Before having any screening test, you may want to discuss the test with your doctor. It is important to know the risks of the test and whether it has been proven to reduce the risk of dying from cancer. Screening may not improve your health or help you live longer if you have cancer that has already spread to the area outside of the prostate or to other places in your body. Some cancers never cause symptoms or become life-threatening, but if found by a screening test, the cancer may be treated. Finding these cancers is called overdiagnosis. It is not known if treatment of these cancers would help you live longer than if no treatment were given. Treatments for prostate cancer, such as radical prostatectomy and radiation therapy, may have long-term side effects in many men. The most common side effects are erectile dysfunction and urinary incontinence. Some studies of patients with newly diagnosed prostate cancer showed these patients had a higher risk of death from cardiovascular (heart and blood vessel) disease or suicide. The risk was greatest in the first weeks or months after diagnosis. If a PSA test is higher than normal, a biopsy of the prostate may be done. Complications from a biopsy of the prostate may include fever, pain, blood in the urine or semen, and urinary tract infection. Even if a biopsy shows that a patient does not have prostate cancer, he may worry more about developing prostate cancer in the future. Magnetic resonance imaging (MRI)−guided biopsy is being studied in the diagnosis of prostate cancer, either in place of, or in addition to, standard prostate needle biopsy. Screening test results may appear to be normal even though prostate cancer is present. A man who receives a false-negative test result (one that shows there is no cancer when there really is) may delay seeking medical care even if he has symptoms. Screening test results may appear to be abnormal even though no cancer is present. A false-positive test result (one that shows there is cancer when there really isn't) can cause anxiety and is usually followed by more tests, (such as biopsy) which also have risks. Your doctor can advise you about your risk for prostate cancer and your need for screening tests. Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about prostate cancer screening. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Screening and Prevention Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Screening and Prevention Editorial Board. PDQ Prostate Cancer Screening. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/prostate/patient/prostate-screening-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389306] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Prostate Cancer Screening (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

Who is at risk for Prostate Cancer? ?

Prostate cancer is the most common nonskin cancer among men in the United States. Prostate cancer is found mainly in older men. Although the number of men with prostate cancer is large, most men diagnosed with this disease do not die from it. Prostate cancer causes more deaths in men than any other cancer except lung cancer and colorectal cancer. Prostate cancer occurs more often in African-American men than in white men. African-American men with prostate cancer are more likely to die from the disease than white men with prostate cancer.

Screening is looking for cancer before a person has any symptoms. This can help find cancer at an early stage. When abnormal tissue or cancer is found early, it may be easier to treat. By the time symptoms appear, cancer may have begun to spread. Scientists are trying to better understand which people are more likely to get certain types of cancer. They also study the things we do and the things around us to see if they cause cancer. This information helps doctors recommend who should be screened for cancer, which screening tests should be used, and how often the tests should be done. It is important to remember that your doctor does not necessarily think you have cancer if he or she suggests a screening test. Screening tests are given when you have no cancer symptoms. Screening tests may be repeated on a regular basis. If a screening test result is abnormal, you may need to have more tests done to find out if you have cancer. These are called diagnostic tests. The prostate is a gland in the male reproductive system located just below the bladder (the organ that collects and empties urine) and in front of the rectum (the lower part of the intestine). It is about the size of a walnut and surrounds part of the urethra (the tube that empties urine from the bladder). The prostate gland produces fluid that makes up part of semen. EnlargeAnatomy of the male reproductive and urinary systems, showing the prostate, testicles, bladder, and other organs. As men age, the prostate may get bigger. A bigger prostate may block the flow of urine from the bladder and cause problems with sexual function. This condition is called benign prostatic hyperplasia (BPH), and although it is not cancer, surgery may be needed to correct it. The symptoms of benign prostatic hyperplasia or of other problems in the prostate may be similar to symptoms of prostate cancer. EnlargeNormal prostate and benign prostatic hyperplasia (BPH). A normal prostate does not block the flow of urine from the bladder. An enlarged prostate presses on the bladder and urethra and blocks the flow of urine. See the following PDQ summaries for more information about prostate cancer: Prostate cancer is found mainly in older men. In the U.S., about one out of every 8 men will be diagnosed with prostate cancer. Most men diagnosed with this disease do not die from it. Prostate cancer causes more deaths in men than any other cancer except lung cancer. Prostate cancer occurs more often in African American men than in White men. African American men with prostate cancer are more likely to die from the disease than White men with prostate cancer. Anything that increases a person's chance of developing a disease is called a risk factor. Anything that decreases your chance of getting a disease is called a protective factor. For information about risk factors and protective factors for prostate cancer, see the PDQ summary on Prostate Cancer Prevention. Scientists study screening tests to find those with the fewest harms and most benefits. Cancer screening trials also are meant to show whether early detection (finding cancer before it causes symptoms) helps a person live longer or decreases a person's chance of dying from the disease. For some types of cancer, the chance of recovery is better if the disease is found and treated at an early stage. Although there are no standard or routine screening tests for prostate cancer, the following tests are being used or studied to screen for it: Digital rectal exam (DRE) is an exam of the rectum. The doctor or nurse inserts a lubricated, gloved finger into the lower part of the rectum to feel the prostate for lumps or anything else that seems unusual.EnlargeDigital rectal exam (DRE). The doctor inserts a gloved, lubricated finger into the rectum and feels the rectum, anus, and prostate (in males) to check for anything abnormal. A prostate-specific antigen (PSA) test is a test that measures the level of PSA in the blood. PSA is a substance made mostly by the prostate that may be found in an increased amount in the blood of men who have prostate cancer. The level of PSA may also be high in men who have an infection or inflammation of the prostate or benign prostatic hyperplasia (BPH; an enlarged, but noncancerous, prostate). A PSA test or a DRE may be able to detect prostate cancer at an early stage, but it is not clear whether early detection and treatment decrease the risk of dying from prostate cancer. Studies are being done to find ways to make PSA testing more accurate for early cancer detection. If a man had a high PSA level and a biopsy of the prostate did not show cancer and the PSA level remains high after the biopsy, a prostate cancer gene 3 (PCA3) RNA test may be done. This test measures the amount of PCA3 RNA in the urine after a DRE. If the PCA3 RNA level is higher than normal, another biopsy may help diagnose prostate cancer. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Decisions about screening tests can be difficult. Not all screening tests are helpful and most have risks. Before having any screening test, you may want to discuss the test with your doctor. It is important to know the risks of the test and whether it has been proven to reduce the risk of dying from cancer. Screening may not improve your health or help you live longer if you have cancer that has already spread to the area outside of the prostate or to other places in your body. Some cancers never cause symptoms or become life-threatening, but if found by a screening test, the cancer may be treated. Finding these cancers is called overdiagnosis. It is not known if treatment of these cancers would help you live longer than if no treatment were given. Treatments for prostate cancer, such as radical prostatectomy and radiation therapy, may have long-term side effects in many men. The most common side effects are erectile dysfunction and urinary incontinence. Some studies of patients with newly diagnosed prostate cancer showed these patients had a higher risk of death from cardiovascular (heart and blood vessel) disease or suicide. The risk was greatest in the first weeks or months after diagnosis. If a PSA test is higher than normal, a biopsy of the prostate may be done. Complications from a biopsy of the prostate may include fever, pain, blood in the urine or semen, and urinary tract infection. Even if a biopsy shows that a patient does not have prostate cancer, he may worry more about developing prostate cancer in the future. Magnetic resonance imaging (MRI)−guided biopsy is being studied in the diagnosis of prostate cancer, either in place of, or in addition to, standard prostate needle biopsy. Screening test results may appear to be normal even though prostate cancer is present. A man who receives a false-negative test result (one that shows there is no cancer when there really is) may delay seeking medical care even if he has symptoms. Screening test results may appear to be abnormal even though no cancer is present. A false-positive test result (one that shows there is cancer when there really isn't) can cause anxiety and is usually followed by more tests, (such as biopsy) which also have risks. Your doctor can advise you about your risk for prostate cancer and your need for screening tests. Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about prostate cancer screening. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Screening and Prevention Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Screening and Prevention Editorial Board. PDQ Prostate Cancer Screening. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/prostate/patient/prostate-screening-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389306] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Prostate Cancer Screening (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

Who is at risk for Prostate Cancer? ?

Different factors increase or decrease the risk of developing prostate cancer. Anything that increases a person&apos;s chance of developing a disease is called a risk factor. Anything that decreases your chance of getting a disease is called a protective factor. For information about risk factors and protective factors for prostate cancer, see the PDQ summary on Prostate Cancer Prevention.

Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In myeloproliferative neoplasms, too many blood stem cells become one or more types of blood cells. The neoplasms usually get worse slowly as the number of extra blood cells increases. The type of myeloproliferative neoplasm is based on whether too many red blood cells, white blood cells, or platelets are being made. Sometimes the body will make too many of more than one type of blood cell, but usually one type of blood cell is affected more than the others are. Chronic myeloproliferative neoplasms include the following 6 types: These types are described below. Chronic myeloproliferative neoplasms sometimes become acute leukemia, in which too many abnormal white blood cells are made. The following tests and procedures may be used: Chronic myelogenous leukemia is a disease in which too many white blood cells are made in the bone marrow. See the PDQ summary on Chronic Myelogenous Leukemia Treatment for information on diagnosis, staging, and treatment. In polycythemia vera, the blood becomes thickened with too many red blood cells. The number of white blood cells and platelets may also increase. These extra blood cells may collect in the spleen and cause it to swell. The increased number of red blood cells, white blood cells, or platelets in the blood can cause bleeding problems and make clots form in blood vessels. This can increase the risk of stroke or heart attack. In patients who are older than 65 years or who have a history of blood clots, the risk of stroke or heart attack is higher. Patients also have an increased risk of acute myeloid leukemia or primary myelofibrosis. Polycythemia vera often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may occur as the number of blood cells increases. Other conditions may cause the same signs and symptoms. Check with your doctor if you have any of the following: In addition to a complete blood count, bone marrow aspiration and biopsy, and cytogenetic analysis, a serum erythropoietin test is used to diagnose polycythemia vera. In this test, a sample of blood is checked for the level of erythropoietin (a hormone that stimulates new red blood cells to be made). In polycythemia vera, the erythropoietin level would be lower than normal because the body does not need to make more red blood cells. The bone marrow is made of tissues that make blood cells (red blood cells, white blood cells, and platelets) and a web of fibers that support the blood-forming tissues. In primary myelofibrosis (also called chronic idiopathic myelofibrosis), large numbers of blood stem cells become blood cells that do not mature properly (blasts). The web of fibers inside the bone marrow also becomes very thick (like scar tissue) and slows the blood-forming tissue’s ability to make blood cells. This causes the blood-forming tissues to make fewer and fewer blood cells. In order to make up for the low number of blood cells made in the bone marrow, the liver and spleen begin to make the blood cells. Primary myelofibrosis often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by primary myelofibrosis or by other conditions. Check with your doctor if you have any of the following: Prognosis depends on the following: Essential thrombocythemia causes an abnormal increase in the number of platelets made in the blood and bone marrow. Essential thrombocythemia often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by essential thrombocythemia or by other conditions. Check with your doctor if you have any of the following: Platelets are sticky. When there are too many platelets, they may clump together and make it hard for the blood to flow. Clots may form in blood vessels and there may also be increased bleeding. These can cause serious health problems such as stroke or heart attack. Prognosis and treatment options depend on the following: Chronic neutrophilic leukemia is a disease in which too many blood stem cells become a type of white blood cell called neutrophils. Neutrophils are infection-fighting blood cells that surround and destroy dead cells and foreign substances (such as bacteria). The spleen and liver may swell because of the extra neutrophils. Chronic neutrophilic leukemia may stay the same or it may progress quickly to acute leukemia. Eosinophils are white blood cells that react to allergens (substances that cause an allergic response) and help fight infections caused by certain parasites. In chronic eosinophilic leukemia, there are too many eosinophils in the blood, bone marrow, and other tissues. Chronic eosinophilic leukemia may stay the same for many years or it may progress quickly to acute leukemia. Chronic eosinophilic leukemia may not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by chronic eosinophilic leukemia or by other conditions. Check with your doctor if you have any of the following: The process used to find out if cancer has spread to other parts of the body is called staging. There is no standard staging system for chronic myeloproliferative neoplasms. It is important to know the type of myeloproliferative neoplasm in order to plan treatment. Different types of treatments are available for patients with chronic myeloproliferative neoplasms. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Watchful waiting is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. Phlebotomy is a procedure in which blood is taken from a vein. A sample of blood may be taken for tests such as a CBC or blood chemistry. Sometimes phlebotomy is used as a treatment and blood is taken from the body to remove extra red blood cells. Phlebotomy is used in this way to treat some chronic myeloproliferative neoplasms. Platelet apheresis is a treatment that uses a special machine to remove platelets from the blood. Blood is taken from the patient and put through a blood cell separator where the platelets are removed. The rest of the blood is then returned to the patient’s bloodstream. Transfusion therapy (blood transfusion) is a method of giving red blood cells, white blood cells, or platelets to replace blood cells destroyed by disease or cancer treatment. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). See Drugs Approved for Myeloproliferative Neoplasms for more information. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body, such as the spleen, with cancer. Prednisone and danazol are drugs that may be used to treat anemia in patients with primary myelofibrosis. Anagrelide therapy is used to reduce the risk of blood clots in patients who have too many platelets in their blood. Low-dose aspirin may also be used to reduce the risk of blood clots. Thalidomide, lenalidomide, and pomalidomide are drugs that prevent blood vessels from growing into areas of tumor cells. Erythropoietic growth factors are used to stimulate the bone marrow to make red blood cells. See Drugs Approved for Myeloproliferative Neoplasms for more information. Splenectomy (surgery to remove the spleen) may be done if the spleen is enlarged. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. See Drugs Approved for Myeloproliferative Neoplasms for more information. Other types of targeted therapies are being studied in clinical trials. High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. EnlargeStem cell transplant. (Step 1): Blood is taken from a vein in the arm of the donor. The patient or another person may be the donor. The blood flows through a machine that removes the stem cells. Then the blood is returned to the donor through a vein in the other arm. (Step 2): The patient receives chemotherapy to kill blood-forming cells. The patient may receive radiation therapy (not shown). (Step 3): The patient receives stem cells through a catheter placed into a blood vessel in the chest. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. See the PDQ summary about Chronic Myelogenous Leukemia Treatment for information. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. The purpose of treatment for polycythemia vera is to reduce the number of extra blood cells. Treatment of polycythemia vera may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of primary myelofibrosis in patients without signs or symptoms is usually watchful waiting. Patients with primary myelofibrosis may have signs or symptoms of anemia. Anemia is usually treated with transfusion of red blood cells to relieve symptoms and improve quality of life. In addition, anemia may be treated with: Treatment of primary myelofibrosis in patients with other signs or symptoms may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of essential thrombocythemia in patients younger than 60 years who have no signs or symptoms and an acceptable platelet count is usually watchful waiting. Treatment of other patients may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic neutrophilic leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic eosinophilic leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about chronic myeloproliferative neoplasms, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of chronic myeloproliferative neoplasms. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Chronic Myeloproliferative Neoplasms Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/myeloproliferative/patient/chronic-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389435] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Chronic Myeloproliferative Neoplasms Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is (are) Primary Myelofibrosis ?

Key Points - Primary myelofibrosis is a disease in which abnormal blood cells and fibers build up inside the bone marrow. - Symptoms of primary myelofibrosis include pain below the ribs on the left side and feeling very tired. - Certain factors affect prognosis (chance of recovery) and treatment options for primary myelofibrosis. Primary myelofibrosis is a disease in which abnormal blood cells and fibers build up inside the bone marrow. The bone marrow is made of tissues that make blood cells (red blood cells, white blood cells, and platelets) and a web of fibers that support the blood-forming tissues. In primary myelofibrosis (also called chronic idiopathic myelofibrosis), large numbers of blood stem cells become blood cells that do not mature properly (blasts). The web of fibers inside the bone marrow also becomes very thick (like scar tissue) and slows the blood-forming tissues ability to make blood cells. This causes the blood-forming tissues to make fewer and fewer blood cells. In order to make up for the low number of blood cells made in the bone marrow, the liver and spleen begin to make the blood cells.

Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In myeloproliferative neoplasms, too many blood stem cells become one or more types of blood cells. The neoplasms usually get worse slowly as the number of extra blood cells increases. The type of myeloproliferative neoplasm is based on whether too many red blood cells, white blood cells, or platelets are being made. Sometimes the body will make too many of more than one type of blood cell, but usually one type of blood cell is affected more than the others are. Chronic myeloproliferative neoplasms include the following 6 types: These types are described below. Chronic myeloproliferative neoplasms sometimes become acute leukemia, in which too many abnormal white blood cells are made. The following tests and procedures may be used: Chronic myelogenous leukemia is a disease in which too many white blood cells are made in the bone marrow. See the PDQ summary on Chronic Myelogenous Leukemia Treatment for information on diagnosis, staging, and treatment. In polycythemia vera, the blood becomes thickened with too many red blood cells. The number of white blood cells and platelets may also increase. These extra blood cells may collect in the spleen and cause it to swell. The increased number of red blood cells, white blood cells, or platelets in the blood can cause bleeding problems and make clots form in blood vessels. This can increase the risk of stroke or heart attack. In patients who are older than 65 years or who have a history of blood clots, the risk of stroke or heart attack is higher. Patients also have an increased risk of acute myeloid leukemia or primary myelofibrosis. Polycythemia vera often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may occur as the number of blood cells increases. Other conditions may cause the same signs and symptoms. Check with your doctor if you have any of the following: In addition to a complete blood count, bone marrow aspiration and biopsy, and cytogenetic analysis, a serum erythropoietin test is used to diagnose polycythemia vera. In this test, a sample of blood is checked for the level of erythropoietin (a hormone that stimulates new red blood cells to be made). In polycythemia vera, the erythropoietin level would be lower than normal because the body does not need to make more red blood cells. The bone marrow is made of tissues that make blood cells (red blood cells, white blood cells, and platelets) and a web of fibers that support the blood-forming tissues. In primary myelofibrosis (also called chronic idiopathic myelofibrosis), large numbers of blood stem cells become blood cells that do not mature properly (blasts). The web of fibers inside the bone marrow also becomes very thick (like scar tissue) and slows the blood-forming tissue’s ability to make blood cells. This causes the blood-forming tissues to make fewer and fewer blood cells. In order to make up for the low number of blood cells made in the bone marrow, the liver and spleen begin to make the blood cells. Primary myelofibrosis often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by primary myelofibrosis or by other conditions. Check with your doctor if you have any of the following: Prognosis depends on the following: Essential thrombocythemia causes an abnormal increase in the number of platelets made in the blood and bone marrow. Essential thrombocythemia often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by essential thrombocythemia or by other conditions. Check with your doctor if you have any of the following: Platelets are sticky. When there are too many platelets, they may clump together and make it hard for the blood to flow. Clots may form in blood vessels and there may also be increased bleeding. These can cause serious health problems such as stroke or heart attack. Prognosis and treatment options depend on the following: Chronic neutrophilic leukemia is a disease in which too many blood stem cells become a type of white blood cell called neutrophils. Neutrophils are infection-fighting blood cells that surround and destroy dead cells and foreign substances (such as bacteria). The spleen and liver may swell because of the extra neutrophils. Chronic neutrophilic leukemia may stay the same or it may progress quickly to acute leukemia. Eosinophils are white blood cells that react to allergens (substances that cause an allergic response) and help fight infections caused by certain parasites. In chronic eosinophilic leukemia, there are too many eosinophils in the blood, bone marrow, and other tissues. Chronic eosinophilic leukemia may stay the same for many years or it may progress quickly to acute leukemia. Chronic eosinophilic leukemia may not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by chronic eosinophilic leukemia or by other conditions. Check with your doctor if you have any of the following: The process used to find out if cancer has spread to other parts of the body is called staging. There is no standard staging system for chronic myeloproliferative neoplasms. It is important to know the type of myeloproliferative neoplasm in order to plan treatment. Different types of treatments are available for patients with chronic myeloproliferative neoplasms. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Watchful waiting is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. Phlebotomy is a procedure in which blood is taken from a vein. A sample of blood may be taken for tests such as a CBC or blood chemistry. Sometimes phlebotomy is used as a treatment and blood is taken from the body to remove extra red blood cells. Phlebotomy is used in this way to treat some chronic myeloproliferative neoplasms. Platelet apheresis is a treatment that uses a special machine to remove platelets from the blood. Blood is taken from the patient and put through a blood cell separator where the platelets are removed. The rest of the blood is then returned to the patient’s bloodstream. Transfusion therapy (blood transfusion) is a method of giving red blood cells, white blood cells, or platelets to replace blood cells destroyed by disease or cancer treatment. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). See Drugs Approved for Myeloproliferative Neoplasms for more information. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body, such as the spleen, with cancer. Prednisone and danazol are drugs that may be used to treat anemia in patients with primary myelofibrosis. Anagrelide therapy is used to reduce the risk of blood clots in patients who have too many platelets in their blood. Low-dose aspirin may also be used to reduce the risk of blood clots. Thalidomide, lenalidomide, and pomalidomide are drugs that prevent blood vessels from growing into areas of tumor cells. Erythropoietic growth factors are used to stimulate the bone marrow to make red blood cells. See Drugs Approved for Myeloproliferative Neoplasms for more information. Splenectomy (surgery to remove the spleen) may be done if the spleen is enlarged. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. See Drugs Approved for Myeloproliferative Neoplasms for more information. Other types of targeted therapies are being studied in clinical trials. High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. EnlargeStem cell transplant. (Step 1): Blood is taken from a vein in the arm of the donor. The patient or another person may be the donor. The blood flows through a machine that removes the stem cells. Then the blood is returned to the donor through a vein in the other arm. (Step 2): The patient receives chemotherapy to kill blood-forming cells. The patient may receive radiation therapy (not shown). (Step 3): The patient receives stem cells through a catheter placed into a blood vessel in the chest. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. See the PDQ summary about Chronic Myelogenous Leukemia Treatment for information. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. The purpose of treatment for polycythemia vera is to reduce the number of extra blood cells. Treatment of polycythemia vera may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of primary myelofibrosis in patients without signs or symptoms is usually watchful waiting. Patients with primary myelofibrosis may have signs or symptoms of anemia. Anemia is usually treated with transfusion of red blood cells to relieve symptoms and improve quality of life. In addition, anemia may be treated with: Treatment of primary myelofibrosis in patients with other signs or symptoms may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of essential thrombocythemia in patients younger than 60 years who have no signs or symptoms and an acceptable platelet count is usually watchful waiting. Treatment of other patients may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic neutrophilic leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic eosinophilic leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about chronic myeloproliferative neoplasms, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of chronic myeloproliferative neoplasms. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Chronic Myeloproliferative Neoplasms Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/myeloproliferative/patient/chronic-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389435] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Chronic Myeloproliferative Neoplasms Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the symptoms of Primary Myelofibrosis ?

Symptoms of primary myelofibrosis include pain below the ribs on the left side and feeling very tired. Primary myelofibrosis often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by primary myelofibrosis or by other conditions. Check with your doctor if you have any of the following: - Feeling pain or fullness below the ribs on the left side. - Feeling full sooner than normal when eating. - Feeling very tired. - Shortness of breath. - Easy bruising or bleeding. - Petechiae (flat, red, pinpoint spots under the skin that are caused by bleeding). - Fever. - Night sweats. - Weight loss.

Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In myeloproliferative neoplasms, too many blood stem cells become one or more types of blood cells. The neoplasms usually get worse slowly as the number of extra blood cells increases. The type of myeloproliferative neoplasm is based on whether too many red blood cells, white blood cells, or platelets are being made. Sometimes the body will make too many of more than one type of blood cell, but usually one type of blood cell is affected more than the others are. Chronic myeloproliferative neoplasms include the following 6 types: These types are described below. Chronic myeloproliferative neoplasms sometimes become acute leukemia, in which too many abnormal white blood cells are made. The following tests and procedures may be used: Chronic myelogenous leukemia is a disease in which too many white blood cells are made in the bone marrow. See the PDQ summary on Chronic Myelogenous Leukemia Treatment for information on diagnosis, staging, and treatment. In polycythemia vera, the blood becomes thickened with too many red blood cells. The number of white blood cells and platelets may also increase. These extra blood cells may collect in the spleen and cause it to swell. The increased number of red blood cells, white blood cells, or platelets in the blood can cause bleeding problems and make clots form in blood vessels. This can increase the risk of stroke or heart attack. In patients who are older than 65 years or who have a history of blood clots, the risk of stroke or heart attack is higher. Patients also have an increased risk of acute myeloid leukemia or primary myelofibrosis. Polycythemia vera often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may occur as the number of blood cells increases. Other conditions may cause the same signs and symptoms. Check with your doctor if you have any of the following: In addition to a complete blood count, bone marrow aspiration and biopsy, and cytogenetic analysis, a serum erythropoietin test is used to diagnose polycythemia vera. In this test, a sample of blood is checked for the level of erythropoietin (a hormone that stimulates new red blood cells to be made). In polycythemia vera, the erythropoietin level would be lower than normal because the body does not need to make more red blood cells. The bone marrow is made of tissues that make blood cells (red blood cells, white blood cells, and platelets) and a web of fibers that support the blood-forming tissues. In primary myelofibrosis (also called chronic idiopathic myelofibrosis), large numbers of blood stem cells become blood cells that do not mature properly (blasts). The web of fibers inside the bone marrow also becomes very thick (like scar tissue) and slows the blood-forming tissue’s ability to make blood cells. This causes the blood-forming tissues to make fewer and fewer blood cells. In order to make up for the low number of blood cells made in the bone marrow, the liver and spleen begin to make the blood cells. Primary myelofibrosis often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by primary myelofibrosis or by other conditions. Check with your doctor if you have any of the following: Prognosis depends on the following: Essential thrombocythemia causes an abnormal increase in the number of platelets made in the blood and bone marrow. Essential thrombocythemia often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by essential thrombocythemia or by other conditions. Check with your doctor if you have any of the following: Platelets are sticky. When there are too many platelets, they may clump together and make it hard for the blood to flow. Clots may form in blood vessels and there may also be increased bleeding. These can cause serious health problems such as stroke or heart attack. Prognosis and treatment options depend on the following: Chronic neutrophilic leukemia is a disease in which too many blood stem cells become a type of white blood cell called neutrophils. Neutrophils are infection-fighting blood cells that surround and destroy dead cells and foreign substances (such as bacteria). The spleen and liver may swell because of the extra neutrophils. Chronic neutrophilic leukemia may stay the same or it may progress quickly to acute leukemia. Eosinophils are white blood cells that react to allergens (substances that cause an allergic response) and help fight infections caused by certain parasites. In chronic eosinophilic leukemia, there are too many eosinophils in the blood, bone marrow, and other tissues. Chronic eosinophilic leukemia may stay the same for many years or it may progress quickly to acute leukemia. Chronic eosinophilic leukemia may not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by chronic eosinophilic leukemia or by other conditions. Check with your doctor if you have any of the following: The process used to find out if cancer has spread to other parts of the body is called staging. There is no standard staging system for chronic myeloproliferative neoplasms. It is important to know the type of myeloproliferative neoplasm in order to plan treatment. Different types of treatments are available for patients with chronic myeloproliferative neoplasms. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Watchful waiting is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. Phlebotomy is a procedure in which blood is taken from a vein. A sample of blood may be taken for tests such as a CBC or blood chemistry. Sometimes phlebotomy is used as a treatment and blood is taken from the body to remove extra red blood cells. Phlebotomy is used in this way to treat some chronic myeloproliferative neoplasms. Platelet apheresis is a treatment that uses a special machine to remove platelets from the blood. Blood is taken from the patient and put through a blood cell separator where the platelets are removed. The rest of the blood is then returned to the patient’s bloodstream. Transfusion therapy (blood transfusion) is a method of giving red blood cells, white blood cells, or platelets to replace blood cells destroyed by disease or cancer treatment. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). See Drugs Approved for Myeloproliferative Neoplasms for more information. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body, such as the spleen, with cancer. Prednisone and danazol are drugs that may be used to treat anemia in patients with primary myelofibrosis. Anagrelide therapy is used to reduce the risk of blood clots in patients who have too many platelets in their blood. Low-dose aspirin may also be used to reduce the risk of blood clots. Thalidomide, lenalidomide, and pomalidomide are drugs that prevent blood vessels from growing into areas of tumor cells. Erythropoietic growth factors are used to stimulate the bone marrow to make red blood cells. See Drugs Approved for Myeloproliferative Neoplasms for more information. Splenectomy (surgery to remove the spleen) may be done if the spleen is enlarged. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. See Drugs Approved for Myeloproliferative Neoplasms for more information. Other types of targeted therapies are being studied in clinical trials. High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. EnlargeStem cell transplant. (Step 1): Blood is taken from a vein in the arm of the donor. The patient or another person may be the donor. The blood flows through a machine that removes the stem cells. Then the blood is returned to the donor through a vein in the other arm. (Step 2): The patient receives chemotherapy to kill blood-forming cells. The patient may receive radiation therapy (not shown). (Step 3): The patient receives stem cells through a catheter placed into a blood vessel in the chest. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. See the PDQ summary about Chronic Myelogenous Leukemia Treatment for information. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. The purpose of treatment for polycythemia vera is to reduce the number of extra blood cells. Treatment of polycythemia vera may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of primary myelofibrosis in patients without signs or symptoms is usually watchful waiting. Patients with primary myelofibrosis may have signs or symptoms of anemia. Anemia is usually treated with transfusion of red blood cells to relieve symptoms and improve quality of life. In addition, anemia may be treated with: Treatment of primary myelofibrosis in patients with other signs or symptoms may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of essential thrombocythemia in patients younger than 60 years who have no signs or symptoms and an acceptable platelet count is usually watchful waiting. Treatment of other patients may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic neutrophilic leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic eosinophilic leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about chronic myeloproliferative neoplasms, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of chronic myeloproliferative neoplasms. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Chronic Myeloproliferative Neoplasms Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/myeloproliferative/patient/chronic-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389435] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Chronic Myeloproliferative Neoplasms Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is the outlook for Primary Myelofibrosis ?

Certain factors affect prognosis (chance of recovery) and treatment options for primary myelofibrosis. Prognosis (chance of recovery) depends on the following: - The age of the patient. - The number of abnormal red blood cells and white blood cells. - The number of blasts in the blood. - Whether there are certain changes in the chromosomes. - Whether the patient has signs such as fever, night sweats, or weight loss.

Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In myeloproliferative neoplasms, too many blood stem cells become one or more types of blood cells. The neoplasms usually get worse slowly as the number of extra blood cells increases. The type of myeloproliferative neoplasm is based on whether too many red blood cells, white blood cells, or platelets are being made. Sometimes the body will make too many of more than one type of blood cell, but usually one type of blood cell is affected more than the others are. Chronic myeloproliferative neoplasms include the following 6 types: These types are described below. Chronic myeloproliferative neoplasms sometimes become acute leukemia, in which too many abnormal white blood cells are made. The following tests and procedures may be used: Chronic myelogenous leukemia is a disease in which too many white blood cells are made in the bone marrow. See the PDQ summary on Chronic Myelogenous Leukemia Treatment for information on diagnosis, staging, and treatment. In polycythemia vera, the blood becomes thickened with too many red blood cells. The number of white blood cells and platelets may also increase. These extra blood cells may collect in the spleen and cause it to swell. The increased number of red blood cells, white blood cells, or platelets in the blood can cause bleeding problems and make clots form in blood vessels. This can increase the risk of stroke or heart attack. In patients who are older than 65 years or who have a history of blood clots, the risk of stroke or heart attack is higher. Patients also have an increased risk of acute myeloid leukemia or primary myelofibrosis. Polycythemia vera often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may occur as the number of blood cells increases. Other conditions may cause the same signs and symptoms. Check with your doctor if you have any of the following: In addition to a complete blood count, bone marrow aspiration and biopsy, and cytogenetic analysis, a serum erythropoietin test is used to diagnose polycythemia vera. In this test, a sample of blood is checked for the level of erythropoietin (a hormone that stimulates new red blood cells to be made). In polycythemia vera, the erythropoietin level would be lower than normal because the body does not need to make more red blood cells. The bone marrow is made of tissues that make blood cells (red blood cells, white blood cells, and platelets) and a web of fibers that support the blood-forming tissues. In primary myelofibrosis (also called chronic idiopathic myelofibrosis), large numbers of blood stem cells become blood cells that do not mature properly (blasts). The web of fibers inside the bone marrow also becomes very thick (like scar tissue) and slows the blood-forming tissue’s ability to make blood cells. This causes the blood-forming tissues to make fewer and fewer blood cells. In order to make up for the low number of blood cells made in the bone marrow, the liver and spleen begin to make the blood cells. Primary myelofibrosis often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by primary myelofibrosis or by other conditions. Check with your doctor if you have any of the following: Prognosis depends on the following: Essential thrombocythemia causes an abnormal increase in the number of platelets made in the blood and bone marrow. Essential thrombocythemia often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by essential thrombocythemia or by other conditions. Check with your doctor if you have any of the following: Platelets are sticky. When there are too many platelets, they may clump together and make it hard for the blood to flow. Clots may form in blood vessels and there may also be increased bleeding. These can cause serious health problems such as stroke or heart attack. Prognosis and treatment options depend on the following: Chronic neutrophilic leukemia is a disease in which too many blood stem cells become a type of white blood cell called neutrophils. Neutrophils are infection-fighting blood cells that surround and destroy dead cells and foreign substances (such as bacteria). The spleen and liver may swell because of the extra neutrophils. Chronic neutrophilic leukemia may stay the same or it may progress quickly to acute leukemia. Eosinophils are white blood cells that react to allergens (substances that cause an allergic response) and help fight infections caused by certain parasites. In chronic eosinophilic leukemia, there are too many eosinophils in the blood, bone marrow, and other tissues. Chronic eosinophilic leukemia may stay the same for many years or it may progress quickly to acute leukemia. Chronic eosinophilic leukemia may not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by chronic eosinophilic leukemia or by other conditions. Check with your doctor if you have any of the following: The process used to find out if cancer has spread to other parts of the body is called staging. There is no standard staging system for chronic myeloproliferative neoplasms. It is important to know the type of myeloproliferative neoplasm in order to plan treatment. Different types of treatments are available for patients with chronic myeloproliferative neoplasms. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Watchful waiting is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. Phlebotomy is a procedure in which blood is taken from a vein. A sample of blood may be taken for tests such as a CBC or blood chemistry. Sometimes phlebotomy is used as a treatment and blood is taken from the body to remove extra red blood cells. Phlebotomy is used in this way to treat some chronic myeloproliferative neoplasms. Platelet apheresis is a treatment that uses a special machine to remove platelets from the blood. Blood is taken from the patient and put through a blood cell separator where the platelets are removed. The rest of the blood is then returned to the patient’s bloodstream. Transfusion therapy (blood transfusion) is a method of giving red blood cells, white blood cells, or platelets to replace blood cells destroyed by disease or cancer treatment. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). See Drugs Approved for Myeloproliferative Neoplasms for more information. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body, such as the spleen, with cancer. Prednisone and danazol are drugs that may be used to treat anemia in patients with primary myelofibrosis. Anagrelide therapy is used to reduce the risk of blood clots in patients who have too many platelets in their blood. Low-dose aspirin may also be used to reduce the risk of blood clots. Thalidomide, lenalidomide, and pomalidomide are drugs that prevent blood vessels from growing into areas of tumor cells. Erythropoietic growth factors are used to stimulate the bone marrow to make red blood cells. See Drugs Approved for Myeloproliferative Neoplasms for more information. Splenectomy (surgery to remove the spleen) may be done if the spleen is enlarged. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. See Drugs Approved for Myeloproliferative Neoplasms for more information. Other types of targeted therapies are being studied in clinical trials. High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. EnlargeStem cell transplant. (Step 1): Blood is taken from a vein in the arm of the donor. The patient or another person may be the donor. The blood flows through a machine that removes the stem cells. Then the blood is returned to the donor through a vein in the other arm. (Step 2): The patient receives chemotherapy to kill blood-forming cells. The patient may receive radiation therapy (not shown). (Step 3): The patient receives stem cells through a catheter placed into a blood vessel in the chest. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. See the PDQ summary about Chronic Myelogenous Leukemia Treatment for information. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. The purpose of treatment for polycythemia vera is to reduce the number of extra blood cells. Treatment of polycythemia vera may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of primary myelofibrosis in patients without signs or symptoms is usually watchful waiting. Patients with primary myelofibrosis may have signs or symptoms of anemia. Anemia is usually treated with transfusion of red blood cells to relieve symptoms and improve quality of life. In addition, anemia may be treated with: Treatment of primary myelofibrosis in patients with other signs or symptoms may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of essential thrombocythemia in patients younger than 60 years who have no signs or symptoms and an acceptable platelet count is usually watchful waiting. Treatment of other patients may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic neutrophilic leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic eosinophilic leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about chronic myeloproliferative neoplasms, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of chronic myeloproliferative neoplasms. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Chronic Myeloproliferative Neoplasms Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/myeloproliferative/patient/chronic-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389435] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Chronic Myeloproliferative Neoplasms Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the treatments for Primary Myelofibrosis ?

Treatment of primary myelofibrosis in patients without signs or symptoms is usually watchful waiting. Patients with primary myelofibrosis may have signs or symptoms of anemia. Anemia is usually treated with transfusion of red blood cells to relieve symptoms and improve quality of life. In addition, anemia may be treated with: - Erythropoietic growth factors. - Prednisone. - Danazol. - Thalidomide, lenalidomide, or pomalidomide, with or without prednisone. Treatment of primary myelofibrosis in patients with other signs or symptoms may include the following: - Targeted therapy with ruxolitinib. - Chemotherapy. - Donor stem cell transplant. - Thalidomide, lenalidomide, or pomalidomide. - Splenectomy. - Radiation therapy to the spleen, lymph nodes, or other areas outside the bone marrow where blood cells are forming. - Biologic therapy using interferon alfa or erythropoietic growth factors. - A clinical trial of other targeted therapy drugs. Check the list of NCI-supported cancer clinical trials that are now accepting patients with primary myelofibrosis. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website.

There are different types of treatment for patients with liver cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. Surveillance is used for lesions smaller than 1 centimeter found during screening. Follow-up every 3 months is common. Surveillance is closely watching a patient’s condition but not giving any treatment unless there are changes in test results that show the condition is getting worse. During active surveillance, certain exams and tests are done on a regular schedule. A partial hepatectomy (surgery to remove the part of the liver where cancer is found) may be done. A wedge of tissue, an entire lobe, or a larger part of the liver, along with some of the healthy tissue around it is removed. The remaining liver tissue takes over the functions of the liver and may regrow. In a liver transplant, the entire liver is removed and replaced with a healthy donated liver. A liver transplant may be done when the disease is in the liver only and a donated liver can be found. If the patient has to wait for a donated liver, other treatment is given as needed. Ablation therapy removes or destroys tissue. Different types of ablation therapy are used for liver cancer: Embolization therapy is used for patients who cannot have surgery to remove the tumor or ablation therapy and whose tumor has not spread outside the liver. Embolization therapy is the use of substances to block or decrease the flow of blood through the hepatic artery to the tumor. When the tumor does not get the oxygen and nutrients it needs, it will not continue to grow. The liver receives blood from the hepatic portal vein and the hepatic artery. Blood that comes into the liver from the hepatic portal vein usually goes to the healthy liver tissue. Blood that comes from the hepatic artery usually goes to the tumor. When the hepatic artery is blocked during embolization therapy, the healthy liver tissue continues to receive blood from the hepatic portal vein. There are two main types of embolization therapy: Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Targeted therapies used to treat advanced liver cancer include the following: To learn more about targeted therapy and its side effects, see Targeted Cancer Therapies. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. Immune checkpoint inhibitors are a type of immunotherapy. Immune checkpoint inhibitors that may be used to treat liver cancer include the following: To learn more about immunotherapy and its side effects, see Immunotherapy to Treat Cancer and Immunotherapy Side Effects. External radiation therapy uses a machine outside the body to send high-energy x-rays or other types of radiation toward the area of the body with cancer. This kills cancer cells or keeps them from growing. Certain ways of giving external radiation therapy can help keep radiation from damaging nearby healthy tissue: To learn more about radiation therapy and its side effects, see Radiation Therapy to Treat Cancer and Radiation Therapy Side Effects. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. For some patients, taking part in a clinical trial may be an option. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. To learn more about clinical trials, see Clinical Trials Information for Patients and Caregivers. Treatment of localized liver cancer may include the following: Treatment of locally advanced or metastatic liver cancer may include the following: Treatment options for recurrent primary liver cancer may include the following: If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Liver Cancer Treatment was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is (are) Adult Primary Liver Cancer ?

Key Points - Adult primary liver cancer is a disease in which malignant (cancer) cells form in the tissues of the liver. - There are two types of adult primary liver cancer. - Having hepatitis or cirrhosis can affect the risk of adult primary liver cancer. - Signs and symptoms of adult primary liver cancer include a lump or pain on the right side. - Tests that examine the liver and the blood are used to detect (find) and diagnose adult primary liver cancer. - Certain factors affect prognosis (chance of recovery) and treatment options. Adult primary liver cancer is a disease in which malignant (cancer) cells form in the tissues of the liver. The liver is one of the largest organs in the body. It has four lobes and fills the upper right side of the abdomen inside the rib cage. Three of the many important functions of the liver are: - To filter harmful substances from the blood so they can be passed from the body in stools and urine. - To make bile to help digest fat that comes from food. - To store glycogen (sugar), which the body uses for energy. There are two types of adult primary liver cancer. The two types of adult primary liver cancer are: - Hepatocellular carcinoma. - Cholangiocarcinoma (bile duct cancer). (See the PDQ summary on Bile Duct Cancer Treatment for more information.) The most common type of adult primary liver cancer is hepatocellular carcinoma. This type of liver cancer is the third leading cause of cancer-related deaths worldwide. This summary is about the treatment of primary liver cancer (cancer that begins in the liver). Treatment of cancer that begins in other parts of the body and spreads to the liver is not covered in this summary. Primary liver cancer can occur in both adults and children. However, treatment for children is different than treatment for adults. (See the PDQ summary on Childhood Liver Cancer Treatment for more information.)

There are different types of treatment for patients with liver cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. Surveillance is used for lesions smaller than 1 centimeter found during screening. Follow-up every 3 months is common. Surveillance is closely watching a patient’s condition but not giving any treatment unless there are changes in test results that show the condition is getting worse. During active surveillance, certain exams and tests are done on a regular schedule. A partial hepatectomy (surgery to remove the part of the liver where cancer is found) may be done. A wedge of tissue, an entire lobe, or a larger part of the liver, along with some of the healthy tissue around it is removed. The remaining liver tissue takes over the functions of the liver and may regrow. In a liver transplant, the entire liver is removed and replaced with a healthy donated liver. A liver transplant may be done when the disease is in the liver only and a donated liver can be found. If the patient has to wait for a donated liver, other treatment is given as needed. Ablation therapy removes or destroys tissue. Different types of ablation therapy are used for liver cancer: Embolization therapy is used for patients who cannot have surgery to remove the tumor or ablation therapy and whose tumor has not spread outside the liver. Embolization therapy is the use of substances to block or decrease the flow of blood through the hepatic artery to the tumor. When the tumor does not get the oxygen and nutrients it needs, it will not continue to grow. The liver receives blood from the hepatic portal vein and the hepatic artery. Blood that comes into the liver from the hepatic portal vein usually goes to the healthy liver tissue. Blood that comes from the hepatic artery usually goes to the tumor. When the hepatic artery is blocked during embolization therapy, the healthy liver tissue continues to receive blood from the hepatic portal vein. There are two main types of embolization therapy: Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Targeted therapies used to treat advanced liver cancer include the following: To learn more about targeted therapy and its side effects, see Targeted Cancer Therapies. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. Immune checkpoint inhibitors are a type of immunotherapy. Immune checkpoint inhibitors that may be used to treat liver cancer include the following: To learn more about immunotherapy and its side effects, see Immunotherapy to Treat Cancer and Immunotherapy Side Effects. External radiation therapy uses a machine outside the body to send high-energy x-rays or other types of radiation toward the area of the body with cancer. This kills cancer cells or keeps them from growing. Certain ways of giving external radiation therapy can help keep radiation from damaging nearby healthy tissue: To learn more about radiation therapy and its side effects, see Radiation Therapy to Treat Cancer and Radiation Therapy Side Effects. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. For some patients, taking part in a clinical trial may be an option. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. To learn more about clinical trials, see Clinical Trials Information for Patients and Caregivers. Treatment of localized liver cancer may include the following: Treatment of locally advanced or metastatic liver cancer may include the following: Treatment options for recurrent primary liver cancer may include the following: If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Liver Cancer Treatment was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

Who is at risk for Adult Primary Liver Cancer? ?

Having hepatitis or cirrhosis can affect the risk of adult primary liver cancer. Anything that increases your chance of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesnt mean that you will not get cancer. Talk with your doctor if you think you may be at risk. The following are risk factors for adult primary liver cancer: - Having hepatitis B or hepatitis C. Having both hepatitis B and hepatitis C increases the risk even more. - Having cirrhosis, which can be caused by: - hepatitis (especially hepatitis C); or - drinking large amounts of alcohol for many years or being an alcoholic. - Having metabolic syndrome, a set of conditions that occur together, including extra fat around the abdomen, high blood sugar, high blood pressure, high levels of triglycerides and low levels of high-density lipoproteins in the blood. - Having liver injury that is long-lasting, especially if it leads to cirrhosis. - Having hemochromatosis, a condition in which the body takes up and stores more iron than it needs. The extra iron is stored in the liver, heart, and pancreas - Eating foods tainted with aflatoxin (poison from a fungus that can grow on foods, such as grains and nuts, that have not been stored properly).

There are different types of treatment for patients with liver cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. Surveillance is used for lesions smaller than 1 centimeter found during screening. Follow-up every 3 months is common. Surveillance is closely watching a patient’s condition but not giving any treatment unless there are changes in test results that show the condition is getting worse. During active surveillance, certain exams and tests are done on a regular schedule. A partial hepatectomy (surgery to remove the part of the liver where cancer is found) may be done. A wedge of tissue, an entire lobe, or a larger part of the liver, along with some of the healthy tissue around it is removed. The remaining liver tissue takes over the functions of the liver and may regrow. In a liver transplant, the entire liver is removed and replaced with a healthy donated liver. A liver transplant may be done when the disease is in the liver only and a donated liver can be found. If the patient has to wait for a donated liver, other treatment is given as needed. Ablation therapy removes or destroys tissue. Different types of ablation therapy are used for liver cancer: Embolization therapy is used for patients who cannot have surgery to remove the tumor or ablation therapy and whose tumor has not spread outside the liver. Embolization therapy is the use of substances to block or decrease the flow of blood through the hepatic artery to the tumor. When the tumor does not get the oxygen and nutrients it needs, it will not continue to grow. The liver receives blood from the hepatic portal vein and the hepatic artery. Blood that comes into the liver from the hepatic portal vein usually goes to the healthy liver tissue. Blood that comes from the hepatic artery usually goes to the tumor. When the hepatic artery is blocked during embolization therapy, the healthy liver tissue continues to receive blood from the hepatic portal vein. There are two main types of embolization therapy: Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Targeted therapies used to treat advanced liver cancer include the following: To learn more about targeted therapy and its side effects, see Targeted Cancer Therapies. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. Immune checkpoint inhibitors are a type of immunotherapy. Immune checkpoint inhibitors that may be used to treat liver cancer include the following: To learn more about immunotherapy and its side effects, see Immunotherapy to Treat Cancer and Immunotherapy Side Effects. External radiation therapy uses a machine outside the body to send high-energy x-rays or other types of radiation toward the area of the body with cancer. This kills cancer cells or keeps them from growing. Certain ways of giving external radiation therapy can help keep radiation from damaging nearby healthy tissue: To learn more about radiation therapy and its side effects, see Radiation Therapy to Treat Cancer and Radiation Therapy Side Effects. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. For some patients, taking part in a clinical trial may be an option. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. To learn more about clinical trials, see Clinical Trials Information for Patients and Caregivers. Treatment of localized liver cancer may include the following: Treatment of locally advanced or metastatic liver cancer may include the following: Treatment options for recurrent primary liver cancer may include the following: If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Liver Cancer Treatment was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the symptoms of Adult Primary Liver Cancer ?

Signs and symptoms of adult primary liver cancer include a lump or pain on the right side. These and other signs and symptoms may be caused by adult primary liver cancer or by other conditions. Check with your doctor if you have any of the following: - A hard lump on the right side just below the rib cage. - Discomfort in the upper abdomen on the right side. - A swollen abdomen. - Pain near the right shoulder blade or in the back. - Jaundice (yellowing of the skin and whites of the eyes). - Easy bruising or bleeding. - Unusual tiredness or weakness. - Nausea and vomiting. - Loss of appetite or feelings of fullness after eating a small meal. - Weight loss for no known reason. - Pale, chalky bowel movements and dark urine. - Fever.

There are different types of treatment for patients with liver cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. Surveillance is used for lesions smaller than 1 centimeter found during screening. Follow-up every 3 months is common. Surveillance is closely watching a patient’s condition but not giving any treatment unless there are changes in test results that show the condition is getting worse. During active surveillance, certain exams and tests are done on a regular schedule. A partial hepatectomy (surgery to remove the part of the liver where cancer is found) may be done. A wedge of tissue, an entire lobe, or a larger part of the liver, along with some of the healthy tissue around it is removed. The remaining liver tissue takes over the functions of the liver and may regrow. In a liver transplant, the entire liver is removed and replaced with a healthy donated liver. A liver transplant may be done when the disease is in the liver only and a donated liver can be found. If the patient has to wait for a donated liver, other treatment is given as needed. Ablation therapy removes or destroys tissue. Different types of ablation therapy are used for liver cancer: Embolization therapy is used for patients who cannot have surgery to remove the tumor or ablation therapy and whose tumor has not spread outside the liver. Embolization therapy is the use of substances to block or decrease the flow of blood through the hepatic artery to the tumor. When the tumor does not get the oxygen and nutrients it needs, it will not continue to grow. The liver receives blood from the hepatic portal vein and the hepatic artery. Blood that comes into the liver from the hepatic portal vein usually goes to the healthy liver tissue. Blood that comes from the hepatic artery usually goes to the tumor. When the hepatic artery is blocked during embolization therapy, the healthy liver tissue continues to receive blood from the hepatic portal vein. There are two main types of embolization therapy: Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Targeted therapies used to treat advanced liver cancer include the following: To learn more about targeted therapy and its side effects, see Targeted Cancer Therapies. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. Immune checkpoint inhibitors are a type of immunotherapy. Immune checkpoint inhibitors that may be used to treat liver cancer include the following: To learn more about immunotherapy and its side effects, see Immunotherapy to Treat Cancer and Immunotherapy Side Effects. External radiation therapy uses a machine outside the body to send high-energy x-rays or other types of radiation toward the area of the body with cancer. This kills cancer cells or keeps them from growing. Certain ways of giving external radiation therapy can help keep radiation from damaging nearby healthy tissue: To learn more about radiation therapy and its side effects, see Radiation Therapy to Treat Cancer and Radiation Therapy Side Effects. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. For some patients, taking part in a clinical trial may be an option. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. To learn more about clinical trials, see Clinical Trials Information for Patients and Caregivers. Treatment of localized liver cancer may include the following: Treatment of locally advanced or metastatic liver cancer may include the following: Treatment options for recurrent primary liver cancer may include the following: If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Liver Cancer Treatment was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

How to diagnose Adult Primary Liver Cancer ?

Tests that examine the liver and the blood are used to detect (find) and diagnose adult primary liver cancer. The following tests and procedures may be used: - Physical exam and history : An exam of the body to check general signs of health, including checking for signs of disease, such as lumps or anything else that seems unusual. A history of the patients health habits and past illnesses and treatments will also be taken. - Serum tumor marker test : A procedure in which a sample of blood is examined to measure the amounts of certain substances released into the blood by organs, tissues, or tumor cells in the body. Certain substances are linked to specific types of cancer when found in increased levels in the blood. These are called tumor markers. An increased level of alpha-fetoprotein (AFP) in the blood may be a sign of liver cancer. Other cancers and certain noncancerous conditions, including cirrhosis and hepatitis, may also increase AFP levels. Sometimes the AFP level is normal even when there is liver cancer. - Liver function tests : A procedure in which a blood sample is checked to measure the amounts of certain substances released into the blood by the liver. A higher than normal amount of a substance can be a sign of liver cancer. - CT scan (CAT scan): A procedure that makes a series of detailed pictures of areas inside the body, such as the abdomen, taken from different angles. The pictures are made by a computer linked to an x-ray machine. A dye may be injected into a vein or swallowed to help the organs or tissues show up more clearly. This procedure is also called computed tomography, computerized tomography, or computerized axial tomography. Images may be taken at three different times after the dye is injected, to get the best picture of abnormal areas in the liver. This is called triple-phase CT. A spiral or helical CT scan makes a series of very detailed pictures of areas inside the body using an x-ray machine that scans the body in a spiral path. - MRI (magnetic resonance imaging): A procedure that uses a magnet, radio waves, and a computer to make a series of detailed pictures of areas inside the body, such as the liver. This procedure is also called nuclear magnetic resonance imaging (NMRI). To create detailed pictures of blood vessels in and near the liver, dye is injected into a vein. This procedure is called MRA (magnetic resonance angiography). Images may be taken at three different times after the dye is injected, to get the best picture of abnormal areas in the liver. This is called triple-phase MRI. - Ultrasound exam: A procedure in which high-energy sound waves (ultrasound) are bounced off internal tissues or organs and make echoes. The echoes form a picture of body tissues called a sonogram. The picture can be printed to be looked at later. - Biopsy : The removal of cells or tissues so they can be viewed under a microscope by a pathologist to check for signs of cancer. Procedures used to collect the sample of cells or tissues include the following: - Fine-needle aspiration biopsy : The removal of cells, tissue or fluid using a thin needle. - Core needle biopsy : The removal of cells or tissue using a slightly wider needle. - Laparoscopy : A surgical procedure to look at the organs inside the abdomen to check for signs of disease. Small incisions (cuts) are made in the wall of the abdomen and a laparoscope (a thin, lighted tube) is inserted into one of the incisions. Another instrument is inserted through the same or another incision to remove the tissue samples. A biopsy is not always needed to diagnose adult primary liver cancer.

There are different types of treatment for patients with liver cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. Surveillance is used for lesions smaller than 1 centimeter found during screening. Follow-up every 3 months is common. Surveillance is closely watching a patient’s condition but not giving any treatment unless there are changes in test results that show the condition is getting worse. During active surveillance, certain exams and tests are done on a regular schedule. A partial hepatectomy (surgery to remove the part of the liver where cancer is found) may be done. A wedge of tissue, an entire lobe, or a larger part of the liver, along with some of the healthy tissue around it is removed. The remaining liver tissue takes over the functions of the liver and may regrow. In a liver transplant, the entire liver is removed and replaced with a healthy donated liver. A liver transplant may be done when the disease is in the liver only and a donated liver can be found. If the patient has to wait for a donated liver, other treatment is given as needed. Ablation therapy removes or destroys tissue. Different types of ablation therapy are used for liver cancer: Embolization therapy is used for patients who cannot have surgery to remove the tumor or ablation therapy and whose tumor has not spread outside the liver. Embolization therapy is the use of substances to block or decrease the flow of blood through the hepatic artery to the tumor. When the tumor does not get the oxygen and nutrients it needs, it will not continue to grow. The liver receives blood from the hepatic portal vein and the hepatic artery. Blood that comes into the liver from the hepatic portal vein usually goes to the healthy liver tissue. Blood that comes from the hepatic artery usually goes to the tumor. When the hepatic artery is blocked during embolization therapy, the healthy liver tissue continues to receive blood from the hepatic portal vein. There are two main types of embolization therapy: Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Targeted therapies used to treat advanced liver cancer include the following: To learn more about targeted therapy and its side effects, see Targeted Cancer Therapies. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. Immune checkpoint inhibitors are a type of immunotherapy. Immune checkpoint inhibitors that may be used to treat liver cancer include the following: To learn more about immunotherapy and its side effects, see Immunotherapy to Treat Cancer and Immunotherapy Side Effects. External radiation therapy uses a machine outside the body to send high-energy x-rays or other types of radiation toward the area of the body with cancer. This kills cancer cells or keeps them from growing. Certain ways of giving external radiation therapy can help keep radiation from damaging nearby healthy tissue: To learn more about radiation therapy and its side effects, see Radiation Therapy to Treat Cancer and Radiation Therapy Side Effects. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. For some patients, taking part in a clinical trial may be an option. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. To learn more about clinical trials, see Clinical Trials Information for Patients and Caregivers. Treatment of localized liver cancer may include the following: Treatment of locally advanced or metastatic liver cancer may include the following: Treatment options for recurrent primary liver cancer may include the following: If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Liver Cancer Treatment was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is the outlook for Adult Primary Liver Cancer ?

Certain factors affect prognosis (chance of recovery) and treatment options. The prognosis (chance of recovery) and treatment options depend on the following: - The stage of the cancer (the size of the tumor, whether it affects part or all of the liver, or has spread to other places in the body). - How well the liver is working. - The patients general health, including whether there is cirrhosis of the liver.

There are different types of treatment for patients with liver cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. Surveillance is used for lesions smaller than 1 centimeter found during screening. Follow-up every 3 months is common. Surveillance is closely watching a patient’s condition but not giving any treatment unless there are changes in test results that show the condition is getting worse. During active surveillance, certain exams and tests are done on a regular schedule. A partial hepatectomy (surgery to remove the part of the liver where cancer is found) may be done. A wedge of tissue, an entire lobe, or a larger part of the liver, along with some of the healthy tissue around it is removed. The remaining liver tissue takes over the functions of the liver and may regrow. In a liver transplant, the entire liver is removed and replaced with a healthy donated liver. A liver transplant may be done when the disease is in the liver only and a donated liver can be found. If the patient has to wait for a donated liver, other treatment is given as needed. Ablation therapy removes or destroys tissue. Different types of ablation therapy are used for liver cancer: Embolization therapy is used for patients who cannot have surgery to remove the tumor or ablation therapy and whose tumor has not spread outside the liver. Embolization therapy is the use of substances to block or decrease the flow of blood through the hepatic artery to the tumor. When the tumor does not get the oxygen and nutrients it needs, it will not continue to grow. The liver receives blood from the hepatic portal vein and the hepatic artery. Blood that comes into the liver from the hepatic portal vein usually goes to the healthy liver tissue. Blood that comes from the hepatic artery usually goes to the tumor. When the hepatic artery is blocked during embolization therapy, the healthy liver tissue continues to receive blood from the hepatic portal vein. There are two main types of embolization therapy: Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Targeted therapies used to treat advanced liver cancer include the following: To learn more about targeted therapy and its side effects, see Targeted Cancer Therapies. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. Immune checkpoint inhibitors are a type of immunotherapy. Immune checkpoint inhibitors that may be used to treat liver cancer include the following: To learn more about immunotherapy and its side effects, see Immunotherapy to Treat Cancer and Immunotherapy Side Effects. External radiation therapy uses a machine outside the body to send high-energy x-rays or other types of radiation toward the area of the body with cancer. This kills cancer cells or keeps them from growing. Certain ways of giving external radiation therapy can help keep radiation from damaging nearby healthy tissue: To learn more about radiation therapy and its side effects, see Radiation Therapy to Treat Cancer and Radiation Therapy Side Effects. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. For some patients, taking part in a clinical trial may be an option. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. To learn more about clinical trials, see Clinical Trials Information for Patients and Caregivers. Treatment of localized liver cancer may include the following: Treatment of locally advanced or metastatic liver cancer may include the following: Treatment options for recurrent primary liver cancer may include the following: If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Liver Cancer Treatment was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the stages of Adult Primary Liver Cancer ?

Key Points - After adult primary liver cancer has been diagnosed, tests are done to find out if cancer cells have spread within the liver or to other parts of the body. - There are three ways that cancer spreads in the body. - Cancer may spread from where it began to other parts of the body. - The Barcelona Clinic Liver Cancer Staging System may be used to stage adult primary liver cancer. - The following groups are used to plan treatment. - BCLC stages 0, A, and B - BCLC stages C and D After adult primary liver cancer has been diagnosed, tests are done to find out if cancer cells have spread within the liver or to other parts of the body. The process used to find out if cancer has spread within the liver or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests and procedures may be used in the staging process: - CT scan (CAT scan): A procedure that makes a series of detailed pictures of areas inside the body, such as the chest, abdomen, and pelvis, taken from different angles. The pictures are made by a computer linked to an x-ray machine. A dye may be injected into a vein or swallowed to help the organs or tissues show up more clearly. This procedure is also called computed tomography, computerized tomography, or computerized axial tomography. - MRI (magnetic resonance imaging): A procedure that uses a magnet, radio waves, and a computer to make a series of detailed pictures of areas inside the body. This procedure is also called nuclear magnetic resonance imaging (NMRI). - PET scan (positron emission tomography scan): A procedure to find malignant tumor cells in the body. A small amount of radioactive glucose (sugar) is injected into a vein. The PET scanner rotates around the body and makes a picture of where glucose is being used in the body. Malignant tumor cells show up brighter in the picture because they are more active and take up more glucose than normal cells do. There are three ways that cancer spreads in the body. Cancer can spread through tissue, the lymph system, and the blood: - Tissue. The cancer spreads from where it began by growing into nearby areas. - Lymph system. The cancer spreads from where it began by getting into the lymph system. The cancer travels through the lymph vessels to other parts of the body. - Blood. The cancer spreads from where it began by getting into the blood. The cancer travels through the blood vessels to other parts of the body. Cancer may spread from where it began to other parts of the body. When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. - Lymph system. The cancer gets into the lymph system, travels through the lymph vessels, and forms a tumor (metastatic tumor) in another part of the body. - Blood. The cancer gets into the blood, travels through the blood vessels, and forms a tumor (metastatic tumor) in another part of the body. The metastatic tumor is the same type of cancer as the primary tumor. For example, if primary liver cancer spreads to the lung, the cancer cells in the lung are actually liver cancer cells. The disease is metastatic liver cancer, not lung cancer. The Barcelona Clinic Liver Cancer Staging System may be used to stage adult primary liver cancer. There are several staging systems for liver cancer. The Barcelona Clinic Liver Cancer (BCLC) Staging System is widely used and is described below. This system is used to predict the patient&apos;s chance of recovery and to plan treatment, based on the following: - Whether the cancer has spread within the liver or to other parts of the body. - How well the liver is working. - The general health and wellness of the patient. - The symptoms caused by the cancer. The BCLC staging system has five stages: - Stage 0: Very early - Stage A: Early - Stage B: Intermediate - Stage C: Advanced - Stage D: End-stage The following groups are used to plan treatment. BCLC stages 0, A, and B Treatment to cure the cancer is given for BCLC stages 0, A, and B. BCLC stages C and D Treatment to relieve the symptoms caused by liver cancer and improve the patient&apos;s quality of life is given for BCLC stages C and D. Treatments are not likely to cure the cancer.

There are different types of treatment for patients with liver cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. Surveillance is used for lesions smaller than 1 centimeter found during screening. Follow-up every 3 months is common. Surveillance is closely watching a patient’s condition but not giving any treatment unless there are changes in test results that show the condition is getting worse. During active surveillance, certain exams and tests are done on a regular schedule. A partial hepatectomy (surgery to remove the part of the liver where cancer is found) may be done. A wedge of tissue, an entire lobe, or a larger part of the liver, along with some of the healthy tissue around it is removed. The remaining liver tissue takes over the functions of the liver and may regrow. In a liver transplant, the entire liver is removed and replaced with a healthy donated liver. A liver transplant may be done when the disease is in the liver only and a donated liver can be found. If the patient has to wait for a donated liver, other treatment is given as needed. Ablation therapy removes or destroys tissue. Different types of ablation therapy are used for liver cancer: Embolization therapy is used for patients who cannot have surgery to remove the tumor or ablation therapy and whose tumor has not spread outside the liver. Embolization therapy is the use of substances to block or decrease the flow of blood through the hepatic artery to the tumor. When the tumor does not get the oxygen and nutrients it needs, it will not continue to grow. The liver receives blood from the hepatic portal vein and the hepatic artery. Blood that comes into the liver from the hepatic portal vein usually goes to the healthy liver tissue. Blood that comes from the hepatic artery usually goes to the tumor. When the hepatic artery is blocked during embolization therapy, the healthy liver tissue continues to receive blood from the hepatic portal vein. There are two main types of embolization therapy: Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Targeted therapies used to treat advanced liver cancer include the following: To learn more about targeted therapy and its side effects, see Targeted Cancer Therapies. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. Immune checkpoint inhibitors are a type of immunotherapy. Immune checkpoint inhibitors that may be used to treat liver cancer include the following: To learn more about immunotherapy and its side effects, see Immunotherapy to Treat Cancer and Immunotherapy Side Effects. External radiation therapy uses a machine outside the body to send high-energy x-rays or other types of radiation toward the area of the body with cancer. This kills cancer cells or keeps them from growing. Certain ways of giving external radiation therapy can help keep radiation from damaging nearby healthy tissue: To learn more about radiation therapy and its side effects, see Radiation Therapy to Treat Cancer and Radiation Therapy Side Effects. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. For some patients, taking part in a clinical trial may be an option. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. To learn more about clinical trials, see Clinical Trials Information for Patients and Caregivers. Treatment of localized liver cancer may include the following: Treatment of locally advanced or metastatic liver cancer may include the following: Treatment options for recurrent primary liver cancer may include the following: If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Liver Cancer Treatment was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

what research (or clinical trials) is being done for Adult Primary Liver Cancer ?

New types of treatment are being tested in clinical trials. Information about clinical trials is available from the NCI website. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials.

There are different types of treatment for patients with liver cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. Surveillance is used for lesions smaller than 1 centimeter found during screening. Follow-up every 3 months is common. Surveillance is closely watching a patient’s condition but not giving any treatment unless there are changes in test results that show the condition is getting worse. During active surveillance, certain exams and tests are done on a regular schedule. A partial hepatectomy (surgery to remove the part of the liver where cancer is found) may be done. A wedge of tissue, an entire lobe, or a larger part of the liver, along with some of the healthy tissue around it is removed. The remaining liver tissue takes over the functions of the liver and may regrow. In a liver transplant, the entire liver is removed and replaced with a healthy donated liver. A liver transplant may be done when the disease is in the liver only and a donated liver can be found. If the patient has to wait for a donated liver, other treatment is given as needed. Ablation therapy removes or destroys tissue. Different types of ablation therapy are used for liver cancer: Embolization therapy is used for patients who cannot have surgery to remove the tumor or ablation therapy and whose tumor has not spread outside the liver. Embolization therapy is the use of substances to block or decrease the flow of blood through the hepatic artery to the tumor. When the tumor does not get the oxygen and nutrients it needs, it will not continue to grow. The liver receives blood from the hepatic portal vein and the hepatic artery. Blood that comes into the liver from the hepatic portal vein usually goes to the healthy liver tissue. Blood that comes from the hepatic artery usually goes to the tumor. When the hepatic artery is blocked during embolization therapy, the healthy liver tissue continues to receive blood from the hepatic portal vein. There are two main types of embolization therapy: Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Targeted therapies used to treat advanced liver cancer include the following: To learn more about targeted therapy and its side effects, see Targeted Cancer Therapies. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. Immune checkpoint inhibitors are a type of immunotherapy. Immune checkpoint inhibitors that may be used to treat liver cancer include the following: To learn more about immunotherapy and its side effects, see Immunotherapy to Treat Cancer and Immunotherapy Side Effects. External radiation therapy uses a machine outside the body to send high-energy x-rays or other types of radiation toward the area of the body with cancer. This kills cancer cells or keeps them from growing. Certain ways of giving external radiation therapy can help keep radiation from damaging nearby healthy tissue: To learn more about radiation therapy and its side effects, see Radiation Therapy to Treat Cancer and Radiation Therapy Side Effects. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. For some patients, taking part in a clinical trial may be an option. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. To learn more about clinical trials, see Clinical Trials Information for Patients and Caregivers. Treatment of localized liver cancer may include the following: Treatment of locally advanced or metastatic liver cancer may include the following: Treatment options for recurrent primary liver cancer may include the following: If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Liver Cancer Treatment was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the treatments for Adult Primary Liver Cancer ?

Key Points - There are different types of treatment for patients with adult primary liver cancer. - Patients with liver cancer are treated by a team of specialists who are experts in treating liver cancer. - Seven types of standard treatment are used: - Surveillance - Surgery - Liver transplant - Ablation therapy - Embolization therapy - Targeted therapy - Radiation therapy - New types of treatment are being tested in clinical trials. - Patients may want to think about taking part in a clinical trial. - Patients can enter clinical trials before, during, or after starting their cancer treatment. - Follow-up tests may be needed. There are different types of treatment for patients with adult primary liver cancer. Different types of treatments are available for patients with adult primary liver cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Patients with liver cancer are treated by a team of specialists who are experts in treating liver cancer. The patient&apos;s treatment will be overseen by a medical oncologist, a doctor who specializes in treating people with cancer. The medical oncologist may refer the patient to other health professionals who have special training in treating patients with liver cancer. These may include the following specialists: - Hepatologist (specialist in liver disease). - Surgical oncologist. - Transplant surgeon. - Radiation oncologist. - Interventional radiologist (a specialist who diagnoses and treats diseases using imaging and the smallest incisions possible). - Pathologist. Seven types of standard treatment are used: Surveillance Surveillance for lesions smaller than 1 centimeter found during screening. Follow-up every three months is common. Surgery A partial hepatectomy (surgery to remove the part of the liver where cancer is found) may be done. A wedge of tissue, an entire lobe, or a larger part of the liver, along with some of the healthy tissue around it is removed. The remaining liver tissue takes over the functions of the liver and may regrow. Liver transplant In a liver transplant, the entire liver is removed and replaced with a healthy donated liver. A liver transplant may be done when the disease is in the liver only and a donated liver can be found. If the patient has to wait for a donated liver, other treatment is given as needed. Ablation therapy Ablation therapy removes or destroys tissue. Different types of ablation therapy are used for liver cancer: - Radiofrequency ablation: The use of special needles that are inserted directly through the skin or through an incision in the abdomen to reach the tumor. High-energy radio waves heat the needles and tumor which kills cancer cells. - Microwave therapy: A type of treatment in which the tumor is exposed to high temperatures created by microwaves. This can damage and kill cancer cells or make them more sensitive to the effects of radiation and certain anticancer drugs. - Percutaneous ethanol injection: A cancer treatment in which a small needle is used to inject ethanol (pure alcohol) directly into a tumor to kill cancer cells. Several treatments may be needed. Usually local anesthesia is used, but if the patient has many tumors in the liver, general anesthesia may be used. - Cryoablation: A treatment that uses an instrument to freeze and destroy cancer cells. This type of treatment is also called cryotherapy and cryosurgery. The doctor may use ultrasound to guide the instrument. - Electroporation therapy: A treatment that sends electrical pulses through an electrode placed in a tumor to kill cancer cells. Electroporation therapy is being studied in clinical trials. Embolization therapy Embolization therapy is the use of substances to block or decrease the flow of blood through the hepatic artery to the tumor. When the tumor does not get the oxygen and nutrients it needs, it will not continue to grow. Embolization therapy is used for patients who cannot have surgery to remove the tumor or ablation therapy and whose tumor has not spread outside the liver. The liver receives blood from the hepatic portal vein and the hepatic artery. Blood that comes into the liver from the hepatic portal vein usually goes to the healthy liver tissue. Blood that comes from the hepatic artery usually goes to the tumor. When the hepatic artery is blocked during embolization therapy, the healthy liver tissue continues to receive blood from the hepatic portal vein. There are two main types of embolization therapy: - Transarterial embolization (TAE): A small incision (cut) is made in the inner thigh and a catheter (thin, flexible tube) is inserted and threaded up into the hepatic artery. Once the catheter is in place, a substance that blocks the hepatic artery and stops blood flow to the tumor is injected. - Transarterial chemoembolization (TACE): This procedure is like TAE except an anticancer drug is also given. The procedure can be done by attaching the anticancer drug to small beads that are injected into the hepatic artery or by injecting the anticancer drug through the catheter into the hepatic artery and then injecting the substance to block the hepatic artery. Most of the anticancer drug is trapped near the tumor and only a small amount of the drug reaches other parts of the body. This type of treatment is also called chemoembolization. Targeted therapy Targeted therapy is a treatment that uses drugs or other substances to identify and attack specific cancer cells without harming normal cells. Adult liver cancer may be treated with a targeted therapy drug that stops cells from dividing and prevents the growth of new blood vessels that tumors need to grow. See Drugs Approved for Liver Cancer for more information. Radiation therapy Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: - External radiation therapy uses a machine outside the body to send radiation toward the cancer. Certain ways of giving radiation therapy can help keep radiation from damaging nearby healthy tissue. These types of external radiation therapy include the following: - Conformal radiation therapy: Conformal radiation therapy is a type of external radiation therapy that uses a computer to make a 3-dimensional (3-D) picture of the tumor and shapes the radiation beams to fit the tumor. This allows a high dose of radiation to reach the tumor and causes less damage to nearby healthy tissue. - Stereotactic body radiation therapy: Stereotactic body radiation therapy is a type of external radiation therapy. Special equipment is used to place the patient in the same position for each radiation treatment. Once a day for several days, a radiation machine aims a larger than usual dose of radiation directly at the tumor. By having the patient in the same position for each treatment, there is less damage to nearby healthy tissue. This procedure is also called stereotactic external-beam radiation therapy and stereotaxic radiation therapy. - Proton beam radiation therapy: Proton-beam therapy is a type of high-energy, external radiation therapy. A radiation therapy machine aims streams of protons (tiny, invisible, positively-charged particles) at the cancer cells to kill them. This type of treatment causes less damage to nearby healthy tissue. - Internal radiation therapy uses a radioactive substance sealed in needles, seeds, wires, or catheters that are placed directly into or near the cancer. The way the radiation therapy is given depends on the type and stage of the cancer being treated. External radiation therapy is used to treat adult primary liver cancer. New types of treatment are being tested in clinical trials. Information about clinical trials is available from the NCI website. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials. Follow-up tests may be needed. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. Treatment Options for Adult Primary Liver Cancer Stages 0, A, and B Adult Primary Liver Cancer Treatment of stages 0, A, and B adult primary liver cancer may include the following: - Surveillance for lesions smaller than 1 centimeter. - Partial hepatectomy. - Total hepatectomy and liver transplant. - Ablation of the tumor using one of the following methods: - Radiofrequency ablation. - Microwave therapy. - Percutaneous ethanol injection. - Cryoablation. - A clinical trial of electroporation therapy. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage 0 adult primary liver cancer (BCLC), stage A adult primary liver cancer (BCLC) and stage B adult primary liver cancer (BCLC). For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Stages C and D Adult Primary Liver Cancer Treatment of stages C and D adult primary liver cancer may include the following: - Embolization therapy using one of the following methods: - Transarterial embolization (TAE). - Transarterial chemoembolization (TACE). - Targeted therapy. - Radiation therapy. - A clinical trial of targeted therapy after chemoembolization or combined with chemotherapy. - A clinical trial of new targeted therapy drugs. - A clinical trial of targeted therapy with or without stereotactic body radiation therapy. - A clinical trial of stereotactic body radiation therapy or proton-beam radiation therapy. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage C adult primary liver cancer (BCLC) and stage D adult primary liver cancer (BCLC). For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website.

The uterus is part of the female reproductive system. The uterus is the hollow, pear-shaped organ in the pelvis, where a fetus grows. The cervix is at the lower, narrow end of the uterus, and leads to the vagina. EnlargeAnatomy of the female reproductive system. The organs in the female reproductive system include the uterus, ovaries, fallopian tubes, cervix, and vagina. The uterus has a muscular outer layer called the myometrium and an inner lining called the endometrium. Uterine sarcoma is a very rare kind of cancer that forms in the uterine muscles or in tissues that support the uterus. (Information about other types of sarcomas can be found in the PDQ summary on Soft Tissue Sarcoma Treatment.) Uterine sarcoma is different from endometrial cancer, a disease in which cancer forms in the tissue that lines the uterus. Carcinosarcoma is a subtype of endometrial cancer and is staged using endometrial cancer definitions. (For more information, seeEndometrial Cancer Treatment.) Uterine sarcomas include leiomyosarcomas, endometrial stromal sarcomas, and adenosarcomas. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for uterine sarcoma include the following: Abnormal bleeding from the vagina and other signs and symptoms may be caused by uterine sarcoma or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: The prognosis and treatment options depend on the following: The process used to find out if cancer has spread within the uterus or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following procedures may be used in the staging process: Surgery is used to diagnose, stage, and treat uterine sarcoma. During this surgery, the doctor removes as much of the cancer as possible. The following procedures may be used to diagnose, stage, and treat uterine sarcoma: Treatment in addition to surgery may be given, as described in the Treatment Option Overview section of this summary. When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if uterine sarcoma spreads to the lung, the cancer cells in the lung are actually uterine sarcoma cells. The disease is metastatic uterine sarcoma, not lung cancer. Cancer can spread through tissue, the lymph system, and the blood: In stage I, the tumor is found in the uterus only. Stage I is divided into stages IA and IB: In stage II, the tumor has spread beyond the uterus but has not spread beyond the pelvis. Stage II is divided into stages IIA and IIB: In stage III, the tumor has spread into tissues in the abdomen. Stage III is divided into stages IIIA, IIIB, and IIIC: Stage IV is divided into stages IVA and IVB: In stage I, the tumor is found in the uterus only. Stage I is divided into stages IA, IB, and IC: In stage II, the tumor has spread outside the uterus into the pelvis. Stage II is divided into stages IIA and IIB: In stage III, the tumor has spread into tissues in the abdomen. Stage III is divided into stages IIIA, IIIB, and IIIC: Stage IV is divided into stages IVA and IVB: The cancer may come back in the uterus, the pelvis, or in other parts of the body. Different types of treatments are available for patients with uterine sarcoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment for uterine sarcoma, as described in the Stages of Uterine Sarcoma section of this summary. After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type and stage of the cancer being treated. External and internal radiation therapy are used to treat uterine sarcoma, and may also be used as palliative therapy to relieve symptoms and improve quality of life. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Hormone therapy is a cancer treatment that removes hormones or blocks their action and stops cancer cells from growing. Hormones are substances produced by glands in the body and circulated in the bloodstream. Some hormones can cause certain cancers to grow. If tests show the cancer cells have places where hormones can attach (receptors), drugs, surgery, or radiation therapy is used to reduce the production of hormones or block them from working. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage I leiomyosarcoma of the uterus, stage I endometrial stromal sarcoma, and stage I adenosarcoma of the uterus may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage II leiomyosarcoma of the uterus, stage II endometrial stromal sarcoma, and stage II adenosarcoma of the uterus may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage III leiomyosarcoma of the uterus, stage III endometrial stromal sarcoma, and stage III adenosarcoma of the uterus may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. There is no standard treatment for patients with stage IV leiomyosarcoma of the uterus, stage IV endometrial stromal sarcoma, or stage IV adenosarcoma of the uterus. Treatment may include a clinical trial using chemotherapy. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. There is no standard treatment for recurrent uterine sarcoma. Treatment may include a clinical trial using chemotherapy. For patients with recurrent carcinosarcoma (a certain type of tumor), treatment may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about uterine sarcoma, see the Uterine Cancer Home Page. For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of uterine sarcoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Uterine Sarcoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/uterine/patient/uterine-sarcoma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389379] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Uterine Sarcoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is (are) Uterine Sarcoma ?

Key Points - Uterine sarcoma is a disease in which malignant (cancer) cells form in the muscles of the uterus or other tissues that support the uterus. - Being exposed to x-rays can increase the risk of uterine sarcoma. - Signs of uterine sarcoma include abnormal bleeding. - Tests that examine the uterus are used to detect (find) and diagnose uterine sarcoma. - Certain factors affect prognosis (chance of recovery) and treatment options. Uterine sarcoma is a disease in which malignant (cancer) cells form in the muscles of the uterus or other tissues that support the uterus. The uterus is part of the female reproductive system. The uterus is the hollow, pear-shaped organ in the pelvis, where a fetus grows. The cervix is at the lower, narrow end of the uterus, and leads to the vagina. Uterine sarcoma is a very rare kind of cancer that forms in the uterine muscles or in tissues that support the uterus. (Information about other types of sarcomas can be found in the PDQ summary on Adult Soft Tissue Sarcoma Treatment.) Uterine sarcoma is different from cancer of the endometrium, a disease in which cancer cells start growing inside the lining of the uterus. (See the PDQ summary on Endometrial Cancer Treatment for information).

The uterus is part of the female reproductive system. The uterus is the hollow, pear-shaped organ in the pelvis, where a fetus grows. The cervix is at the lower, narrow end of the uterus, and leads to the vagina. EnlargeAnatomy of the female reproductive system. The organs in the female reproductive system include the uterus, ovaries, fallopian tubes, cervix, and vagina. The uterus has a muscular outer layer called the myometrium and an inner lining called the endometrium. Uterine sarcoma is a very rare kind of cancer that forms in the uterine muscles or in tissues that support the uterus. (Information about other types of sarcomas can be found in the PDQ summary on Soft Tissue Sarcoma Treatment.) Uterine sarcoma is different from endometrial cancer, a disease in which cancer forms in the tissue that lines the uterus. Carcinosarcoma is a subtype of endometrial cancer and is staged using endometrial cancer definitions. (For more information, seeEndometrial Cancer Treatment.) Uterine sarcomas include leiomyosarcomas, endometrial stromal sarcomas, and adenosarcomas. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for uterine sarcoma include the following: Abnormal bleeding from the vagina and other signs and symptoms may be caused by uterine sarcoma or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: The prognosis and treatment options depend on the following: The process used to find out if cancer has spread within the uterus or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following procedures may be used in the staging process: Surgery is used to diagnose, stage, and treat uterine sarcoma. During this surgery, the doctor removes as much of the cancer as possible. The following procedures may be used to diagnose, stage, and treat uterine sarcoma: Treatment in addition to surgery may be given, as described in the Treatment Option Overview section of this summary. When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if uterine sarcoma spreads to the lung, the cancer cells in the lung are actually uterine sarcoma cells. The disease is metastatic uterine sarcoma, not lung cancer. Cancer can spread through tissue, the lymph system, and the blood: In stage I, the tumor is found in the uterus only. Stage I is divided into stages IA and IB: In stage II, the tumor has spread beyond the uterus but has not spread beyond the pelvis. Stage II is divided into stages IIA and IIB: In stage III, the tumor has spread into tissues in the abdomen. Stage III is divided into stages IIIA, IIIB, and IIIC: Stage IV is divided into stages IVA and IVB: In stage I, the tumor is found in the uterus only. Stage I is divided into stages IA, IB, and IC: In stage II, the tumor has spread outside the uterus into the pelvis. Stage II is divided into stages IIA and IIB: In stage III, the tumor has spread into tissues in the abdomen. Stage III is divided into stages IIIA, IIIB, and IIIC: Stage IV is divided into stages IVA and IVB: The cancer may come back in the uterus, the pelvis, or in other parts of the body. Different types of treatments are available for patients with uterine sarcoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment for uterine sarcoma, as described in the Stages of Uterine Sarcoma section of this summary. After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type and stage of the cancer being treated. External and internal radiation therapy are used to treat uterine sarcoma, and may also be used as palliative therapy to relieve symptoms and improve quality of life. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Hormone therapy is a cancer treatment that removes hormones or blocks their action and stops cancer cells from growing. Hormones are substances produced by glands in the body and circulated in the bloodstream. Some hormones can cause certain cancers to grow. If tests show the cancer cells have places where hormones can attach (receptors), drugs, surgery, or radiation therapy is used to reduce the production of hormones or block them from working. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage I leiomyosarcoma of the uterus, stage I endometrial stromal sarcoma, and stage I adenosarcoma of the uterus may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage II leiomyosarcoma of the uterus, stage II endometrial stromal sarcoma, and stage II adenosarcoma of the uterus may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage III leiomyosarcoma of the uterus, stage III endometrial stromal sarcoma, and stage III adenosarcoma of the uterus may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. There is no standard treatment for patients with stage IV leiomyosarcoma of the uterus, stage IV endometrial stromal sarcoma, or stage IV adenosarcoma of the uterus. Treatment may include a clinical trial using chemotherapy. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. There is no standard treatment for recurrent uterine sarcoma. Treatment may include a clinical trial using chemotherapy. For patients with recurrent carcinosarcoma (a certain type of tumor), treatment may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about uterine sarcoma, see the Uterine Cancer Home Page. For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of uterine sarcoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Uterine Sarcoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/uterine/patient/uterine-sarcoma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389379] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Uterine Sarcoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

Who is at risk for Uterine Sarcoma? ?

Being exposed to x-rays can increase the risk of uterine sarcoma. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesnt mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for uterine sarcoma include the following: - Past treatment with radiation therapy to the pelvis. - Treatment with tamoxifen for breast cancer. If you are taking this drug, have a pelvic exam every year and report any vaginal bleeding (other than menstrual bleeding) as soon as possible.

The uterus is part of the female reproductive system. The uterus is the hollow, pear-shaped organ in the pelvis, where a fetus grows. The cervix is at the lower, narrow end of the uterus, and leads to the vagina. EnlargeAnatomy of the female reproductive system. The organs in the female reproductive system include the uterus, ovaries, fallopian tubes, cervix, and vagina. The uterus has a muscular outer layer called the myometrium and an inner lining called the endometrium. Uterine sarcoma is a very rare kind of cancer that forms in the uterine muscles or in tissues that support the uterus. (Information about other types of sarcomas can be found in the PDQ summary on Soft Tissue Sarcoma Treatment.) Uterine sarcoma is different from endometrial cancer, a disease in which cancer forms in the tissue that lines the uterus. Carcinosarcoma is a subtype of endometrial cancer and is staged using endometrial cancer definitions. (For more information, seeEndometrial Cancer Treatment.) Uterine sarcomas include leiomyosarcomas, endometrial stromal sarcomas, and adenosarcomas. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for uterine sarcoma include the following: Abnormal bleeding from the vagina and other signs and symptoms may be caused by uterine sarcoma or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: The prognosis and treatment options depend on the following: The process used to find out if cancer has spread within the uterus or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following procedures may be used in the staging process: Surgery is used to diagnose, stage, and treat uterine sarcoma. During this surgery, the doctor removes as much of the cancer as possible. The following procedures may be used to diagnose, stage, and treat uterine sarcoma: Treatment in addition to surgery may be given, as described in the Treatment Option Overview section of this summary. When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if uterine sarcoma spreads to the lung, the cancer cells in the lung are actually uterine sarcoma cells. The disease is metastatic uterine sarcoma, not lung cancer. Cancer can spread through tissue, the lymph system, and the blood: In stage I, the tumor is found in the uterus only. Stage I is divided into stages IA and IB: In stage II, the tumor has spread beyond the uterus but has not spread beyond the pelvis. Stage II is divided into stages IIA and IIB: In stage III, the tumor has spread into tissues in the abdomen. Stage III is divided into stages IIIA, IIIB, and IIIC: Stage IV is divided into stages IVA and IVB: In stage I, the tumor is found in the uterus only. Stage I is divided into stages IA, IB, and IC: In stage II, the tumor has spread outside the uterus into the pelvis. Stage II is divided into stages IIA and IIB: In stage III, the tumor has spread into tissues in the abdomen. Stage III is divided into stages IIIA, IIIB, and IIIC: Stage IV is divided into stages IVA and IVB: The cancer may come back in the uterus, the pelvis, or in other parts of the body. Different types of treatments are available for patients with uterine sarcoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment for uterine sarcoma, as described in the Stages of Uterine Sarcoma section of this summary. After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type and stage of the cancer being treated. External and internal radiation therapy are used to treat uterine sarcoma, and may also be used as palliative therapy to relieve symptoms and improve quality of life. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Hormone therapy is a cancer treatment that removes hormones or blocks their action and stops cancer cells from growing. Hormones are substances produced by glands in the body and circulated in the bloodstream. Some hormones can cause certain cancers to grow. If tests show the cancer cells have places where hormones can attach (receptors), drugs, surgery, or radiation therapy is used to reduce the production of hormones or block them from working. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage I leiomyosarcoma of the uterus, stage I endometrial stromal sarcoma, and stage I adenosarcoma of the uterus may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage II leiomyosarcoma of the uterus, stage II endometrial stromal sarcoma, and stage II adenosarcoma of the uterus may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage III leiomyosarcoma of the uterus, stage III endometrial stromal sarcoma, and stage III adenosarcoma of the uterus may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. There is no standard treatment for patients with stage IV leiomyosarcoma of the uterus, stage IV endometrial stromal sarcoma, or stage IV adenosarcoma of the uterus. Treatment may include a clinical trial using chemotherapy. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. There is no standard treatment for recurrent uterine sarcoma. Treatment may include a clinical trial using chemotherapy. For patients with recurrent carcinosarcoma (a certain type of tumor), treatment may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about uterine sarcoma, see the Uterine Cancer Home Page. For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of uterine sarcoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Uterine Sarcoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/uterine/patient/uterine-sarcoma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389379] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Uterine Sarcoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the symptoms of Uterine Sarcoma ?

Signs of uterine sarcoma include abnormal bleeding. Abnormal bleeding from the vagina and other signs and symptoms may be caused by uterine sarcoma or by other conditions. Check with your doctor if you have any of the following: - Bleeding that is not part of menstrual periods. - Bleeding after menopause. - A mass in the vagina. - Pain or a feeling of fullness in the abdomen. - Frequent urination.

The uterus is part of the female reproductive system. The uterus is the hollow, pear-shaped organ in the pelvis, where a fetus grows. The cervix is at the lower, narrow end of the uterus, and leads to the vagina. EnlargeAnatomy of the female reproductive system. The organs in the female reproductive system include the uterus, ovaries, fallopian tubes, cervix, and vagina. The uterus has a muscular outer layer called the myometrium and an inner lining called the endometrium. Uterine sarcoma is a very rare kind of cancer that forms in the uterine muscles or in tissues that support the uterus. (Information about other types of sarcomas can be found in the PDQ summary on Soft Tissue Sarcoma Treatment.) Uterine sarcoma is different from endometrial cancer, a disease in which cancer forms in the tissue that lines the uterus. Carcinosarcoma is a subtype of endometrial cancer and is staged using endometrial cancer definitions. (For more information, seeEndometrial Cancer Treatment.) Uterine sarcomas include leiomyosarcomas, endometrial stromal sarcomas, and adenosarcomas. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for uterine sarcoma include the following: Abnormal bleeding from the vagina and other signs and symptoms may be caused by uterine sarcoma or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: The prognosis and treatment options depend on the following: The process used to find out if cancer has spread within the uterus or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following procedures may be used in the staging process: Surgery is used to diagnose, stage, and treat uterine sarcoma. During this surgery, the doctor removes as much of the cancer as possible. The following procedures may be used to diagnose, stage, and treat uterine sarcoma: Treatment in addition to surgery may be given, as described in the Treatment Option Overview section of this summary. When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if uterine sarcoma spreads to the lung, the cancer cells in the lung are actually uterine sarcoma cells. The disease is metastatic uterine sarcoma, not lung cancer. Cancer can spread through tissue, the lymph system, and the blood: In stage I, the tumor is found in the uterus only. Stage I is divided into stages IA and IB: In stage II, the tumor has spread beyond the uterus but has not spread beyond the pelvis. Stage II is divided into stages IIA and IIB: In stage III, the tumor has spread into tissues in the abdomen. Stage III is divided into stages IIIA, IIIB, and IIIC: Stage IV is divided into stages IVA and IVB: In stage I, the tumor is found in the uterus only. Stage I is divided into stages IA, IB, and IC: In stage II, the tumor has spread outside the uterus into the pelvis. Stage II is divided into stages IIA and IIB: In stage III, the tumor has spread into tissues in the abdomen. Stage III is divided into stages IIIA, IIIB, and IIIC: Stage IV is divided into stages IVA and IVB: The cancer may come back in the uterus, the pelvis, or in other parts of the body. Different types of treatments are available for patients with uterine sarcoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment for uterine sarcoma, as described in the Stages of Uterine Sarcoma section of this summary. After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type and stage of the cancer being treated. External and internal radiation therapy are used to treat uterine sarcoma, and may also be used as palliative therapy to relieve symptoms and improve quality of life. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Hormone therapy is a cancer treatment that removes hormones or blocks their action and stops cancer cells from growing. Hormones are substances produced by glands in the body and circulated in the bloodstream. Some hormones can cause certain cancers to grow. If tests show the cancer cells have places where hormones can attach (receptors), drugs, surgery, or radiation therapy is used to reduce the production of hormones or block them from working. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage I leiomyosarcoma of the uterus, stage I endometrial stromal sarcoma, and stage I adenosarcoma of the uterus may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage II leiomyosarcoma of the uterus, stage II endometrial stromal sarcoma, and stage II adenosarcoma of the uterus may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage III leiomyosarcoma of the uterus, stage III endometrial stromal sarcoma, and stage III adenosarcoma of the uterus may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. There is no standard treatment for patients with stage IV leiomyosarcoma of the uterus, stage IV endometrial stromal sarcoma, or stage IV adenosarcoma of the uterus. Treatment may include a clinical trial using chemotherapy. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. There is no standard treatment for recurrent uterine sarcoma. Treatment may include a clinical trial using chemotherapy. For patients with recurrent carcinosarcoma (a certain type of tumor), treatment may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about uterine sarcoma, see the Uterine Cancer Home Page. For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of uterine sarcoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Uterine Sarcoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/uterine/patient/uterine-sarcoma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389379] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Uterine Sarcoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

How to diagnose Uterine Sarcoma ?

Tests that examine the uterus are used to detect (find) and diagnose uterine sarcoma. The following tests and procedures may be used: - Physical exam and history : An exam of the body to check general signs of health, including checking for signs of disease, such as lumps or anything else that seems unusual. A history of the patients health habits and past illnesses and treatments will also be taken. - Pelvic exam: An exam of the vagina, cervix, uterus, fallopian tubes, ovaries, and rectum. A speculum is inserted into the vagina and the doctor or nurse looks at the vagina and cervix for signs of disease. A Pap test of the cervix is usually done. The doctor or nurse also inserts one or two lubricated, gloved fingers of one hand into the vagina and places the other hand over the lower abdomen to feel the size, shape, and position of the uterus and ovaries. The doctor or nurse also inserts a lubricated, gloved finger into the rectum to feel for lumps or abnormal areas. - Pap test: A procedure to collect cells from the surface of the cervix and vagina. A piece of cotton, a brush, or a small wooden stick is used to gently scrape cells from the cervix and vagina. The cells are viewed under a microscope to find out if they are abnormal. This procedure is also called a Pap smear. Because uterine sarcoma begins inside the uterus, this cancer may not show up on the Pap test. - Transvaginal ultrasound exam: A procedure used to examine the vagina, uterus, fallopian tubes, and bladder. An ultrasound transducer (probe) is inserted into the vagina and used to bounce high-energy sound waves (ultrasound) off internal tissues or organs and make echoes. The echoes form a picture of body tissues called a sonogram. The doctor can identify tumors by looking at the sonogram. - Dilatation and curettage : A procedure to remove samples of tissue from the inner lining of the uterus. The cervix is dilated and a curette (spoon-shaped instrument) is inserted into the uterus to remove tissue. The tissue samples are checked under a microscope for signs of disease. This procedure is also called a D&amp;C. - Endometrial biopsy : The removal of tissue from the endometrium (inner lining of the uterus) by inserting a thin, flexible tube through the cervix and into the uterus. The tube is used to gently scrape a small amount of tissue from the endometrium and then remove the tissue samples. A pathologist views the tissue under a microscope to look for cancer cells.

The uterus is part of the female reproductive system. The uterus is the hollow, pear-shaped organ in the pelvis, where a fetus grows. The cervix is at the lower, narrow end of the uterus, and leads to the vagina. EnlargeAnatomy of the female reproductive system. The organs in the female reproductive system include the uterus, ovaries, fallopian tubes, cervix, and vagina. The uterus has a muscular outer layer called the myometrium and an inner lining called the endometrium. Uterine sarcoma is a very rare kind of cancer that forms in the uterine muscles or in tissues that support the uterus. (Information about other types of sarcomas can be found in the PDQ summary on Soft Tissue Sarcoma Treatment.) Uterine sarcoma is different from endometrial cancer, a disease in which cancer forms in the tissue that lines the uterus. Carcinosarcoma is a subtype of endometrial cancer and is staged using endometrial cancer definitions. (For more information, seeEndometrial Cancer Treatment.) Uterine sarcomas include leiomyosarcomas, endometrial stromal sarcomas, and adenosarcomas. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for uterine sarcoma include the following: Abnormal bleeding from the vagina and other signs and symptoms may be caused by uterine sarcoma or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: The prognosis and treatment options depend on the following: The process used to find out if cancer has spread within the uterus or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following procedures may be used in the staging process: Surgery is used to diagnose, stage, and treat uterine sarcoma. During this surgery, the doctor removes as much of the cancer as possible. The following procedures may be used to diagnose, stage, and treat uterine sarcoma: Treatment in addition to surgery may be given, as described in the Treatment Option Overview section of this summary. When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if uterine sarcoma spreads to the lung, the cancer cells in the lung are actually uterine sarcoma cells. The disease is metastatic uterine sarcoma, not lung cancer. Cancer can spread through tissue, the lymph system, and the blood: In stage I, the tumor is found in the uterus only. Stage I is divided into stages IA and IB: In stage II, the tumor has spread beyond the uterus but has not spread beyond the pelvis. Stage II is divided into stages IIA and IIB: In stage III, the tumor has spread into tissues in the abdomen. Stage III is divided into stages IIIA, IIIB, and IIIC: Stage IV is divided into stages IVA and IVB: In stage I, the tumor is found in the uterus only. Stage I is divided into stages IA, IB, and IC: In stage II, the tumor has spread outside the uterus into the pelvis. Stage II is divided into stages IIA and IIB: In stage III, the tumor has spread into tissues in the abdomen. Stage III is divided into stages IIIA, IIIB, and IIIC: Stage IV is divided into stages IVA and IVB: The cancer may come back in the uterus, the pelvis, or in other parts of the body. Different types of treatments are available for patients with uterine sarcoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment for uterine sarcoma, as described in the Stages of Uterine Sarcoma section of this summary. After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type and stage of the cancer being treated. External and internal radiation therapy are used to treat uterine sarcoma, and may also be used as palliative therapy to relieve symptoms and improve quality of life. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Hormone therapy is a cancer treatment that removes hormones or blocks their action and stops cancer cells from growing. Hormones are substances produced by glands in the body and circulated in the bloodstream. Some hormones can cause certain cancers to grow. If tests show the cancer cells have places where hormones can attach (receptors), drugs, surgery, or radiation therapy is used to reduce the production of hormones or block them from working. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage I leiomyosarcoma of the uterus, stage I endometrial stromal sarcoma, and stage I adenosarcoma of the uterus may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage II leiomyosarcoma of the uterus, stage II endometrial stromal sarcoma, and stage II adenosarcoma of the uterus may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage III leiomyosarcoma of the uterus, stage III endometrial stromal sarcoma, and stage III adenosarcoma of the uterus may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. There is no standard treatment for patients with stage IV leiomyosarcoma of the uterus, stage IV endometrial stromal sarcoma, or stage IV adenosarcoma of the uterus. Treatment may include a clinical trial using chemotherapy. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. There is no standard treatment for recurrent uterine sarcoma. Treatment may include a clinical trial using chemotherapy. For patients with recurrent carcinosarcoma (a certain type of tumor), treatment may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about uterine sarcoma, see the Uterine Cancer Home Page. For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of uterine sarcoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Uterine Sarcoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/uterine/patient/uterine-sarcoma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389379] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Uterine Sarcoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is the outlook for Uterine Sarcoma ?

Certain factors affect prognosis (chance of recovery) and treatment options. The prognosis (chance of recovery) and treatment options depend on the following: - The stage of the cancer. - The type and size of the tumor. - The patient&apos;s general health. - Whether the cancer has just been diagnosed or has recurred (come back).

The uterus is part of the female reproductive system. The uterus is the hollow, pear-shaped organ in the pelvis, where a fetus grows. The cervix is at the lower, narrow end of the uterus, and leads to the vagina. EnlargeAnatomy of the female reproductive system. The organs in the female reproductive system include the uterus, ovaries, fallopian tubes, cervix, and vagina. The uterus has a muscular outer layer called the myometrium and an inner lining called the endometrium. Uterine sarcoma is a very rare kind of cancer that forms in the uterine muscles or in tissues that support the uterus. (Information about other types of sarcomas can be found in the PDQ summary on Soft Tissue Sarcoma Treatment.) Uterine sarcoma is different from endometrial cancer, a disease in which cancer forms in the tissue that lines the uterus. Carcinosarcoma is a subtype of endometrial cancer and is staged using endometrial cancer definitions. (For more information, seeEndometrial Cancer Treatment.) Uterine sarcomas include leiomyosarcomas, endometrial stromal sarcomas, and adenosarcomas. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for uterine sarcoma include the following: Abnormal bleeding from the vagina and other signs and symptoms may be caused by uterine sarcoma or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: The prognosis and treatment options depend on the following: The process used to find out if cancer has spread within the uterus or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following procedures may be used in the staging process: Surgery is used to diagnose, stage, and treat uterine sarcoma. During this surgery, the doctor removes as much of the cancer as possible. The following procedures may be used to diagnose, stage, and treat uterine sarcoma: Treatment in addition to surgery may be given, as described in the Treatment Option Overview section of this summary. When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if uterine sarcoma spreads to the lung, the cancer cells in the lung are actually uterine sarcoma cells. The disease is metastatic uterine sarcoma, not lung cancer. Cancer can spread through tissue, the lymph system, and the blood: In stage I, the tumor is found in the uterus only. Stage I is divided into stages IA and IB: In stage II, the tumor has spread beyond the uterus but has not spread beyond the pelvis. Stage II is divided into stages IIA and IIB: In stage III, the tumor has spread into tissues in the abdomen. Stage III is divided into stages IIIA, IIIB, and IIIC: Stage IV is divided into stages IVA and IVB: In stage I, the tumor is found in the uterus only. Stage I is divided into stages IA, IB, and IC: In stage II, the tumor has spread outside the uterus into the pelvis. Stage II is divided into stages IIA and IIB: In stage III, the tumor has spread into tissues in the abdomen. Stage III is divided into stages IIIA, IIIB, and IIIC: Stage IV is divided into stages IVA and IVB: The cancer may come back in the uterus, the pelvis, or in other parts of the body. Different types of treatments are available for patients with uterine sarcoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment for uterine sarcoma, as described in the Stages of Uterine Sarcoma section of this summary. After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type and stage of the cancer being treated. External and internal radiation therapy are used to treat uterine sarcoma, and may also be used as palliative therapy to relieve symptoms and improve quality of life. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Hormone therapy is a cancer treatment that removes hormones or blocks their action and stops cancer cells from growing. Hormones are substances produced by glands in the body and circulated in the bloodstream. Some hormones can cause certain cancers to grow. If tests show the cancer cells have places where hormones can attach (receptors), drugs, surgery, or radiation therapy is used to reduce the production of hormones or block them from working. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage I leiomyosarcoma of the uterus, stage I endometrial stromal sarcoma, and stage I adenosarcoma of the uterus may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage II leiomyosarcoma of the uterus, stage II endometrial stromal sarcoma, and stage II adenosarcoma of the uterus may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage III leiomyosarcoma of the uterus, stage III endometrial stromal sarcoma, and stage III adenosarcoma of the uterus may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. There is no standard treatment for patients with stage IV leiomyosarcoma of the uterus, stage IV endometrial stromal sarcoma, or stage IV adenosarcoma of the uterus. Treatment may include a clinical trial using chemotherapy. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. There is no standard treatment for recurrent uterine sarcoma. Treatment may include a clinical trial using chemotherapy. For patients with recurrent carcinosarcoma (a certain type of tumor), treatment may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about uterine sarcoma, see the Uterine Cancer Home Page. For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of uterine sarcoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Uterine Sarcoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/uterine/patient/uterine-sarcoma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389379] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Uterine Sarcoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the stages of Uterine Sarcoma ?

Key Points - After uterine sarcoma has been diagnosed, tests are done to find out if cancer cells have spread within the uterus or to other parts of the body. - Uterine sarcoma may be diagnosed, staged, and treated in the same surgery. - There are three ways that cancer spreads in the body. - Cancer may spread from where it began to other parts of the body. - The following stages are used for uterine sarcoma: - Stage I - Stage II - Stage III - Stage IV After uterine sarcoma has been diagnosed, tests are done to find out if cancer cells have spread within the uterus or to other parts of the body. The process used to find out if cancer has spread within the uterus or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following procedures may be used in the staging process: - Blood chemistry studies : A procedure in which a blood sample is checked to measure the amounts of certain substances released into the blood by organs and tissues in the body. An unusual (higher or lower than normal) amount of a substance can be a sign of disease. - CA 125 assay : A test that measures the level of CA 125 in the blood. CA 125 is a substance released by cells into the bloodstream. An increased CA 125 level is sometimes a sign of cancer or other condition. - Chest x-ray : An x-ray of the organs and bones inside the chest. An x-ray is a type of energy beam that can go through the body and onto film, making a picture of areas inside the body. - Transvaginal ultrasound exam: A procedure used to examine the vagina, uterus, fallopian tubes, and bladder. An ultrasound transducer (probe) is inserted into the vagina and used to bounce high-energy sound waves (ultrasound) off internal tissues or organs and make echoes. The echoes form a picture of body tissues called a sonogram. The doctor can identify tumors by looking at the sonogram. - CT scan (CAT scan): A procedure that makes a series of detailed pictures of areas inside the body, such as the abdomen and pelvis, taken from different angles. The pictures are made by a computer linked to an x-ray machine. A dye may be injected into a vein or swallowed to help the organs or tissues to show up more clearly. This procedure is also called computed tomography, computerized tomography, or computerized axial tomography. - Cystoscopy : A procedure to look inside the bladder and urethra to check for abnormal areas. A cystoscope is inserted through the urethra into the bladder. A cystoscope is a thin, tube-like instrument with a light and a lens for viewing. It may also have a tool to remove tissue samples, which are checked under a microscope for signs of cancer. Uterine sarcoma may be diagnosed, staged, and treated in the same surgery. Surgery is used to diagnose, stage, and treat uterine sarcoma. During this surgery, the doctor removes as much of the cancer as possible. The following procedures may be used to diagnose, stage, and treat uterine sarcoma: - Laparotomy: A surgical procedure in which an incision (cut) is made in the wall of the abdomen to check the inside of the abdomen for signs of disease. The size of the incision depends on the reason the laparotomy is being done. Sometimes organs are removed or tissue samples are taken and checked under a microscope for signs of disease. - Abdominal and pelvic washings: A procedure in which a saline solution is placed into the abdominal and pelvic body cavities. After a short time, the fluid is removed and viewed under a microscope to check for cancer cells. - Total abdominal hysterectomy: A surgical procedure to remove the uterus and cervix through a large incision (cut) in the abdomen. - Bilateral salpingo-oophorectomy: Surgery to remove both ovaries and both fallopian tubes. - Lymphadenectomy: A surgical procedure in which lymph nodes are removed and checked under a microscope for signs of cancer. For a regional lymphadenectomy, some of the lymph nodes in the tumor area are removed. For a radical lymphadenectomy, most or all of the lymph nodes in the tumor area are removed. This procedure is also called lymph node dissection. Treatment in addition to surgery may be given, as described in the Treatment Option Overview section of this summary. There are three ways that cancer spreads in the body. Cancer can spread through tissue, the lymph system, and the blood: - Tissue. The cancer spreads from where it began by growing into nearby areas. - Lymph system. The cancer spreads from where it began by getting into the lymph system. The cancer travels through the lymph vessels to other parts of the body. - Blood. The cancer spreads from where it began by getting into the blood. The cancer travels through the blood vessels to other parts of the body. Cancer may spread from where it began to other parts of the body. When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. - Lymph system. The cancer gets into the lymph system, travels through the lymph vessels, and forms a tumor (metastatic tumor) in another part of the body. - Blood. The cancer gets into the blood, travels through the blood vessels, and forms a tumor (metastatic tumor) in another part of the body. The metastatic tumor is the same type of cancer as the primary tumor. For example, if uterine sarcoma spreads to the lung, the cancer cells in the lung are actually uterine sarcoma cells. The disease is metastatic uterine sarcoma, not lung cancer. The following stages are used for uterine sarcoma: Stage I In stage I, cancer is found in the uterus only. Stage I is divided into stages IA and IB, based on how far the cancer has spread. - Stage IA: Cancer is in the endometrium only or less than halfway through the myometrium (muscle layer of the uterus). - Stage IB: Cancer has spread halfway or more into the myometrium. Stage II In stage II, cancer has spread into connective tissue of the cervix, but has not spread outside the uterus. Stage III In stage III, cancer has spread beyond the uterus and cervix, but has not spread beyond the pelvis. Stage III is divided into stages IIIA, IIIB, and IIIC, based on how far the cancer has spread within the pelvis. - Stage IIIA: Cancer has spread to the outer layer of the uterus and/or to the fallopian tubes, ovaries, and ligaments of the uterus. - Stage IIIB: Cancer has spread to the vagina or to the parametrium (connective tissue and fat around the uterus). - Stage IIIC: Cancer has spread to lymph nodes in the pelvis and/or around the aorta (largest artery in the body, which carries blood away from the heart). Stage IV In stage IV, cancer has spread beyond the pelvis. Stage IV is divided into stages IVA and IVB, based on how far the cancer has spread. - Stage IVA: Cancer has spread to the bladder and/or bowel wall. - Stage IVB: Cancer has spread to other parts of the body beyond the pelvis, including the abdomen and/or lymph nodes in the groin.

The uterus is part of the female reproductive system. The uterus is the hollow, pear-shaped organ in the pelvis, where a fetus grows. The cervix is at the lower, narrow end of the uterus, and leads to the vagina. EnlargeAnatomy of the female reproductive system. The organs in the female reproductive system include the uterus, ovaries, fallopian tubes, cervix, and vagina. The uterus has a muscular outer layer called the myometrium and an inner lining called the endometrium. Uterine sarcoma is a very rare kind of cancer that forms in the uterine muscles or in tissues that support the uterus. (Information about other types of sarcomas can be found in the PDQ summary on Soft Tissue Sarcoma Treatment.) Uterine sarcoma is different from endometrial cancer, a disease in which cancer forms in the tissue that lines the uterus. Carcinosarcoma is a subtype of endometrial cancer and is staged using endometrial cancer definitions. (For more information, seeEndometrial Cancer Treatment.) Uterine sarcomas include leiomyosarcomas, endometrial stromal sarcomas, and adenosarcomas. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for uterine sarcoma include the following: Abnormal bleeding from the vagina and other signs and symptoms may be caused by uterine sarcoma or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: The prognosis and treatment options depend on the following: The process used to find out if cancer has spread within the uterus or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following procedures may be used in the staging process: Surgery is used to diagnose, stage, and treat uterine sarcoma. During this surgery, the doctor removes as much of the cancer as possible. The following procedures may be used to diagnose, stage, and treat uterine sarcoma: Treatment in addition to surgery may be given, as described in the Treatment Option Overview section of this summary. When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if uterine sarcoma spreads to the lung, the cancer cells in the lung are actually uterine sarcoma cells. The disease is metastatic uterine sarcoma, not lung cancer. Cancer can spread through tissue, the lymph system, and the blood: In stage I, the tumor is found in the uterus only. Stage I is divided into stages IA and IB: In stage II, the tumor has spread beyond the uterus but has not spread beyond the pelvis. Stage II is divided into stages IIA and IIB: In stage III, the tumor has spread into tissues in the abdomen. Stage III is divided into stages IIIA, IIIB, and IIIC: Stage IV is divided into stages IVA and IVB: In stage I, the tumor is found in the uterus only. Stage I is divided into stages IA, IB, and IC: In stage II, the tumor has spread outside the uterus into the pelvis. Stage II is divided into stages IIA and IIB: In stage III, the tumor has spread into tissues in the abdomen. Stage III is divided into stages IIIA, IIIB, and IIIC: Stage IV is divided into stages IVA and IVB: The cancer may come back in the uterus, the pelvis, or in other parts of the body. Different types of treatments are available for patients with uterine sarcoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment for uterine sarcoma, as described in the Stages of Uterine Sarcoma section of this summary. After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type and stage of the cancer being treated. External and internal radiation therapy are used to treat uterine sarcoma, and may also be used as palliative therapy to relieve symptoms and improve quality of life. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Hormone therapy is a cancer treatment that removes hormones or blocks their action and stops cancer cells from growing. Hormones are substances produced by glands in the body and circulated in the bloodstream. Some hormones can cause certain cancers to grow. If tests show the cancer cells have places where hormones can attach (receptors), drugs, surgery, or radiation therapy is used to reduce the production of hormones or block them from working. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage I leiomyosarcoma of the uterus, stage I endometrial stromal sarcoma, and stage I adenosarcoma of the uterus may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage II leiomyosarcoma of the uterus, stage II endometrial stromal sarcoma, and stage II adenosarcoma of the uterus may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage III leiomyosarcoma of the uterus, stage III endometrial stromal sarcoma, and stage III adenosarcoma of the uterus may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. There is no standard treatment for patients with stage IV leiomyosarcoma of the uterus, stage IV endometrial stromal sarcoma, or stage IV adenosarcoma of the uterus. Treatment may include a clinical trial using chemotherapy. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. There is no standard treatment for recurrent uterine sarcoma. Treatment may include a clinical trial using chemotherapy. For patients with recurrent carcinosarcoma (a certain type of tumor), treatment may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about uterine sarcoma, see the Uterine Cancer Home Page. For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of uterine sarcoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Uterine Sarcoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/uterine/patient/uterine-sarcoma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389379] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Uterine Sarcoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the treatments for Uterine Sarcoma ?

Key Points - There are different types of treatment for patients with uterine sarcoma. - Four types of standard treatment are used: - Surgery - Radiation therapy - Chemotherapy - Hormone therapy - New types of treatment are being tested in clinical trials. - Patients may want to think about taking part in a clinical trial. - Patients can enter clinical trials before, during, or after starting their cancer treatment. - Follow-up tests may be needed. There are different types of treatment for patients with uterine sarcoma. Different types of treatments are available for patients with uterine sarcoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Four types of standard treatment are used: Surgery Surgery is the most common treatment for uterine sarcoma, as described in the Stages of Uterine Sarcoma section of this summary. Even if the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy Radiation therapy is a cancer treatment that uses high energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: - External radiation therapy uses a machine outside the body to send radiation toward the cancer. - Internal radiation therapy uses a radioactive substance sealed in needles, seeds, wires, or catheters that are placed directly into or near the cancer. The way the radiation therapy is given depends on the type and stage of the cancer being treated. External and internal radiation therapy are used to treat uterine sarcoma, and may also be used as palliative therapy to relieve symptoms and improve quality of life. Chemotherapy Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on the type and stage of the cancer being treated. Hormone therapy Hormone therapy is a cancer treatment that removes hormones or blocks their action and stops cancer cells from growing. Hormones are substances produced by glands in the body and circulated in the bloodstream. Some hormones can cause certain cancers to grow. If tests show the cancer cells have places where hormones can attach (receptors), drugs, surgery, or radiation therapy is used to reduce the production of hormones or block them from working. New types of treatment are being tested in clinical trials. Information about clinical trials is available from the NCI website. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials. Follow-up tests may be needed. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. Treatment Options by Stage Stage I Uterine Sarcoma Treatment of stage I uterine sarcoma may include the following: - Surgery (total abdominal hysterectomy, bilateral salpingo-oophorectomy, and lymphadenectomy). - Surgery followed by radiation therapy to the pelvis. - Surgery followed by chemotherapy. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage I uterine sarcoma. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Stage II Uterine Sarcoma Treatment of stage II uterine sarcoma may include the following: - Surgery (total abdominal hysterectomy, bilateral salpingo-oophorectomy, and lymphadenectomy). - Surgery followed by radiation therapy to the pelvis. - Surgery followed by chemotherapy. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage II uterine sarcoma. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Stage III Uterine Sarcoma Treatment of stage III uterine sarcoma may include the following: - Surgery (total abdominal hysterectomy, bilateral salpingo-oophorectomy, and lymphadenectomy). - A clinical trial of surgery followed by radiation therapy to the pelvis. - A clinical trial of surgery followed by chemotherapy. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage III uterine sarcoma. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Stage IV Uterine Sarcoma There is no standard treatment for patients with stage IV uterine sarcoma. Treatment may include a clinical trial using chemotherapy. Check the list of NCI-supported cancer clinical trials that are now accepting patients with stage IV uterine sarcoma. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website.

The uterus is part of the female reproductive system. The uterus is the hollow, pear-shaped organ in the pelvis, where a fetus grows. The cervix is at the lower, narrow end of the uterus, and leads to the vagina. EnlargeAnatomy of the female reproductive system. The organs in the female reproductive system include the uterus, ovaries, fallopian tubes, cervix, and vagina. The uterus has a muscular outer layer called the myometrium and an inner lining called the endometrium. Uterine sarcoma is a very rare kind of cancer that forms in the uterine muscles or in tissues that support the uterus. (Information about other types of sarcomas can be found in the PDQ summary on Soft Tissue Sarcoma Treatment.) Uterine sarcoma is different from endometrial cancer, a disease in which cancer forms in the tissue that lines the uterus. Carcinosarcoma is a subtype of endometrial cancer and is staged using endometrial cancer definitions. (For more information, seeEndometrial Cancer Treatment.) Uterine sarcomas include leiomyosarcomas, endometrial stromal sarcomas, and adenosarcomas. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for uterine sarcoma include the following: Abnormal bleeding from the vagina and other signs and symptoms may be caused by uterine sarcoma or by other conditions. Check with your doctor if you have any of the following: The following tests and procedures may be used: The prognosis and treatment options depend on the following: The process used to find out if cancer has spread within the uterus or to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following procedures may be used in the staging process: Surgery is used to diagnose, stage, and treat uterine sarcoma. During this surgery, the doctor removes as much of the cancer as possible. The following procedures may be used to diagnose, stage, and treat uterine sarcoma: Treatment in addition to surgery may be given, as described in the Treatment Option Overview section of this summary. When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood. The metastatic tumor is the same type of cancer as the primary tumor. For example, if uterine sarcoma spreads to the lung, the cancer cells in the lung are actually uterine sarcoma cells. The disease is metastatic uterine sarcoma, not lung cancer. Cancer can spread through tissue, the lymph system, and the blood: In stage I, the tumor is found in the uterus only. Stage I is divided into stages IA and IB: In stage II, the tumor has spread beyond the uterus but has not spread beyond the pelvis. Stage II is divided into stages IIA and IIB: In stage III, the tumor has spread into tissues in the abdomen. Stage III is divided into stages IIIA, IIIB, and IIIC: Stage IV is divided into stages IVA and IVB: In stage I, the tumor is found in the uterus only. Stage I is divided into stages IA, IB, and IC: In stage II, the tumor has spread outside the uterus into the pelvis. Stage II is divided into stages IIA and IIB: In stage III, the tumor has spread into tissues in the abdomen. Stage III is divided into stages IIIA, IIIB, and IIIC: Stage IV is divided into stages IVA and IVB: The cancer may come back in the uterus, the pelvis, or in other parts of the body. Different types of treatments are available for patients with uterine sarcoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Surgery is the most common treatment for uterine sarcoma, as described in the Stages of Uterine Sarcoma section of this summary. After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy. Radiation therapy is a cancer treatment that uses high energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type and stage of the cancer being treated. External and internal radiation therapy are used to treat uterine sarcoma, and may also be used as palliative therapy to relieve symptoms and improve quality of life. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Hormone therapy is a cancer treatment that removes hormones or blocks their action and stops cancer cells from growing. Hormones are substances produced by glands in the body and circulated in the bloodstream. Some hormones can cause certain cancers to grow. If tests show the cancer cells have places where hormones can attach (receptors), drugs, surgery, or radiation therapy is used to reduce the production of hormones or block them from working. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage I leiomyosarcoma of the uterus, stage I endometrial stromal sarcoma, and stage I adenosarcoma of the uterus may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage II leiomyosarcoma of the uterus, stage II endometrial stromal sarcoma, and stage II adenosarcoma of the uterus may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of stage III leiomyosarcoma of the uterus, stage III endometrial stromal sarcoma, and stage III adenosarcoma of the uterus may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. There is no standard treatment for patients with stage IV leiomyosarcoma of the uterus, stage IV endometrial stromal sarcoma, or stage IV adenosarcoma of the uterus. Treatment may include a clinical trial using chemotherapy. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. There is no standard treatment for recurrent uterine sarcoma. Treatment may include a clinical trial using chemotherapy. For patients with recurrent carcinosarcoma (a certain type of tumor), treatment may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about uterine sarcoma, see the Uterine Cancer Home Page. For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of uterine sarcoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Uterine Sarcoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/uterine/patient/uterine-sarcoma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389379] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Uterine Sarcoma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

what research (or clinical trials) is being done for Uterine Sarcoma ?

New types of treatment are being tested in clinical trials. Information about clinical trials is available from the NCI website. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials.

Myelodysplastic/myeloproliferative neoplasms are diseases of the blood and bone marrow. Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In myelodysplastic diseases, the blood stem cells do not mature into healthy red blood cells, white blood cells, or platelets. The immature blood cells, called blasts, do not work the way they should and die in the bone marrow or soon after they enter the blood. As a result, there are fewer healthy red blood cells, white blood cells, and platelets. In myeloproliferative diseases, a greater than normal number of blood stem cells become one or more types of blood cells and the total number of blood cells slowly increases. This summary is about neoplasms that have features of both myelodysplastic and myeloproliferative diseases. See the following PDQ summaries for more information about related diseases: The 3 main types of myelodysplastic/myeloproliferative neoplasms include the following: When a myelodysplastic/myeloproliferative neoplasm does not match any of these types, it is called myelodysplastic/myeloproliferative neoplasm, unclassifiable (MDS/MPN-UC). Myelodysplastic/myeloproliferative neoplasms may progress to acute leukemia. The following tests and procedures may be used: The following tests may be done on the sample of tissue that is removed: In chronic myelomonocytic leukemia (CMML), the body tells too many blood stem cells to become two types of white blood cells called myelocytes and monocytes. Some of these blood stem cells never become mature white blood cells. These immature white blood cells are called blasts. Over time, the myelocytes, monocytes, and blasts crowd out the red blood cells and platelets in the bone marrow. When this happens, infection, anemia, or easy bleeding may occur. Anything that increases your chance of getting a disease is called a risk factor. Possible risk factors for CMML include the following: These and other signs and symptoms may be caused by CMML or by other conditions. Check with your doctor if you have any of the following: The prognosis and treatment options for CMML depend on the following: Juvenile myelomonocytic leukemia (JMML) is a rare childhood cancer that occurs more often in children younger than 2 years. Children who have neurofibromatosis type 1 and males have an increased risk of juvenile myelomonocytic leukemia. In JMML, the body tells too many blood stem cells to become two types of white blood cells called myelocytes and monocytes. Some of these blood stem cells never become mature white blood cells. These immature white blood cells are called blasts. Over time, the myelocytes, monocytes, and blasts crowd out the red blood cells and platelets in the bone marrow. When this happens, infection, anemia, or easy bleeding may occur. These and other signs and symptoms may be caused by JMML or by other conditions. Check with your doctor if you have any of the following: The prognosis and treatment options for JMML depend on the following: In atypical chronic myelogenous leukemia (CML), the body tells too many blood stem cells to become a type of white blood cell called granulocytes. Some of these blood stem cells never become mature white blood cells. These immature white blood cells are called blasts. Over time, the granulocytes and blasts crowd out the red blood cells and platelets in the bone marrow. The leukemia cells in atypical CML and CML look alike under a microscope. However, in atypical CML a certain chromosome change, called the "Philadelphia chromosome" is not there. These and other signs and symptoms may be caused by atypical CML or by other conditions. Check with your doctor if you have any of the following: The prognosis for atypical CML depends on the number of red blood cells and platelets in the blood. In myelodysplastic/myeloproliferative neoplasm, unclassifiable (MDS/MPD-UC), the body tells too many blood stem cells to become red blood cells, white blood cells, or platelets. Some of these blood stem cells never become mature blood cells. These immature blood cells are called blasts. Over time, the abnormal blood cells and blasts in the bone marrow crowd out the healthy red blood cells, white blood cells, and platelets. MDS/MPN-UC is a very rare disease. Because it is so rare, the factors that affect risk and prognosis are not known. These and other signs and symptoms may be caused by MDS/MPN-UC or by other conditions. Check with your doctor if you have any of the following: The process used to find out if cancer has spread is called staging. There is no standard staging system for myelodysplastic/myeloproliferative neoplasms. It is important to know the type of myelodysplastic/myeloproliferative neoplasm in order to plan treatment. Different types of treatments are available for patients with myelodysplastic/myeloproliferative neoplasms. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Combination chemotherapy is treatment using more than one anticancer drug. See Drugs Approved for Myeloproliferative Neoplasms for more information. 13-cis retinoic acid is a vitamin-like drug that slows the cancer's ability to make more cancer cells and changes the way these cells look and act. Chemotherapy is given to kill abnormal cells or cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. EnlargeStem cell transplant. (Step 1): Blood is taken from a vein in the arm of the donor. The patient or another person may be the donor. The blood flows through a machine that removes the stem cells. Then the blood is returned to the donor through a vein in the other arm. (Step 2): The patient receives chemotherapy to kill blood-forming cells. The patient may receive radiation therapy (not shown). (Step 3): The patient receives stem cells through a catheter placed into a blood vessel in the chest. Supportive care is given to lessen the problems caused by the disease or its treatment. Supportive care may include transfusion therapy or drug therapy, such as antibiotics to fight infection. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. See Drugs Approved for Myeloproliferative Neoplasms for more information. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic myelomonocytic leukemia (CMML) may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of juvenile myelomonocytic leukemia (JMML) may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of atypical chronic myelogenous leukemia (CML) may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Because myelodysplastic/myeloproliferative neoplasm, unclassifiable (MDS/MPN-UC) is a rare disease, little is known about its treatment. Treatment may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about myelodysplastic/myeloproliferative neoplasms, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of myelodysplastic/ myeloproliferative neoplasms. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Myelodysplastic/ Myeloproliferative Neoplasms Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/myeloproliferative/patient/mds-mpd-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389360] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Myelodysplastic/ Myeloproliferative Neoplasms Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is (are) Juvenile Myelomonocytic Leukemia ?

Key Points - Juvenile myelomonocytic leukemia is a childhood disease in which too many myelocytes and monocytes (immature white blood cells) are made in the bone marrow. - Signs and symptoms of juvenile myelomonocytic leukemia include fever, weight loss, and feeling very tired. - Certain factors affect prognosis (chance of recovery) and treatment options. Juvenile myelomonocytic leukemia is a childhood disease in which too many myelocytes and monocytes (immature white blood cells) are made in the bone marrow. Juvenile myelomonocytic leukemia (JMML) is a rare childhood cancer that occurs more often in children younger than 2 years. Children who have neurofibromatosis type 1 and males have an increased risk of juvenile myelomonocytic leukemia. In JMML, the body tells too many blood stem cells to become two types of white blood cells called myelocytes and monocytes. Some of these blood stem cells never become mature white blood cells. These immature white blood cells are called blasts. Over time, the myelocytes, monocytes, and blasts crowd out the red blood cells and platelets in the bone marrow. When this happens, infection, anemia, or easy bleeding may occur.

Myelodysplastic/myeloproliferative neoplasms are diseases of the blood and bone marrow. Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In myelodysplastic diseases, the blood stem cells do not mature into healthy red blood cells, white blood cells, or platelets. The immature blood cells, called blasts, do not work the way they should and die in the bone marrow or soon after they enter the blood. As a result, there are fewer healthy red blood cells, white blood cells, and platelets. In myeloproliferative diseases, a greater than normal number of blood stem cells become one or more types of blood cells and the total number of blood cells slowly increases. This summary is about neoplasms that have features of both myelodysplastic and myeloproliferative diseases. See the following PDQ summaries for more information about related diseases: The 3 main types of myelodysplastic/myeloproliferative neoplasms include the following: When a myelodysplastic/myeloproliferative neoplasm does not match any of these types, it is called myelodysplastic/myeloproliferative neoplasm, unclassifiable (MDS/MPN-UC). Myelodysplastic/myeloproliferative neoplasms may progress to acute leukemia. The following tests and procedures may be used: The following tests may be done on the sample of tissue that is removed: In chronic myelomonocytic leukemia (CMML), the body tells too many blood stem cells to become two types of white blood cells called myelocytes and monocytes. Some of these blood stem cells never become mature white blood cells. These immature white blood cells are called blasts. Over time, the myelocytes, monocytes, and blasts crowd out the red blood cells and platelets in the bone marrow. When this happens, infection, anemia, or easy bleeding may occur. Anything that increases your chance of getting a disease is called a risk factor. Possible risk factors for CMML include the following: These and other signs and symptoms may be caused by CMML or by other conditions. Check with your doctor if you have any of the following: The prognosis and treatment options for CMML depend on the following: Juvenile myelomonocytic leukemia (JMML) is a rare childhood cancer that occurs more often in children younger than 2 years. Children who have neurofibromatosis type 1 and males have an increased risk of juvenile myelomonocytic leukemia. In JMML, the body tells too many blood stem cells to become two types of white blood cells called myelocytes and monocytes. Some of these blood stem cells never become mature white blood cells. These immature white blood cells are called blasts. Over time, the myelocytes, monocytes, and blasts crowd out the red blood cells and platelets in the bone marrow. When this happens, infection, anemia, or easy bleeding may occur. These and other signs and symptoms may be caused by JMML or by other conditions. Check with your doctor if you have any of the following: The prognosis and treatment options for JMML depend on the following: In atypical chronic myelogenous leukemia (CML), the body tells too many blood stem cells to become a type of white blood cell called granulocytes. Some of these blood stem cells never become mature white blood cells. These immature white blood cells are called blasts. Over time, the granulocytes and blasts crowd out the red blood cells and platelets in the bone marrow. The leukemia cells in atypical CML and CML look alike under a microscope. However, in atypical CML a certain chromosome change, called the "Philadelphia chromosome" is not there. These and other signs and symptoms may be caused by atypical CML or by other conditions. Check with your doctor if you have any of the following: The prognosis for atypical CML depends on the number of red blood cells and platelets in the blood. In myelodysplastic/myeloproliferative neoplasm, unclassifiable (MDS/MPD-UC), the body tells too many blood stem cells to become red blood cells, white blood cells, or platelets. Some of these blood stem cells never become mature blood cells. These immature blood cells are called blasts. Over time, the abnormal blood cells and blasts in the bone marrow crowd out the healthy red blood cells, white blood cells, and platelets. MDS/MPN-UC is a very rare disease. Because it is so rare, the factors that affect risk and prognosis are not known. These and other signs and symptoms may be caused by MDS/MPN-UC or by other conditions. Check with your doctor if you have any of the following: The process used to find out if cancer has spread is called staging. There is no standard staging system for myelodysplastic/myeloproliferative neoplasms. It is important to know the type of myelodysplastic/myeloproliferative neoplasm in order to plan treatment. Different types of treatments are available for patients with myelodysplastic/myeloproliferative neoplasms. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Combination chemotherapy is treatment using more than one anticancer drug. See Drugs Approved for Myeloproliferative Neoplasms for more information. 13-cis retinoic acid is a vitamin-like drug that slows the cancer's ability to make more cancer cells and changes the way these cells look and act. Chemotherapy is given to kill abnormal cells or cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. EnlargeStem cell transplant. (Step 1): Blood is taken from a vein in the arm of the donor. The patient or another person may be the donor. The blood flows through a machine that removes the stem cells. Then the blood is returned to the donor through a vein in the other arm. (Step 2): The patient receives chemotherapy to kill blood-forming cells. The patient may receive radiation therapy (not shown). (Step 3): The patient receives stem cells through a catheter placed into a blood vessel in the chest. Supportive care is given to lessen the problems caused by the disease or its treatment. Supportive care may include transfusion therapy or drug therapy, such as antibiotics to fight infection. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. See Drugs Approved for Myeloproliferative Neoplasms for more information. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic myelomonocytic leukemia (CMML) may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of juvenile myelomonocytic leukemia (JMML) may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of atypical chronic myelogenous leukemia (CML) may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Because myelodysplastic/myeloproliferative neoplasm, unclassifiable (MDS/MPN-UC) is a rare disease, little is known about its treatment. Treatment may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about myelodysplastic/myeloproliferative neoplasms, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of myelodysplastic/ myeloproliferative neoplasms. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Myelodysplastic/ Myeloproliferative Neoplasms Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/myeloproliferative/patient/mds-mpd-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389360] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Myelodysplastic/ Myeloproliferative Neoplasms Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the symptoms of Juvenile Myelomonocytic Leukemia ?

Signs and symptoms of juvenile myelomonocytic leukemia include fever, weight loss, and feeling very tired. These and other signs and symptoms may be caused by JMML or by other conditions. Check with your doctor if you have any of the following: - Fever for no known reason. - Having infections, such as bronchitis or tonsillitis. - Feeling very tired. - Easy bruising or bleeding. - Skin rash. - Painless swelling of the lymph nodes in the neck, underarm, stomach, or groin. - Pain or a feeling of fullness below the ribs.

Myelodysplastic/myeloproliferative neoplasms are diseases of the blood and bone marrow. Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In myelodysplastic diseases, the blood stem cells do not mature into healthy red blood cells, white blood cells, or platelets. The immature blood cells, called blasts, do not work the way they should and die in the bone marrow or soon after they enter the blood. As a result, there are fewer healthy red blood cells, white blood cells, and platelets. In myeloproliferative diseases, a greater than normal number of blood stem cells become one or more types of blood cells and the total number of blood cells slowly increases. This summary is about neoplasms that have features of both myelodysplastic and myeloproliferative diseases. See the following PDQ summaries for more information about related diseases: The 3 main types of myelodysplastic/myeloproliferative neoplasms include the following: When a myelodysplastic/myeloproliferative neoplasm does not match any of these types, it is called myelodysplastic/myeloproliferative neoplasm, unclassifiable (MDS/MPN-UC). Myelodysplastic/myeloproliferative neoplasms may progress to acute leukemia. The following tests and procedures may be used: The following tests may be done on the sample of tissue that is removed: In chronic myelomonocytic leukemia (CMML), the body tells too many blood stem cells to become two types of white blood cells called myelocytes and monocytes. Some of these blood stem cells never become mature white blood cells. These immature white blood cells are called blasts. Over time, the myelocytes, monocytes, and blasts crowd out the red blood cells and platelets in the bone marrow. When this happens, infection, anemia, or easy bleeding may occur. Anything that increases your chance of getting a disease is called a risk factor. Possible risk factors for CMML include the following: These and other signs and symptoms may be caused by CMML or by other conditions. Check with your doctor if you have any of the following: The prognosis and treatment options for CMML depend on the following: Juvenile myelomonocytic leukemia (JMML) is a rare childhood cancer that occurs more often in children younger than 2 years. Children who have neurofibromatosis type 1 and males have an increased risk of juvenile myelomonocytic leukemia. In JMML, the body tells too many blood stem cells to become two types of white blood cells called myelocytes and monocytes. Some of these blood stem cells never become mature white blood cells. These immature white blood cells are called blasts. Over time, the myelocytes, monocytes, and blasts crowd out the red blood cells and platelets in the bone marrow. When this happens, infection, anemia, or easy bleeding may occur. These and other signs and symptoms may be caused by JMML or by other conditions. Check with your doctor if you have any of the following: The prognosis and treatment options for JMML depend on the following: In atypical chronic myelogenous leukemia (CML), the body tells too many blood stem cells to become a type of white blood cell called granulocytes. Some of these blood stem cells never become mature white blood cells. These immature white blood cells are called blasts. Over time, the granulocytes and blasts crowd out the red blood cells and platelets in the bone marrow. The leukemia cells in atypical CML and CML look alike under a microscope. However, in atypical CML a certain chromosome change, called the "Philadelphia chromosome" is not there. These and other signs and symptoms may be caused by atypical CML or by other conditions. Check with your doctor if you have any of the following: The prognosis for atypical CML depends on the number of red blood cells and platelets in the blood. In myelodysplastic/myeloproliferative neoplasm, unclassifiable (MDS/MPD-UC), the body tells too many blood stem cells to become red blood cells, white blood cells, or platelets. Some of these blood stem cells never become mature blood cells. These immature blood cells are called blasts. Over time, the abnormal blood cells and blasts in the bone marrow crowd out the healthy red blood cells, white blood cells, and platelets. MDS/MPN-UC is a very rare disease. Because it is so rare, the factors that affect risk and prognosis are not known. These and other signs and symptoms may be caused by MDS/MPN-UC or by other conditions. Check with your doctor if you have any of the following: The process used to find out if cancer has spread is called staging. There is no standard staging system for myelodysplastic/myeloproliferative neoplasms. It is important to know the type of myelodysplastic/myeloproliferative neoplasm in order to plan treatment. Different types of treatments are available for patients with myelodysplastic/myeloproliferative neoplasms. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Combination chemotherapy is treatment using more than one anticancer drug. See Drugs Approved for Myeloproliferative Neoplasms for more information. 13-cis retinoic acid is a vitamin-like drug that slows the cancer's ability to make more cancer cells and changes the way these cells look and act. Chemotherapy is given to kill abnormal cells or cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. EnlargeStem cell transplant. (Step 1): Blood is taken from a vein in the arm of the donor. The patient or another person may be the donor. The blood flows through a machine that removes the stem cells. Then the blood is returned to the donor through a vein in the other arm. (Step 2): The patient receives chemotherapy to kill blood-forming cells. The patient may receive radiation therapy (not shown). (Step 3): The patient receives stem cells through a catheter placed into a blood vessel in the chest. Supportive care is given to lessen the problems caused by the disease or its treatment. Supportive care may include transfusion therapy or drug therapy, such as antibiotics to fight infection. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. See Drugs Approved for Myeloproliferative Neoplasms for more information. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic myelomonocytic leukemia (CMML) may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of juvenile myelomonocytic leukemia (JMML) may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of atypical chronic myelogenous leukemia (CML) may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Because myelodysplastic/myeloproliferative neoplasm, unclassifiable (MDS/MPN-UC) is a rare disease, little is known about its treatment. Treatment may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about myelodysplastic/myeloproliferative neoplasms, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of myelodysplastic/ myeloproliferative neoplasms. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Myelodysplastic/ Myeloproliferative Neoplasms Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/myeloproliferative/patient/mds-mpd-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389360] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Myelodysplastic/ Myeloproliferative Neoplasms Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is the outlook for Juvenile Myelomonocytic Leukemia ?

Certain factors affect prognosis (chance of recovery) and treatment options. The prognosis (chance of recovery) and treatment options for JMML depend on the following: - The age of the child at diagnosis. - The number of platelets in the blood. - The amount of a certain type of hemoglobin in red blood cells.

Myelodysplastic/myeloproliferative neoplasms are diseases of the blood and bone marrow. Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In myelodysplastic diseases, the blood stem cells do not mature into healthy red blood cells, white blood cells, or platelets. The immature blood cells, called blasts, do not work the way they should and die in the bone marrow or soon after they enter the blood. As a result, there are fewer healthy red blood cells, white blood cells, and platelets. In myeloproliferative diseases, a greater than normal number of blood stem cells become one or more types of blood cells and the total number of blood cells slowly increases. This summary is about neoplasms that have features of both myelodysplastic and myeloproliferative diseases. See the following PDQ summaries for more information about related diseases: The 3 main types of myelodysplastic/myeloproliferative neoplasms include the following: When a myelodysplastic/myeloproliferative neoplasm does not match any of these types, it is called myelodysplastic/myeloproliferative neoplasm, unclassifiable (MDS/MPN-UC). Myelodysplastic/myeloproliferative neoplasms may progress to acute leukemia. The following tests and procedures may be used: The following tests may be done on the sample of tissue that is removed: In chronic myelomonocytic leukemia (CMML), the body tells too many blood stem cells to become two types of white blood cells called myelocytes and monocytes. Some of these blood stem cells never become mature white blood cells. These immature white blood cells are called blasts. Over time, the myelocytes, monocytes, and blasts crowd out the red blood cells and platelets in the bone marrow. When this happens, infection, anemia, or easy bleeding may occur. Anything that increases your chance of getting a disease is called a risk factor. Possible risk factors for CMML include the following: These and other signs and symptoms may be caused by CMML or by other conditions. Check with your doctor if you have any of the following: The prognosis and treatment options for CMML depend on the following: Juvenile myelomonocytic leukemia (JMML) is a rare childhood cancer that occurs more often in children younger than 2 years. Children who have neurofibromatosis type 1 and males have an increased risk of juvenile myelomonocytic leukemia. In JMML, the body tells too many blood stem cells to become two types of white blood cells called myelocytes and monocytes. Some of these blood stem cells never become mature white blood cells. These immature white blood cells are called blasts. Over time, the myelocytes, monocytes, and blasts crowd out the red blood cells and platelets in the bone marrow. When this happens, infection, anemia, or easy bleeding may occur. These and other signs and symptoms may be caused by JMML or by other conditions. Check with your doctor if you have any of the following: The prognosis and treatment options for JMML depend on the following: In atypical chronic myelogenous leukemia (CML), the body tells too many blood stem cells to become a type of white blood cell called granulocytes. Some of these blood stem cells never become mature white blood cells. These immature white blood cells are called blasts. Over time, the granulocytes and blasts crowd out the red blood cells and platelets in the bone marrow. The leukemia cells in atypical CML and CML look alike under a microscope. However, in atypical CML a certain chromosome change, called the "Philadelphia chromosome" is not there. These and other signs and symptoms may be caused by atypical CML or by other conditions. Check with your doctor if you have any of the following: The prognosis for atypical CML depends on the number of red blood cells and platelets in the blood. In myelodysplastic/myeloproliferative neoplasm, unclassifiable (MDS/MPD-UC), the body tells too many blood stem cells to become red blood cells, white blood cells, or platelets. Some of these blood stem cells never become mature blood cells. These immature blood cells are called blasts. Over time, the abnormal blood cells and blasts in the bone marrow crowd out the healthy red blood cells, white blood cells, and platelets. MDS/MPN-UC is a very rare disease. Because it is so rare, the factors that affect risk and prognosis are not known. These and other signs and symptoms may be caused by MDS/MPN-UC or by other conditions. Check with your doctor if you have any of the following: The process used to find out if cancer has spread is called staging. There is no standard staging system for myelodysplastic/myeloproliferative neoplasms. It is important to know the type of myelodysplastic/myeloproliferative neoplasm in order to plan treatment. Different types of treatments are available for patients with myelodysplastic/myeloproliferative neoplasms. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Combination chemotherapy is treatment using more than one anticancer drug. See Drugs Approved for Myeloproliferative Neoplasms for more information. 13-cis retinoic acid is a vitamin-like drug that slows the cancer's ability to make more cancer cells and changes the way these cells look and act. Chemotherapy is given to kill abnormal cells or cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. EnlargeStem cell transplant. (Step 1): Blood is taken from a vein in the arm of the donor. The patient or another person may be the donor. The blood flows through a machine that removes the stem cells. Then the blood is returned to the donor through a vein in the other arm. (Step 2): The patient receives chemotherapy to kill blood-forming cells. The patient may receive radiation therapy (not shown). (Step 3): The patient receives stem cells through a catheter placed into a blood vessel in the chest. Supportive care is given to lessen the problems caused by the disease or its treatment. Supportive care may include transfusion therapy or drug therapy, such as antibiotics to fight infection. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. See Drugs Approved for Myeloproliferative Neoplasms for more information. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic myelomonocytic leukemia (CMML) may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of juvenile myelomonocytic leukemia (JMML) may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of atypical chronic myelogenous leukemia (CML) may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Because myelodysplastic/myeloproliferative neoplasm, unclassifiable (MDS/MPN-UC) is a rare disease, little is known about its treatment. Treatment may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about myelodysplastic/myeloproliferative neoplasms, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of myelodysplastic/ myeloproliferative neoplasms. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Myelodysplastic/ Myeloproliferative Neoplasms Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/myeloproliferative/patient/mds-mpd-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389360] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Myelodysplastic/ Myeloproliferative Neoplasms Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the treatments for Juvenile Myelomonocytic Leukemia ?

Treatment of juvenile myelomonocytic leukemia (JMML) may include the following: - Combination chemotherapy. - Stem cell transplant. - 13-cis-retinoic acid therapy. - A clinical trial of a new treatment, such as targeted therapy. Check the list of NCI-supported cancer clinical trials that are now accepting patients with juvenile myelomonocytic leukemia. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website.

Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. It is the most common type of acute leukemia in adults. This type of cancer usually gets worse quickly if it is not treated. AML is also called acute myelogenous leukemia and acute nonlymphocytic leukemia. Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In AML, the myeloid stem cells usually become a type of immature white blood cell called myeloblasts (or myeloid blasts). The myeloblasts in AML are abnormal and do not become healthy white blood cells. Sometimes in AML, too many stem cells become abnormal red blood cells or platelets. These abnormal white blood cells, red blood cells, or platelets are also called leukemia cells or blasts. Leukemia cells can build up in the bone marrow and blood so there is less room for healthy white blood cells, red blood cells, and platelets. When this happens, infection, anemia, or easy bleeding may occur. The leukemia cells can spread outside the blood to other parts of the body, including the central nervous system (brain and spinal cord), skin, and gums. Sometimes leukemia cells form a solid tumor called a myeloid sarcoma. Myeloid sarcoma is also called extramedullary myeloid tumor, granulocytic sarcoma, or chloroma. This summary is about adult AML. For more information about leukemia, see the following: Most AML subtypes are based on how mature (developed) the cancer cells are at the time of diagnosis and how different they are from normal cells. Acute promyelocytic leukemia (APL) is a subtype of AML. This leukemia occurs when genes on chromosome 15 switch places with some genes on chromosome 17 and an abnormal gene called PML-RARA is made. The PML-RARA gene sends a message that stops promyelocytes (a type of white blood cell) from maturing. Problems with severe bleeding and blood clots may occur. This is a serious health problem that needs treatment as soon as possible. APL usually occurs in middle-aged adults. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Possible risk factors for AML include the following: The early signs and symptoms of AML may be like those caused by the flu or other common diseases. Check with your doctor if you have any of the following: Less common signs or symptoms may be caused by clusters of leukemia cells in the central nervous system (CNS) or testicles, or a tumor of myeloid cells called a chloroma. Symptoms of acute leukemia often develop between 4 and 6 weeks before diagnosis. The following tests and procedures may be used: The prognosis and treatment options depend on: It is important that acute leukemia be treated right away. The extent or spread of cancer is usually described as stages. In acute myeloid leukemia (AML), the subtype of AML and whether the leukemia has spread outside the blood and bone marrow are used instead of the stage to plan treatment. The following tests and procedures may be used to determine if the leukemia has spread: The disease is described as untreated, in remission, refractory, or recurrent. Newly diagnosed (untreated) AML In untreated AML, the disease is newly diagnosed. It has not been treated except to relieve signs and symptoms such as fever, bleeding, or pain, and the following are true: AML in remission In AML in remission, the disease has been treated and the following are true: Refractory or recurrent AML After treatment with chemotherapy, some patients with newly diagnosed AML will not go into remission. This is called refractory cancer. In contrast, recurrent AML is cancer that has recurred (come back) after remission. The AML may come back in the blood or bone marrow. Different types of treatment are available for patients with acute myeloid leukemia (AML). Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. The two treatment phases of AML are: Patients must be closely monitored during treatment of AML. Myelosuppression, a condition which results in fewer red blood cells, white blood cells, and platelets, is a side effect of both AML and treatment with chemotherapy. Supportive care during remission induction therapy may include: Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Intrathecal chemotherapy may be used to treat adult AML that has spread to the brain and spinal cord. Combination chemotherapy is treatment using more than one anticancer drug. The way the chemotherapy is given depends on the subtype of AML being treated and whether leukemia cells have spread to the brain and spinal cord.EnlargeIntrathecal chemotherapy. Anticancer drugs are injected into the intrathecal space, which is the space that holds the cerebrospinal fluid (CSF, shown in blue). There are two different ways to do this. One way, shown in the top part of the figure, is to inject the drugs into an Ommaya reservoir (a dome-shaped container that is placed under the scalp during surgery; it holds the drugs as they flow through a small tube into the brain). The other way, shown in the bottom part of the figure, is to inject the drugs directly into the CSF in the lower part of the spinal column, after a small area on the lower back is numbed. For more information, see Drugs Approved for Acute Myeloid Leukemia. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Total-body irradiation sends radiation toward the whole body. It is a type of external radiation that may be used to prepare the body for a stem cell transplant when the leukemia has recurred. Chemotherapy is given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy and/or total-body irradiation, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. There are different types of targeted therapy. Monoclonal antibodies: immune system proteins made in the laboratory to treat many diseases, including cancer. As a cancer treatment, these antibodies can attach to a specific target on cancer cells or other cells that may help cancer cells grow. The antibodies are able to then kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. Other targeted therapies include: Less-intensive targeted therapies in older or frail patients who cannot receive other treatments include: For more information, see Drugs Approved for Acute Myeloid Leukemia. Arsenic trioxide and all-trans retinoic acid (ATRA) are anticancer drugs that kill leukemia cells, stop the leukemia cells from dividing, or help the leukemia cells mature into white blood cells. These drugs are used in the treatment of a subtype of AML called acute promyelocytic leukemia. For more information, see Drugs Approved for Acute Myeloid Leukemia. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Standard treatment of untreated acute myeloid leukemia (AML) during the remission induction phase depends on the subtype of AML and may include the following: For older adults or patients too frail to receive intensive chemotherapy, the following may be continued as long as the patient benefits or until toxic effects occur: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of acute myeloid leukemia (AML) during the remission phase depends on the subtype of AML and may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. There is no standard treatment for refractory or recurrent acute myeloid leukemia (AML). Treatment depends on the subtype of AML and may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed acute promyelocytic leukemia (APL) includes: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent acute promyelocytic leukemia includes: For more information from the National Cancer Institute about adult acute myeloid leukemia, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of adult acute myeloid leukemia. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Acute Myeloid Leukemia Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/leukemia/patient/adult-aml-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389377] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Acute Myeloid Leukemia Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is (are) Adult Acute Myeloid Leukemia ?

Key Points - Adult acute myeloid leukemia (AML) is a type of cancer in which the bone marrow makes abnormal myeloblasts (a type of white blood cell), red blood cells, or platelets. - Leukemia may affect red blood cells, white blood cells, and platelets. - There are different subtypes of AML. - Smoking, previous chemotherapy treatment, and exposure to radiation may affect the risk of adult AML. - Signs and symptoms of adult AML include fever, feeling tired, and easy bruising or bleeding. - Tests that examine the blood and bone marrow are used to detect (find) and diagnose adult AML. - Certain factors affect prognosis (chance of recovery) and treatment options. Adult acute myeloid leukemia (AML) is a type of cancer in which the bone marrow makes abnormal myeloblasts (a type of white blood cell), red blood cells, or platelets. Adult acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. This type of cancer usually gets worse quickly if it is not treated. It is the most common type of acute leukemia in adults. AML is also called acute myelogenous leukemia, acute myeloblastic leukemia, acute granulocytic leukemia, and acute nonlymphocytic leukemia. Leukemia may affect red blood cells, white blood cells, and platelets. Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: - Red blood cells that carry oxygen and other substances to all tissues of the body. - White blood cells that fight infection and disease. - Platelets that form blood clots to stop bleeding. In AML, the myeloid stem cells usually become a type of immature white blood cell called myeloblasts (or myeloid blasts). The myeloblasts in AML are abnormal and do not become healthy white blood cells. Sometimes in AML, too many stem cells become abnormal red blood cells or platelets. These abnormal white blood cells, red blood cells, or platelets are also called leukemia cells or blasts. Leukemia cells can build up in the bone marrow and blood so there is less room for healthy white blood cells, red blood cells, and platelets. When this happens, infection, anemia, or easy bleeding may occur. The leukemia cells can spread outside the blood to other parts of the body, including the central nervous system (brain and spinal cord), skin, and gums. This summary is about adult AML. See the following PDQ summaries for information about other types of leukemia: - Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment - Chronic Myelogenous Leukemia Treatment - Adult Acute Lymphoblastic Leukemia Treatment - Childhood Acute Lymphoblastic Leukemia Treatment - Chronic Lymphocytic Leukemia Treatment - Hairy Cell Leukemia Treatment There are different subtypes of AML. Most AML subtypes are based on how mature (developed) the cancer cells are at the time of diagnosis and how different they are from normal cells. Acute promyelocytic leukemia (APL) is a subtype of AML that occurs when parts of two genes stick together. APL usually occurs in middle-aged adults. Signs of APL may include both bleeding and forming blood clots.

Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. It is the most common type of acute leukemia in adults. This type of cancer usually gets worse quickly if it is not treated. AML is also called acute myelogenous leukemia and acute nonlymphocytic leukemia. Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In AML, the myeloid stem cells usually become a type of immature white blood cell called myeloblasts (or myeloid blasts). The myeloblasts in AML are abnormal and do not become healthy white blood cells. Sometimes in AML, too many stem cells become abnormal red blood cells or platelets. These abnormal white blood cells, red blood cells, or platelets are also called leukemia cells or blasts. Leukemia cells can build up in the bone marrow and blood so there is less room for healthy white blood cells, red blood cells, and platelets. When this happens, infection, anemia, or easy bleeding may occur. The leukemia cells can spread outside the blood to other parts of the body, including the central nervous system (brain and spinal cord), skin, and gums. Sometimes leukemia cells form a solid tumor called a myeloid sarcoma. Myeloid sarcoma is also called extramedullary myeloid tumor, granulocytic sarcoma, or chloroma. This summary is about adult AML. For more information about leukemia, see the following: Most AML subtypes are based on how mature (developed) the cancer cells are at the time of diagnosis and how different they are from normal cells. Acute promyelocytic leukemia (APL) is a subtype of AML. This leukemia occurs when genes on chromosome 15 switch places with some genes on chromosome 17 and an abnormal gene called PML-RARA is made. The PML-RARA gene sends a message that stops promyelocytes (a type of white blood cell) from maturing. Problems with severe bleeding and blood clots may occur. This is a serious health problem that needs treatment as soon as possible. APL usually occurs in middle-aged adults. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Possible risk factors for AML include the following: The early signs and symptoms of AML may be like those caused by the flu or other common diseases. Check with your doctor if you have any of the following: Less common signs or symptoms may be caused by clusters of leukemia cells in the central nervous system (CNS) or testicles, or a tumor of myeloid cells called a chloroma. Symptoms of acute leukemia often develop between 4 and 6 weeks before diagnosis. The following tests and procedures may be used: The prognosis and treatment options depend on: It is important that acute leukemia be treated right away. The extent or spread of cancer is usually described as stages. In acute myeloid leukemia (AML), the subtype of AML and whether the leukemia has spread outside the blood and bone marrow are used instead of the stage to plan treatment. The following tests and procedures may be used to determine if the leukemia has spread: The disease is described as untreated, in remission, refractory, or recurrent. Newly diagnosed (untreated) AML In untreated AML, the disease is newly diagnosed. It has not been treated except to relieve signs and symptoms such as fever, bleeding, or pain, and the following are true: AML in remission In AML in remission, the disease has been treated and the following are true: Refractory or recurrent AML After treatment with chemotherapy, some patients with newly diagnosed AML will not go into remission. This is called refractory cancer. In contrast, recurrent AML is cancer that has recurred (come back) after remission. The AML may come back in the blood or bone marrow. Different types of treatment are available for patients with acute myeloid leukemia (AML). Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. The two treatment phases of AML are: Patients must be closely monitored during treatment of AML. Myelosuppression, a condition which results in fewer red blood cells, white blood cells, and platelets, is a side effect of both AML and treatment with chemotherapy. Supportive care during remission induction therapy may include: Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Intrathecal chemotherapy may be used to treat adult AML that has spread to the brain and spinal cord. Combination chemotherapy is treatment using more than one anticancer drug. The way the chemotherapy is given depends on the subtype of AML being treated and whether leukemia cells have spread to the brain and spinal cord.EnlargeIntrathecal chemotherapy. Anticancer drugs are injected into the intrathecal space, which is the space that holds the cerebrospinal fluid (CSF, shown in blue). There are two different ways to do this. One way, shown in the top part of the figure, is to inject the drugs into an Ommaya reservoir (a dome-shaped container that is placed under the scalp during surgery; it holds the drugs as they flow through a small tube into the brain). The other way, shown in the bottom part of the figure, is to inject the drugs directly into the CSF in the lower part of the spinal column, after a small area on the lower back is numbed. For more information, see Drugs Approved for Acute Myeloid Leukemia. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Total-body irradiation sends radiation toward the whole body. It is a type of external radiation that may be used to prepare the body for a stem cell transplant when the leukemia has recurred. Chemotherapy is given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy and/or total-body irradiation, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. There are different types of targeted therapy. Monoclonal antibodies: immune system proteins made in the laboratory to treat many diseases, including cancer. As a cancer treatment, these antibodies can attach to a specific target on cancer cells or other cells that may help cancer cells grow. The antibodies are able to then kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. Other targeted therapies include: Less-intensive targeted therapies in older or frail patients who cannot receive other treatments include: For more information, see Drugs Approved for Acute Myeloid Leukemia. Arsenic trioxide and all-trans retinoic acid (ATRA) are anticancer drugs that kill leukemia cells, stop the leukemia cells from dividing, or help the leukemia cells mature into white blood cells. These drugs are used in the treatment of a subtype of AML called acute promyelocytic leukemia. For more information, see Drugs Approved for Acute Myeloid Leukemia. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Standard treatment of untreated acute myeloid leukemia (AML) during the remission induction phase depends on the subtype of AML and may include the following: For older adults or patients too frail to receive intensive chemotherapy, the following may be continued as long as the patient benefits or until toxic effects occur: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of acute myeloid leukemia (AML) during the remission phase depends on the subtype of AML and may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. There is no standard treatment for refractory or recurrent acute myeloid leukemia (AML). Treatment depends on the subtype of AML and may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed acute promyelocytic leukemia (APL) includes: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent acute promyelocytic leukemia includes: For more information from the National Cancer Institute about adult acute myeloid leukemia, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of adult acute myeloid leukemia. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Acute Myeloid Leukemia Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/leukemia/patient/adult-aml-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389377] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Acute Myeloid Leukemia Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

Who is at risk for Adult Acute Myeloid Leukemia? ?

Smoking, previous chemotherapy treatment, and exposure to radiation may affect the risk of adult AML. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesnt mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Possible risk factors for AML include the following: - Being male. - Smoking, especially after age 60. - Having had treatment with chemotherapy or radiation therapy in the past. - Having had treatment for childhood acute lymphoblastic leukemia (ALL) in the past. - Being exposed to radiation from an atomic bomb or to the chemical benzene. - Having a history of a blood disorder such as myelodysplastic syndrome.

Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. It is the most common type of acute leukemia in adults. This type of cancer usually gets worse quickly if it is not treated. AML is also called acute myelogenous leukemia and acute nonlymphocytic leukemia. Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In AML, the myeloid stem cells usually become a type of immature white blood cell called myeloblasts (or myeloid blasts). The myeloblasts in AML are abnormal and do not become healthy white blood cells. Sometimes in AML, too many stem cells become abnormal red blood cells or platelets. These abnormal white blood cells, red blood cells, or platelets are also called leukemia cells or blasts. Leukemia cells can build up in the bone marrow and blood so there is less room for healthy white blood cells, red blood cells, and platelets. When this happens, infection, anemia, or easy bleeding may occur. The leukemia cells can spread outside the blood to other parts of the body, including the central nervous system (brain and spinal cord), skin, and gums. Sometimes leukemia cells form a solid tumor called a myeloid sarcoma. Myeloid sarcoma is also called extramedullary myeloid tumor, granulocytic sarcoma, or chloroma. This summary is about adult AML. For more information about leukemia, see the following: Most AML subtypes are based on how mature (developed) the cancer cells are at the time of diagnosis and how different they are from normal cells. Acute promyelocytic leukemia (APL) is a subtype of AML. This leukemia occurs when genes on chromosome 15 switch places with some genes on chromosome 17 and an abnormal gene called PML-RARA is made. The PML-RARA gene sends a message that stops promyelocytes (a type of white blood cell) from maturing. Problems with severe bleeding and blood clots may occur. This is a serious health problem that needs treatment as soon as possible. APL usually occurs in middle-aged adults. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Possible risk factors for AML include the following: The early signs and symptoms of AML may be like those caused by the flu or other common diseases. Check with your doctor if you have any of the following: Less common signs or symptoms may be caused by clusters of leukemia cells in the central nervous system (CNS) or testicles, or a tumor of myeloid cells called a chloroma. Symptoms of acute leukemia often develop between 4 and 6 weeks before diagnosis. The following tests and procedures may be used: The prognosis and treatment options depend on: It is important that acute leukemia be treated right away. The extent or spread of cancer is usually described as stages. In acute myeloid leukemia (AML), the subtype of AML and whether the leukemia has spread outside the blood and bone marrow are used instead of the stage to plan treatment. The following tests and procedures may be used to determine if the leukemia has spread: The disease is described as untreated, in remission, refractory, or recurrent. Newly diagnosed (untreated) AML In untreated AML, the disease is newly diagnosed. It has not been treated except to relieve signs and symptoms such as fever, bleeding, or pain, and the following are true: AML in remission In AML in remission, the disease has been treated and the following are true: Refractory or recurrent AML After treatment with chemotherapy, some patients with newly diagnosed AML will not go into remission. This is called refractory cancer. In contrast, recurrent AML is cancer that has recurred (come back) after remission. The AML may come back in the blood or bone marrow. Different types of treatment are available for patients with acute myeloid leukemia (AML). Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. The two treatment phases of AML are: Patients must be closely monitored during treatment of AML. Myelosuppression, a condition which results in fewer red blood cells, white blood cells, and platelets, is a side effect of both AML and treatment with chemotherapy. Supportive care during remission induction therapy may include: Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Intrathecal chemotherapy may be used to treat adult AML that has spread to the brain and spinal cord. Combination chemotherapy is treatment using more than one anticancer drug. The way the chemotherapy is given depends on the subtype of AML being treated and whether leukemia cells have spread to the brain and spinal cord.EnlargeIntrathecal chemotherapy. Anticancer drugs are injected into the intrathecal space, which is the space that holds the cerebrospinal fluid (CSF, shown in blue). There are two different ways to do this. One way, shown in the top part of the figure, is to inject the drugs into an Ommaya reservoir (a dome-shaped container that is placed under the scalp during surgery; it holds the drugs as they flow through a small tube into the brain). The other way, shown in the bottom part of the figure, is to inject the drugs directly into the CSF in the lower part of the spinal column, after a small area on the lower back is numbed. For more information, see Drugs Approved for Acute Myeloid Leukemia. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Total-body irradiation sends radiation toward the whole body. It is a type of external radiation that may be used to prepare the body for a stem cell transplant when the leukemia has recurred. Chemotherapy is given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy and/or total-body irradiation, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. There are different types of targeted therapy. Monoclonal antibodies: immune system proteins made in the laboratory to treat many diseases, including cancer. As a cancer treatment, these antibodies can attach to a specific target on cancer cells or other cells that may help cancer cells grow. The antibodies are able to then kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. Other targeted therapies include: Less-intensive targeted therapies in older or frail patients who cannot receive other treatments include: For more information, see Drugs Approved for Acute Myeloid Leukemia. Arsenic trioxide and all-trans retinoic acid (ATRA) are anticancer drugs that kill leukemia cells, stop the leukemia cells from dividing, or help the leukemia cells mature into white blood cells. These drugs are used in the treatment of a subtype of AML called acute promyelocytic leukemia. For more information, see Drugs Approved for Acute Myeloid Leukemia. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Standard treatment of untreated acute myeloid leukemia (AML) during the remission induction phase depends on the subtype of AML and may include the following: For older adults or patients too frail to receive intensive chemotherapy, the following may be continued as long as the patient benefits or until toxic effects occur: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of acute myeloid leukemia (AML) during the remission phase depends on the subtype of AML and may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. There is no standard treatment for refractory or recurrent acute myeloid leukemia (AML). Treatment depends on the subtype of AML and may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed acute promyelocytic leukemia (APL) includes: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent acute promyelocytic leukemia includes: For more information from the National Cancer Institute about adult acute myeloid leukemia, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of adult acute myeloid leukemia. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Acute Myeloid Leukemia Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/leukemia/patient/adult-aml-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389377] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Acute Myeloid Leukemia Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the symptoms of Adult Acute Myeloid Leukemia ?

Signs and symptoms of adult AML include fever, feeling tired, and easy bruising or bleeding. The early signs and symptoms of AML may be like those caused by the flu or other common diseases. Check with your doctor if you have any of the following: - Fever. - Shortness of breath. - Easy bruising or bleeding. - Petechiae (flat, pinpoint spots under the skin caused by bleeding). - Weakness or feeling tired. - Weight loss or loss of appetite.

Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. It is the most common type of acute leukemia in adults. This type of cancer usually gets worse quickly if it is not treated. AML is also called acute myelogenous leukemia and acute nonlymphocytic leukemia. Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In AML, the myeloid stem cells usually become a type of immature white blood cell called myeloblasts (or myeloid blasts). The myeloblasts in AML are abnormal and do not become healthy white blood cells. Sometimes in AML, too many stem cells become abnormal red blood cells or platelets. These abnormal white blood cells, red blood cells, or platelets are also called leukemia cells or blasts. Leukemia cells can build up in the bone marrow and blood so there is less room for healthy white blood cells, red blood cells, and platelets. When this happens, infection, anemia, or easy bleeding may occur. The leukemia cells can spread outside the blood to other parts of the body, including the central nervous system (brain and spinal cord), skin, and gums. Sometimes leukemia cells form a solid tumor called a myeloid sarcoma. Myeloid sarcoma is also called extramedullary myeloid tumor, granulocytic sarcoma, or chloroma. This summary is about adult AML. For more information about leukemia, see the following: Most AML subtypes are based on how mature (developed) the cancer cells are at the time of diagnosis and how different they are from normal cells. Acute promyelocytic leukemia (APL) is a subtype of AML. This leukemia occurs when genes on chromosome 15 switch places with some genes on chromosome 17 and an abnormal gene called PML-RARA is made. The PML-RARA gene sends a message that stops promyelocytes (a type of white blood cell) from maturing. Problems with severe bleeding and blood clots may occur. This is a serious health problem that needs treatment as soon as possible. APL usually occurs in middle-aged adults. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Possible risk factors for AML include the following: The early signs and symptoms of AML may be like those caused by the flu or other common diseases. Check with your doctor if you have any of the following: Less common signs or symptoms may be caused by clusters of leukemia cells in the central nervous system (CNS) or testicles, or a tumor of myeloid cells called a chloroma. Symptoms of acute leukemia often develop between 4 and 6 weeks before diagnosis. The following tests and procedures may be used: The prognosis and treatment options depend on: It is important that acute leukemia be treated right away. The extent or spread of cancer is usually described as stages. In acute myeloid leukemia (AML), the subtype of AML and whether the leukemia has spread outside the blood and bone marrow are used instead of the stage to plan treatment. The following tests and procedures may be used to determine if the leukemia has spread: The disease is described as untreated, in remission, refractory, or recurrent. Newly diagnosed (untreated) AML In untreated AML, the disease is newly diagnosed. It has not been treated except to relieve signs and symptoms such as fever, bleeding, or pain, and the following are true: AML in remission In AML in remission, the disease has been treated and the following are true: Refractory or recurrent AML After treatment with chemotherapy, some patients with newly diagnosed AML will not go into remission. This is called refractory cancer. In contrast, recurrent AML is cancer that has recurred (come back) after remission. The AML may come back in the blood or bone marrow. Different types of treatment are available for patients with acute myeloid leukemia (AML). Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. The two treatment phases of AML are: Patients must be closely monitored during treatment of AML. Myelosuppression, a condition which results in fewer red blood cells, white blood cells, and platelets, is a side effect of both AML and treatment with chemotherapy. Supportive care during remission induction therapy may include: Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Intrathecal chemotherapy may be used to treat adult AML that has spread to the brain and spinal cord. Combination chemotherapy is treatment using more than one anticancer drug. The way the chemotherapy is given depends on the subtype of AML being treated and whether leukemia cells have spread to the brain and spinal cord.EnlargeIntrathecal chemotherapy. Anticancer drugs are injected into the intrathecal space, which is the space that holds the cerebrospinal fluid (CSF, shown in blue). There are two different ways to do this. One way, shown in the top part of the figure, is to inject the drugs into an Ommaya reservoir (a dome-shaped container that is placed under the scalp during surgery; it holds the drugs as they flow through a small tube into the brain). The other way, shown in the bottom part of the figure, is to inject the drugs directly into the CSF in the lower part of the spinal column, after a small area on the lower back is numbed. For more information, see Drugs Approved for Acute Myeloid Leukemia. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Total-body irradiation sends radiation toward the whole body. It is a type of external radiation that may be used to prepare the body for a stem cell transplant when the leukemia has recurred. Chemotherapy is given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy and/or total-body irradiation, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. There are different types of targeted therapy. Monoclonal antibodies: immune system proteins made in the laboratory to treat many diseases, including cancer. As a cancer treatment, these antibodies can attach to a specific target on cancer cells or other cells that may help cancer cells grow. The antibodies are able to then kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. Other targeted therapies include: Less-intensive targeted therapies in older or frail patients who cannot receive other treatments include: For more information, see Drugs Approved for Acute Myeloid Leukemia. Arsenic trioxide and all-trans retinoic acid (ATRA) are anticancer drugs that kill leukemia cells, stop the leukemia cells from dividing, or help the leukemia cells mature into white blood cells. These drugs are used in the treatment of a subtype of AML called acute promyelocytic leukemia. For more information, see Drugs Approved for Acute Myeloid Leukemia. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Standard treatment of untreated acute myeloid leukemia (AML) during the remission induction phase depends on the subtype of AML and may include the following: For older adults or patients too frail to receive intensive chemotherapy, the following may be continued as long as the patient benefits or until toxic effects occur: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of acute myeloid leukemia (AML) during the remission phase depends on the subtype of AML and may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. There is no standard treatment for refractory or recurrent acute myeloid leukemia (AML). Treatment depends on the subtype of AML and may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed acute promyelocytic leukemia (APL) includes: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent acute promyelocytic leukemia includes: For more information from the National Cancer Institute about adult acute myeloid leukemia, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of adult acute myeloid leukemia. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Acute Myeloid Leukemia Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/leukemia/patient/adult-aml-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389377] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Acute Myeloid Leukemia Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

How to diagnose Adult Acute Myeloid Leukemia ?

Tests that examine the blood and bone marrow are used to detect (find) and diagnose adult AML. The following tests and procedures may be used: - Physical exam and history : An exam of the body to check general signs of health, including checking for signs of disease, such as lumps or anything else that seems unusual. A history of the patients health habits and past illnesses and treatments will also be taken. - Complete blood count (CBC): A procedure in which a sample of blood is drawn and checked for the following: - The number of red blood cells, white blood cells, and platelets. - The amount of hemoglobin (the protein that carries oxygen) in the red blood cells. - The portion of the sample made up of red blood cells. - Peripheral blood smear : A procedure in which a sample of blood is checked for blast cells, the number and kinds of white blood cells, the number of platelets, and changes in the shape of blood cells. - Bone marrow aspiration and biopsy : The removal of bone marrow, blood, and a small piece of bone by inserting a hollow needle into the hipbone or breastbone. A pathologist views the bone marrow, blood, and bone under a microscope to look for signs of cancer. - Cytogenetic analysis : A laboratory test in which the cells in a sample of blood or bone marrow are viewed under a microscope to look for certain changes in the chromosomes. Other tests, such as fluorescence in situ hybridization (FISH), may also be done to look for certain changes in the chromosomes. - Immunophenotyping : A process used to identify cells, based on the types of antigens or markers on the surface of the cell. This process is used to diagnose the subtype of AML by comparing the cancer cells to normal cells of the immune system. For example, a cytochemistry study may test the cells in a sample of tissue using chemicals (dyes) to look for certain changes in the sample. A chemical may cause a color change in one type of leukemia cell but not in another type of leukemia cell. - Reverse transcriptionpolymerase chain reaction test (RTPCR): A laboratory test in which cells in a sample of tissue are studied using chemicals to look for certain changes in the structure or function of genes. This test is used to diagnose certain types of AML including acute promyelocytic leukemia (APL).

Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. It is the most common type of acute leukemia in adults. This type of cancer usually gets worse quickly if it is not treated. AML is also called acute myelogenous leukemia and acute nonlymphocytic leukemia. Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In AML, the myeloid stem cells usually become a type of immature white blood cell called myeloblasts (or myeloid blasts). The myeloblasts in AML are abnormal and do not become healthy white blood cells. Sometimes in AML, too many stem cells become abnormal red blood cells or platelets. These abnormal white blood cells, red blood cells, or platelets are also called leukemia cells or blasts. Leukemia cells can build up in the bone marrow and blood so there is less room for healthy white blood cells, red blood cells, and platelets. When this happens, infection, anemia, or easy bleeding may occur. The leukemia cells can spread outside the blood to other parts of the body, including the central nervous system (brain and spinal cord), skin, and gums. Sometimes leukemia cells form a solid tumor called a myeloid sarcoma. Myeloid sarcoma is also called extramedullary myeloid tumor, granulocytic sarcoma, or chloroma. This summary is about adult AML. For more information about leukemia, see the following: Most AML subtypes are based on how mature (developed) the cancer cells are at the time of diagnosis and how different they are from normal cells. Acute promyelocytic leukemia (APL) is a subtype of AML. This leukemia occurs when genes on chromosome 15 switch places with some genes on chromosome 17 and an abnormal gene called PML-RARA is made. The PML-RARA gene sends a message that stops promyelocytes (a type of white blood cell) from maturing. Problems with severe bleeding and blood clots may occur. This is a serious health problem that needs treatment as soon as possible. APL usually occurs in middle-aged adults. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Possible risk factors for AML include the following: The early signs and symptoms of AML may be like those caused by the flu or other common diseases. Check with your doctor if you have any of the following: Less common signs or symptoms may be caused by clusters of leukemia cells in the central nervous system (CNS) or testicles, or a tumor of myeloid cells called a chloroma. Symptoms of acute leukemia often develop between 4 and 6 weeks before diagnosis. The following tests and procedures may be used: The prognosis and treatment options depend on: It is important that acute leukemia be treated right away. The extent or spread of cancer is usually described as stages. In acute myeloid leukemia (AML), the subtype of AML and whether the leukemia has spread outside the blood and bone marrow are used instead of the stage to plan treatment. The following tests and procedures may be used to determine if the leukemia has spread: The disease is described as untreated, in remission, refractory, or recurrent. Newly diagnosed (untreated) AML In untreated AML, the disease is newly diagnosed. It has not been treated except to relieve signs and symptoms such as fever, bleeding, or pain, and the following are true: AML in remission In AML in remission, the disease has been treated and the following are true: Refractory or recurrent AML After treatment with chemotherapy, some patients with newly diagnosed AML will not go into remission. This is called refractory cancer. In contrast, recurrent AML is cancer that has recurred (come back) after remission. The AML may come back in the blood or bone marrow. Different types of treatment are available for patients with acute myeloid leukemia (AML). Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. The two treatment phases of AML are: Patients must be closely monitored during treatment of AML. Myelosuppression, a condition which results in fewer red blood cells, white blood cells, and platelets, is a side effect of both AML and treatment with chemotherapy. Supportive care during remission induction therapy may include: Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Intrathecal chemotherapy may be used to treat adult AML that has spread to the brain and spinal cord. Combination chemotherapy is treatment using more than one anticancer drug. The way the chemotherapy is given depends on the subtype of AML being treated and whether leukemia cells have spread to the brain and spinal cord.EnlargeIntrathecal chemotherapy. Anticancer drugs are injected into the intrathecal space, which is the space that holds the cerebrospinal fluid (CSF, shown in blue). There are two different ways to do this. One way, shown in the top part of the figure, is to inject the drugs into an Ommaya reservoir (a dome-shaped container that is placed under the scalp during surgery; it holds the drugs as they flow through a small tube into the brain). The other way, shown in the bottom part of the figure, is to inject the drugs directly into the CSF in the lower part of the spinal column, after a small area on the lower back is numbed. For more information, see Drugs Approved for Acute Myeloid Leukemia. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Total-body irradiation sends radiation toward the whole body. It is a type of external radiation that may be used to prepare the body for a stem cell transplant when the leukemia has recurred. Chemotherapy is given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy and/or total-body irradiation, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. There are different types of targeted therapy. Monoclonal antibodies: immune system proteins made in the laboratory to treat many diseases, including cancer. As a cancer treatment, these antibodies can attach to a specific target on cancer cells or other cells that may help cancer cells grow. The antibodies are able to then kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. Other targeted therapies include: Less-intensive targeted therapies in older or frail patients who cannot receive other treatments include: For more information, see Drugs Approved for Acute Myeloid Leukemia. Arsenic trioxide and all-trans retinoic acid (ATRA) are anticancer drugs that kill leukemia cells, stop the leukemia cells from dividing, or help the leukemia cells mature into white blood cells. These drugs are used in the treatment of a subtype of AML called acute promyelocytic leukemia. For more information, see Drugs Approved for Acute Myeloid Leukemia. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Standard treatment of untreated acute myeloid leukemia (AML) during the remission induction phase depends on the subtype of AML and may include the following: For older adults or patients too frail to receive intensive chemotherapy, the following may be continued as long as the patient benefits or until toxic effects occur: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of acute myeloid leukemia (AML) during the remission phase depends on the subtype of AML and may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. There is no standard treatment for refractory or recurrent acute myeloid leukemia (AML). Treatment depends on the subtype of AML and may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed acute promyelocytic leukemia (APL) includes: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent acute promyelocytic leukemia includes: For more information from the National Cancer Institute about adult acute myeloid leukemia, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of adult acute myeloid leukemia. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Acute Myeloid Leukemia Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/leukemia/patient/adult-aml-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389377] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Acute Myeloid Leukemia Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is the outlook for Adult Acute Myeloid Leukemia ?

Certain factors affect prognosis (chance of recovery) and treatment options. The prognosis (chance of recovery) and treatment options depend on: - The age of the patient. - The subtype of AML. - Whether the patient received chemotherapy in the past to treat a different cancer. - Whether there is a history of a blood disorder such as myelodysplastic syndrome. - Whether the cancer has spread to the central nervous system. - Whether the cancer has been treated before or recurred (come back). It is important that acute leukemia be treated right away.

Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. It is the most common type of acute leukemia in adults. This type of cancer usually gets worse quickly if it is not treated. AML is also called acute myelogenous leukemia and acute nonlymphocytic leukemia. Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In AML, the myeloid stem cells usually become a type of immature white blood cell called myeloblasts (or myeloid blasts). The myeloblasts in AML are abnormal and do not become healthy white blood cells. Sometimes in AML, too many stem cells become abnormal red blood cells or platelets. These abnormal white blood cells, red blood cells, or platelets are also called leukemia cells or blasts. Leukemia cells can build up in the bone marrow and blood so there is less room for healthy white blood cells, red blood cells, and platelets. When this happens, infection, anemia, or easy bleeding may occur. The leukemia cells can spread outside the blood to other parts of the body, including the central nervous system (brain and spinal cord), skin, and gums. Sometimes leukemia cells form a solid tumor called a myeloid sarcoma. Myeloid sarcoma is also called extramedullary myeloid tumor, granulocytic sarcoma, or chloroma. This summary is about adult AML. For more information about leukemia, see the following: Most AML subtypes are based on how mature (developed) the cancer cells are at the time of diagnosis and how different they are from normal cells. Acute promyelocytic leukemia (APL) is a subtype of AML. This leukemia occurs when genes on chromosome 15 switch places with some genes on chromosome 17 and an abnormal gene called PML-RARA is made. The PML-RARA gene sends a message that stops promyelocytes (a type of white blood cell) from maturing. Problems with severe bleeding and blood clots may occur. This is a serious health problem that needs treatment as soon as possible. APL usually occurs in middle-aged adults. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Possible risk factors for AML include the following: The early signs and symptoms of AML may be like those caused by the flu or other common diseases. Check with your doctor if you have any of the following: Less common signs or symptoms may be caused by clusters of leukemia cells in the central nervous system (CNS) or testicles, or a tumor of myeloid cells called a chloroma. Symptoms of acute leukemia often develop between 4 and 6 weeks before diagnosis. The following tests and procedures may be used: The prognosis and treatment options depend on: It is important that acute leukemia be treated right away. The extent or spread of cancer is usually described as stages. In acute myeloid leukemia (AML), the subtype of AML and whether the leukemia has spread outside the blood and bone marrow are used instead of the stage to plan treatment. The following tests and procedures may be used to determine if the leukemia has spread: The disease is described as untreated, in remission, refractory, or recurrent. Newly diagnosed (untreated) AML In untreated AML, the disease is newly diagnosed. It has not been treated except to relieve signs and symptoms such as fever, bleeding, or pain, and the following are true: AML in remission In AML in remission, the disease has been treated and the following are true: Refractory or recurrent AML After treatment with chemotherapy, some patients with newly diagnosed AML will not go into remission. This is called refractory cancer. In contrast, recurrent AML is cancer that has recurred (come back) after remission. The AML may come back in the blood or bone marrow. Different types of treatment are available for patients with acute myeloid leukemia (AML). Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. The two treatment phases of AML are: Patients must be closely monitored during treatment of AML. Myelosuppression, a condition which results in fewer red blood cells, white blood cells, and platelets, is a side effect of both AML and treatment with chemotherapy. Supportive care during remission induction therapy may include: Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Intrathecal chemotherapy may be used to treat adult AML that has spread to the brain and spinal cord. Combination chemotherapy is treatment using more than one anticancer drug. The way the chemotherapy is given depends on the subtype of AML being treated and whether leukemia cells have spread to the brain and spinal cord.EnlargeIntrathecal chemotherapy. Anticancer drugs are injected into the intrathecal space, which is the space that holds the cerebrospinal fluid (CSF, shown in blue). There are two different ways to do this. One way, shown in the top part of the figure, is to inject the drugs into an Ommaya reservoir (a dome-shaped container that is placed under the scalp during surgery; it holds the drugs as they flow through a small tube into the brain). The other way, shown in the bottom part of the figure, is to inject the drugs directly into the CSF in the lower part of the spinal column, after a small area on the lower back is numbed. For more information, see Drugs Approved for Acute Myeloid Leukemia. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Total-body irradiation sends radiation toward the whole body. It is a type of external radiation that may be used to prepare the body for a stem cell transplant when the leukemia has recurred. Chemotherapy is given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy and/or total-body irradiation, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. There are different types of targeted therapy. Monoclonal antibodies: immune system proteins made in the laboratory to treat many diseases, including cancer. As a cancer treatment, these antibodies can attach to a specific target on cancer cells or other cells that may help cancer cells grow. The antibodies are able to then kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. Other targeted therapies include: Less-intensive targeted therapies in older or frail patients who cannot receive other treatments include: For more information, see Drugs Approved for Acute Myeloid Leukemia. Arsenic trioxide and all-trans retinoic acid (ATRA) are anticancer drugs that kill leukemia cells, stop the leukemia cells from dividing, or help the leukemia cells mature into white blood cells. These drugs are used in the treatment of a subtype of AML called acute promyelocytic leukemia. For more information, see Drugs Approved for Acute Myeloid Leukemia. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Standard treatment of untreated acute myeloid leukemia (AML) during the remission induction phase depends on the subtype of AML and may include the following: For older adults or patients too frail to receive intensive chemotherapy, the following may be continued as long as the patient benefits or until toxic effects occur: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of acute myeloid leukemia (AML) during the remission phase depends on the subtype of AML and may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. There is no standard treatment for refractory or recurrent acute myeloid leukemia (AML). Treatment depends on the subtype of AML and may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed acute promyelocytic leukemia (APL) includes: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent acute promyelocytic leukemia includes: For more information from the National Cancer Institute about adult acute myeloid leukemia, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of adult acute myeloid leukemia. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Acute Myeloid Leukemia Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/leukemia/patient/adult-aml-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389377] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Acute Myeloid Leukemia Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the stages of Adult Acute Myeloid Leukemia ?

Key Points - Once adult acute myeloid leukemia (AML) has been diagnosed, tests are done to find out if the cancer has spread to other parts of the body. - There is no standard staging system for adult AML. Once adult acute myeloid leukemia (AML) has been diagnosed, tests are done to find out if the cancer has spread to other parts of the body. The extent or spread of cancer is usually described as stages. In adult acute myeloid leukemia (AML), the subtype of AML and whether the leukemia has spread outside the blood and bone marrow are used instead of the stage to plan treatment. The following tests and procedures may be used to determine if the leukemia has spread: - Lumbar puncture : A procedure used to collect a sample of cerebrospinal fluid (CSF) from the spinal column. This is done by placing a needle between two bones in the spine and into the CSF around the spinal cord and removing a sample of the fluid. The sample of CSF is checked under a microscope for signs that leukemia cells have spread to the brain and spinal cord. This procedure is also called an LP or spinal tap. - CT scan (CAT scan): A procedure that makes a series of detailed pictures of the abdomen, taken from different angles. The pictures are made by a computer linked to an x-ray machine. A dye may be injected into a vein or swallowed to help the organs or tissues show up more clearly. This procedure is also called computed tomography, computerized tomography, or computerized axial tomography. There is no standard staging system for adult AML. The disease is described as untreated, in remission, or recurrent. Untreated adult AML In untreated adult AML, the disease is newly diagnosed. It has not been treated except to relieve signs and symptoms such as fever, bleeding, or pain, and the following are true: - The complete blood count is abnormal. - At least 20% of the cells in the bone marrow are blasts (leukemia cells). - There are signs or symptoms of leukemia. Adult AML in remission In adult AML in remission, the disease has been treated and the following are true: - The complete blood count is normal. - Less than 5% of the cells in the bone marrow are blasts (leukemia cells). - There are no signs or symptoms of leukemia in the brain and spinal cord or elsewhere in the body. Recurrent Adult AML Recurrent AML is cancer that has recurred (come back) after it has been treated. The AML may come back in the blood or bone marrow.

Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. It is the most common type of acute leukemia in adults. This type of cancer usually gets worse quickly if it is not treated. AML is also called acute myelogenous leukemia and acute nonlymphocytic leukemia. Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In AML, the myeloid stem cells usually become a type of immature white blood cell called myeloblasts (or myeloid blasts). The myeloblasts in AML are abnormal and do not become healthy white blood cells. Sometimes in AML, too many stem cells become abnormal red blood cells or platelets. These abnormal white blood cells, red blood cells, or platelets are also called leukemia cells or blasts. Leukemia cells can build up in the bone marrow and blood so there is less room for healthy white blood cells, red blood cells, and platelets. When this happens, infection, anemia, or easy bleeding may occur. The leukemia cells can spread outside the blood to other parts of the body, including the central nervous system (brain and spinal cord), skin, and gums. Sometimes leukemia cells form a solid tumor called a myeloid sarcoma. Myeloid sarcoma is also called extramedullary myeloid tumor, granulocytic sarcoma, or chloroma. This summary is about adult AML. For more information about leukemia, see the following: Most AML subtypes are based on how mature (developed) the cancer cells are at the time of diagnosis and how different they are from normal cells. Acute promyelocytic leukemia (APL) is a subtype of AML. This leukemia occurs when genes on chromosome 15 switch places with some genes on chromosome 17 and an abnormal gene called PML-RARA is made. The PML-RARA gene sends a message that stops promyelocytes (a type of white blood cell) from maturing. Problems with severe bleeding and blood clots may occur. This is a serious health problem that needs treatment as soon as possible. APL usually occurs in middle-aged adults. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Possible risk factors for AML include the following: The early signs and symptoms of AML may be like those caused by the flu or other common diseases. Check with your doctor if you have any of the following: Less common signs or symptoms may be caused by clusters of leukemia cells in the central nervous system (CNS) or testicles, or a tumor of myeloid cells called a chloroma. Symptoms of acute leukemia often develop between 4 and 6 weeks before diagnosis. The following tests and procedures may be used: The prognosis and treatment options depend on: It is important that acute leukemia be treated right away. The extent or spread of cancer is usually described as stages. In acute myeloid leukemia (AML), the subtype of AML and whether the leukemia has spread outside the blood and bone marrow are used instead of the stage to plan treatment. The following tests and procedures may be used to determine if the leukemia has spread: The disease is described as untreated, in remission, refractory, or recurrent. Newly diagnosed (untreated) AML In untreated AML, the disease is newly diagnosed. It has not been treated except to relieve signs and symptoms such as fever, bleeding, or pain, and the following are true: AML in remission In AML in remission, the disease has been treated and the following are true: Refractory or recurrent AML After treatment with chemotherapy, some patients with newly diagnosed AML will not go into remission. This is called refractory cancer. In contrast, recurrent AML is cancer that has recurred (come back) after remission. The AML may come back in the blood or bone marrow. Different types of treatment are available for patients with acute myeloid leukemia (AML). Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. The two treatment phases of AML are: Patients must be closely monitored during treatment of AML. Myelosuppression, a condition which results in fewer red blood cells, white blood cells, and platelets, is a side effect of both AML and treatment with chemotherapy. Supportive care during remission induction therapy may include: Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Intrathecal chemotherapy may be used to treat adult AML that has spread to the brain and spinal cord. Combination chemotherapy is treatment using more than one anticancer drug. The way the chemotherapy is given depends on the subtype of AML being treated and whether leukemia cells have spread to the brain and spinal cord.EnlargeIntrathecal chemotherapy. Anticancer drugs are injected into the intrathecal space, which is the space that holds the cerebrospinal fluid (CSF, shown in blue). There are two different ways to do this. One way, shown in the top part of the figure, is to inject the drugs into an Ommaya reservoir (a dome-shaped container that is placed under the scalp during surgery; it holds the drugs as they flow through a small tube into the brain). The other way, shown in the bottom part of the figure, is to inject the drugs directly into the CSF in the lower part of the spinal column, after a small area on the lower back is numbed. For more information, see Drugs Approved for Acute Myeloid Leukemia. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Total-body irradiation sends radiation toward the whole body. It is a type of external radiation that may be used to prepare the body for a stem cell transplant when the leukemia has recurred. Chemotherapy is given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy and/or total-body irradiation, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. There are different types of targeted therapy. Monoclonal antibodies: immune system proteins made in the laboratory to treat many diseases, including cancer. As a cancer treatment, these antibodies can attach to a specific target on cancer cells or other cells that may help cancer cells grow. The antibodies are able to then kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. Other targeted therapies include: Less-intensive targeted therapies in older or frail patients who cannot receive other treatments include: For more information, see Drugs Approved for Acute Myeloid Leukemia. Arsenic trioxide and all-trans retinoic acid (ATRA) are anticancer drugs that kill leukemia cells, stop the leukemia cells from dividing, or help the leukemia cells mature into white blood cells. These drugs are used in the treatment of a subtype of AML called acute promyelocytic leukemia. For more information, see Drugs Approved for Acute Myeloid Leukemia. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Standard treatment of untreated acute myeloid leukemia (AML) during the remission induction phase depends on the subtype of AML and may include the following: For older adults or patients too frail to receive intensive chemotherapy, the following may be continued as long as the patient benefits or until toxic effects occur: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of acute myeloid leukemia (AML) during the remission phase depends on the subtype of AML and may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. There is no standard treatment for refractory or recurrent acute myeloid leukemia (AML). Treatment depends on the subtype of AML and may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed acute promyelocytic leukemia (APL) includes: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent acute promyelocytic leukemia includes: For more information from the National Cancer Institute about adult acute myeloid leukemia, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of adult acute myeloid leukemia. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Acute Myeloid Leukemia Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/leukemia/patient/adult-aml-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389377] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. 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In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Acute Myeloid Leukemia Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the treatments for Adult Acute Myeloid Leukemia ?

Key Points - There are different types of treatment for patients with adult acute myeloid leukemia. - The treatment of adult AML usually has 2 phases. - Four types of standard treatment are used: - Chemotherapy - Radiation therapy - Stem cell transplant - Other drug therapy - New types of treatment are being tested in clinical trials. - Targeted therapy - Patients may want to think about taking part in a clinical trial. - Patients can enter clinical trials before, during, or after starting their cancer treatment. - Follow-up tests may be needed. There are different types of treatment for patients with adult acute myeloid leukemia. Different types of treatment are available for patients with adult acute myeloid leukemia (AML). Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. The treatment of adult AML usually has 2 phases. The 2 treatment phases of adult AML are: - Remission induction therapy: This is the first phase of treatment. The goal is to kill the leukemia cells in the blood and bone marrow. This puts the leukemia into remission. - Post-remission therapy: This is the second phase of treatment. It begins after the leukemia is in remission. The goal of post-remission therapy is to kill any remaining leukemia cells that may not be active but could begin to regrow and cause a relapse. This phase is also called remission continuation therapy. Four types of standard treatment are used: Chemotherapy Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Intrathecal chemotherapy may be used to treat adult AML that has spread to the brain and spinal cord. Combination chemotherapy is treatment using more than one anticancer drug. The way the chemotherapy is given depends on the subtype of AML being treated and whether leukemia cells have spread to the brain and spinal cord. See Drugs Approved for Acute Myeloid Leukemia for more information. Radiation therapy Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: - External radiation therapy uses a machine outside the body to send radiation toward the cancer. - Internal radiation therapy uses a radioactive substance sealed in needles, seeds, wires, or catheters that are placed directly into or near the cancer. The way the radiation therapy is given depends on the type of cancer being treated and whether leukemia cells have spread to the brain and spinal cord. External radiation therapy is used to treat adult AML. Stem cell transplant Stem cell transplant is a method of giving chemotherapy and replacing blood -forming cells that are abnormal or destroyed by the cancer treatment. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the chemotherapy is completed, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body&apos;s blood cells. Other drug therapy Arsenic trioxide and all-trans retinoic acid (ATRA) are anticancer drugs that kill leukemia cells, stop the leukemia cells from dividing, or help the leukemia cells mature into white blood cells. These drugs are used in the treatment of a subtype of AML called acute promyelocytic leukemia. See Drugs Approved for Acute Myeloid Leukemia for more information. New types of treatment are being tested in clinical trials. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Targeted therapy Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells without harming normal cells. Monoclonal antibody therapy is one type of targeted therapy being studied in the treatment of adult AML. Monoclonal antibody therapy is a cancer treatment that uses antibodies made in the laboratory from a single type of immune system cell. These antibodies can identify substances on cancer cells or normal substances that may help cancer cells grow. The antibodies attach to the substances and kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials. Follow-up tests may be needed. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. Treatment Options for Adult Acute Myeloid Leukemia Untreated Adult Acute Myeloid Leukemia Standard treatment of untreated adult acute myeloid leukemia (AML) during the remission induction phase depends on the subtype of AML and may include the following: - Combination chemotherapy. - High-dose combination chemotherapy. - Low-dose chemotherapy. - Intrathecal chemotherapy. - All-trans retinoic acid (ATRA) plus arsenic trioxide for the treatment of acute promyelocytic leukemia (APL). - ATRA plus combination chemotherapy followed by arsenic trioxide for the treatment of APL. Check the list of NCI-supported cancer clinical trials that are now accepting patients with untreated adult acute myeloid leukemia. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Adult Acute Myeloid Leukemia in Remission Treatment of adult AML during the remission phase depends on the subtype of AML and may include the following: - Combination chemotherapy. - High-dose chemotherapy, with or without radiation therapy, and stem cell transplant using the patient&apos;s stem cells. - High-dose chemotherapy and stem cell transplant using donor stem cells. - A clinical trial of arsenic trioxide. Check the list of NCI-supported cancer clinical trials that are now accepting patients with adult acute myeloid leukemia in remission. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website. Recurrent Adult Acute Myeloid Leukemia There is no standard treatment for recurrent adult AML. Treatment depends on the subtype of AML and may include the following: - Combination chemotherapy. - Targeted therapy with monoclonal antibodies. - Stem cell transplant. - Arsenic trioxide therapy. - A clinical trial of arsenic trioxide therapy followed by stem cell transplant. Check the list of NCI-supported cancer clinical trials that are now accepting patients with recurrent adult acute myeloid leukemia. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI website.

Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. It is the most common type of acute leukemia in adults. This type of cancer usually gets worse quickly if it is not treated. AML is also called acute myelogenous leukemia and acute nonlymphocytic leukemia. Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In AML, the myeloid stem cells usually become a type of immature white blood cell called myeloblasts (or myeloid blasts). The myeloblasts in AML are abnormal and do not become healthy white blood cells. Sometimes in AML, too many stem cells become abnormal red blood cells or platelets. These abnormal white blood cells, red blood cells, or platelets are also called leukemia cells or blasts. Leukemia cells can build up in the bone marrow and blood so there is less room for healthy white blood cells, red blood cells, and platelets. When this happens, infection, anemia, or easy bleeding may occur. The leukemia cells can spread outside the blood to other parts of the body, including the central nervous system (brain and spinal cord), skin, and gums. Sometimes leukemia cells form a solid tumor called a myeloid sarcoma. Myeloid sarcoma is also called extramedullary myeloid tumor, granulocytic sarcoma, or chloroma. This summary is about adult AML. For more information about leukemia, see the following: Most AML subtypes are based on how mature (developed) the cancer cells are at the time of diagnosis and how different they are from normal cells. Acute promyelocytic leukemia (APL) is a subtype of AML. This leukemia occurs when genes on chromosome 15 switch places with some genes on chromosome 17 and an abnormal gene called PML-RARA is made. The PML-RARA gene sends a message that stops promyelocytes (a type of white blood cell) from maturing. Problems with severe bleeding and blood clots may occur. This is a serious health problem that needs treatment as soon as possible. APL usually occurs in middle-aged adults. Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Possible risk factors for AML include the following: The early signs and symptoms of AML may be like those caused by the flu or other common diseases. Check with your doctor if you have any of the following: Less common signs or symptoms may be caused by clusters of leukemia cells in the central nervous system (CNS) or testicles, or a tumor of myeloid cells called a chloroma. Symptoms of acute leukemia often develop between 4 and 6 weeks before diagnosis. The following tests and procedures may be used: The prognosis and treatment options depend on: It is important that acute leukemia be treated right away. The extent or spread of cancer is usually described as stages. In acute myeloid leukemia (AML), the subtype of AML and whether the leukemia has spread outside the blood and bone marrow are used instead of the stage to plan treatment. The following tests and procedures may be used to determine if the leukemia has spread: The disease is described as untreated, in remission, refractory, or recurrent. Newly diagnosed (untreated) AML In untreated AML, the disease is newly diagnosed. It has not been treated except to relieve signs and symptoms such as fever, bleeding, or pain, and the following are true: AML in remission In AML in remission, the disease has been treated and the following are true: Refractory or recurrent AML After treatment with chemotherapy, some patients with newly diagnosed AML will not go into remission. This is called refractory cancer. In contrast, recurrent AML is cancer that has recurred (come back) after remission. The AML may come back in the blood or bone marrow. Different types of treatment are available for patients with acute myeloid leukemia (AML). Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. The two treatment phases of AML are: Patients must be closely monitored during treatment of AML. Myelosuppression, a condition which results in fewer red blood cells, white blood cells, and platelets, is a side effect of both AML and treatment with chemotherapy. Supportive care during remission induction therapy may include: Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Intrathecal chemotherapy may be used to treat adult AML that has spread to the brain and spinal cord. Combination chemotherapy is treatment using more than one anticancer drug. The way the chemotherapy is given depends on the subtype of AML being treated and whether leukemia cells have spread to the brain and spinal cord.EnlargeIntrathecal chemotherapy. Anticancer drugs are injected into the intrathecal space, which is the space that holds the cerebrospinal fluid (CSF, shown in blue). There are two different ways to do this. One way, shown in the top part of the figure, is to inject the drugs into an Ommaya reservoir (a dome-shaped container that is placed under the scalp during surgery; it holds the drugs as they flow through a small tube into the brain). The other way, shown in the bottom part of the figure, is to inject the drugs directly into the CSF in the lower part of the spinal column, after a small area on the lower back is numbed. For more information, see Drugs Approved for Acute Myeloid Leukemia. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Total-body irradiation sends radiation toward the whole body. It is a type of external radiation that may be used to prepare the body for a stem cell transplant when the leukemia has recurred. Chemotherapy is given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy and/or total-body irradiation, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. There are different types of targeted therapy. Monoclonal antibodies: immune system proteins made in the laboratory to treat many diseases, including cancer. As a cancer treatment, these antibodies can attach to a specific target on cancer cells or other cells that may help cancer cells grow. The antibodies are able to then kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. Other targeted therapies include: Less-intensive targeted therapies in older or frail patients who cannot receive other treatments include: For more information, see Drugs Approved for Acute Myeloid Leukemia. Arsenic trioxide and all-trans retinoic acid (ATRA) are anticancer drugs that kill leukemia cells, stop the leukemia cells from dividing, or help the leukemia cells mature into white blood cells. These drugs are used in the treatment of a subtype of AML called acute promyelocytic leukemia. For more information, see Drugs Approved for Acute Myeloid Leukemia. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. For information about the treatments listed below, see the Treatment Option Overview section. Standard treatment of untreated acute myeloid leukemia (AML) during the remission induction phase depends on the subtype of AML and may include the following: For older adults or patients too frail to receive intensive chemotherapy, the following may be continued as long as the patient benefits or until toxic effects occur: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of acute myeloid leukemia (AML) during the remission phase depends on the subtype of AML and may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. There is no standard treatment for refractory or recurrent acute myeloid leukemia (AML). Treatment depends on the subtype of AML and may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of newly diagnosed acute promyelocytic leukemia (APL) includes: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of recurrent acute promyelocytic leukemia includes: For more information from the National Cancer Institute about adult acute myeloid leukemia, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of adult acute myeloid leukemia. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Acute Myeloid Leukemia Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/leukemia/patient/adult-aml-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389377] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Acute Myeloid Leukemia Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

what research (or clinical trials) is being done for Adult Acute Myeloid Leukemia ?

New types of treatment are being tested in clinical trials. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Targeted therapy Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells without harming normal cells. Monoclonal antibody therapy is one type of targeted therapy being studied in the treatment of adult AML. Monoclonal antibody therapy is a cancer treatment that uses antibodies made in the laboratory from a single type of immune system cell. These antibodies can identify substances on cancer cells or normal substances that may help cancer cells grow. The antibodies attach to the substances and kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials.

Gliomas are tumors formed from glial cells. Glial cells in the brain hold nerve cells in place, bring food and oxygen to nerve cells, and help protect nerve cells from disease, such as infection. In brain stem glioma, the glial cells in the brain stem are affected. The brain stem is made up of the midbrain, pons, and medulla. It is the lowest part of the brain and connects to the spinal cord, just above the back of the neck. The brain stem controls vital functions such as breathing, heart rate, and blood pressure, and the nerves and muscles used in seeing, hearing, walking, talking, and eating. Most childhood brain stem gliomas are diffuse intrinsic pontine gliomas (DIPG), which form in the pons. Focal gliomas form in other parts of the brain stem. Brain tumors are the second most common type of cancer in children. This summary is about the treatment of primary brain tumors (tumors that begin in the brain). Treatment for metastatic brain tumors, which are tumors formed by cancer cells that begin in other parts of the body and spread to the brain, is not discussed in this summary. Brain tumors can occur in both children and adults; however, treatment for children may be different than treatment for adults. For information on treatment of brain tumors in adults, see the PDQ summary Adult Central Nervous System Tumors Treatment. Even though DIPG and focal brain stem glioma form in the same type of cell, they act differently: Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your child's doctor if you think your child may be at risk. Possible risk factors for brain stem glioma include: Signs and symptoms depend on the following: Signs and symptoms may be caused by childhood brain stem gliomas or by other conditions. Check with your child's doctor if your child has any of the following: The following tests and procedures may be used: If the MRI scan looks like the tumor is a DIPG, a biopsy is usually not done and the tumor is not removed. When the MRI scan results are uncertain, a biopsy may be done. If the MRI scan looks like a focal brain stem glioma, a biopsy may be done. A part of the skull is removed and a needle is used to remove a sample of the brain tissue. Sometimes, the needle is guided by a computer. A pathologist views the tissue under a microscope to look for cancer cells. If cancer cells are found, the doctor will remove as much tumor as safely possible during the same surgery. The following test may be done on the tissue sample that was removed during biopsy or surgery: The child's prognosis depends on the following: Most children with DIPG live less than 18 months after diagnosis. Children with a focal glioma usually live longer than 5 years. Staging is the process used to find out how much cancer there is and if cancer has spread. It is important to know the stage in order to plan treatment. There is no standard staging system for childhood brain stem glioma. Treatment is based on the following: A focal brain stem glioma may recur many years after first being treated. The tumor may come back in the brain or in other parts of the central nervous system. Before cancer treatment is given, imaging tests, a biopsy, or surgery may be done to make sure there is cancer and find out how much cancer there is. Different types of treatment are available for children with brain stem glioma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating children with brain tumors and who specialize in certain areas of medicine. These may include the following specialists: Biopsy or surgery to remove DIPG is not usually done because of the following: A biopsy to diagnose or surgery to remove the tumor may be used for childhood focal brain stem glioma. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type of the cancer being treated. External radiation therapy is used to treat DIPG. External and/or internal radiation therapy may be used to treat focal brain stem gliomas. Several months after radiation therapy to the brain, imaging tests may show changes to the brain tissue. These changes may be caused by the radiation therapy or may mean the tumor is growing. It is important to be sure the tumor is growing before any more treatment is given. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Because radiation therapy to the brain can affect growth and brain development in young children, chemotherapy may be given to delay or reduce the need for radiation therapy. Cerebrospinal fluid diversion is a method used to drain fluid that has built up in the brain. A shunt (long, thin tube) is placed in a ventricle (fluid-filled space) of the brain and threaded under the skin to another part of the body, usually the abdomen. The shunt carries extra fluid away from the brain so it may be absorbed elsewhere in the body.EnlargeCerebrospinal fluid (CSF) diversion. Extra CSF is removed from a ventricle in the brain through a shunt (tube) and is emptied into the abdomen. A valve controls the flow of CSF. Observation is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. There are different types of targeted therapy being studied in the treatment of brain stem gliomas: A monoclonal antibody, APX005M, binds to CD40, a cell surface receptor found on certain immune cells and some cancer cells. It may fight cancer by boosting the immune system and by slowing cancer cell growth. It is being studied in the treatment of pediatric brain tumors that are growing, spreading, or getting worse (progressive), or in newly diagnosed DIPG. For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information). Information about clinical trials is available from the NCI website. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. If the results of imaging tests done after treatment for DIPG show a mass in the brain, a biopsy may be done to find out if it is made up of dead tumor cells or if new cancer cells are growing. In children who are expected to live a long time, regular MRIs may be done to see if the cancer has come back. For information about the treatments listed below, see the Treatment Option Overview section. Newly diagnosed childhood diffuse intrinsic brain stem glioma (DIPG) is a tumor for which no treatment has been given. The child may have received drugs or treatment to relieve signs or symptoms caused by the tumor. Standard treatment of DIPG may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Newly diagnosed childhood focal glioma is a tumor for which no treatment has been given. The child may have received drugs or treatment to relieve signs or symptoms caused by the tumor. Treatment of focal glioma may include the following: Treatment of brain stem glioma in children with neurofibromatosis type 1 may be observation. The tumors are slow-growing in these children and may not need specific treatment for years. When cancer does not get better with treatment or comes back, palliative care is an important part of the child's treatment plan. It includes physical, psychological, social, and spiritual support for the child and family. The goal of palliative care is to help control symptoms and give the child the best quality of life possible. Parents may not be sure about whether to continue treatment or what kind of treatment is best for their child. The healthcare team can give parents information to help them make these decisions. For information about the treatments listed below, see the Treatment Option Overview section. More radiation therapy may be given to children with progressive or recurrent diffuse intrinsic pontine glioma (DIPG) who responded when first treated with radiation therapy. Treatment of progressive or recurrent DIPG may also include the following: Treatment of recurrent focal childhood brain stem glioma may include the following: For more information about childhood brain tumors, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood brain stem glioma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Brain Stem Glioma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/brain/patient/child-glioma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389295] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Childhood Brain Stem Glioma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is (are) Childhood Brain Stem Glioma ?

Key Points - Childhood brain stem glioma is a disease in which benign (noncancer) or malignant (cancer) cells form in the tissues of the brain stem. - Brain tumors may be benign (not cancer) or malignant (cancer). - There are two types of brain stem gliomas in children. - The cause of most childhood brain tumors is unknown. - The signs and symptoms of brain stem glioma are not the same in every child. - Tests that examine the brain are used to detect (find) childhood brain stem glioma. - A biopsy may be done to diagnose certain types of brain stem glioma. - Certain factors affect prognosis (chance of recovery) and treatment options. Childhood brain stem glioma is a disease in which benign (noncancer) or malignant (cancer) cells form in the tissues of the brain stem. Gliomas are tumors formed from glial cells. Glial cells in the brain hold nerve cells in place, bring food and oxygen to them, and help protect them from disease, such as infection. The brain stem is the part of the brain connected to the spinal cord. It is in the lowest part of the brain, just above the back of the neck. The brain stem is the part of the brain that controls breathing, heart rate, and the nerves and muscles used in seeing, hearing, walking, talking, and eating. Most childhood brain stem gliomas are pontine gliomas, which form in a part of the brain stem called the pons. Brain tumors are the third most common type of cancer in children. This summary refers to the treatment of primary brain tumors (tumors that begin in the brain). Treatment for metastatic brain tumors, which are tumors formed by cancer cells that begin in other parts of the body and spread to the brain, is not discussed in this summary. Brain tumors can occur in both children and adults; however, treatment for children may be different than treatment for adults. See the following PDQ treatment summaries for more information: - Childhood Brain and Spinal Cord Tumors Treatment Overview - Adult Central Nervous System Tumors Treatment Brain tumors may be benign (not cancer) or malignant (cancer). Benign brain tumors grow and press on nearby areas of the brain. They rarely spread into other tissues. Malignant brain tumors are likely to grow quickly and spread into other brain tissue. When a tumor grows into or presses on an area of the brain, it may stop that part of the brain from working the way it should. Both benign and malignant brain tumors can cause signs and symptoms and need treatment. There are two types of brain stem gliomas in children. Childhood brain stem glioma is either a diffuse intrinsic pontine glioma (DIPG) or a focal glioma. - DIPG is a high-grade tumor that is fast-growing and spreads all through the brain stem. It is hard to treat and has a poor prognosis (chance of recovery). Children younger than 3 years diagnosed with DIPG may have a better prognosis than children who are 3 years and older. - A focal glioma is slow-growing and is in one area of the brain stem. It is easier to treat than DIPG and has a better prognosis.

Gliomas are tumors formed from glial cells. Glial cells in the brain hold nerve cells in place, bring food and oxygen to nerve cells, and help protect nerve cells from disease, such as infection. In brain stem glioma, the glial cells in the brain stem are affected. The brain stem is made up of the midbrain, pons, and medulla. It is the lowest part of the brain and connects to the spinal cord, just above the back of the neck. The brain stem controls vital functions such as breathing, heart rate, and blood pressure, and the nerves and muscles used in seeing, hearing, walking, talking, and eating. Most childhood brain stem gliomas are diffuse intrinsic pontine gliomas (DIPG), which form in the pons. Focal gliomas form in other parts of the brain stem. Brain tumors are the second most common type of cancer in children. This summary is about the treatment of primary brain tumors (tumors that begin in the brain). Treatment for metastatic brain tumors, which are tumors formed by cancer cells that begin in other parts of the body and spread to the brain, is not discussed in this summary. Brain tumors can occur in both children and adults; however, treatment for children may be different than treatment for adults. For information on treatment of brain tumors in adults, see the PDQ summary Adult Central Nervous System Tumors Treatment. Even though DIPG and focal brain stem glioma form in the same type of cell, they act differently: Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your child's doctor if you think your child may be at risk. Possible risk factors for brain stem glioma include: Signs and symptoms depend on the following: Signs and symptoms may be caused by childhood brain stem gliomas or by other conditions. Check with your child's doctor if your child has any of the following: The following tests and procedures may be used: If the MRI scan looks like the tumor is a DIPG, a biopsy is usually not done and the tumor is not removed. When the MRI scan results are uncertain, a biopsy may be done. If the MRI scan looks like a focal brain stem glioma, a biopsy may be done. A part of the skull is removed and a needle is used to remove a sample of the brain tissue. Sometimes, the needle is guided by a computer. A pathologist views the tissue under a microscope to look for cancer cells. If cancer cells are found, the doctor will remove as much tumor as safely possible during the same surgery. The following test may be done on the tissue sample that was removed during biopsy or surgery: The child's prognosis depends on the following: Most children with DIPG live less than 18 months after diagnosis. Children with a focal glioma usually live longer than 5 years. Staging is the process used to find out how much cancer there is and if cancer has spread. It is important to know the stage in order to plan treatment. There is no standard staging system for childhood brain stem glioma. Treatment is based on the following: A focal brain stem glioma may recur many years after first being treated. The tumor may come back in the brain or in other parts of the central nervous system. Before cancer treatment is given, imaging tests, a biopsy, or surgery may be done to make sure there is cancer and find out how much cancer there is. Different types of treatment are available for children with brain stem glioma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating children with brain tumors and who specialize in certain areas of medicine. These may include the following specialists: Biopsy or surgery to remove DIPG is not usually done because of the following: A biopsy to diagnose or surgery to remove the tumor may be used for childhood focal brain stem glioma. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type of the cancer being treated. External radiation therapy is used to treat DIPG. External and/or internal radiation therapy may be used to treat focal brain stem gliomas. Several months after radiation therapy to the brain, imaging tests may show changes to the brain tissue. These changes may be caused by the radiation therapy or may mean the tumor is growing. It is important to be sure the tumor is growing before any more treatment is given. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Because radiation therapy to the brain can affect growth and brain development in young children, chemotherapy may be given to delay or reduce the need for radiation therapy. Cerebrospinal fluid diversion is a method used to drain fluid that has built up in the brain. A shunt (long, thin tube) is placed in a ventricle (fluid-filled space) of the brain and threaded under the skin to another part of the body, usually the abdomen. The shunt carries extra fluid away from the brain so it may be absorbed elsewhere in the body.EnlargeCerebrospinal fluid (CSF) diversion. Extra CSF is removed from a ventricle in the brain through a shunt (tube) and is emptied into the abdomen. A valve controls the flow of CSF. Observation is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. There are different types of targeted therapy being studied in the treatment of brain stem gliomas: A monoclonal antibody, APX005M, binds to CD40, a cell surface receptor found on certain immune cells and some cancer cells. It may fight cancer by boosting the immune system and by slowing cancer cell growth. It is being studied in the treatment of pediatric brain tumors that are growing, spreading, or getting worse (progressive), or in newly diagnosed DIPG. For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information). Information about clinical trials is available from the NCI website. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. If the results of imaging tests done after treatment for DIPG show a mass in the brain, a biopsy may be done to find out if it is made up of dead tumor cells or if new cancer cells are growing. In children who are expected to live a long time, regular MRIs may be done to see if the cancer has come back. For information about the treatments listed below, see the Treatment Option Overview section. Newly diagnosed childhood diffuse intrinsic brain stem glioma (DIPG) is a tumor for which no treatment has been given. The child may have received drugs or treatment to relieve signs or symptoms caused by the tumor. Standard treatment of DIPG may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Newly diagnosed childhood focal glioma is a tumor for which no treatment has been given. The child may have received drugs or treatment to relieve signs or symptoms caused by the tumor. Treatment of focal glioma may include the following: Treatment of brain stem glioma in children with neurofibromatosis type 1 may be observation. The tumors are slow-growing in these children and may not need specific treatment for years. When cancer does not get better with treatment or comes back, palliative care is an important part of the child's treatment plan. It includes physical, psychological, social, and spiritual support for the child and family. The goal of palliative care is to help control symptoms and give the child the best quality of life possible. Parents may not be sure about whether to continue treatment or what kind of treatment is best for their child. The healthcare team can give parents information to help them make these decisions. For information about the treatments listed below, see the Treatment Option Overview section. More radiation therapy may be given to children with progressive or recurrent diffuse intrinsic pontine glioma (DIPG) who responded when first treated with radiation therapy. Treatment of progressive or recurrent DIPG may also include the following: Treatment of recurrent focal childhood brain stem glioma may include the following: For more information about childhood brain tumors, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood brain stem glioma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Brain Stem Glioma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/brain/patient/child-glioma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389295] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Childhood Brain Stem Glioma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What causes Childhood Brain Stem Glioma ?

The cause of most childhood brain tumors is unknown.

Gliomas are tumors formed from glial cells. Glial cells in the brain hold nerve cells in place, bring food and oxygen to nerve cells, and help protect nerve cells from disease, such as infection. In brain stem glioma, the glial cells in the brain stem are affected. The brain stem is made up of the midbrain, pons, and medulla. It is the lowest part of the brain and connects to the spinal cord, just above the back of the neck. The brain stem controls vital functions such as breathing, heart rate, and blood pressure, and the nerves and muscles used in seeing, hearing, walking, talking, and eating. Most childhood brain stem gliomas are diffuse intrinsic pontine gliomas (DIPG), which form in the pons. Focal gliomas form in other parts of the brain stem. Brain tumors are the second most common type of cancer in children. This summary is about the treatment of primary brain tumors (tumors that begin in the brain). Treatment for metastatic brain tumors, which are tumors formed by cancer cells that begin in other parts of the body and spread to the brain, is not discussed in this summary. Brain tumors can occur in both children and adults; however, treatment for children may be different than treatment for adults. For information on treatment of brain tumors in adults, see the PDQ summary Adult Central Nervous System Tumors Treatment. Even though DIPG and focal brain stem glioma form in the same type of cell, they act differently: Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your child's doctor if you think your child may be at risk. Possible risk factors for brain stem glioma include: Signs and symptoms depend on the following: Signs and symptoms may be caused by childhood brain stem gliomas or by other conditions. Check with your child's doctor if your child has any of the following: The following tests and procedures may be used: If the MRI scan looks like the tumor is a DIPG, a biopsy is usually not done and the tumor is not removed. When the MRI scan results are uncertain, a biopsy may be done. If the MRI scan looks like a focal brain stem glioma, a biopsy may be done. A part of the skull is removed and a needle is used to remove a sample of the brain tissue. Sometimes, the needle is guided by a computer. A pathologist views the tissue under a microscope to look for cancer cells. If cancer cells are found, the doctor will remove as much tumor as safely possible during the same surgery. The following test may be done on the tissue sample that was removed during biopsy or surgery: The child's prognosis depends on the following: Most children with DIPG live less than 18 months after diagnosis. Children with a focal glioma usually live longer than 5 years. Staging is the process used to find out how much cancer there is and if cancer has spread. It is important to know the stage in order to plan treatment. There is no standard staging system for childhood brain stem glioma. Treatment is based on the following: A focal brain stem glioma may recur many years after first being treated. The tumor may come back in the brain or in other parts of the central nervous system. Before cancer treatment is given, imaging tests, a biopsy, or surgery may be done to make sure there is cancer and find out how much cancer there is. Different types of treatment are available for children with brain stem glioma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating children with brain tumors and who specialize in certain areas of medicine. These may include the following specialists: Biopsy or surgery to remove DIPG is not usually done because of the following: A biopsy to diagnose or surgery to remove the tumor may be used for childhood focal brain stem glioma. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type of the cancer being treated. External radiation therapy is used to treat DIPG. External and/or internal radiation therapy may be used to treat focal brain stem gliomas. Several months after radiation therapy to the brain, imaging tests may show changes to the brain tissue. These changes may be caused by the radiation therapy or may mean the tumor is growing. It is important to be sure the tumor is growing before any more treatment is given. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Because radiation therapy to the brain can affect growth and brain development in young children, chemotherapy may be given to delay or reduce the need for radiation therapy. Cerebrospinal fluid diversion is a method used to drain fluid that has built up in the brain. A shunt (long, thin tube) is placed in a ventricle (fluid-filled space) of the brain and threaded under the skin to another part of the body, usually the abdomen. The shunt carries extra fluid away from the brain so it may be absorbed elsewhere in the body.EnlargeCerebrospinal fluid (CSF) diversion. Extra CSF is removed from a ventricle in the brain through a shunt (tube) and is emptied into the abdomen. A valve controls the flow of CSF. Observation is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. There are different types of targeted therapy being studied in the treatment of brain stem gliomas: A monoclonal antibody, APX005M, binds to CD40, a cell surface receptor found on certain immune cells and some cancer cells. It may fight cancer by boosting the immune system and by slowing cancer cell growth. It is being studied in the treatment of pediatric brain tumors that are growing, spreading, or getting worse (progressive), or in newly diagnosed DIPG. For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information). Information about clinical trials is available from the NCI website. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. If the results of imaging tests done after treatment for DIPG show a mass in the brain, a biopsy may be done to find out if it is made up of dead tumor cells or if new cancer cells are growing. In children who are expected to live a long time, regular MRIs may be done to see if the cancer has come back. For information about the treatments listed below, see the Treatment Option Overview section. Newly diagnosed childhood diffuse intrinsic brain stem glioma (DIPG) is a tumor for which no treatment has been given. The child may have received drugs or treatment to relieve signs or symptoms caused by the tumor. Standard treatment of DIPG may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Newly diagnosed childhood focal glioma is a tumor for which no treatment has been given. The child may have received drugs or treatment to relieve signs or symptoms caused by the tumor. Treatment of focal glioma may include the following: Treatment of brain stem glioma in children with neurofibromatosis type 1 may be observation. The tumors are slow-growing in these children and may not need specific treatment for years. When cancer does not get better with treatment or comes back, palliative care is an important part of the child's treatment plan. It includes physical, psychological, social, and spiritual support for the child and family. The goal of palliative care is to help control symptoms and give the child the best quality of life possible. Parents may not be sure about whether to continue treatment or what kind of treatment is best for their child. The healthcare team can give parents information to help them make these decisions. For information about the treatments listed below, see the Treatment Option Overview section. More radiation therapy may be given to children with progressive or recurrent diffuse intrinsic pontine glioma (DIPG) who responded when first treated with radiation therapy. Treatment of progressive or recurrent DIPG may also include the following: Treatment of recurrent focal childhood brain stem glioma may include the following: For more information about childhood brain tumors, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood brain stem glioma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Brain Stem Glioma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/brain/patient/child-glioma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389295] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Childhood Brain Stem Glioma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

Who is at risk for Childhood Brain Stem Glioma? ?

Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesnt mean that you will not get cancer. Talk with your child&apos;s doctor if you think your child may be at risk. Possible risk factors for brain stem glioma include: - Having certain genetic disorders, such as neurofibromatosis type 1 (NF1).

Gliomas are tumors formed from glial cells. Glial cells in the brain hold nerve cells in place, bring food and oxygen to nerve cells, and help protect nerve cells from disease, such as infection. In brain stem glioma, the glial cells in the brain stem are affected. The brain stem is made up of the midbrain, pons, and medulla. It is the lowest part of the brain and connects to the spinal cord, just above the back of the neck. The brain stem controls vital functions such as breathing, heart rate, and blood pressure, and the nerves and muscles used in seeing, hearing, walking, talking, and eating. Most childhood brain stem gliomas are diffuse intrinsic pontine gliomas (DIPG), which form in the pons. Focal gliomas form in other parts of the brain stem. Brain tumors are the second most common type of cancer in children. This summary is about the treatment of primary brain tumors (tumors that begin in the brain). Treatment for metastatic brain tumors, which are tumors formed by cancer cells that begin in other parts of the body and spread to the brain, is not discussed in this summary. Brain tumors can occur in both children and adults; however, treatment for children may be different than treatment for adults. For information on treatment of brain tumors in adults, see the PDQ summary Adult Central Nervous System Tumors Treatment. Even though DIPG and focal brain stem glioma form in the same type of cell, they act differently: Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your child's doctor if you think your child may be at risk. Possible risk factors for brain stem glioma include: Signs and symptoms depend on the following: Signs and symptoms may be caused by childhood brain stem gliomas or by other conditions. Check with your child's doctor if your child has any of the following: The following tests and procedures may be used: If the MRI scan looks like the tumor is a DIPG, a biopsy is usually not done and the tumor is not removed. When the MRI scan results are uncertain, a biopsy may be done. If the MRI scan looks like a focal brain stem glioma, a biopsy may be done. A part of the skull is removed and a needle is used to remove a sample of the brain tissue. Sometimes, the needle is guided by a computer. A pathologist views the tissue under a microscope to look for cancer cells. If cancer cells are found, the doctor will remove as much tumor as safely possible during the same surgery. The following test may be done on the tissue sample that was removed during biopsy or surgery: The child's prognosis depends on the following: Most children with DIPG live less than 18 months after diagnosis. Children with a focal glioma usually live longer than 5 years. Staging is the process used to find out how much cancer there is and if cancer has spread. It is important to know the stage in order to plan treatment. There is no standard staging system for childhood brain stem glioma. Treatment is based on the following: A focal brain stem glioma may recur many years after first being treated. The tumor may come back in the brain or in other parts of the central nervous system. Before cancer treatment is given, imaging tests, a biopsy, or surgery may be done to make sure there is cancer and find out how much cancer there is. Different types of treatment are available for children with brain stem glioma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating children with brain tumors and who specialize in certain areas of medicine. These may include the following specialists: Biopsy or surgery to remove DIPG is not usually done because of the following: A biopsy to diagnose or surgery to remove the tumor may be used for childhood focal brain stem glioma. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type of the cancer being treated. External radiation therapy is used to treat DIPG. External and/or internal radiation therapy may be used to treat focal brain stem gliomas. Several months after radiation therapy to the brain, imaging tests may show changes to the brain tissue. These changes may be caused by the radiation therapy or may mean the tumor is growing. It is important to be sure the tumor is growing before any more treatment is given. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Because radiation therapy to the brain can affect growth and brain development in young children, chemotherapy may be given to delay or reduce the need for radiation therapy. Cerebrospinal fluid diversion is a method used to drain fluid that has built up in the brain. A shunt (long, thin tube) is placed in a ventricle (fluid-filled space) of the brain and threaded under the skin to another part of the body, usually the abdomen. The shunt carries extra fluid away from the brain so it may be absorbed elsewhere in the body.EnlargeCerebrospinal fluid (CSF) diversion. Extra CSF is removed from a ventricle in the brain through a shunt (tube) and is emptied into the abdomen. A valve controls the flow of CSF. Observation is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. There are different types of targeted therapy being studied in the treatment of brain stem gliomas: A monoclonal antibody, APX005M, binds to CD40, a cell surface receptor found on certain immune cells and some cancer cells. It may fight cancer by boosting the immune system and by slowing cancer cell growth. It is being studied in the treatment of pediatric brain tumors that are growing, spreading, or getting worse (progressive), or in newly diagnosed DIPG. For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information). Information about clinical trials is available from the NCI website. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. If the results of imaging tests done after treatment for DIPG show a mass in the brain, a biopsy may be done to find out if it is made up of dead tumor cells or if new cancer cells are growing. In children who are expected to live a long time, regular MRIs may be done to see if the cancer has come back. For information about the treatments listed below, see the Treatment Option Overview section. Newly diagnosed childhood diffuse intrinsic brain stem glioma (DIPG) is a tumor for which no treatment has been given. The child may have received drugs or treatment to relieve signs or symptoms caused by the tumor. Standard treatment of DIPG may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Newly diagnosed childhood focal glioma is a tumor for which no treatment has been given. The child may have received drugs or treatment to relieve signs or symptoms caused by the tumor. Treatment of focal glioma may include the following: Treatment of brain stem glioma in children with neurofibromatosis type 1 may be observation. The tumors are slow-growing in these children and may not need specific treatment for years. When cancer does not get better with treatment or comes back, palliative care is an important part of the child's treatment plan. It includes physical, psychological, social, and spiritual support for the child and family. The goal of palliative care is to help control symptoms and give the child the best quality of life possible. Parents may not be sure about whether to continue treatment or what kind of treatment is best for their child. The healthcare team can give parents information to help them make these decisions. For information about the treatments listed below, see the Treatment Option Overview section. More radiation therapy may be given to children with progressive or recurrent diffuse intrinsic pontine glioma (DIPG) who responded when first treated with radiation therapy. Treatment of progressive or recurrent DIPG may also include the following: Treatment of recurrent focal childhood brain stem glioma may include the following: For more information about childhood brain tumors, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood brain stem glioma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Brain Stem Glioma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/brain/patient/child-glioma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389295] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Childhood Brain Stem Glioma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the symptoms of Childhood Brain Stem Glioma ?

The signs and symptoms of brain stem glioma are not the same in every child. Signs and symptoms depend on the following: - Where the tumor forms in the brain. - The size of the tumor and whether it has spread all through the brain stem. - How fast the tumor grows. - The child&apos;s age and development. Some tumors do not cause signs or symptoms. Signs and symptoms may be caused by childhood brain stem gliomas or by other conditions. Check with your child&apos;s doctor if your child has any of the following: - Loss of ability to move one side of the face and/or body. - Loss of balance and trouble walking. - Vision and hearing problems. - Morning headache or headache that goes away after vomiting. - Nausea and vomiting. - Unusual sleepiness. - More or less energy than usual. - Changes in behavior. - Trouble learning in school.

Gliomas are tumors formed from glial cells. Glial cells in the brain hold nerve cells in place, bring food and oxygen to nerve cells, and help protect nerve cells from disease, such as infection. In brain stem glioma, the glial cells in the brain stem are affected. The brain stem is made up of the midbrain, pons, and medulla. It is the lowest part of the brain and connects to the spinal cord, just above the back of the neck. The brain stem controls vital functions such as breathing, heart rate, and blood pressure, and the nerves and muscles used in seeing, hearing, walking, talking, and eating. Most childhood brain stem gliomas are diffuse intrinsic pontine gliomas (DIPG), which form in the pons. Focal gliomas form in other parts of the brain stem. Brain tumors are the second most common type of cancer in children. This summary is about the treatment of primary brain tumors (tumors that begin in the brain). Treatment for metastatic brain tumors, which are tumors formed by cancer cells that begin in other parts of the body and spread to the brain, is not discussed in this summary. Brain tumors can occur in both children and adults; however, treatment for children may be different than treatment for adults. For information on treatment of brain tumors in adults, see the PDQ summary Adult Central Nervous System Tumors Treatment. Even though DIPG and focal brain stem glioma form in the same type of cell, they act differently: Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your child's doctor if you think your child may be at risk. Possible risk factors for brain stem glioma include: Signs and symptoms depend on the following: Signs and symptoms may be caused by childhood brain stem gliomas or by other conditions. Check with your child's doctor if your child has any of the following: The following tests and procedures may be used: If the MRI scan looks like the tumor is a DIPG, a biopsy is usually not done and the tumor is not removed. When the MRI scan results are uncertain, a biopsy may be done. If the MRI scan looks like a focal brain stem glioma, a biopsy may be done. A part of the skull is removed and a needle is used to remove a sample of the brain tissue. Sometimes, the needle is guided by a computer. A pathologist views the tissue under a microscope to look for cancer cells. If cancer cells are found, the doctor will remove as much tumor as safely possible during the same surgery. The following test may be done on the tissue sample that was removed during biopsy or surgery: The child's prognosis depends on the following: Most children with DIPG live less than 18 months after diagnosis. Children with a focal glioma usually live longer than 5 years. Staging is the process used to find out how much cancer there is and if cancer has spread. It is important to know the stage in order to plan treatment. There is no standard staging system for childhood brain stem glioma. Treatment is based on the following: A focal brain stem glioma may recur many years after first being treated. The tumor may come back in the brain or in other parts of the central nervous system. Before cancer treatment is given, imaging tests, a biopsy, or surgery may be done to make sure there is cancer and find out how much cancer there is. Different types of treatment are available for children with brain stem glioma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating children with brain tumors and who specialize in certain areas of medicine. These may include the following specialists: Biopsy or surgery to remove DIPG is not usually done because of the following: A biopsy to diagnose or surgery to remove the tumor may be used for childhood focal brain stem glioma. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type of the cancer being treated. External radiation therapy is used to treat DIPG. External and/or internal radiation therapy may be used to treat focal brain stem gliomas. Several months after radiation therapy to the brain, imaging tests may show changes to the brain tissue. These changes may be caused by the radiation therapy or may mean the tumor is growing. It is important to be sure the tumor is growing before any more treatment is given. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Because radiation therapy to the brain can affect growth and brain development in young children, chemotherapy may be given to delay or reduce the need for radiation therapy. Cerebrospinal fluid diversion is a method used to drain fluid that has built up in the brain. A shunt (long, thin tube) is placed in a ventricle (fluid-filled space) of the brain and threaded under the skin to another part of the body, usually the abdomen. The shunt carries extra fluid away from the brain so it may be absorbed elsewhere in the body.EnlargeCerebrospinal fluid (CSF) diversion. Extra CSF is removed from a ventricle in the brain through a shunt (tube) and is emptied into the abdomen. A valve controls the flow of CSF. Observation is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. There are different types of targeted therapy being studied in the treatment of brain stem gliomas: A monoclonal antibody, APX005M, binds to CD40, a cell surface receptor found on certain immune cells and some cancer cells. It may fight cancer by boosting the immune system and by slowing cancer cell growth. It is being studied in the treatment of pediatric brain tumors that are growing, spreading, or getting worse (progressive), or in newly diagnosed DIPG. For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information). Information about clinical trials is available from the NCI website. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. If the results of imaging tests done after treatment for DIPG show a mass in the brain, a biopsy may be done to find out if it is made up of dead tumor cells or if new cancer cells are growing. In children who are expected to live a long time, regular MRIs may be done to see if the cancer has come back. For information about the treatments listed below, see the Treatment Option Overview section. Newly diagnosed childhood diffuse intrinsic brain stem glioma (DIPG) is a tumor for which no treatment has been given. The child may have received drugs or treatment to relieve signs or symptoms caused by the tumor. Standard treatment of DIPG may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Newly diagnosed childhood focal glioma is a tumor for which no treatment has been given. The child may have received drugs or treatment to relieve signs or symptoms caused by the tumor. Treatment of focal glioma may include the following: Treatment of brain stem glioma in children with neurofibromatosis type 1 may be observation. The tumors are slow-growing in these children and may not need specific treatment for years. When cancer does not get better with treatment or comes back, palliative care is an important part of the child's treatment plan. It includes physical, psychological, social, and spiritual support for the child and family. The goal of palliative care is to help control symptoms and give the child the best quality of life possible. Parents may not be sure about whether to continue treatment or what kind of treatment is best for their child. The healthcare team can give parents information to help them make these decisions. For information about the treatments listed below, see the Treatment Option Overview section. More radiation therapy may be given to children with progressive or recurrent diffuse intrinsic pontine glioma (DIPG) who responded when first treated with radiation therapy. Treatment of progressive or recurrent DIPG may also include the following: Treatment of recurrent focal childhood brain stem glioma may include the following: For more information about childhood brain tumors, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood brain stem glioma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Brain Stem Glioma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/brain/patient/child-glioma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389295] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Childhood Brain Stem Glioma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

How to diagnose Childhood Brain Stem Glioma ?

Tests that examine the brain are used to detect (find) childhood brain stem glioma. The following tests and procedures may be used: - Physical exam and history : An exam of the body to check general signs of health, including checking for signs of disease, such as lumps or anything else that seems unusual. A history of the patients health habits and past illnesses and treatments will also be taken. - Neurological exam : A series of questions and tests to check the brain, spinal cord, and nerve function. The exam checks a persons mental status, coordination, and ability to walk normally, and how well the muscles, senses, and reflexes work. This may also be called a neuro exam or a neurologic exam. - MRI (magnetic resonance imaging) with gadolinium : A procedure that uses a magnet, radio waves, and a computer to make a series of detailed pictures of areas inside the brain. A substance called gadolinium is injected into a vein. The gadolinium collects around the cancer cells so they show up brighter in the picture. This procedure is also called nuclear magnetic resonance imaging (NMRI). - A biopsy may be done to diagnose certain types of brain stem glioma. If the MRI scan looks like the tumor is a DIPG, a biopsy is usually not done and the tumor is not removed. If the MRI scan looks like a focal brain stem glioma, a biopsy may be done. A part of the skull is removed and a needle is used to remove a sample of the brain tissue. Sometimes, the needle is guided by a computer. A pathologist views the tissue under a microscope to look for cancer cells. If cancer cells are found, the doctor will remove as much tumor as safely possible during the same surgery. The following test may be done on the tissue that was removed: - Immunohistochemistry : A test that uses antibodies to check for certain antigens in a sample of tissue. The antibody is usually linked to a radioactive substance or a dye that causes the tissue to light up under a microscope. This type of test may be used to tell the difference between brain stem glioma and other brain tumors.

Gliomas are tumors formed from glial cells. Glial cells in the brain hold nerve cells in place, bring food and oxygen to nerve cells, and help protect nerve cells from disease, such as infection. In brain stem glioma, the glial cells in the brain stem are affected. The brain stem is made up of the midbrain, pons, and medulla. It is the lowest part of the brain and connects to the spinal cord, just above the back of the neck. The brain stem controls vital functions such as breathing, heart rate, and blood pressure, and the nerves and muscles used in seeing, hearing, walking, talking, and eating. Most childhood brain stem gliomas are diffuse intrinsic pontine gliomas (DIPG), which form in the pons. Focal gliomas form in other parts of the brain stem. Brain tumors are the second most common type of cancer in children. This summary is about the treatment of primary brain tumors (tumors that begin in the brain). Treatment for metastatic brain tumors, which are tumors formed by cancer cells that begin in other parts of the body and spread to the brain, is not discussed in this summary. Brain tumors can occur in both children and adults; however, treatment for children may be different than treatment for adults. For information on treatment of brain tumors in adults, see the PDQ summary Adult Central Nervous System Tumors Treatment. Even though DIPG and focal brain stem glioma form in the same type of cell, they act differently: Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your child's doctor if you think your child may be at risk. Possible risk factors for brain stem glioma include: Signs and symptoms depend on the following: Signs and symptoms may be caused by childhood brain stem gliomas or by other conditions. Check with your child's doctor if your child has any of the following: The following tests and procedures may be used: If the MRI scan looks like the tumor is a DIPG, a biopsy is usually not done and the tumor is not removed. When the MRI scan results are uncertain, a biopsy may be done. If the MRI scan looks like a focal brain stem glioma, a biopsy may be done. A part of the skull is removed and a needle is used to remove a sample of the brain tissue. Sometimes, the needle is guided by a computer. A pathologist views the tissue under a microscope to look for cancer cells. If cancer cells are found, the doctor will remove as much tumor as safely possible during the same surgery. The following test may be done on the tissue sample that was removed during biopsy or surgery: The child's prognosis depends on the following: Most children with DIPG live less than 18 months after diagnosis. Children with a focal glioma usually live longer than 5 years. Staging is the process used to find out how much cancer there is and if cancer has spread. It is important to know the stage in order to plan treatment. There is no standard staging system for childhood brain stem glioma. Treatment is based on the following: A focal brain stem glioma may recur many years after first being treated. The tumor may come back in the brain or in other parts of the central nervous system. Before cancer treatment is given, imaging tests, a biopsy, or surgery may be done to make sure there is cancer and find out how much cancer there is. Different types of treatment are available for children with brain stem glioma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating children with brain tumors and who specialize in certain areas of medicine. These may include the following specialists: Biopsy or surgery to remove DIPG is not usually done because of the following: A biopsy to diagnose or surgery to remove the tumor may be used for childhood focal brain stem glioma. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type of the cancer being treated. External radiation therapy is used to treat DIPG. External and/or internal radiation therapy may be used to treat focal brain stem gliomas. Several months after radiation therapy to the brain, imaging tests may show changes to the brain tissue. These changes may be caused by the radiation therapy or may mean the tumor is growing. It is important to be sure the tumor is growing before any more treatment is given. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Because radiation therapy to the brain can affect growth and brain development in young children, chemotherapy may be given to delay or reduce the need for radiation therapy. Cerebrospinal fluid diversion is a method used to drain fluid that has built up in the brain. A shunt (long, thin tube) is placed in a ventricle (fluid-filled space) of the brain and threaded under the skin to another part of the body, usually the abdomen. The shunt carries extra fluid away from the brain so it may be absorbed elsewhere in the body.EnlargeCerebrospinal fluid (CSF) diversion. Extra CSF is removed from a ventricle in the brain through a shunt (tube) and is emptied into the abdomen. A valve controls the flow of CSF. Observation is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. There are different types of targeted therapy being studied in the treatment of brain stem gliomas: A monoclonal antibody, APX005M, binds to CD40, a cell surface receptor found on certain immune cells and some cancer cells. It may fight cancer by boosting the immune system and by slowing cancer cell growth. It is being studied in the treatment of pediatric brain tumors that are growing, spreading, or getting worse (progressive), or in newly diagnosed DIPG. For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information). Information about clinical trials is available from the NCI website. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. If the results of imaging tests done after treatment for DIPG show a mass in the brain, a biopsy may be done to find out if it is made up of dead tumor cells or if new cancer cells are growing. In children who are expected to live a long time, regular MRIs may be done to see if the cancer has come back. For information about the treatments listed below, see the Treatment Option Overview section. Newly diagnosed childhood diffuse intrinsic brain stem glioma (DIPG) is a tumor for which no treatment has been given. The child may have received drugs or treatment to relieve signs or symptoms caused by the tumor. Standard treatment of DIPG may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Newly diagnosed childhood focal glioma is a tumor for which no treatment has been given. The child may have received drugs or treatment to relieve signs or symptoms caused by the tumor. Treatment of focal glioma may include the following: Treatment of brain stem glioma in children with neurofibromatosis type 1 may be observation. The tumors are slow-growing in these children and may not need specific treatment for years. When cancer does not get better with treatment or comes back, palliative care is an important part of the child's treatment plan. It includes physical, psychological, social, and spiritual support for the child and family. The goal of palliative care is to help control symptoms and give the child the best quality of life possible. Parents may not be sure about whether to continue treatment or what kind of treatment is best for their child. The healthcare team can give parents information to help them make these decisions. For information about the treatments listed below, see the Treatment Option Overview section. More radiation therapy may be given to children with progressive or recurrent diffuse intrinsic pontine glioma (DIPG) who responded when first treated with radiation therapy. Treatment of progressive or recurrent DIPG may also include the following: Treatment of recurrent focal childhood brain stem glioma may include the following: For more information about childhood brain tumors, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood brain stem glioma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Brain Stem Glioma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/brain/patient/child-glioma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389295] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Childhood Brain Stem Glioma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is the outlook for Childhood Brain Stem Glioma ?

Certain factors affect prognosis (chance of recovery) and treatment options. The prognosis and treatment options depend on: - The type of brain stem glioma. - Where the tumor is found in the brain and if it has spread within the brain stem. - The age of the child when diagnosed. - Whether or not the child has a condition called neurofibromatosis type 1. - Whether the tumor has just been diagnosed or has recurred (come back).

Gliomas are tumors formed from glial cells. Glial cells in the brain hold nerve cells in place, bring food and oxygen to nerve cells, and help protect nerve cells from disease, such as infection. In brain stem glioma, the glial cells in the brain stem are affected. The brain stem is made up of the midbrain, pons, and medulla. It is the lowest part of the brain and connects to the spinal cord, just above the back of the neck. The brain stem controls vital functions such as breathing, heart rate, and blood pressure, and the nerves and muscles used in seeing, hearing, walking, talking, and eating. Most childhood brain stem gliomas are diffuse intrinsic pontine gliomas (DIPG), which form in the pons. Focal gliomas form in other parts of the brain stem. Brain tumors are the second most common type of cancer in children. This summary is about the treatment of primary brain tumors (tumors that begin in the brain). Treatment for metastatic brain tumors, which are tumors formed by cancer cells that begin in other parts of the body and spread to the brain, is not discussed in this summary. Brain tumors can occur in both children and adults; however, treatment for children may be different than treatment for adults. For information on treatment of brain tumors in adults, see the PDQ summary Adult Central Nervous System Tumors Treatment. Even though DIPG and focal brain stem glioma form in the same type of cell, they act differently: Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your child's doctor if you think your child may be at risk. Possible risk factors for brain stem glioma include: Signs and symptoms depend on the following: Signs and symptoms may be caused by childhood brain stem gliomas or by other conditions. Check with your child's doctor if your child has any of the following: The following tests and procedures may be used: If the MRI scan looks like the tumor is a DIPG, a biopsy is usually not done and the tumor is not removed. When the MRI scan results are uncertain, a biopsy may be done. If the MRI scan looks like a focal brain stem glioma, a biopsy may be done. A part of the skull is removed and a needle is used to remove a sample of the brain tissue. Sometimes, the needle is guided by a computer. A pathologist views the tissue under a microscope to look for cancer cells. If cancer cells are found, the doctor will remove as much tumor as safely possible during the same surgery. The following test may be done on the tissue sample that was removed during biopsy or surgery: The child's prognosis depends on the following: Most children with DIPG live less than 18 months after diagnosis. Children with a focal glioma usually live longer than 5 years. Staging is the process used to find out how much cancer there is and if cancer has spread. It is important to know the stage in order to plan treatment. There is no standard staging system for childhood brain stem glioma. Treatment is based on the following: A focal brain stem glioma may recur many years after first being treated. The tumor may come back in the brain or in other parts of the central nervous system. Before cancer treatment is given, imaging tests, a biopsy, or surgery may be done to make sure there is cancer and find out how much cancer there is. Different types of treatment are available for children with brain stem glioma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating children with brain tumors and who specialize in certain areas of medicine. These may include the following specialists: Biopsy or surgery to remove DIPG is not usually done because of the following: A biopsy to diagnose or surgery to remove the tumor may be used for childhood focal brain stem glioma. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type of the cancer being treated. External radiation therapy is used to treat DIPG. External and/or internal radiation therapy may be used to treat focal brain stem gliomas. Several months after radiation therapy to the brain, imaging tests may show changes to the brain tissue. These changes may be caused by the radiation therapy or may mean the tumor is growing. It is important to be sure the tumor is growing before any more treatment is given. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Because radiation therapy to the brain can affect growth and brain development in young children, chemotherapy may be given to delay or reduce the need for radiation therapy. Cerebrospinal fluid diversion is a method used to drain fluid that has built up in the brain. A shunt (long, thin tube) is placed in a ventricle (fluid-filled space) of the brain and threaded under the skin to another part of the body, usually the abdomen. The shunt carries extra fluid away from the brain so it may be absorbed elsewhere in the body.EnlargeCerebrospinal fluid (CSF) diversion. Extra CSF is removed from a ventricle in the brain through a shunt (tube) and is emptied into the abdomen. A valve controls the flow of CSF. Observation is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. There are different types of targeted therapy being studied in the treatment of brain stem gliomas: A monoclonal antibody, APX005M, binds to CD40, a cell surface receptor found on certain immune cells and some cancer cells. It may fight cancer by boosting the immune system and by slowing cancer cell growth. It is being studied in the treatment of pediatric brain tumors that are growing, spreading, or getting worse (progressive), or in newly diagnosed DIPG. For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information). Information about clinical trials is available from the NCI website. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. If the results of imaging tests done after treatment for DIPG show a mass in the brain, a biopsy may be done to find out if it is made up of dead tumor cells or if new cancer cells are growing. In children who are expected to live a long time, regular MRIs may be done to see if the cancer has come back. For information about the treatments listed below, see the Treatment Option Overview section. Newly diagnosed childhood diffuse intrinsic brain stem glioma (DIPG) is a tumor for which no treatment has been given. The child may have received drugs or treatment to relieve signs or symptoms caused by the tumor. Standard treatment of DIPG may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Newly diagnosed childhood focal glioma is a tumor for which no treatment has been given. The child may have received drugs or treatment to relieve signs or symptoms caused by the tumor. Treatment of focal glioma may include the following: Treatment of brain stem glioma in children with neurofibromatosis type 1 may be observation. The tumors are slow-growing in these children and may not need specific treatment for years. When cancer does not get better with treatment or comes back, palliative care is an important part of the child's treatment plan. It includes physical, psychological, social, and spiritual support for the child and family. The goal of palliative care is to help control symptoms and give the child the best quality of life possible. Parents may not be sure about whether to continue treatment or what kind of treatment is best for their child. The healthcare team can give parents information to help them make these decisions. For information about the treatments listed below, see the Treatment Option Overview section. More radiation therapy may be given to children with progressive or recurrent diffuse intrinsic pontine glioma (DIPG) who responded when first treated with radiation therapy. Treatment of progressive or recurrent DIPG may also include the following: Treatment of recurrent focal childhood brain stem glioma may include the following: For more information about childhood brain tumors, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood brain stem glioma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Brain Stem Glioma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/brain/patient/child-glioma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389295] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Childhood Brain Stem Glioma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the stages of Childhood Brain Stem Glioma ?

Key Points - The plan for cancer treatment depends on whether the tumor is in one area of the brain or has spread all through the brain. The plan for cancer treatment depends on whether the tumor is in one area of the brain or has spread all through the brain. Staging is the process used to find out how much cancer there is and if cancer has spread. It is important to know the stage in order to plan treatment. There is no standard staging system for childhood brain stem glioma. Treatment is based on the following: - Whether the tumor is newly diagnosed or recurrent (has come back after treatment). - The type of tumor (either a diffuse intrinsic pontine glioma or a focal glioma).

Gliomas are tumors formed from glial cells. Glial cells in the brain hold nerve cells in place, bring food and oxygen to nerve cells, and help protect nerve cells from disease, such as infection. In brain stem glioma, the glial cells in the brain stem are affected. The brain stem is made up of the midbrain, pons, and medulla. It is the lowest part of the brain and connects to the spinal cord, just above the back of the neck. The brain stem controls vital functions such as breathing, heart rate, and blood pressure, and the nerves and muscles used in seeing, hearing, walking, talking, and eating. Most childhood brain stem gliomas are diffuse intrinsic pontine gliomas (DIPG), which form in the pons. Focal gliomas form in other parts of the brain stem. Brain tumors are the second most common type of cancer in children. This summary is about the treatment of primary brain tumors (tumors that begin in the brain). Treatment for metastatic brain tumors, which are tumors formed by cancer cells that begin in other parts of the body and spread to the brain, is not discussed in this summary. Brain tumors can occur in both children and adults; however, treatment for children may be different than treatment for adults. For information on treatment of brain tumors in adults, see the PDQ summary Adult Central Nervous System Tumors Treatment. Even though DIPG and focal brain stem glioma form in the same type of cell, they act differently: Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your child's doctor if you think your child may be at risk. Possible risk factors for brain stem glioma include: Signs and symptoms depend on the following: Signs and symptoms may be caused by childhood brain stem gliomas or by other conditions. Check with your child's doctor if your child has any of the following: The following tests and procedures may be used: If the MRI scan looks like the tumor is a DIPG, a biopsy is usually not done and the tumor is not removed. When the MRI scan results are uncertain, a biopsy may be done. If the MRI scan looks like a focal brain stem glioma, a biopsy may be done. A part of the skull is removed and a needle is used to remove a sample of the brain tissue. Sometimes, the needle is guided by a computer. A pathologist views the tissue under a microscope to look for cancer cells. If cancer cells are found, the doctor will remove as much tumor as safely possible during the same surgery. The following test may be done on the tissue sample that was removed during biopsy or surgery: The child's prognosis depends on the following: Most children with DIPG live less than 18 months after diagnosis. Children with a focal glioma usually live longer than 5 years. Staging is the process used to find out how much cancer there is and if cancer has spread. It is important to know the stage in order to plan treatment. There is no standard staging system for childhood brain stem glioma. Treatment is based on the following: A focal brain stem glioma may recur many years after first being treated. The tumor may come back in the brain or in other parts of the central nervous system. Before cancer treatment is given, imaging tests, a biopsy, or surgery may be done to make sure there is cancer and find out how much cancer there is. Different types of treatment are available for children with brain stem glioma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating children with brain tumors and who specialize in certain areas of medicine. These may include the following specialists: Biopsy or surgery to remove DIPG is not usually done because of the following: A biopsy to diagnose or surgery to remove the tumor may be used for childhood focal brain stem glioma. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type of the cancer being treated. External radiation therapy is used to treat DIPG. External and/or internal radiation therapy may be used to treat focal brain stem gliomas. Several months after radiation therapy to the brain, imaging tests may show changes to the brain tissue. These changes may be caused by the radiation therapy or may mean the tumor is growing. It is important to be sure the tumor is growing before any more treatment is given. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Because radiation therapy to the brain can affect growth and brain development in young children, chemotherapy may be given to delay or reduce the need for radiation therapy. Cerebrospinal fluid diversion is a method used to drain fluid that has built up in the brain. A shunt (long, thin tube) is placed in a ventricle (fluid-filled space) of the brain and threaded under the skin to another part of the body, usually the abdomen. The shunt carries extra fluid away from the brain so it may be absorbed elsewhere in the body.EnlargeCerebrospinal fluid (CSF) diversion. Extra CSF is removed from a ventricle in the brain through a shunt (tube) and is emptied into the abdomen. A valve controls the flow of CSF. Observation is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. There are different types of targeted therapy being studied in the treatment of brain stem gliomas: A monoclonal antibody, APX005M, binds to CD40, a cell surface receptor found on certain immune cells and some cancer cells. It may fight cancer by boosting the immune system and by slowing cancer cell growth. It is being studied in the treatment of pediatric brain tumors that are growing, spreading, or getting worse (progressive), or in newly diagnosed DIPG. For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information). Information about clinical trials is available from the NCI website. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. If the results of imaging tests done after treatment for DIPG show a mass in the brain, a biopsy may be done to find out if it is made up of dead tumor cells or if new cancer cells are growing. In children who are expected to live a long time, regular MRIs may be done to see if the cancer has come back. For information about the treatments listed below, see the Treatment Option Overview section. Newly diagnosed childhood diffuse intrinsic brain stem glioma (DIPG) is a tumor for which no treatment has been given. The child may have received drugs or treatment to relieve signs or symptoms caused by the tumor. Standard treatment of DIPG may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Newly diagnosed childhood focal glioma is a tumor for which no treatment has been given. The child may have received drugs or treatment to relieve signs or symptoms caused by the tumor. Treatment of focal glioma may include the following: Treatment of brain stem glioma in children with neurofibromatosis type 1 may be observation. The tumors are slow-growing in these children and may not need specific treatment for years. When cancer does not get better with treatment or comes back, palliative care is an important part of the child's treatment plan. It includes physical, psychological, social, and spiritual support for the child and family. The goal of palliative care is to help control symptoms and give the child the best quality of life possible. Parents may not be sure about whether to continue treatment or what kind of treatment is best for their child. The healthcare team can give parents information to help them make these decisions. For information about the treatments listed below, see the Treatment Option Overview section. More radiation therapy may be given to children with progressive or recurrent diffuse intrinsic pontine glioma (DIPG) who responded when first treated with radiation therapy. Treatment of progressive or recurrent DIPG may also include the following: Treatment of recurrent focal childhood brain stem glioma may include the following: For more information about childhood brain tumors, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood brain stem glioma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Brain Stem Glioma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/brain/patient/child-glioma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389295] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Childhood Brain Stem Glioma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

what research (or clinical trials) is being done for Childhood Brain Stem Glioma ?

New types of treatment are being tested in clinical trials. Information about clinical trials is available from the NCI website. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials.

Gliomas are tumors formed from glial cells. Glial cells in the brain hold nerve cells in place, bring food and oxygen to nerve cells, and help protect nerve cells from disease, such as infection. In brain stem glioma, the glial cells in the brain stem are affected. The brain stem is made up of the midbrain, pons, and medulla. It is the lowest part of the brain and connects to the spinal cord, just above the back of the neck. The brain stem controls vital functions such as breathing, heart rate, and blood pressure, and the nerves and muscles used in seeing, hearing, walking, talking, and eating. Most childhood brain stem gliomas are diffuse intrinsic pontine gliomas (DIPG), which form in the pons. Focal gliomas form in other parts of the brain stem. Brain tumors are the second most common type of cancer in children. This summary is about the treatment of primary brain tumors (tumors that begin in the brain). Treatment for metastatic brain tumors, which are tumors formed by cancer cells that begin in other parts of the body and spread to the brain, is not discussed in this summary. Brain tumors can occur in both children and adults; however, treatment for children may be different than treatment for adults. For information on treatment of brain tumors in adults, see the PDQ summary Adult Central Nervous System Tumors Treatment. Even though DIPG and focal brain stem glioma form in the same type of cell, they act differently: Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your child's doctor if you think your child may be at risk. Possible risk factors for brain stem glioma include: Signs and symptoms depend on the following: Signs and symptoms may be caused by childhood brain stem gliomas or by other conditions. Check with your child's doctor if your child has any of the following: The following tests and procedures may be used: If the MRI scan looks like the tumor is a DIPG, a biopsy is usually not done and the tumor is not removed. When the MRI scan results are uncertain, a biopsy may be done. If the MRI scan looks like a focal brain stem glioma, a biopsy may be done. A part of the skull is removed and a needle is used to remove a sample of the brain tissue. Sometimes, the needle is guided by a computer. A pathologist views the tissue under a microscope to look for cancer cells. If cancer cells are found, the doctor will remove as much tumor as safely possible during the same surgery. The following test may be done on the tissue sample that was removed during biopsy or surgery: The child's prognosis depends on the following: Most children with DIPG live less than 18 months after diagnosis. Children with a focal glioma usually live longer than 5 years. Staging is the process used to find out how much cancer there is and if cancer has spread. It is important to know the stage in order to plan treatment. There is no standard staging system for childhood brain stem glioma. Treatment is based on the following: A focal brain stem glioma may recur many years after first being treated. The tumor may come back in the brain or in other parts of the central nervous system. Before cancer treatment is given, imaging tests, a biopsy, or surgery may be done to make sure there is cancer and find out how much cancer there is. Different types of treatment are available for children with brain stem glioma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating children with brain tumors and who specialize in certain areas of medicine. These may include the following specialists: Biopsy or surgery to remove DIPG is not usually done because of the following: A biopsy to diagnose or surgery to remove the tumor may be used for childhood focal brain stem glioma. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: The way the radiation therapy is given depends on the type of the cancer being treated. External radiation therapy is used to treat DIPG. External and/or internal radiation therapy may be used to treat focal brain stem gliomas. Several months after radiation therapy to the brain, imaging tests may show changes to the brain tissue. These changes may be caused by the radiation therapy or may mean the tumor is growing. It is important to be sure the tumor is growing before any more treatment is given. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Because radiation therapy to the brain can affect growth and brain development in young children, chemotherapy may be given to delay or reduce the need for radiation therapy. Cerebrospinal fluid diversion is a method used to drain fluid that has built up in the brain. A shunt (long, thin tube) is placed in a ventricle (fluid-filled space) of the brain and threaded under the skin to another part of the body, usually the abdomen. The shunt carries extra fluid away from the brain so it may be absorbed elsewhere in the body.EnlargeCerebrospinal fluid (CSF) diversion. Extra CSF is removed from a ventricle in the brain through a shunt (tube) and is emptied into the abdomen. A valve controls the flow of CSF. Observation is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. There are different types of targeted therapy being studied in the treatment of brain stem gliomas: A monoclonal antibody, APX005M, binds to CD40, a cell surface receptor found on certain immune cells and some cancer cells. It may fight cancer by boosting the immune system and by slowing cancer cell growth. It is being studied in the treatment of pediatric brain tumors that are growing, spreading, or getting worse (progressive), or in newly diagnosed DIPG. For information about side effects that begin during treatment for cancer, see our Side Effects page. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects may include the following: Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information). Information about clinical trials is available from the NCI website. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. If the results of imaging tests done after treatment for DIPG show a mass in the brain, a biopsy may be done to find out if it is made up of dead tumor cells or if new cancer cells are growing. In children who are expected to live a long time, regular MRIs may be done to see if the cancer has come back. For information about the treatments listed below, see the Treatment Option Overview section. Newly diagnosed childhood diffuse intrinsic brain stem glioma (DIPG) is a tumor for which no treatment has been given. The child may have received drugs or treatment to relieve signs or symptoms caused by the tumor. Standard treatment of DIPG may include the following: For information about the treatments listed below, see the Treatment Option Overview section. Newly diagnosed childhood focal glioma is a tumor for which no treatment has been given. The child may have received drugs or treatment to relieve signs or symptoms caused by the tumor. Treatment of focal glioma may include the following: Treatment of brain stem glioma in children with neurofibromatosis type 1 may be observation. The tumors are slow-growing in these children and may not need specific treatment for years. When cancer does not get better with treatment or comes back, palliative care is an important part of the child's treatment plan. It includes physical, psychological, social, and spiritual support for the child and family. The goal of palliative care is to help control symptoms and give the child the best quality of life possible. Parents may not be sure about whether to continue treatment or what kind of treatment is best for their child. The healthcare team can give parents information to help them make these decisions. For information about the treatments listed below, see the Treatment Option Overview section. More radiation therapy may be given to children with progressive or recurrent diffuse intrinsic pontine glioma (DIPG) who responded when first treated with radiation therapy. Treatment of progressive or recurrent DIPG may also include the following: Treatment of recurrent focal childhood brain stem glioma may include the following: For more information about childhood brain tumors, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood brain stem glioma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Brain Stem Glioma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/brain/patient/child-glioma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389295] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Childhood Brain Stem Glioma Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the treatments for Childhood Brain Stem Glioma ?

Key Points - There are different types of treatment for children with brain stem glioma. - Children with brain stem glioma should have their treatment planned by a team of health care providers who are experts in treating childhood brain tumors. - Childhood brain stem gliomas may cause signs or symptoms that begin before the cancer is diagnosed and continue for months or years. - Some cancer treatments cause side effects months or years after treatment has ended. - Six types of standard treatment are used: - Surgery - Radiation therapy - Chemotherapy - Cerebrospinal fluid diversion - Observation - Targeted therapy - New types of treatment are being tested in clinical trials. - Patients may want to think about taking part in a clinical trial. - Patients can enter clinical trials before, during, or after starting their cancer treatment. - Follow-up tests may be needed. There are different types of treatment for children with brain stem glioma. Different types of treatment are available for children with brain stem glioma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment. Children with brain stem glioma should have their treatment planned by a team of health care providers who are experts in treating childhood brain tumors. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating children with brain tumors and who specialize in certain areas of medicine. These may include the following specialists: - Pediatrician. - Neurosurgeon. - Neuropathologist. - Radiation oncologist. - Neuro-oncologist. - Neurologist. - Rehabilitation specialist. - Neuroradiologist. - Endocrinologist. - Psychologist. Childhood brain stem gliomas may cause signs or symptoms that begin before the cancer is diagnosed and continue for months or years. Childhood brain stem gliomas may cause signs or symptoms that continue for months or years. Signs or symptoms caused by the tumor may begin before diagnosis. Signs or symptoms caused by treatment may begin during or right after treatment. Some cancer treatments cause side effects months or years after treatment has ended. These are called late effects. Late effects may include the following: - Physical problems. - Changes in mood, feelings, thinking, learning, or memory. - Second cancers (new types of cancer). Some late effects may be treated or controlled. It is important to talk with your child&apos;s doctors about the effects cancer treatment can have on your child. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information). Six types of standard treatment are used: Surgery Surgery may be used to diagnose and treat childhood brain stem glioma as discussed in the General Information section of this summary. Radiation therapy Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy: - External radiation therapy uses a machine outside the body to send radiation toward the cancer. - Internal radiation therapy uses a radioactive substance sealed in needles, seeds, wires, or catheters that are placed directly into or near the cancer. The way the radiation therapy is given depends on the type of the cancer being treated. External radiation therapy is used to treat DIPG. External and/or internal radiation therapy may be used to treat focal brain stem gliomas. Several months after radiation therapy to the brain, imaging tests may show changes to the brain tissue. These changes may be caused by the radiation therapy or may mean the tumor is growing. It is important to be sure the tumor is growing before any more treatment is given. Chemotherapy Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly in the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on the type of the cancer being treated. Because radiation therapy to the brain can affect growth and brain development in young children, clinical trials are studying ways of using chemotherapy to delay or reduce the need for radiation therapy. Cerebrospinal fluid diversion Cerebrospinal fluid diversion is a method used to drain fluid that has built up in the brain. A shunt (long, thin tube) is placed in a ventricle (fluid-filled space) of the brain and threaded under the skin to another part of the body, usually the abdomen. The shunt carries extra fluid away from the brain so it may be absorbed elsewhere in the body. Observation Observation is closely monitoring a patients condition without giving any treatment until signs or symptoms appear or change. Targeted therapy Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells without harming normal cells. Some focal brain stem gliomas that cannot be removed by surgery may be treated with BRAF kinase inhibitor therapy. BRAF kinase inhibitors block the BRAF protein. BRAF proteins help control cell growth and may be mutated (changed) in some types of brain stem glioma. Blocking mutated BRAF kinase proteins may help keep cancer cells from growing. New types of treatment are being tested in clinical trials. Information about clinical trials is available from the NCI website. Patients may want to think about taking part in a clinical trial. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today&apos;s standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Patients can enter clinical trials before, during, or after starting their cancer treatment. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI&apos;s listing of clinical trials. Follow-up tests may be needed. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child&apos;s condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. If the results of imaging tests done after treatment show a mass in the brain, a biopsy may be done to find out if it is made up of dead tumor cells or if new cancer cells are growing. In children who are expected to live a long time, regular MRIs may be done to see if the cancer has come back. Treatment Options for Childhood Brain Stem Glioma Newly Diagnosed Childhood Brain Stem Glioma Newly diagnosed childhood brain stem glioma is a tumor for which no treatment has been given. The child may have received drugs or treatment to relieve signs or symptoms caused by the tumor. Standard treatment of diffuse intrinsic pontine glioma (DIPG) may include the following: - Radiation therapy. - Chemotherapy (in infants). Standard treatment of focal glioma may include the following: - Surgery that may be followed by chemotherapy and/or radiation therapy. - Observation for small tumors that grow slowly. Cerebrospinal fluid diversion may be done when there is extra fluid in the brain. - Internal radiation therapy with radioactive seeds, with or without chemotherapy, when the tumor cannot be removed by surgery. - Targeted therapy with a BRAF kinase inhibitor, for certain tumors that cannot be removed by surgery. Treatment of brain stem glioma in children with neurofibromatosis type 1 may be observation. The tumors are slow-growing in these children and may not need specific treatment for years. Check the list of NCI-supported cancer clinical trials that are now accepting patients with untreated childhood brain stem glioma. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your child&apos;s doctor about clinical trials that may be right for your child. General information about clinical trials is available from the NCI website. Progressive or Recurrent Childhood Brain Stem Glioma When cancer does not get better with treatment or comes back, palliative care is an important part of the child&apos;s treatment plan. It includes physical, psychological, social, and spiritual support for the child and family. The goal of palliative care is to help control symptoms and give the child the best quality of life possible. Parents may not be sure about whether to continue treatment or what kind of treatment is best for their child. The healthcare team can give parents information to help them make these decisions. There is no standard treatment for progressive or recurrent diffuse intrinsic pontine glioma. The child may be treated in a clinical trial of a new treatment. Treatment of recurrent focal childhood brain stem glioma may include the following: - A second surgery to remove the tumor. - External radiation therapy. - Chemotherapy. - A clinical trial of a new treatment. Check the list of NCI-supported cancer clinical trials that are now accepting patients with recurrent childhood brain stem glioma. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your child&apos;s doctor about clinical trials that may be right for your child. General information about clinical trials is available from the NCI website.

Screening is looking for cancer before a person has any symptoms. This can help find cancer at an early stage. When abnormal tissue or cancer is found early, it may be easier to treat. By the time symptoms appear, cancer may have begun to spread. Scientists are trying to better understand which people are more likely to get certain types of cancer. They also study the things we do and the things around us to see if they cause cancer. This information helps doctors recommend who should be screened for cancer, which screening tests should be used, and how often the tests should be done. It is important to remember that your doctor does not necessarily think you have cancer if he or she suggests a screening test. Screening tests are given when you have no cancer symptoms. If a screening test result is abnormal, you may need to have more tests done to find out if you have cancer. These are called diagnostic tests. The ovaries are a pair of organs in the female reproductive system. They are located in the pelvis, one on each side of the uterus (the hollow, pear-shaped organ where a fetus grows). Each ovary is about the size and shape of an almond. The ovaries produce eggs and female hormones (chemicals that control the way certain cells or organs function). The fallopian tubes are a pair of long, slender tubes, one on each side of the uterus. Eggs pass from the ovaries, through the fallopian tubes, to the uterus. Cancer sometimes begins at the end of the fallopian tube near the ovary and spreads to the ovary. The peritoneum is the tissue that lines the abdominal wall and covers organs in the abdomen. Primary peritoneal cancer is cancer that forms in the peritoneum and has not spread there from another part of the body. Cancer sometimes begins in the peritoneum and spreads to the ovary. Ovarian epithelial cancer, fallopian tube cancer, and primary peritoneal cancer form in the same type of tissue. Studies of screening tests look at these cancers together. See the following PDQ summaries for more information about ovarian, fallopian tube, and primary peritoneal cancers: While ovarian cancer is rare, it is the leading cause of death from cancer of the female reproductive system. The number of new cases of ovarian cancer decreased slightly each year from 1990 to the mid-2010s, but increased slightly each year from 2014 to 2018. There was a slight decrease in the number of deaths from ovarian cancer each year from 2015 to 2019. Anything that increases your chance of getting a disease is called a risk factor. Anything that decreases your chance of getting a disease is called a protective factor. For information about risk factors and protective factors for ovarian cancer, see the Ovarian, Fallopian Tube, and Primary Peritoneal Cancer Prevention summary. Talk to your doctor about your risk of ovarian cancer. Scientists study screening tests to find those with the fewest harms and most benefits. Cancer screening trials also are meant to show whether early detection (finding cancer before it causes symptoms) helps a person live longer or decreases a person's chance of dying from the disease. For some types of cancer, the chance of recovery is better if the disease is found and treated at an early stage. Pelvic exams to screen for ovarian cancer have not been shown to decrease the number of deaths from the disease. A pelvic exam is an exam of the vagina, cervix, uterus, fallopian tubes, ovaries, and rectum. A speculum is inserted into the vagina and the doctor or nurse looks at the vagina and cervix for signs of disease. The doctor or nurse also inserts one or two lubricated, gloved fingers of one hand into the vagina and places the other hand over the lower abdomen to feel the size, shape, and position of the uterus and ovaries. The doctor or nurse also inserts a lubricated, gloved finger into the rectum to feel for lumps or abnormal areas.EnlargePelvic exam. A doctor or nurse inserts one or two lubricated, gloved fingers of one hand into the vagina and presses on the lower abdomen with the other hand. This is done to feel the size, shape, and position of the uterus and ovaries. The vagina, cervix, fallopian tubes, and rectum are also checked. Transvaginal ultrasound (TVU) to screen for ovarian cancer has not been shown to decrease the number of deaths from the disease. TVU is a procedure used to examine the vagina, uterus, fallopian tubes, and bladder. An ultrasound transducer (probe) is inserted into the vagina and used to bounce high-energy sound waves (ultrasound) off internal tissues or organs and make echoes. The echoes form a picture of body tissues called a sonogram. EnlargeTransvaginal ultrasound. An ultrasound probe connected to a computer is inserted into the vagina and is gently moved to show different organs. The probe bounces sound waves off internal organs and tissues to make echoes that form a sonogram (computer picture). A CA-125 assay is a test that measures the level of CA-125 in the blood. CA-125 is a type of biomarker (a substance released by cells into the bloodstream that may be a sign of a condition or disease). An increased CA-125 level is sometimes a sign of certain types of cancer, including ovarian cancer, or other conditions. Studies have also shown that using CA-125 levels and TVU together to screen for ovarian cancer does not decrease the number of deaths from ovarian cancer. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Decisions about screening tests can be difficult. Not all screening tests are helpful and most have risks. Before having any screening test, you may want to talk about the test with your doctor. It is important to know the risks of the test and whether it has been proven to reduce the risk of dying from cancer. Screening may not improve your health or help you live longer if you have advanced ovarian cancer or if it has already spread to other places in your body. Screening test results may appear to be normal even though ovarian cancer is present. A woman who receives a false-negative test result (one that shows there is no cancer when there really is) may delay seeking medical care even if she has symptoms. Screening test results may appear to be abnormal even though no cancer is present. A false-positive test result (one that shows there is cancer when there really isn't) can cause anxiety and is usually followed by more tests (such as a laparoscopy or a laparotomy to see if cancer is present), which also have risks. Problems caused by tests used to diagnose ovarian cancer include infection, blood loss, bowel injury, and heart and blood vessel problems. A false-positive test result can also lead to an unneeded oophorectomy (removal of one or both ovaries). Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about ovarian, fallopian tube, and primary peritoneal cancer screening. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Screening and Prevention Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Screening and Prevention Editorial Board. PDQ Ovarian, Fallopian Tube, and Primary Peritoneal Cancer Screening. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/ovarian/patient/ovarian-screening-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389490] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Ovarian, Fallopian Tube, and Primary Peritoneal Cancer Screening (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is (are) Ovarian, Fallopian Tube, and Primary Peritoneal Cancer ?

Key Points - Ovarian, fallopian tube, and primary peritoneal cancers are diseases in which malignant (cancer) cells form in the ovaries, fallopian tubes, or peritoneum. - In the United States, ovarian cancer is the fifth leading cause of cancer death in women. - Different factors increase or decrease the risk of getting ovarian, fallopian tube, and primary peritoneal cancer. Ovarian, fallopian tube, and primary peritoneal cancers are diseases in which malignant (cancer) cells form in the ovaries, fallopian tubes, or peritoneum. The ovaries are a pair of organs in the female reproductive system. They are located in the pelvis, one on each side of the uterus (the hollow, pear-shaped organ where a fetus grows). Each ovary is about the size and shape of an almond. The ovaries produce eggs and female hormones (chemicals that control the way certain cells or organs function). The fallopian tubes are a pair of long, slender tubes, one on each side of the uterus. Eggs pass from the ovaries, through the fallopian tubes, to the uterus. Cancer sometimes begins at the end of the fallopian tube near the ovary and spreads to the ovary. The peritoneum is the tissue that lines the abdominal wall and covers organs in the abdomen. Primary peritoneal cancer is cancer that forms in the peritoneum and has not spread there from another part of the body. Cancer sometimes begins in the peritoneum and spreads to the ovary. Ovarian epithelial cancer, fallopian tube cancer, and primary peritoneal cancer form in the same type of tissue. Studies of screening tests look at these cancers together. See the following PDQ summaries for more information about ovarian, fallopian tube, and primary peritoneal cancers: - Ovarian, Fallopian Tube, and Primary Peritoneal Cancer Prevention - Genetics of Breast and Gynecologic Cancers - Ovarian Epithelial, Fallopian Tube, and Primary Peritoneal Cancer Treatment - Ovarian Germ Cell Tumors Treatment - Ovarian Low Malignant Potential Tumors Treatment In the United States, ovarian cancer is the fifth leading cause of cancer death in women. Ovarian cancer is also the leading cause of death from cancer of the female reproductive system. Over the last 20 years, the number of new cases of ovarian cancer has gone down slightly in white women and in black women. Since 2005, the number of deaths from ovarian cancer also decreased slightly in white and black women.

Screening is looking for cancer before a person has any symptoms. This can help find cancer at an early stage. When abnormal tissue or cancer is found early, it may be easier to treat. By the time symptoms appear, cancer may have begun to spread. Scientists are trying to better understand which people are more likely to get certain types of cancer. They also study the things we do and the things around us to see if they cause cancer. This information helps doctors recommend who should be screened for cancer, which screening tests should be used, and how often the tests should be done. It is important to remember that your doctor does not necessarily think you have cancer if he or she suggests a screening test. Screening tests are given when you have no cancer symptoms. If a screening test result is abnormal, you may need to have more tests done to find out if you have cancer. These are called diagnostic tests. The ovaries are a pair of organs in the female reproductive system. They are located in the pelvis, one on each side of the uterus (the hollow, pear-shaped organ where a fetus grows). Each ovary is about the size and shape of an almond. The ovaries produce eggs and female hormones (chemicals that control the way certain cells or organs function). The fallopian tubes are a pair of long, slender tubes, one on each side of the uterus. Eggs pass from the ovaries, through the fallopian tubes, to the uterus. Cancer sometimes begins at the end of the fallopian tube near the ovary and spreads to the ovary. The peritoneum is the tissue that lines the abdominal wall and covers organs in the abdomen. Primary peritoneal cancer is cancer that forms in the peritoneum and has not spread there from another part of the body. Cancer sometimes begins in the peritoneum and spreads to the ovary. Ovarian epithelial cancer, fallopian tube cancer, and primary peritoneal cancer form in the same type of tissue. Studies of screening tests look at these cancers together. See the following PDQ summaries for more information about ovarian, fallopian tube, and primary peritoneal cancers: While ovarian cancer is rare, it is the leading cause of death from cancer of the female reproductive system. The number of new cases of ovarian cancer decreased slightly each year from 1990 to the mid-2010s, but increased slightly each year from 2014 to 2018. There was a slight decrease in the number of deaths from ovarian cancer each year from 2015 to 2019. Anything that increases your chance of getting a disease is called a risk factor. Anything that decreases your chance of getting a disease is called a protective factor. For information about risk factors and protective factors for ovarian cancer, see the Ovarian, Fallopian Tube, and Primary Peritoneal Cancer Prevention summary. Talk to your doctor about your risk of ovarian cancer. Scientists study screening tests to find those with the fewest harms and most benefits. Cancer screening trials also are meant to show whether early detection (finding cancer before it causes symptoms) helps a person live longer or decreases a person's chance of dying from the disease. For some types of cancer, the chance of recovery is better if the disease is found and treated at an early stage. Pelvic exams to screen for ovarian cancer have not been shown to decrease the number of deaths from the disease. A pelvic exam is an exam of the vagina, cervix, uterus, fallopian tubes, ovaries, and rectum. A speculum is inserted into the vagina and the doctor or nurse looks at the vagina and cervix for signs of disease. The doctor or nurse also inserts one or two lubricated, gloved fingers of one hand into the vagina and places the other hand over the lower abdomen to feel the size, shape, and position of the uterus and ovaries. The doctor or nurse also inserts a lubricated, gloved finger into the rectum to feel for lumps or abnormal areas.EnlargePelvic exam. A doctor or nurse inserts one or two lubricated, gloved fingers of one hand into the vagina and presses on the lower abdomen with the other hand. This is done to feel the size, shape, and position of the uterus and ovaries. The vagina, cervix, fallopian tubes, and rectum are also checked. Transvaginal ultrasound (TVU) to screen for ovarian cancer has not been shown to decrease the number of deaths from the disease. TVU is a procedure used to examine the vagina, uterus, fallopian tubes, and bladder. An ultrasound transducer (probe) is inserted into the vagina and used to bounce high-energy sound waves (ultrasound) off internal tissues or organs and make echoes. The echoes form a picture of body tissues called a sonogram. EnlargeTransvaginal ultrasound. An ultrasound probe connected to a computer is inserted into the vagina and is gently moved to show different organs. The probe bounces sound waves off internal organs and tissues to make echoes that form a sonogram (computer picture). A CA-125 assay is a test that measures the level of CA-125 in the blood. CA-125 is a type of biomarker (a substance released by cells into the bloodstream that may be a sign of a condition or disease). An increased CA-125 level is sometimes a sign of certain types of cancer, including ovarian cancer, or other conditions. Studies have also shown that using CA-125 levels and TVU together to screen for ovarian cancer does not decrease the number of deaths from ovarian cancer. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Decisions about screening tests can be difficult. Not all screening tests are helpful and most have risks. Before having any screening test, you may want to talk about the test with your doctor. It is important to know the risks of the test and whether it has been proven to reduce the risk of dying from cancer. Screening may not improve your health or help you live longer if you have advanced ovarian cancer or if it has already spread to other places in your body. Screening test results may appear to be normal even though ovarian cancer is present. A woman who receives a false-negative test result (one that shows there is no cancer when there really is) may delay seeking medical care even if she has symptoms. Screening test results may appear to be abnormal even though no cancer is present. A false-positive test result (one that shows there is cancer when there really isn't) can cause anxiety and is usually followed by more tests (such as a laparoscopy or a laparotomy to see if cancer is present), which also have risks. Problems caused by tests used to diagnose ovarian cancer include infection, blood loss, bowel injury, and heart and blood vessel problems. A false-positive test result can also lead to an unneeded oophorectomy (removal of one or both ovaries). Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about ovarian, fallopian tube, and primary peritoneal cancer screening. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Screening and Prevention Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Screening and Prevention Editorial Board. PDQ Ovarian, Fallopian Tube, and Primary Peritoneal Cancer Screening. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/ovarian/patient/ovarian-screening-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389490] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Ovarian, Fallopian Tube, and Primary Peritoneal Cancer Screening (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

Who is at risk for Ovarian, Fallopian Tube, and Primary Peritoneal Cancer? ?

Different factors increase or decrease the risk of getting ovarian, fallopian tube, and primary peritoneal cancer. Anything that increases your chance of getting a disease is called a risk factor. Anything that decreases your chance of getting a disease is called a protective factor. For information about risk factors and protective factors for ovarian cancer, see the Ovarian, Fallopian Tube, and Primary Peritoneal Cancer Prevention summary. Talk to your doctor about your risk of ovarian cancer. Screening tests have risks. Decisions about screening tests can be difficult. Not all screening tests are helpful and most have risks. Before having any screening test, you may want to talk about the test with your doctor. It is important to know the risks of the test and whether it has been proven to reduce the risk of dying from cancer. The risks of ovarian, fallopian tube, and primary peritoneal cancer screening tests include the following: Finding ovarian, fallopian tube, and primary peritoneal cancer may not improve health or help a woman live longer. Screening may not improve your health or help you live longer if you have advanced ovarian cancer or if it has already spread to other places in your body. False-negative test results can occur. Screening test results may appear to be normal even though ovarian cancer is present. A woman who receives a false-negative test result (one that shows there is no cancer when there really is) may delay seeking medical care even if she has symptoms. False-positive test results can occur. Screening test results may appear to be abnormal even though no cancer is present. A false-positive test result (one that shows there is cancer when there really isn&apos;t) can cause anxiety and is usually followed by more tests (such as a laparoscopy or a laparotomy to see if cancer is present), which also have risks. Problems caused by tests used to diagnose ovarian cancer include infection, blood loss, bowel injury, and heart and blood vessel problems. A false-positive test result can also lead to an unneeded oophorectomy (removal of one or both ovaries).

Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In myeloproliferative neoplasms, too many blood stem cells become one or more types of blood cells. The neoplasms usually get worse slowly as the number of extra blood cells increases. The type of myeloproliferative neoplasm is based on whether too many red blood cells, white blood cells, or platelets are being made. Sometimes the body will make too many of more than one type of blood cell, but usually one type of blood cell is affected more than the others are. Chronic myeloproliferative neoplasms include the following 6 types: These types are described below. Chronic myeloproliferative neoplasms sometimes become acute leukemia, in which too many abnormal white blood cells are made. The following tests and procedures may be used: Chronic myelogenous leukemia is a disease in which too many white blood cells are made in the bone marrow. See the PDQ summary on Chronic Myelogenous Leukemia Treatment for information on diagnosis, staging, and treatment. In polycythemia vera, the blood becomes thickened with too many red blood cells. The number of white blood cells and platelets may also increase. These extra blood cells may collect in the spleen and cause it to swell. The increased number of red blood cells, white blood cells, or platelets in the blood can cause bleeding problems and make clots form in blood vessels. This can increase the risk of stroke or heart attack. In patients who are older than 65 years or who have a history of blood clots, the risk of stroke or heart attack is higher. Patients also have an increased risk of acute myeloid leukemia or primary myelofibrosis. Polycythemia vera often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may occur as the number of blood cells increases. Other conditions may cause the same signs and symptoms. Check with your doctor if you have any of the following: In addition to a complete blood count, bone marrow aspiration and biopsy, and cytogenetic analysis, a serum erythropoietin test is used to diagnose polycythemia vera. In this test, a sample of blood is checked for the level of erythropoietin (a hormone that stimulates new red blood cells to be made). In polycythemia vera, the erythropoietin level would be lower than normal because the body does not need to make more red blood cells. The bone marrow is made of tissues that make blood cells (red blood cells, white blood cells, and platelets) and a web of fibers that support the blood-forming tissues. In primary myelofibrosis (also called chronic idiopathic myelofibrosis), large numbers of blood stem cells become blood cells that do not mature properly (blasts). The web of fibers inside the bone marrow also becomes very thick (like scar tissue) and slows the blood-forming tissue’s ability to make blood cells. This causes the blood-forming tissues to make fewer and fewer blood cells. In order to make up for the low number of blood cells made in the bone marrow, the liver and spleen begin to make the blood cells. Primary myelofibrosis often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by primary myelofibrosis or by other conditions. Check with your doctor if you have any of the following: Prognosis depends on the following: Essential thrombocythemia causes an abnormal increase in the number of platelets made in the blood and bone marrow. Essential thrombocythemia often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by essential thrombocythemia or by other conditions. Check with your doctor if you have any of the following: Platelets are sticky. When there are too many platelets, they may clump together and make it hard for the blood to flow. Clots may form in blood vessels and there may also be increased bleeding. These can cause serious health problems such as stroke or heart attack. Prognosis and treatment options depend on the following: Chronic neutrophilic leukemia is a disease in which too many blood stem cells become a type of white blood cell called neutrophils. Neutrophils are infection-fighting blood cells that surround and destroy dead cells and foreign substances (such as bacteria). The spleen and liver may swell because of the extra neutrophils. Chronic neutrophilic leukemia may stay the same or it may progress quickly to acute leukemia. Eosinophils are white blood cells that react to allergens (substances that cause an allergic response) and help fight infections caused by certain parasites. In chronic eosinophilic leukemia, there are too many eosinophils in the blood, bone marrow, and other tissues. Chronic eosinophilic leukemia may stay the same for many years or it may progress quickly to acute leukemia. Chronic eosinophilic leukemia may not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by chronic eosinophilic leukemia or by other conditions. Check with your doctor if you have any of the following: The process used to find out if cancer has spread to other parts of the body is called staging. There is no standard staging system for chronic myeloproliferative neoplasms. It is important to know the type of myeloproliferative neoplasm in order to plan treatment. Different types of treatments are available for patients with chronic myeloproliferative neoplasms. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Watchful waiting is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. Phlebotomy is a procedure in which blood is taken from a vein. A sample of blood may be taken for tests such as a CBC or blood chemistry. Sometimes phlebotomy is used as a treatment and blood is taken from the body to remove extra red blood cells. Phlebotomy is used in this way to treat some chronic myeloproliferative neoplasms. Platelet apheresis is a treatment that uses a special machine to remove platelets from the blood. Blood is taken from the patient and put through a blood cell separator where the platelets are removed. The rest of the blood is then returned to the patient’s bloodstream. Transfusion therapy (blood transfusion) is a method of giving red blood cells, white blood cells, or platelets to replace blood cells destroyed by disease or cancer treatment. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). See Drugs Approved for Myeloproliferative Neoplasms for more information. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body, such as the spleen, with cancer. Prednisone and danazol are drugs that may be used to treat anemia in patients with primary myelofibrosis. Anagrelide therapy is used to reduce the risk of blood clots in patients who have too many platelets in their blood. Low-dose aspirin may also be used to reduce the risk of blood clots. Thalidomide, lenalidomide, and pomalidomide are drugs that prevent blood vessels from growing into areas of tumor cells. Erythropoietic growth factors are used to stimulate the bone marrow to make red blood cells. See Drugs Approved for Myeloproliferative Neoplasms for more information. Splenectomy (surgery to remove the spleen) may be done if the spleen is enlarged. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. See Drugs Approved for Myeloproliferative Neoplasms for more information. Other types of targeted therapies are being studied in clinical trials. High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. EnlargeStem cell transplant. (Step 1): Blood is taken from a vein in the arm of the donor. The patient or another person may be the donor. The blood flows through a machine that removes the stem cells. Then the blood is returned to the donor through a vein in the other arm. (Step 2): The patient receives chemotherapy to kill blood-forming cells. The patient may receive radiation therapy (not shown). (Step 3): The patient receives stem cells through a catheter placed into a blood vessel in the chest. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. See the PDQ summary about Chronic Myelogenous Leukemia Treatment for information. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. The purpose of treatment for polycythemia vera is to reduce the number of extra blood cells. Treatment of polycythemia vera may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of primary myelofibrosis in patients without signs or symptoms is usually watchful waiting. Patients with primary myelofibrosis may have signs or symptoms of anemia. Anemia is usually treated with transfusion of red blood cells to relieve symptoms and improve quality of life. In addition, anemia may be treated with: Treatment of primary myelofibrosis in patients with other signs or symptoms may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of essential thrombocythemia in patients younger than 60 years who have no signs or symptoms and an acceptable platelet count is usually watchful waiting. Treatment of other patients may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic neutrophilic leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic eosinophilic leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about chronic myeloproliferative neoplasms, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of chronic myeloproliferative neoplasms. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Chronic Myeloproliferative Neoplasms Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/myeloproliferative/patient/chronic-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389435] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Chronic Myeloproliferative Neoplasms Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is (are) Chronic Myelogenous Leukemia ?

Chronic myelogenous leukemia is a disease in which too many white blood cells are made in the bone marrow. See the PDQ summary on Chronic Myelogenous Leukemia Treatment for information on diagnosis, staging, and treatment.

Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells: In myeloproliferative neoplasms, too many blood stem cells become one or more types of blood cells. The neoplasms usually get worse slowly as the number of extra blood cells increases. The type of myeloproliferative neoplasm is based on whether too many red blood cells, white blood cells, or platelets are being made. Sometimes the body will make too many of more than one type of blood cell, but usually one type of blood cell is affected more than the others are. Chronic myeloproliferative neoplasms include the following 6 types: These types are described below. Chronic myeloproliferative neoplasms sometimes become acute leukemia, in which too many abnormal white blood cells are made. The following tests and procedures may be used: Chronic myelogenous leukemia is a disease in which too many white blood cells are made in the bone marrow. See the PDQ summary on Chronic Myelogenous Leukemia Treatment for information on diagnosis, staging, and treatment. In polycythemia vera, the blood becomes thickened with too many red blood cells. The number of white blood cells and platelets may also increase. These extra blood cells may collect in the spleen and cause it to swell. The increased number of red blood cells, white blood cells, or platelets in the blood can cause bleeding problems and make clots form in blood vessels. This can increase the risk of stroke or heart attack. In patients who are older than 65 years or who have a history of blood clots, the risk of stroke or heart attack is higher. Patients also have an increased risk of acute myeloid leukemia or primary myelofibrosis. Polycythemia vera often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may occur as the number of blood cells increases. Other conditions may cause the same signs and symptoms. Check with your doctor if you have any of the following: In addition to a complete blood count, bone marrow aspiration and biopsy, and cytogenetic analysis, a serum erythropoietin test is used to diagnose polycythemia vera. In this test, a sample of blood is checked for the level of erythropoietin (a hormone that stimulates new red blood cells to be made). In polycythemia vera, the erythropoietin level would be lower than normal because the body does not need to make more red blood cells. The bone marrow is made of tissues that make blood cells (red blood cells, white blood cells, and platelets) and a web of fibers that support the blood-forming tissues. In primary myelofibrosis (also called chronic idiopathic myelofibrosis), large numbers of blood stem cells become blood cells that do not mature properly (blasts). The web of fibers inside the bone marrow also becomes very thick (like scar tissue) and slows the blood-forming tissue’s ability to make blood cells. This causes the blood-forming tissues to make fewer and fewer blood cells. In order to make up for the low number of blood cells made in the bone marrow, the liver and spleen begin to make the blood cells. Primary myelofibrosis often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by primary myelofibrosis or by other conditions. Check with your doctor if you have any of the following: Prognosis depends on the following: Essential thrombocythemia causes an abnormal increase in the number of platelets made in the blood and bone marrow. Essential thrombocythemia often does not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by essential thrombocythemia or by other conditions. Check with your doctor if you have any of the following: Platelets are sticky. When there are too many platelets, they may clump together and make it hard for the blood to flow. Clots may form in blood vessels and there may also be increased bleeding. These can cause serious health problems such as stroke or heart attack. Prognosis and treatment options depend on the following: Chronic neutrophilic leukemia is a disease in which too many blood stem cells become a type of white blood cell called neutrophils. Neutrophils are infection-fighting blood cells that surround and destroy dead cells and foreign substances (such as bacteria). The spleen and liver may swell because of the extra neutrophils. Chronic neutrophilic leukemia may stay the same or it may progress quickly to acute leukemia. Eosinophils are white blood cells that react to allergens (substances that cause an allergic response) and help fight infections caused by certain parasites. In chronic eosinophilic leukemia, there are too many eosinophils in the blood, bone marrow, and other tissues. Chronic eosinophilic leukemia may stay the same for many years or it may progress quickly to acute leukemia. Chronic eosinophilic leukemia may not cause early signs or symptoms. It may be found during a routine blood test. Signs and symptoms may be caused by chronic eosinophilic leukemia or by other conditions. Check with your doctor if you have any of the following: The process used to find out if cancer has spread to other parts of the body is called staging. There is no standard staging system for chronic myeloproliferative neoplasms. It is important to know the type of myeloproliferative neoplasm in order to plan treatment. Different types of treatments are available for patients with chronic myeloproliferative neoplasms. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Watchful waiting is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. Phlebotomy is a procedure in which blood is taken from a vein. A sample of blood may be taken for tests such as a CBC or blood chemistry. Sometimes phlebotomy is used as a treatment and blood is taken from the body to remove extra red blood cells. Phlebotomy is used in this way to treat some chronic myeloproliferative neoplasms. Platelet apheresis is a treatment that uses a special machine to remove platelets from the blood. Blood is taken from the patient and put through a blood cell separator where the platelets are removed. The rest of the blood is then returned to the patient’s bloodstream. Transfusion therapy (blood transfusion) is a method of giving red blood cells, white blood cells, or platelets to replace blood cells destroyed by disease or cancer treatment. Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). See Drugs Approved for Myeloproliferative Neoplasms for more information. Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body, such as the spleen, with cancer. Prednisone and danazol are drugs that may be used to treat anemia in patients with primary myelofibrosis. Anagrelide therapy is used to reduce the risk of blood clots in patients who have too many platelets in their blood. Low-dose aspirin may also be used to reduce the risk of blood clots. Thalidomide, lenalidomide, and pomalidomide are drugs that prevent blood vessels from growing into areas of tumor cells. Erythropoietic growth factors are used to stimulate the bone marrow to make red blood cells. See Drugs Approved for Myeloproliferative Neoplasms for more information. Splenectomy (surgery to remove the spleen) may be done if the spleen is enlarged. Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy. Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. See Drugs Approved for Myeloproliferative Neoplasms for more information. Other types of targeted therapies are being studied in clinical trials. High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells. EnlargeStem cell transplant. (Step 1): Blood is taken from a vein in the arm of the donor. The patient or another person may be the donor. The blood flows through a machine that removes the stem cells. Then the blood is returned to the donor through a vein in the other arm. (Step 2): The patient receives chemotherapy to kill blood-forming cells. The patient may receive radiation therapy (not shown). (Step 3): The patient receives stem cells through a catheter placed into a blood vessel in the chest. Information about clinical trials is available from the NCI website. For information about side effects caused by treatment for cancer, see our Side Effects page. For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment. Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment. Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward. Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment. Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website. Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups. See the PDQ summary about Chronic Myelogenous Leukemia Treatment for information. Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. The purpose of treatment for polycythemia vera is to reduce the number of extra blood cells. Treatment of polycythemia vera may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of primary myelofibrosis in patients without signs or symptoms is usually watchful waiting. Patients with primary myelofibrosis may have signs or symptoms of anemia. Anemia is usually treated with transfusion of red blood cells to relieve symptoms and improve quality of life. In addition, anemia may be treated with: Treatment of primary myelofibrosis in patients with other signs or symptoms may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of essential thrombocythemia in patients younger than 60 years who have no signs or symptoms and an acceptable platelet count is usually watchful waiting. Treatment of other patients may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic neutrophilic leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For information about the treatments listed below, see the Treatment Option Overview section. Treatment of chronic eosinophilic leukemia may include the following: Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available. For more information from the National Cancer Institute about chronic myeloproliferative neoplasms, see the following: For general cancer information and other resources from the National Cancer Institute, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of chronic myeloproliferative neoplasms. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Adult Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Adult Treatment Editorial Board. PDQ Chronic Myeloproliferative Neoplasms Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/myeloproliferative/patient/chronic-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389435] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Chronic Myeloproliferative Neoplasms Treatment (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the treatments for Chronic Myelogenous Leukemia ?

See the PDQ summary about Chronic Myelogenous Leukemia Treatment for information.

There are many types of childhood brain and spinal cord tumors. The tumors are formed by the abnormal growth of cells and may begin in different areas of the brain or spinal cord. The tumors may be benign (not cancer) or malignant (cancer). Benign brain tumors may grow and press on nearby areas of the brain. They rarely spread into other brain tissue. Malignant brain tumors may be low grade or high grade. High-grade tumors are likely to grow quickly and spread into other brain tissue. Low-grade tumors tend to grow and spread more slowly than high-grade tumors. When a tumor grows into or presses on an area of the brain, it may stop that part of the brain from working the way it should. Both benign and malignant brain tumors can cause signs or symptoms, need treatment, and can recur (come back). Together, the brain and spinal cord make up the central nervous system (CNS). This summary is about primary benign and malignant brain and spinal cord tumors. The brain has three major parts: The spinal cord is a column of nerve tissue that runs from the brain stem down the center of the back. It is covered by three thin layers of tissue called membranes. The spinal cord and membranes are surrounded by the vertebrae (back bones). Spinal cord nerves carry messages between the brain and the rest of the body, such as a message from the brain to cause muscles to move or a message from the skin to the brain to feel touch. Although cancer is rare in children, brain and spinal cord tumors are the second most common type of childhood cancer, after leukemia. Brain tumors can occur in both children and adults. Treatment for children is usually different than treatment for adults. (See the PDQ summary on Adult Central Nervous System Tumors Treatment for more information about the treatment of adults.) Metastatic tumors are formed by cancer cells that begin in other parts of the body and spread to the brain or spinal cord. Treatment of metastatic brain and spinal cord tumors is not covered in this summary. Signs and symptoms depend on the following: Signs and symptoms may be caused by childhood brain and spinal cord tumors or by other conditions. Check with your child's doctor if your child has any of the following: Brain Tumor Signs and Symptoms Spinal Cord Tumor Signs and Symptoms In addition to these signs and symptoms of brain and spinal cord tumors, some children are unable to reach certain growth and development milestones such as sitting up, walking, and talking in sentences. The following tests and procedures may be used: If doctors think there might be a brain tumor, a biopsy may be done to remove a sample of tissue. For tumors in the brain, a part of the skull is removed and a needle is used to remove a sample of tissue. Sometimes, the needle is guided by a computer. A pathologist views the tissue under a microscope to look for cancer cells. If cancer cells are found, the doctor may remove as much tumor as safely possible during the same surgery. The pathologist checks the cancer cells to find out the type and grade of brain tumor. The grade of the tumor is based on how abnormal the cancer cells look under a microscope and how quickly the tumor is likely to grow and spread. The following test may be done on the sample of tissue that is removed: Sometimes a biopsy or surgery cannot be done safely because of where the tumor formed in the brain or spinal cord. These tumors are diagnosed based on the results of imaging tests and other procedures. The prognosis depends on the following: The brain and spinal cord are made of different kinds of cells. Childhood brain tumors and spinal cord tumors can be benign or malignant and are grouped and treated based on the type of cell the tumor formed in and where the tumor began growing in the central nervous system (CNS). Some types of tumors are divided into subtypes based on how the tumor looks under a microscope and whether it has certain gene changes. See the list below for more information about staging and treatment of newly diagnosed and recurrent childhood brain and spinal cord tumors. See the PDQ summary on Childhood Astrocytomas Treatment for more information on astrocytomas, gliomas, xanthroastrocytomas, and neurofibromatosis type 1 (NF1). See the PDQ summary on Childhood Brain Stem Glioma Treatment for more information on diffuse intrinsic pontine gliomas and focal gliomas. See the PDQ summary on Childhood Central Nervous System Atypical Teratoid/Rhabdoid Tumor Treatment for more information. See the PDQ summary on Childhood Central Nervous System Germ Cell Tumors Treatment for more information on germinomas, embryonal carcinomas, yolk sac tumors, choriocarcinoma, mature teratomas, immature teratomas, teratoma with malignant transformation, and mixed germ cell tumors. See the PDQ summary on Childhood Craniopharyngioma Treatment for more information. See the PDQ summary on Childhood Ependymoma Treatment for more information. See the PDQ summary on Childhood Medulloblastoma and Other Central Nervous System Embryonal Tumors Treatment for more information on medulloblastomas, embryonal tumors, and pineoblastomas. For more information about childhood brain and spinal cord tumors, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood brain and spinal cord tumors. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Brain and Spinal Cord Tumors Treatment Overview. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/brain/patient/child-brain-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389351] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Childhood Brain and Spinal Cord Tumors Treatment Overview (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What is (are) Childhood Brain and Spinal Cord Tumors ?

Key Points - A childhood brain or spinal cord tumor is a disease in which abnormal cells form in the tissues of the brain or spinal cord. - The brain controls many important body functions. - The spinal cord connects the brain with nerves in most parts of the body. - Brain and spinal cord tumors are a common type of childhood cancer. - The cause of most childhood brain and spinal cord tumors is unknown. - The signs and symptoms of childhood brain and spinal cord tumors are not the same in every child. - Tests that examine the brain and spinal cord are used to detect (find) childhood brain and spinal cord tumors. - Most childhood brain tumors are diagnosed and removed in surgery. - Some childhood brain and spinal cord tumors are diagnosed by imaging tests. - Certain factors affect prognosis (chance of recovery). A childhood brain or spinal cord tumor is a disease in which abnormal cells form in the tissues of the brain or spinal cord. There are many types of childhood brain and spinal cord tumors. The tumors are formed by the abnormal growth of cells and may begin in different areas of the brain or spinal cord. The tumors may be benign (not cancer) or malignant (cancer). Benign brain tumors grow and press on nearby areas of the brain. They rarely spread into other tissues. Malignant brain tumors are likely to grow quickly and spread into other brain tissue. When a tumor grows into or presses on an area of the brain, it may stop that part of the brain from working the way it should. Both benign and malignant brain tumors can cause signs or symptoms and need treatment. Together, the brain and spinal cord make up the central nervous system (CNS). The brain controls many important body functions. The brain has three major parts: - The cerebrum is the largest part of the brain. It is at the top of the head. The cerebrum controls thinking, learning, problem solving, emotions, speech, reading, writing, and voluntary movement. - The cerebellum is in the lower back of the brain (near the middle of the back of the head). It controls movement, balance, and posture. - The brain stem connects the brain to the spinal cord. It is in the lowest part of the brain (just above the back of the neck). The brain stem controls breathing, heart rate, and the nerves and muscles used in seeing, hearing, walking, talking, and eating. The spinal cord connects the brain with nerves in most parts of the body. The spinal cord is a column of nerve tissue that runs from the brain stem down the center of the back. It is covered by three thin layers of tissue called membranes. These membranes are surrounded by the vertebrae (back bones). Spinal cord nerves carry messages between the brain and the rest of the body, such as a message from the brain to cause muscles to move or a message from the skin to the brain to feel touch. Brain and spinal cord tumors are a common type of childhood cancer. Although cancer is rare in children, brain and spinal cord tumors are the third most common type of childhood cancer, after leukemia and lymphoma. Brain tumors can occur in both children and adults. Treatment for children is usually different than treatment for adults. (See the PDQ summary on Adult Central Nervous System Tumors Treatment for more information about the treatment of adults.) This summary describes the treatment of primary brain and spinal cord tumors (tumors that begin in the brain and spinal cord). Treatment of metastatic brain and spinal cord tumors is not covered in this summary. Metastatic tumors are formed by cancer cells that begin in other parts of the body and spread to the brain or spinal cord.

There are many types of childhood brain and spinal cord tumors. The tumors are formed by the abnormal growth of cells and may begin in different areas of the brain or spinal cord. The tumors may be benign (not cancer) or malignant (cancer). Benign brain tumors may grow and press on nearby areas of the brain. They rarely spread into other brain tissue. Malignant brain tumors may be low grade or high grade. High-grade tumors are likely to grow quickly and spread into other brain tissue. Low-grade tumors tend to grow and spread more slowly than high-grade tumors. When a tumor grows into or presses on an area of the brain, it may stop that part of the brain from working the way it should. Both benign and malignant brain tumors can cause signs or symptoms, need treatment, and can recur (come back). Together, the brain and spinal cord make up the central nervous system (CNS). This summary is about primary benign and malignant brain and spinal cord tumors. The brain has three major parts: The spinal cord is a column of nerve tissue that runs from the brain stem down the center of the back. It is covered by three thin layers of tissue called membranes. The spinal cord and membranes are surrounded by the vertebrae (back bones). Spinal cord nerves carry messages between the brain and the rest of the body, such as a message from the brain to cause muscles to move or a message from the skin to the brain to feel touch. Although cancer is rare in children, brain and spinal cord tumors are the second most common type of childhood cancer, after leukemia. Brain tumors can occur in both children and adults. Treatment for children is usually different than treatment for adults. (See the PDQ summary on Adult Central Nervous System Tumors Treatment for more information about the treatment of adults.) Metastatic tumors are formed by cancer cells that begin in other parts of the body and spread to the brain or spinal cord. Treatment of metastatic brain and spinal cord tumors is not covered in this summary. Signs and symptoms depend on the following: Signs and symptoms may be caused by childhood brain and spinal cord tumors or by other conditions. Check with your child's doctor if your child has any of the following: Brain Tumor Signs and Symptoms Spinal Cord Tumor Signs and Symptoms In addition to these signs and symptoms of brain and spinal cord tumors, some children are unable to reach certain growth and development milestones such as sitting up, walking, and talking in sentences. The following tests and procedures may be used: If doctors think there might be a brain tumor, a biopsy may be done to remove a sample of tissue. For tumors in the brain, a part of the skull is removed and a needle is used to remove a sample of tissue. Sometimes, the needle is guided by a computer. A pathologist views the tissue under a microscope to look for cancer cells. If cancer cells are found, the doctor may remove as much tumor as safely possible during the same surgery. The pathologist checks the cancer cells to find out the type and grade of brain tumor. The grade of the tumor is based on how abnormal the cancer cells look under a microscope and how quickly the tumor is likely to grow and spread. The following test may be done on the sample of tissue that is removed: Sometimes a biopsy or surgery cannot be done safely because of where the tumor formed in the brain or spinal cord. These tumors are diagnosed based on the results of imaging tests and other procedures. The prognosis depends on the following: The brain and spinal cord are made of different kinds of cells. Childhood brain tumors and spinal cord tumors can be benign or malignant and are grouped and treated based on the type of cell the tumor formed in and where the tumor began growing in the central nervous system (CNS). Some types of tumors are divided into subtypes based on how the tumor looks under a microscope and whether it has certain gene changes. See the list below for more information about staging and treatment of newly diagnosed and recurrent childhood brain and spinal cord tumors. See the PDQ summary on Childhood Astrocytomas Treatment for more information on astrocytomas, gliomas, xanthroastrocytomas, and neurofibromatosis type 1 (NF1). See the PDQ summary on Childhood Brain Stem Glioma Treatment for more information on diffuse intrinsic pontine gliomas and focal gliomas. See the PDQ summary on Childhood Central Nervous System Atypical Teratoid/Rhabdoid Tumor Treatment for more information. See the PDQ summary on Childhood Central Nervous System Germ Cell Tumors Treatment for more information on germinomas, embryonal carcinomas, yolk sac tumors, choriocarcinoma, mature teratomas, immature teratomas, teratoma with malignant transformation, and mixed germ cell tumors. See the PDQ summary on Childhood Craniopharyngioma Treatment for more information. See the PDQ summary on Childhood Ependymoma Treatment for more information. See the PDQ summary on Childhood Medulloblastoma and Other Central Nervous System Embryonal Tumors Treatment for more information on medulloblastomas, embryonal tumors, and pineoblastomas. For more information about childhood brain and spinal cord tumors, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood brain and spinal cord tumors. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Brain and Spinal Cord Tumors Treatment Overview. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/brain/patient/child-brain-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389351] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Childhood Brain and Spinal Cord Tumors Treatment Overview (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What causes Childhood Brain and Spinal Cord Tumors ?

The cause of most childhood brain and spinal cord tumors is unknown.

There are many types of childhood brain and spinal cord tumors. The tumors are formed by the abnormal growth of cells and may begin in different areas of the brain or spinal cord. The tumors may be benign (not cancer) or malignant (cancer). Benign brain tumors may grow and press on nearby areas of the brain. They rarely spread into other brain tissue. Malignant brain tumors may be low grade or high grade. High-grade tumors are likely to grow quickly and spread into other brain tissue. Low-grade tumors tend to grow and spread more slowly than high-grade tumors. When a tumor grows into or presses on an area of the brain, it may stop that part of the brain from working the way it should. Both benign and malignant brain tumors can cause signs or symptoms, need treatment, and can recur (come back). Together, the brain and spinal cord make up the central nervous system (CNS). This summary is about primary benign and malignant brain and spinal cord tumors. The brain has three major parts: The spinal cord is a column of nerve tissue that runs from the brain stem down the center of the back. It is covered by three thin layers of tissue called membranes. The spinal cord and membranes are surrounded by the vertebrae (back bones). Spinal cord nerves carry messages between the brain and the rest of the body, such as a message from the brain to cause muscles to move or a message from the skin to the brain to feel touch. Although cancer is rare in children, brain and spinal cord tumors are the second most common type of childhood cancer, after leukemia. Brain tumors can occur in both children and adults. Treatment for children is usually different than treatment for adults. (See the PDQ summary on Adult Central Nervous System Tumors Treatment for more information about the treatment of adults.) Metastatic tumors are formed by cancer cells that begin in other parts of the body and spread to the brain or spinal cord. Treatment of metastatic brain and spinal cord tumors is not covered in this summary. Signs and symptoms depend on the following: Signs and symptoms may be caused by childhood brain and spinal cord tumors or by other conditions. Check with your child's doctor if your child has any of the following: Brain Tumor Signs and Symptoms Spinal Cord Tumor Signs and Symptoms In addition to these signs and symptoms of brain and spinal cord tumors, some children are unable to reach certain growth and development milestones such as sitting up, walking, and talking in sentences. The following tests and procedures may be used: If doctors think there might be a brain tumor, a biopsy may be done to remove a sample of tissue. For tumors in the brain, a part of the skull is removed and a needle is used to remove a sample of tissue. Sometimes, the needle is guided by a computer. A pathologist views the tissue under a microscope to look for cancer cells. If cancer cells are found, the doctor may remove as much tumor as safely possible during the same surgery. The pathologist checks the cancer cells to find out the type and grade of brain tumor. The grade of the tumor is based on how abnormal the cancer cells look under a microscope and how quickly the tumor is likely to grow and spread. The following test may be done on the sample of tissue that is removed: Sometimes a biopsy or surgery cannot be done safely because of where the tumor formed in the brain or spinal cord. These tumors are diagnosed based on the results of imaging tests and other procedures. The prognosis depends on the following: The brain and spinal cord are made of different kinds of cells. Childhood brain tumors and spinal cord tumors can be benign or malignant and are grouped and treated based on the type of cell the tumor formed in and where the tumor began growing in the central nervous system (CNS). Some types of tumors are divided into subtypes based on how the tumor looks under a microscope and whether it has certain gene changes. See the list below for more information about staging and treatment of newly diagnosed and recurrent childhood brain and spinal cord tumors. See the PDQ summary on Childhood Astrocytomas Treatment for more information on astrocytomas, gliomas, xanthroastrocytomas, and neurofibromatosis type 1 (NF1). See the PDQ summary on Childhood Brain Stem Glioma Treatment for more information on diffuse intrinsic pontine gliomas and focal gliomas. See the PDQ summary on Childhood Central Nervous System Atypical Teratoid/Rhabdoid Tumor Treatment for more information. See the PDQ summary on Childhood Central Nervous System Germ Cell Tumors Treatment for more information on germinomas, embryonal carcinomas, yolk sac tumors, choriocarcinoma, mature teratomas, immature teratomas, teratoma with malignant transformation, and mixed germ cell tumors. See the PDQ summary on Childhood Craniopharyngioma Treatment for more information. See the PDQ summary on Childhood Ependymoma Treatment for more information. See the PDQ summary on Childhood Medulloblastoma and Other Central Nervous System Embryonal Tumors Treatment for more information on medulloblastomas, embryonal tumors, and pineoblastomas. For more information about childhood brain and spinal cord tumors, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood brain and spinal cord tumors. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Brain and Spinal Cord Tumors Treatment Overview. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/brain/patient/child-brain-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389351] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Childhood Brain and Spinal Cord Tumors Treatment Overview (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

What are the symptoms of Childhood Brain and Spinal Cord Tumors ?

The signs and symptoms of childhood brain and spinal cord tumors are not the same in every child. Signs and symptoms depend on the following: - Where the tumor forms in the brain or spinal cord. - The size of the tumor. - How fast the tumor grows. - The child&apos;s age and development. Signs and symptoms may be caused by childhood brain and spinal cord tumors or by other conditions, including cancer that has spread to the brain. Check with your child&apos;s doctor if your child has any of the following: Brain Tumor Signs and Symptoms - Morning headache or headache that goes away after vomiting. - Frequent nausea and vomiting. - Vision, hearing, and speech problems. - Loss of balance and trouble walking. - Unusual sleepiness or change in activity level. - Unusual changes in personality or behavior. - Seizures. - Increase in the head size (in infants). Spinal Cord Tumor Signs and Symptoms - Back pain or pain that spreads from the back towards the arms or legs. - A change in bowel habits or trouble urinating. - Weakness in the legs. - Trouble walking. In addition to these signs and symptoms of brain and spinal cord tumors, some children are unable to reach certain growth and development milestones such as sitting up, walking, and talking in sentences.

There are many types of childhood brain and spinal cord tumors. The tumors are formed by the abnormal growth of cells and may begin in different areas of the brain or spinal cord. The tumors may be benign (not cancer) or malignant (cancer). Benign brain tumors may grow and press on nearby areas of the brain. They rarely spread into other brain tissue. Malignant brain tumors may be low grade or high grade. High-grade tumors are likely to grow quickly and spread into other brain tissue. Low-grade tumors tend to grow and spread more slowly than high-grade tumors. When a tumor grows into or presses on an area of the brain, it may stop that part of the brain from working the way it should. Both benign and malignant brain tumors can cause signs or symptoms, need treatment, and can recur (come back). Together, the brain and spinal cord make up the central nervous system (CNS). This summary is about primary benign and malignant brain and spinal cord tumors. The brain has three major parts: The spinal cord is a column of nerve tissue that runs from the brain stem down the center of the back. It is covered by three thin layers of tissue called membranes. The spinal cord and membranes are surrounded by the vertebrae (back bones). Spinal cord nerves carry messages between the brain and the rest of the body, such as a message from the brain to cause muscles to move or a message from the skin to the brain to feel touch. Although cancer is rare in children, brain and spinal cord tumors are the second most common type of childhood cancer, after leukemia. Brain tumors can occur in both children and adults. Treatment for children is usually different than treatment for adults. (See the PDQ summary on Adult Central Nervous System Tumors Treatment for more information about the treatment of adults.) Metastatic tumors are formed by cancer cells that begin in other parts of the body and spread to the brain or spinal cord. Treatment of metastatic brain and spinal cord tumors is not covered in this summary. Signs and symptoms depend on the following: Signs and symptoms may be caused by childhood brain and spinal cord tumors or by other conditions. Check with your child's doctor if your child has any of the following: Brain Tumor Signs and Symptoms Spinal Cord Tumor Signs and Symptoms In addition to these signs and symptoms of brain and spinal cord tumors, some children are unable to reach certain growth and development milestones such as sitting up, walking, and talking in sentences. The following tests and procedures may be used: If doctors think there might be a brain tumor, a biopsy may be done to remove a sample of tissue. For tumors in the brain, a part of the skull is removed and a needle is used to remove a sample of tissue. Sometimes, the needle is guided by a computer. A pathologist views the tissue under a microscope to look for cancer cells. If cancer cells are found, the doctor may remove as much tumor as safely possible during the same surgery. The pathologist checks the cancer cells to find out the type and grade of brain tumor. The grade of the tumor is based on how abnormal the cancer cells look under a microscope and how quickly the tumor is likely to grow and spread. The following test may be done on the sample of tissue that is removed: Sometimes a biopsy or surgery cannot be done safely because of where the tumor formed in the brain or spinal cord. These tumors are diagnosed based on the results of imaging tests and other procedures. The prognosis depends on the following: The brain and spinal cord are made of different kinds of cells. Childhood brain tumors and spinal cord tumors can be benign or malignant and are grouped and treated based on the type of cell the tumor formed in and where the tumor began growing in the central nervous system (CNS). Some types of tumors are divided into subtypes based on how the tumor looks under a microscope and whether it has certain gene changes. See the list below for more information about staging and treatment of newly diagnosed and recurrent childhood brain and spinal cord tumors. See the PDQ summary on Childhood Astrocytomas Treatment for more information on astrocytomas, gliomas, xanthroastrocytomas, and neurofibromatosis type 1 (NF1). See the PDQ summary on Childhood Brain Stem Glioma Treatment for more information on diffuse intrinsic pontine gliomas and focal gliomas. See the PDQ summary on Childhood Central Nervous System Atypical Teratoid/Rhabdoid Tumor Treatment for more information. See the PDQ summary on Childhood Central Nervous System Germ Cell Tumors Treatment for more information on germinomas, embryonal carcinomas, yolk sac tumors, choriocarcinoma, mature teratomas, immature teratomas, teratoma with malignant transformation, and mixed germ cell tumors. See the PDQ summary on Childhood Craniopharyngioma Treatment for more information. See the PDQ summary on Childhood Ependymoma Treatment for more information. See the PDQ summary on Childhood Medulloblastoma and Other Central Nervous System Embryonal Tumors Treatment for more information on medulloblastomas, embryonal tumors, and pineoblastomas. For more information about childhood brain and spinal cord tumors, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish. PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH. This PDQ cancer information summary has current information about the treatment of childhood brain and spinal cord tumors. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care. Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change. The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board. A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment. Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237). PDQ is a registered trademark. The content of PDQ documents can be used freely as text. It cannot be identified as an NCI PDQ cancer information summary unless the whole summary is shown and it is updated regularly. However, a user would be allowed to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks in the following way: [include excerpt from the summary].” The best way to cite this PDQ summary is: PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Brain and Spinal Cord Tumors Treatment Overview. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/brain/patient/child-brain-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389351] Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images. The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page. More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website’s E-mail Us. If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Childhood Brain and Spinal Cord Tumors Treatment Overview (PDQ®)–Patient Version was originally published by the National Cancer Institute.” Want to use this content on your website or other digital platform? Our syndication services page shows you how.

How to diagnose Childhood Brain and Spinal Cord Tumors ?

Tests that examine the brain and spinal cord are used to detect (find) childhood brain and spinal cord tumors. The following tests and procedures may be used: - Physical exam and history : An exam of the body to check general signs of health, including checking for signs of disease, such as lumps or anything else that seems unusual. A history of the patients health habits and past illnesses and treatments will also be taken. - Neurological exam : A series of questions and tests to check the brain, spinal cord, and nerve function. The exam checks a persons mental status, coordination, and ability to walk normally, and how well the muscles, senses, and reflexes work. This may also be called a neuro exam or a neurologic exam. - MRI (magnetic resonance imaging) with gadolinium : A procedure that uses a magnet, radio waves, and a computer to make a series of detailed pictures of the brain and spinal cord. A substance called gadolinium is injected into a vein. The gadolinium collects around the cancer cells so they show up brighter in the picture. This procedure is also called nuclear magnetic resonance imaging (NMRI). - Serum tumor marker test : A procedure in which a sample of blood is examined to measure the amounts of certain substances released into the blood by organs, tissues, or tumor cells in the body. Certain substances are linked to specific types of cancer when found in increased levels in the blood. These are called tumor markers. Most childhood brain tumors are diagnosed and removed in surgery. If doctors think there might be a brain tumor, a biopsy may be done to remove a sample of tissue. For tumors in the brain, the biopsy is done by removing part of the skull and using a needle to remove a sample of tissue. A pathologist views the tissue under a microscope to look for cancer cells. If cancer cells are found, the doctor may remove as much tumor as safely possible during the same surgery. The pathologist checks the cancer cells to find out the type and grade of brain tumor. The grade of the tumor is based on how abnormal the cancer cells look under a microscope and how quickly the tumor is likely to grow and spread. The following test may be done on the sample of tissue that is removed: - Immunohistochemistry : A test that uses antibodies to check for certain antigens in a sample of tissue. The antibody is usually linked to a radioactive substance or a dye that causes the tissue to light up under a microscope. This type of test may be used to tell the difference between different types of cancer. Some childhood brain and spinal cord tumors are diagnosed by imaging tests. Sometimes a biopsy or surgery cannot be done safely because of where the tumor formed in the brain or spinal cord. These tumors are diagnosed based on the results of imaging tests and other procedures.

There are many types of childhood brain and spinal cord tumors. The tumors are formed by the abnormal growth of cells and may begin in different areas of the brain or spinal cord. The tumors may be benign (not cancer) or malignant (cancer). Benign brain tumors may grow and press on nearby areas of the brain. They rarely spread into other brain tissue. Malignant brain tumors may be low grade or high grade. High-grade tumors are likely to grow quickly and spread into other brain tissue. Low-grade tumors tend to grow and spread more slowly than high-grade tumors. When a tumor grows into or presses on an area of the brain, it may stop that part of the brain from working the way it should. Both benign and malignant brain tumors can cause signs or symptoms, need treatment, and can recur (come back). Together, the brain and spinal cord make up the central nervous system (CNS). This summary is about primary benign and malignant brain and spinal cord tumors. The brain has three major parts: The spinal cord is a column of nerve tissue that runs from the brain stem down the center of the back. It is covered by three thin layers of tissue called membranes. The spinal cord and membranes are surrounded by the vertebrae (back bones). Spinal cord nerves carry messages between the brain and the rest of the body, such as a message from the brain to cause muscles to move or a message from the skin to the brain to feel touch. Although cancer is rare in children, brain and spinal cord tumors are the second most common type of childhood cancer, after leukemia. Brain tumors can occur in both children and adults. Treatment for children is usually different than treatment for adults. (See the PDQ summary on Adult Central Nervous System Tumors Treatment for more information about the treatment of adults.) Metastatic tumors are formed by cancer cells that begin in other parts of the body and spread to the brain or spinal cord. Treatment of metastatic brain and spinal cord tumors is not covered in this summary. Signs and symptoms depend on the following: Signs and symptoms may be caused by childhood brain and spinal cord tumors or by other conditions. Check with your child's doctor if your child has any of the following: Brain Tumor Signs and Symptoms Spinal Cord Tumor Signs and Symptoms In addition to these signs and symptoms of brain and spinal cord tumors, some children are unable to reach certain growth and development milestones such as sitting up, walking, and talking in sentences. The following tests and procedures may be used: If doctors think there might be a brain tumor, a biopsy may be done to remove a sample of tissue. For tumors in the brain, a part of the skull is removed and a needle is used to remove a sample of tissue. Sometimes, the needle is guided by a computer. A pathologist views the tissue under a microscope to look for cancer cells. If cancer cells are found, the doctor may remove as much tumor as safely possible during the same surgery. The pathologist checks the cancer cells to find out the type and grade of brain tumor. The grade of the tumor is based on how abnormal the cancer cells look under a microscope and how quickly the tumor is likely to grow and spread. The following test may be done on the sample of tissue that is removed: Sometimes a biopsy or surgery cannot be done safely because of where the tumor formed in the brain or spinal cord. These tumors are diagnosed based on the results of imaging tests and other procedures. The prognosis depends on the following: The brain and spinal cord are made of different kinds of cells. Childhood brain tumors and spinal cord tumors can be benign or malignant and are grouped and treated based on the type of cell the tumor formed in and where the tumor began growing in the central nervous system (CNS). Some types of tumors are divided into subtypes based on how the tumor looks under a microscope and whether it has certain gene changes. See the list below for more information about staging and treatment of newly diagnosed and recurrent childhood brain and spinal cord tumors. See the PDQ summary on Childhood Astrocytomas Treatment for more information on astrocytomas, gliomas, xanthroastrocytomas, and neurofibromatosis type 1 (NF1). See the PDQ summary on Childhood Brain Stem Glioma Treatment for more information on diffuse intrinsic pontine gliomas and focal gliomas. See the PDQ summary on Childhood Central Nervous System Atypical Teratoid/Rhabdoid Tumor Treatment for more information. See the PDQ summary on Childhood Central Nervous System Germ Cell Tumors Treatment for more information on germinomas, embryonal carcinomas, yolk sac tumors, choriocarcinoma, mature teratomas, immature teratomas, teratoma with malignant transformation, and mixed germ cell tumors. See the PDQ summary on Childhood Craniopharyngioma Treatment for more information. See the PDQ summary on Childhood Ependymoma Treatment for more information. See the PDQ summary on Childhood Medulloblastoma and Other Central Nervous System Embryonal Tumors Treatment for more information on medulloblastomas, embryonal tumors, and pineoblastomas. For more information about childhood brain and spinal cord tumors, see the following: For more childhood cancer information and other general cancer resources, see the following: Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. 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What is the outlook for Childhood Brain and Spinal Cord Tumors ?

Certain factors affect prognosis (chance of recovery). The prognosis (chance of recovery) depends on the following: - Whether there are any cancer cells left after surgery. - The type of tumor. - Where the tumor is in the body. - The child&apos;s age. - Whether the tumor has just been diagnosed or has recurred (come back).