Medulloblastoma and other central nervous system (CNS) embryonal tumors may begin in embryonic (fetal) cells that remain in the brain after birth.

Medulloblastoma is a fast-growing tumor that forms in the cerebellum (the lower, back part of the brain). Medulloblastoma is the most common type of CNS embryonal tumor. CNS embryonal tumors are uncontrolled growths of cells in the brain. These tumors form in cells that are left over from fetal development, called embryonal cells. Pineoblastoma is a fast-growing type of brain tumor that forms in or around a tiny organ near the center of the brain called the pineal gland.

These tumors may be benign (not cancer) or malignant (cancer). Benign brain tumors grow and press on nearby areas of the brain but rarely spread to other parts of the brain. Malignant brain tumors are likely to grow quickly and spread into other parts of the brain. They may also spread to other parts of the body, but this is rare. When a tumor grows into and presses on an area of the brain or spreads to other parts 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 serious signs or symptoms and need treatment.

Most medulloblastomas, other CNS embryonal tumors, and pineoblastomas in children are malignant. These tumors tend to spread through the cerebrospinal fluid to other parts of the brain and spinal cord.

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).

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There are different types of CNS embryonal tumors.

The different types of CNS embryonal tumors include:

CNS atypical teratoid/rhabdoid tumor is also a type of embryonal tumor, but it is treated differently than other childhood CNS embryonal tumors. Learn more at Childhood Central Nervous System Atypical Teratoid/Rhabdoid Tumor Treatment.

Pineoblastoma forms 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. Pineoblastoma are usually malignant 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 similar to 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. It is rare for pineoblastoma to spread to the bone, bone marrow, lung, or other parts of the body.

Certain genetic conditions increase the risk of childhood medulloblastoma.

Childhood medulloblastoma is caused by certain changes to the way brain cells function, especially how they grow and divide into new cells. Often, the exact cause of the cell changes is unknown. Learn more about how cancer develops at What Is Cancer?

A risk factor is anything that increases the chance of getting a disease. Not every child with one or more of these risk factors will develop medulloblastoma. And it will develop in some children who don’t have a known risk factor.

The risk for medulloblastoma is increased in people who have any of the following inherited diseases:

Talk with your child’s doctor if you think your child may be at risk.

Genetic counseling may be done for children with medulloblastoma or pineoblastoma.

It may not be clear from the family medical history whether a child with a brain tumor has an inherited condition that increased their risk. Genetic counselors and other specially trained health professionals can discuss your child’s diagnosis and the family’s medical history to understand:

Genetic counselors can also help you cope with your child’s genetic testing results, including how to discuss the results with family members.

Learn more about Genetic Testing for Inherited Cancer Risk.

Symptoms of medulloblastoma, other CNS embryonal tumors, and pineoblastoma depend on the child’s age and where the tumor is.

Children may not have symptoms of medulloblastoma, other CNS embryonal tumors, or pineoblastoma until the tumor has grown bigger. It’s important to check with your child’s doctor if your child has:

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 may also cause an increase in the size of an infant’s head.

These symptoms may be caused by problems other than medulloblastoma, other CNS embryonal tumors, or pineoblastoma. The only way to know is to see your child’s doctor.

Tests that examine the brain and spinal cord are used to diagnose childhood medulloblastoma, other CNS embryonal tumors, and pineoblastoma.

If your child has symptoms that suggest medulloblastoma, another type of CNS embryonal tumor, or pineoblastoma, the doctor will need to find out if these are due to cancer or another problem. They will ask about your child’s personal and family health history and do a physical exam. Depending on the results, they may recommend other tests. If your child is diagnosed with medulloblastoma, another type of CNS embryonal tumor, or pineoblastoma, the results of these tests will help you and your child’s doctor plan treatment.

The tests used to diagnose medulloblastoma, other CNS embryonal tumors, and pineoblastoma may include:

A biopsy may be done to be sure of the diagnosis.

If doctors think your child may have medulloblastoma, another type of CNS embryonal tumor, or pineoblastoma, a biopsy may be done. 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.

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The following tests may be done on the sample of tissue that is removed:

Certain factors affect prognosis (chance of recovery) and treatment options.

If your child has been diagnosed with medulloblastoma, other CNS embryonal tumor, or pineoblastoma, you likely have questions about how serious the cancer is and your child’s chances of survival. The likely outcome or course of a disease is called prognosis.

The prognosis and treatment options depend on:

No two people are alike, and responses to treatment can vary greatly. Your child’s cancer care team is in the best position to talk with you about your child’s prognosis.

You may want to get a second opinion.

You may want to get a second opinion to confirm your child’s diagnosis and treatment plan. If you seek a second opinion, you will need to get important medical test results and reports from the first doctor to share with the second doctor. The second doctor will review the genetic test results, pathology report, slides, and scans. This doctor may agree with the first doctor, suggest changes to the treatment plan, or provide more information about your child’s tumor.

To learn more about choosing a doctor and getting a second opinion, see Finding Cancer Care. You can contact NCI’s Cancer Information Service via chat, email, or phone (both in English and Spanish) for help finding a doctor or hospital that can provide a second opinion. For questions you might want to ask at your child’s appointments, see Questions to Ask Your Doctor About Cancer.

Medulloblastoma, other CNS embryonal tumors, and pineoblastoma in children are treated based on the tumor type and the child’s age.

Cancer stage describes the extent of cancer in the body, such as the size of the tumor, whether it has spread, and how far it has spread from where it first formed. There is no staging system used for childhood medulloblastoma, other central nervous system (CNS) embryonal tumors, or pineoblastoma, but the tests and procedures done to diagnose the cancer are also used to help plan treatment.

Treatment of other CNS embryonal tumors and pineoblastoma in children is based on the child’s age. Children aged 3 years and younger may be given different treatment than children older than 3 years.

Treatment of medulloblastoma in children older than 3 years also depends on whether the tumor is average risk or high risk.

The results of the tests and procedures done to diagnose medulloblastoma, other CNS embryonal tumors, and pineoblastoma in children are used to plan cancer treatment.

If your child is diagnosed with medulloblastoma, another type of CNS embryonal tumor, or pineoblastoma, they will be referred to a pediatric oncologist/neuro-oncologist. This is a doctor who specializes in staging and treating childhood cancers. They will recommend tests to determine the extent (stage) of cancer. Some of the tests used to diagnose the cancer are repeated after surgery. This is to find out how much tumor remains after surgery and to see if the cancer has spread from the brain to the spine or other parts of the body. It is important to know if the cancer has spread in order to plan the best treatment. Learn more about diagnostic tests in the General Information section.

The following tests may be used to find out if the cancer has spread beyond the brain and spinal cord:

Sometimes childhood medulloblastoma and other central nervous system embryonal tumors come back after treatment.

Childhood medulloblastoma and other types of 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.

There are different types of treatment for children who have medulloblastoma and other central nervous system (CNS) embryonal tumors.

There are different types of treatment for children and adolescents with medulloblastoma, other types of CNS embryonal tumors, or pineoblastoma. You and your child’s cancer care team will work together to decide treatment. Many factors will be considered, such as your child’s overall health and whether the tumor is newly diagnosed or has come back.

Children who have medulloblastoma, other CNS embryonal tumors, and pineoblastoma should have their treatment planned by a team of health care providers who are experts in treating brain tumors in children.

A pediatric oncologist, a doctor who specializes in treating children with cancer, oversees treatment of medulloblastoma, other CNS embryonal tumors, and pineoblastoma. 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. Other specialists may include:

Your child’s treatment plan will include information about the cancer, the goals of treatment, treatment options, and the possible side effects. It will be helpful to talk with your child’s cancer care team before treatment begins about what to expect. For help every step of the way, see our downloadable booklet, Children with Cancer: A Guide for Parents.

The following types of treatment may be used:

New types of treatment 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. For some patients, taking part in a clinical trial may be the best treatment choice.

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. 

Learn more at Clinical Trials Information for Patients and Caregivers. 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.

The tumor and the treatment may cause symptoms that continue after treatment ends.

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.

Cancer treatments can cause side effects. Which side effects your child might have depends on the type of treatment they receive, the dose, and how their body reacts. Talk with your child’s treatment team about which side effects to look for and ways to manage them.

To learn more about side effects that begin during treatment for cancer, visit Side Effects.

Problems from cancer treatment that begin 6 months or later after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include:

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:

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. Learn more about Late Effects of Treatment for Childhood Cancer.

About PDQ

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.

Purpose of This Summary

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.

Reviewers and Updates

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.

Clinical Trial Information

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).

Permission to Use This Summary

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: /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.

Disclaimer

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.

Contact Us

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.

An adult central nervous system (CNS) tumor is a disease in which abnormal cells form in the tissues of the brain and/or spinal cord.

There are many types of brain and spinal cord tumors. The tumors are formed by the abnormal growth of cells and may begin in different parts of the brain or spinal cord. Together, the brain and spinal cord make up the central nervous system (CNS).

The tumors may be either benign (not cancer) or malignant (cancer):

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 cause signs and symptoms and need treatment.

Brain and spinal cord tumors can occur in both adults and children. However, treatment for children may be different than treatment for adults.

For information about lymphoma that begins in the brain, see Primary CNS Lymphoma Treatment.

A tumor that starts in another part of the body and spreads to the brain is called a metastatic brain tumor.

Tumors that start in the brain are called primary brain tumors. Primary brain tumors may spread to other parts of the brain or to the spine. They rarely spread to other parts of the body.

Often, tumors found in the brain have started somewhere else in the body and spread to one or more parts of the brain. These are called metastatic brain tumors (or brain metastases). Metastatic brain tumors are more common than primary brain tumors. Up to half of metastatic brain tumors are from lung cancer.

Cancer may spread to the leptomeninges (the two innermost membranes covering the brain and spinal cord). This is called leptomeningeal carcinomatosis.

The brain controls many important body functions.

The brain has three major parts:

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The spinal cord connects the brain to 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.

There are different types of brain and spinal cord tumors.

Brain and spinal cord tumors are named based on the type of cell they formed in and where the tumor first formed in the CNS. The grade of a tumor may be used to tell the difference between slow-growing and fast-growing types of the tumor. The World Health Organization (WHO) tumor grades are based on how abnormal the cancer cells look under a microscope and how quickly the tumor is likely to grow and spread.

The following types of primary tumors can form in the brain or spinal cord:

Having certain genetic syndromes may increase the risk of a CNS tumor.

Anything that increases a person’s chance of getting a disease is called a risk factor. Not every person with one or more of these risk factors will develop a brain or spinal cord tumor, and they can develop in people who don’t have any known risk factors. Talk with your doctor if you think you may be at risk. There are few known risk factors for brain tumors. The following conditions may increase the risk of certain types of brain tumors:

The cause of most adult brain and spinal cord tumors is not known.

The signs and symptoms of adult brain and spinal cord tumors are not the same in every person.

Signs and symptoms depend on the following:

These and other signs and symptoms may be caused by CNS tumors or by other conditions, including cancer that has spread to the brain. Check with your doctor if you have any of the following:

Tests that examine the brain and spinal cord are used to diagnose adult brain and spinal cord tumors.

In addition to asking about your personal and family health history and doing a physical exam, your doctor may perform the following tests and procedures:

A biopsy is also used to diagnose a brain tumor.

If imaging tests show there may be a brain tumor, a biopsy is usually done. One of the following types of biopsies may be used:

The pathologist checks the biopsy sample to find out the type and grade of the brain tumor. The grade of the tumor is based on how the tumor cells look under a microscope, and how quickly the tumor is likely to grow and spread.

The following tests may be done on the tumor tissue that is removed:

Certain factors affect prognosis (chance of recovery) and treatment options.

The prognosis and treatment options for primary brain and spinal cord tumors depend on the following:

The prognosis and treatment options for metastatic brain and spinal cord tumors depend on the following:

There is no standard staging system for adult brain and spinal cord tumors.

The process used to find out if cancer has spread to other areas of the brain or to other parts of the body is called staging. Brain tumors that begin in the brain rarely spread to other parts of the body. There is no standard staging system for brain and spinal cord tumors.

Treatment of primary brain and spinal cord tumors is based on the following:

Treatment of tumors that have spread to the brain from other parts of the body is based on the number of tumors in the brain.

Imaging tests may be repeated after surgery to help plan more treatment.

Some of the tests and procedures used to diagnose a brain or spinal cord tumor may be repeated after treatment to find out how much tumor is left.

Central nervous system (CNS) tumors often recur, sometimes many years after treatment.

A recurrent CNS tumor is a tumor that has recurred (come back) after it has been treated. The tumor may recur at the same place as the first tumor or in other parts of the CNS.

There are different types of treatment for patients with adult brain and spinal cord tumors.

Different types of treatment are available for patients with adult brain and spinal cord 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. 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 following types of treatment 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. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer.

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Certain ways of giving external 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 uses a computer to make a 3-dimensional (3-D) picture of the tumor and shapes the radiation beams to fit the tumor.
• Intensity-modulated radiation therapy (IMRT): IMRT is a type of 3-dimensional (3-D) radiation therapy that uses a computer to make pictures of the size and shape of the tumor. Thin beams of radiation of different intensities (strengths) are aimed at the tumor from many angles.
• Stereotactic radiosurgery: Stereotactic radiosurgery uses a rigid head frame that is attached to the skull to keep the head still during the radiation treatment. A machine aims a single large dose of radiation directly at the tumor. This procedure does not involve surgery. It is also called stereotaxic radiosurgery, radiosurgery, and radiation surgery.

Supportive care is given to lessen the problems caused by the disease or its treatment.

This therapy controls problems or side effects caused by the disease or its treatment and improves quality of life. For brain tumors, supportive care includes drugs to control seizures and fluid buildup or swelling in the brain.

New types of treatment are being tested in clinical trials.

This summary section refers to new treatments being studied in clinical trials, but it may not mention every new treatment being studied. Information about clinical trials is available from the NCI website.

Treatment for adult central nervous system tumors may cause side effects.

For information about side effects caused by treatment for cancer, visit our Side Effects page.

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’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. 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.

Follow-up tests may be needed.

As you go through treatment, you will have follow-up tests or check-ups. Some tests that were done to diagnose or stage the cancer may be repeated 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).

The following tests and procedures may be used to check whether a brain tumor has come back after treatment:

Astrocytic Tumors

Oligodendroglial Tumors

Treatment of oligodendrogliomas may include surgery with or without radiation therapy. Chemotherapy may be given after radiation therapy.

Treatment of anaplastic oligodendroglioma 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.

Mixed Gliomas

Treatment of mixed gliomas may include surgery and radiation therapy. Sometimes, chemotherapy is also given.

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.

Ependymal Tumors

Treatment of grade I and grade II ependymomas may include surgery to remove the tumor. Radiation therapy may also be given if some of the tumor remains after surgery.

Treatment of grade III anaplastic ependymoma may include surgery and radiation therapy.

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.

Medulloblastomas

Treatment of medulloblastomas 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.

Pineal Parenchymal Tumors

Treatment of pineal parenchymal 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.

Meningeal Tumors

Treatment of grade I meningiomas may include the following:

Treatment of grade II and III meningiomas and hemangiopericytomas 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.

Germ Cell Tumors

There is no standard treatment for germ cell tumors (germinoma, embryonal carcinoma, choriocarcinoma, and teratoma). Treatment depends on what the tumor cells look like under a microscope, the tumor markers, where the tumor is in the brain, and whether it can be removed by surgery.

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.

Craniopharyngiomas

Treatment of craniopharyngiomas 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.

About PDQ

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.

Purpose of This Summary

This PDQ cancer information summary has current information about the treatment of adult central nervous system 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.

Reviewers and Updates

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.

Clinical Trial Information

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).

Permission to Use This Summary

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 Adult Central Nervous System Tumors Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: /types/brain/patient/adult-brain-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389458]

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.

Disclaimer

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.

Contact Us

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.

Incidence and Mortality

Brain tumors account for 85% to 90% of all primary central nervous system (CNS) tumors.[1] Estimated new cases and deaths from brain tumors and other nervous system tumors in the United States in 2025:[2]

Data from the Surveillance, Epidemiology, and End Results (SEER) Program database for 2017 to 2021 indicated that the combined incidence of brain and other CNS tumors in the United States was 6.2 per 100,000 people per year. The mortality rate was 4.4 deaths per 100,000 people per year based on age-adjusted deaths from 2018 to 2022.[3] Worldwide, approximately 321,476 new cases of brain and other CNS tumors were diagnosed in the year 2022, with an estimated 248,305 deaths.[4]

In general, the incidence of primary CNS tumors is higher in White individuals than in Black individuals, and mortality is higher in men than in women.[3]

Primary brain tumors include the following in decreasing order of frequency:[1]

Primary spinal tumors include the following in decreasing order of frequency:

Primary brain tumors rarely spread to other areas of the body, but they can spread to other parts of the brain and to the spinal axis.

Anatomy

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Risk Factors

Few definitive observations have been made about environmental or occupational causes of primary CNS tumors.[1]

The following potential risk factors have been considered:

The familial tumor syndromes and related chromosomal abnormalities that are associated with CNS neoplasms include the following:[6,7]

Clinical Features

The clinical presentation of various brain tumors is best appreciated by considering the relationship of signs and symptoms to anatomy.[1]

General signs and symptoms include the following:

Seizures are a presenting symptom in approximately 20% of patients with supratentorial brain tumors and may antedate the clinical diagnosis by months to years in patients with slow-growing tumors. Among all patients with brain tumors, 70% with primary parenchymal tumors and 40% with metastatic brain tumors develop seizures at some time during the clinical course.[8]

Diagnostic Evaluation

All brain tumors, whether primary, metastatic, malignant, or benign, must be differentiated from other space-occupying lesions that can have similar clinical presentations, such as abscesses, arteriovenous malformations, and infarctions.[9]

Prognostic Factors

Several genetic alterations have emerged as powerful prognostic factors in diffuse glioma (astrocytoma, oligodendroglioma, mixed glioma, and glioblastoma), and these alterations may guide patient management. Specific alterations include the following:

Other prognostic factors that confer poor prognosis include the following:[11,12]

In an exploratory analysis of 318 patients with low-grade glioma treated with either radiation therapy alone or temozolomide chemotherapy alone, a combination of these prognostic factors demonstrated the following:[11]

For more information, see the Treatment of Primary Central Nervous System Tumors by Tumor Type section.

Genomic Alterations

Alterations in the BRAF, IDH1, and IDH2 genes, and genomic 1p/19q codeletion, appear to be hallmark aberrations in particular glioma subtypes. Assessment for the presence of these variants aids diagnosis and prognosis and, with regard to 1p/19q codeletion, predicts for response to chemotherapy.

In pilocytic astrocytomas (WHO grade I), tandem duplication at 7q34 leading to a KIAA1549::BRAF gene fusion is found in approximately 70% of pilocytic astrocytomas.[6–8] Activating single nucleotide variants in BRAF (V600E) are found in an additional 5% to 9% of these tumors. Overall, RAF alterations occur in approximately 80% of pilocytic astrocytomas.

BRAF V600E variants are observed (in about 60%) of other benign gliomas, including pleomorphic xanthoastrocytoma and ganglioglioma, while BRAF tandem duplications are not found in these variant glioma tumors.[9–11]

Most WHO grade II and III diffuse gliomas (astrocytomas, oligodendrogliomas, and oligoastrocytomas) and 5% to 10% of glioblastomas (WHO grade IV) harbor single nucleotide variants in the R132 position of IDH1 or, rarely, the analogous codon in IDH2 (R172).[12–16] The presence of an IDH1 or IDH2 variant is a strong prognostic factor. Patients with these tumor variants have significantly longer survival independent of WHO grade or histological subtype.

Deletion of chromosomes 1p and 19q occurs through a translocation event [17] and is common in oligodendrogliomas. 1p/19q codeletion is a powerful prognostic factor and may predict for response to chemotherapy. For more information, see the Anaplastic oligodendrogliomas treatment section.

These genetic alterations have potential diagnostic utility. Presence of the IDH1 and IDH2 variants may distinguish diffuse gliomas from other gliomas, which often have BRAF genetic alterations, and nonneoplastic reactive astrocytosis.[18] Most (90%) IDH variants in gliomas result in an R132H substitution, which can be detected with a highly sensitive and specific monoclonal antibody. A rapid immunohistochemical analysis using the variant-specific IDH1 antibody can aid diagnostic analysis.[19]

Other CNS tumors are associated with characteristic patterns of altered oncogenes, altered tumor suppressor genes, and chromosomal abnormalities. Familial tumor syndromes with defined chromosomal abnormalities are associated with gliomas.

Primary CNS Tumors

This section discusses general treatment modalities for primary central nervous system (CNS) tumors. For a description of specific treatment options for each tumor type, see the Treatment of Primary Central Nervous System Tumors by Tumor Type section.

Radiation therapy and chemotherapy options vary according to histology and anatomical site of the CNS tumor. For glioblastoma, combined modality therapy with resection, radiation, and chemotherapy is standard. Anaplastic astrocytomas, anaplastic oligodendrogliomas, and anaplastic oligoastrocytomas represent only a small proportion of CNS gliomas; therefore, phase III randomized trials restricted to these tumor types are not generally practical. The natural histories of these tumors are variable, depending on histological and molecular factors; therefore, treatment guidelines are evolving. Therapy involving surgically implanted carmustine-impregnated polymer wafers combined with postoperative external-beam radiation therapy (EBRT) may play a role in the treatment of high-grade (grades III and IV) gliomas in some patients.[1]

Treatment options for primary CNS tumors include the following:

Astrocytic Tumors Treatment

Oligodendroglial Tumors Treatment

Mixed Gliomas Treatment

Patients with mixed glial tumors, which include oligoastrocytoma (WHO grade II) and anaplastic oligoastrocytoma (WHO grade III), have highly variable prognoses based on their status of the IDH1 and IDH2 genes and 1p/19q chromosomes.[27–29] Therefore, the optimal treatment for these tumors as a group is uncertain. Often, they are treated similarly to astrocytic tumors because a subset of tumors may have outcomes similar to WHO grade III astrocytic or WHO grade IV glioblastoma tumors. Testing for these known, strong prognostic molecular markers should be performed, which may help to guide the assessment of risk and subsequent management.

Treatment options for mixed gliomas include the following:

For more information, see the Astrocytic Tumors Treatment section.

Ependymal Tumors Treatment

Ependymal tumors (WHO grade I) and ependymomas (WHO grade II)—i.e., subependymomas and myxopapillary ependymomas—are often curable.

Treatment options for grades I and II ependymal tumors include the following:

Patients with anaplastic ependymomas (WHO grade III) have variable prognoses that depend on the location and extent of disease. Frequently, but not invariably, patients with anaplastic ependymomas have worse prognoses than do those patients with lower-grade ependymal tumors.

Treatment options for anaplastic ependymomas include the following:

Embryonal Cell Tumors (Medulloblastomas) Treatment

Medulloblastoma occurs primarily in children but may also occur in adults.[31] For more information, see Childhood Medulloblastoma and Other Central Nervous System Embryonal Tumors Treatment.

Treatment options for medulloblastomas include the following:

Pineal Parenchymal Tumors Treatment

Pineocytomas (WHO grade II), pineoblastomas (WHO grade IV), and pineal parenchymal tumors of intermediate differentiation are diverse tumors that require special consideration. Pineocytomas are slow-growing tumors and prognosis varies.

Pineoblastomas grow more rapidly and patients with these tumors have worse prognoses. Pineal parenchymal tumors of intermediate differentiation have unpredictable growth and clinical behavior.

Treatment options for pineal parenchymal tumors include the following:

Meningeal Tumors Treatment

WHO grade I meningiomas are usually curable when they are resectable. With the increasing use of sensitive neuroimaging tools, there has been more detection of asymptomatic low-grade meningiomas. Most appear to show minimal growth and can often be safely observed while therapy is deferred until growth or the development of symptoms.[33,34]

Treatment options for meningeal tumors include the following:

The prognoses for patients with WHO grade II meningiomas (atypical, clear cell, and chordoid), WHO grade III meningiomas (anaplastic/malignant, rhabdoid, and papillary), and hemangiopericytomas are worse than the prognoses for patients with low-grade meningiomas because complete resections are less commonly feasible, and the proliferative capacity is greater.

Treatment options for grades II and III meningiomas and hemangiopericytomas include the following:

Germ Cell Tumors Treatment

The prognoses and treatment of patients with germ cell tumors—which include germinomas, embryonal carcinomas, choriocarcinomas, and teratomas—depend on tumor histology, tumor location, presence and levels of biological markers, and surgical resectability.

Treatment of Tumors of the Sellar Region

Craniopharyngiomas (WHO grade I) are often curable.

Treatment options for craniopharyngiomas include the following:

Treatment Options Under Clinical Evaluation for Primary CNS Tumors

Patients who have central nervous system (CNS) tumors that are either infrequently curable or unresectable should consider enrollment in clinical trials. Information about ongoing clinical trials is available from the NCI website.

Heavy-particle radiation, such as proton-beam therapy, carries the theoretical advantage of delivering high doses of ionizing radiation to the tumor bed while sparing surrounding brain tissue. The data are preliminary for this investigational technique and are not widely available.

Novel biological therapies under clinical evaluation for patients with CNS tumors include the following:[36]

Current Clinical Trials

Use our advanced clinical trial search to find NCI-supported cancer clinical trials that are now enrolling patients. The search can be narrowed by location of the trial, type of treatment, name of the drug, and other criteria. General information about clinical trials is also available.

General Information About Metastatic Brain Tumors

Brain metastases outnumber primary neoplasms by at least 10 to 1, and they occur in 20% to 40% of cancer patients, with subsequent median survival generally less than 6 months.[1] The exact incidence is unknown because no national cancer registry documents brain metastases, but it has been estimated that 98,000 to 170,000 new cases are diagnosed in the United States each year.[2,3] This number may be increasing because of the capacity of magnetic resonance imaging (MRI) to detect small metastases and because of prolonged survival resulting from improved systemic therapy.[1,2]

The most common primary tumors with brain metastases and the percentage of patients affected are as follows:[1,2]

Eighty percent of brain metastases occur in the cerebral hemispheres, 15% occur in the cerebellum, and 5% occur in the brain stem.[2] Metastases to the brain are multiple in more than 70% of cases, but solitary metastases also occur.[1]

Brain involvement can occur with cancers of the nasopharyngeal region by direct extension along the cranial nerves or through the foramina at the base of the skull. Dural metastases may constitute as much as 9% of total brain metastases.

Clinical Features

The diagnosis of brain metastases in cancer patients is based on the following:

Patients may describe any of the following:

Often, family members or friends may notice the following:

Diagnostic Evaluation

A physical examination may show objective neurological findings or only minor cognitive changes. The presence of multiple lesions and a high predilection of primary tumor metastasis may be sufficient to make the diagnosis of brain metastasis.

A lesion in the brain should not be assumed to be a metastasis just because a patient has had a previous cancer; such an assumption could result in overlooking appropriate treatment of a curable tumor.

Treatment of Metastatic Brain Tumors

The optimal therapy for patients with brain metastases continues to evolve.[1,2,5] The following treatments have been used in the management of metastatic brain tumors:

Because most cases of brain metastases involve multiple metastases, a mainstay of therapy has historically been whole-brain radiation therapy (WBRT). However, stereotactic radiosurgery has become increasingly common. The role of radiosurgery continues to be defined. Stereotactic radiosurgery in combination with WBRT has been assessed.

Surgery is indicated to obtain tissue from a metastasis with an unknown primary tumor or to decompress a symptomatic dominant lesion that is causing significant mass effect.

Chemotherapy is usually not the primary therapy for most patients; however, it may have a role in the treatment of patients with brain metastases from chemosensitive tumors and can even be curative when combined with radiation for metastatic testicular germ cell tumors.[1,6] Intrathecal chemotherapy is also used for meningeal spread of metastatic tumors.

Leptomeningeal Carcinomatosis (LC)

LC occurs in about 5% of all cancer patients. The most common types of cancer to spread to the leptomeninges are:

Diagnosis includes a combination of neurospinal axis imaging and cerebrospinal fluid (CSF) cytology. Median OS is in the range of 10 to 12 weeks.

The management of LC includes the following:

In a series of 149 patients with metastatic non-small cell lung carcinoma, cytologically proven LC, poor performance status, high protein level in the CSF, and a high initial CSF white blood cell count were significant poor prognostic factors for survival.[19] Patients received active treatment, including intrathecal chemotherapy, WBRT, or epidermal growth factor receptor-tyrosine kinase inhibitors, or underwent a ventriculoperitoneal shunt procedure.

In a retrospective series of 38 patients with metastatic breast cancer and LC, the proportion of LC cases varied by breast cancer subtype:[20]

Patients with triple-negative breast cancer had a shorter interval between metastatic breast cancer diagnosis and the development of LC. Median survival did not differ across breast cancer subtypes. Consideration of intrathecal administration of trastuzumab in patients with HER2-positive LC has also been described in case reports.[21]

Chemotherapy

Antiangiogenesis Therapy

In 2009, the U.S. Food and Drug Administration (FDA) granted accelerated approval of bevacizumab monotherapy for patients with progressive glioblastoma. The indication was granted under the FDA’s accelerated approval program that permits the use of certain surrogate end points or an effect on a clinical end point other than survival or irreversible morbidity as bases for approvals of products intended for serious or life-threatening illnesses or conditions.

The approval was based on the demonstration of improved objective response rates observed in two historically controlled, single-arm, or noncomparative phase II trials.[3,4][Level of evidence C3] Based on these data and the FDA approval, bevacizumab monotherapy has become standard therapy for recurrent glioblastoma.

Evidence (antiangiogenesis therapy):

No data are available from prospective randomized controlled trials demonstrating improvement in health outcomes, such as disease-related symptoms or increased survival with the use of bevacizumab to treat glioblastoma.

Radiation Therapy

Because there are no randomized trials, the role of repeat radiation after disease progression or the development of radiation-induced cancers is also ill defined. Interpretation is difficult because the literature is limited to small retrospective case series.[5] The decision must be made carefully because of the risk of neurocognitive deficits and radiation necrosis.

Surgery

Re-resection of recurrent CNS tumors is an option for some patients. However, most patients do not qualify because of a deteriorating condition or technically inoperable tumors. The evidence is limited to noncontrolled studies and case series of patients who are healthy enough and have tumors that are small enough to technically debulk. The impact on survival of reoperation versus patient selection is not known.

Current Clinical Trials

Use our advanced clinical trial search to find NCI-supported cancer clinical trials that are now enrolling patients. The search can be narrowed by location of the trial, type of treatment, name of the drug, and other criteria. General information about clinical trials is also available.

Purpose of This Summary

This PDQ cancer information summary for health professionals provides comprehensive, peer-reviewed, evidence-based information about the treatment of adult central nervous system tumors. It is intended as a resource to inform and assist clinicians in the care of their patients. It does not provide formal guidelines or recommendations for making health care decisions.

Reviewers and Updates

This summary is reviewed regularly and updated as necessary by the PDQ Adult Treatment Editorial Board, which is editorially independent of the National Cancer Institute (NCI). The summary reflects an independent review of the literature and does not represent a policy statement of NCI or the National Institutes of Health (NIH).

Board members review recently published articles each month to determine whether an article should:

Changes to the summaries are made through a consensus process in which Board members evaluate the strength of the evidence in the published articles and determine how the article should be included in the summary.

The lead reviewers for Adult Central Nervous System Tumors Treatment are:

Any comments or questions about the summary content should be submitted to Cancer.gov through the NCI website’s Email Us. Do not contact the individual Board Members with questions or comments about the summaries. Board members will not respond to individual inquiries.

Levels of Evidence

Some of the reference citations in this summary are accompanied by a level-of-evidence designation. These designations are intended to help readers assess the strength of the evidence supporting the use of specific interventions or approaches. The PDQ Adult Treatment Editorial Board uses a formal evidence ranking system in developing its level-of-evidence designations.

Permission to Use This Summary

PDQ is a registered trademark. Although the content of PDQ documents can be used freely as text, it cannot be identified as an NCI PDQ cancer information summary unless it is presented in its entirety and is regularly updated. However, an author would be permitted to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks succinctly: [include excerpt from the summary].”

The preferred citation for this PDQ summary is:

PDQ® Adult Treatment Editorial Board. PDQ Adult Central Nervous System Tumors Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: /types/brain/hp/adult-brain-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389419]

Images in this summary are used with permission of the author(s), artist, and/or publisher for use within the PDQ summaries only. Permission to use images outside the context of PDQ information must be obtained from the owner(s) and cannot be granted by the National Cancer Institute. Information about using the illustrations in this summary, along with many other cancer-related images, is available in Visuals Online, a collection of over 2,000 scientific images.

Disclaimer

Based on the strength of the available evidence, treatment options may be described as either “standard” or “under clinical evaluation.” These classifications should not be used as a basis for insurance reimbursement determinations. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page.

Contact Us

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 Email Us.

Anal cancer is a disease in which malignant (cancer) cells form in the tissues of the anus.

The anus is the end of the large intestine, below the rectum. It is where stool (solid waste) leaves the body. The anus is formed partly from the outer skin layers of the body and partly from the intestine. The anus is connected to the rectum by the anal canal, which is about 1–1½ inches long. This area is controlled by two ring-like sphincter muscles, which open and close to let stool pass out of the body.

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The skin around the outside of the anus is called the perianal area. Tumors in this area are skin tumors, not anal cancer.

To learn more about anal cancer, visit Anal Cancer Treatment.

Squamous cell carcinoma is the most common type of anal cancer.

In the United States, squamous cell carcinoma is the most common type of anal cancer. Studies show that human papillomavirus (HPV) infection is the main cause of this type of anal cancer.

Another type of anal cancer, called anal adenocarcinoma, is very rare and is not discussed in this summary.

In the United States, the number of new cases of anal cancer has increased in recent years.

Between 2012 and 2021, the number of new cases of anal cancer increased each year. Between 2013 and 2022, the rate of deaths from this disease also increased each year.

Avoiding risk factors and increasing protective factors may help prevent cancer.

Avoiding cancer risk factors may help prevent certain cancers. Risk factors include smoking, having overweight, and not getting enough exercise. Increasing protective factors such as quitting smoking and exercising may also help prevent some cancers. Talk to your doctor or other health care professional about how you might lower your risk of cancer.

The following are risk factors for anal cancer:

The following protective factors decrease the risk of anal cancer:

It is not clear if the following protective factor decreases the risk of anal cancer:

Cancer prevention clinical trials are used to study ways to prevent cancer.

Cancer prevention clinical trials are used to study ways to lower the risk of developing certain types of cancer. Some cancer prevention trials include healthy people who may or may not have an increased risk of cancer. Other prevention trials include people who have had cancer and are trying to prevent recurrence or a second cancer.

The purpose of some cancer prevention clinical trials is to find out whether actions people take can prevent cancer. These may include eating fruits and vegetables, exercising, quitting smoking, or taking certain medicines, vitamins, minerals, or food supplements.

New ways to prevent anal cancer are being studied in clinical trials.

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.

About PDQ

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.

Purpose of This Summary

This PDQ cancer information summary has current information about anal cancer prevention. 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.

Reviewers and Updates

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.

Clinical Trial Information

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).

Permission to Use This Summary

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 Anal Cancer Prevention. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: /types/anal/patient/anal-prevention-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389512]

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.

Disclaimer

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.

Contact Us

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.

Anal cancer is a type of cancer that forms in the tissues of the anus.

The anus is the end of the large intestine. It is where stool (solid waste) leaves the body. The anus is formed partly from the outer skin layers of the body and partly from the intestine. The anus is connected to the rectum by the anal canal, which is about 1 to 1½ inches long. This area is controlled by two ring-like sphincter muscles, which contract to hold stool in and relax to allow its passage out of the body.

Enlarge

Anal cancer can start in the lining of the anal canal, called the mucosa, or in the perianal skin, the squamous cells outside of the anus that contain hair follicles and sweat glands.

Tumors of the perianal skin that do not involve the anal sphincter are usually treated the same as anal cancers, although local therapy (treatment directed to a limited area of skin) may be used for some.

Most anal cancers are related to human papillomavirus (HPV) infection.

Anal cancer is caused by certain changes to the way anal cells function, especially how they grow and divide into new cells. There are many risk factors for anal cancer, but many do not directly cause cancer. Instead, they increase the chance of DNA damage in cells that may lead to anal cancer. Learn more about how cancer develops at What Is Cancer?

A risk factor is anything that increases the chance of getting a disease. Some risk factors for anal cancer can be changed. However, risk factors also include things people cannot change, like getting older and their health history. Learning about risk factors for anal cancer can help you make changes that might lower your risk of getting it.

Risk factors for anal cancer include:

Talk with your doctor if you think you may be at risk.

Signs of anal cancer include bleeding from the anus or rectum or a lump near the anus.

These and other signs and symptoms may be caused by anal cancer or by other conditions. Check with your doctor if you have:

Tests that examine the rectum and anus are used to diagnose anal cancer.

In addition to asking about your personal and family health history and doing a physical exam, your doctor may perform the following tests and procedures:

After anal cancer has been diagnosed, tests are done to find out if cancer cells have spread within the anus or to other parts of the body.

The process used to find out if cancer has spread within the anus or to other parts of the body is called staging. The information gathered from this staging process determines the stage of the disease. It is important to know the stage in order to plan treatment. The following tests may be used in the staging process:

Some people decide to get a second opinion.

You may want to get a second opinion to confirm your anal cancer diagnosis and treatment plan. If you seek a second opinion, you will need to get medical test results and reports from the first doctor to share with the second doctor. The second doctor will review the pathology report, slides, and scans. They may agree with the first doctor, suggest changes or another treatment approach, or provide more information about your cancer.

Learn more about choosing a doctor and getting a second opinion at Finding Cancer Care. You can contact NCI’s Cancer Information Service via chat, email, or phone (both in English and Spanish) for help finding a doctor, hospital, or getting a second opinion. For questions you might want to ask at your appointments, visit Questions to Ask Your Doctor About Cancer.

Certain factors affect the prognosis (chance of recovery) and treatment options.

The prognosis depends on:

The treatment options depend on:

The following stages are used for anal cancer:

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Anal cancer can recur (come back) after it has been treated.

Recurrent anal cancer is cancer that has come back after it has been treated. If anal cancer comes back, it may come back in the anus or in other parts of the body, such as the liver or lungs. Tests will be done to help determine where the cancer has returned. The type of treatment for recurrent anal cancer will depend on where it has come back.

Learn more in Recurrent Cancer: When Cancer Comes Back.

There are different types of treatment for people with anal cancer.

Different types of treatments are available for anal cancer. You and your cancer care team will work together to decide your treatment plan, which may include more than one type of treatment. Many factors will be considered, such as the stage of the cancer, your overall health, and your preferences. Your plan will include information about your cancer, the goals of treatment, your treatment options and the possible side effects, and the expected length of treatment.

Talking with your cancer care team before treatment begins about what to expect will be helpful. You’ll want to learn what you need to do before treatment begins, how you’ll feel while going through it, and what kind of help you will need. To learn more, visit Questions to Ask Your Doctor About Treatment.

The following types of treatment are used:

New types of treatment are being tested in clinical trials.

For some people, joining a clinical trial may be an option. There are different types of clinical trials for people with cancer. For example, a treatment trial tests new treatments or new ways of using current treatments. Supportive care and palliative care trials look at ways to improve quality of life, especially for those who have side effects from cancer and its treatment.

You can use the clinical trial search to find NCI-supported cancer clinical trials accepting participants. The search allows you to filter trials based on the type of cancer, your age, and where the trials are being done. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website.

Learn more about clinical trials, including how to find and join one, at Clinical Trials Information for Patients and Caregivers.

Treatment for anal cancer may cause side effects.

For information about side effects caused by treatment for cancer, visit our Side Effects page.

Follow-up care may be needed.

As you go through treatment, you will have follow-up tests or check-ups. Some tests that were done to diagnose or stage the cancer may be repeated 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).

About PDQ

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.

Purpose of This Summary

This PDQ cancer information summary has current information about the treatment of anal 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.

Reviewers and Updates

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.

Clinical Trial Information

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).

Permission to Use This Summary

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 Anal Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: /types/anal/patient/anal-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389368]

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.

Disclaimer

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.

Contact Us

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.

Who Is at Risk?

Human papillomavirus (HPV) infection is the strongest risk factor for anal cancer and is accepted as a causal agent of squamous cell carcinoma of the anus and its precursor lesions.[1] Behaviors or medical conditions that either indicate HPV infection or facilitate HPV transmission or persistence are associated with increased risk. These behaviors and conditions include a history of HPV-related cancers, high-risk sexual practices such as sex between men, receptive anal intercourse and numerous sexual partners, human immunodeficiency virus (HIV) infection, and chronic immunosuppressive states.[2] Cigarette smoking is also a risk factor.[3]

Factors Associated With Increased Risk of Anal Cancer

Interventions Associated With a Decreased Risk of Anal Cancer

Interventions With Inadequate Evidence as to Whether They Reduce the Risk of Anal Cancer

United States

The Surveillance, Epidemiology, and End Results (SEER) Program age-adjusted annual incidence rate of anal cancer in the United States from 2017 to 2021 was 1.9 cases per 100,000 persons per year, and the mortality rate was 0.4 cases per 100,000 persons per year from 2018 to 2022. Incidence rates were slightly higher for women than for men (2.3 vs. 1.6 per 100,000 person-years, respectively), but mortality rates were about the same.[1] In 2025, it is estimated that 10,930 Americans will be diagnosed with anal cancer and 2,030 will die of this disease.[2] Incidence rates increased annually from 2012 to 2021 (average increase, 1.3%), and mortality rates increased annually from 2013 to 2022 (average increase, 5.1%). All incidence and mortality increases were statistically different from zero.[3] The 5-year survival rate has remained fairly constant since 1975, and based on data from 2014 to 2020, it was 70.6%.[1]

World

An estimated 27,000 new cases of anal cancer were diagnosed worldwide in 2008.[4] No global incidence rates, mortality rates, or survival statistics are available.

Precursor Lesions

Squamous cell cancer of the anus is preceded by grade 2 or 3 anal intraepithelial neoplasia (AIN), also referred to as high-grade AIN. Grade 1 AIN is not considered a precursor lesion of anal cancer but may precede high-grade AIN.[4] The cytological terms for low- and high-grade AIN are low-grade squamous cell intraepithelial lesions (LSIL) and high-grade squamous cell intraepithelial lesions (HSIL).[4]

One study reported that 11% of AIN cases progressed to invasive disease over an 8-year period.[5] However, results from another study suggested that progression is much less frequent. Using AIN prevalence and anal cancer incidence data, the investigators estimated hypothetical annual rates of progression from high-grade AIN to anal cancer. For men who have sex with men (MSM) and who are human immunodeficiency virus (HIV) positive, the rate was about 1 case in 600 patients. For HIV-negative MSM, the rate was 1 case in 4,000 patients.[6] Using meta-analysis techniques to combine data from numerous studies worldwide, the investigators estimated that the prevalence of LSIL is 27.5% (95% confidence interval [CI], 21.9%–33.2%) and the prevalence of HSIL is 6.7% (95% CI, 4.4%–9.0%) in HIV-positive MSM. Among HIV-negative MSM, the prevalence of LSIL was 6.6% (95% CI, 1.1%–12.1%), and the prevalence of HSIL was 2.7% (95% CI, 0.0%–5.1%).[6]

Factors Associated With Increased Risk of Anal Cancer

HPV Vaccination

Because human papillomavirus (HPV) is a causal condition for squamous cell anal cancer development, vaccination against the oncogenic strains of HPV before exposure may reduce the risk of anal cancer. Conducted from 2004 to 2008, a multicountry trial randomly assigned 4,065 boys and men to receive either the three-shot quadrivalent HPV vaccine regimen (for HPV-6, -11, -16, and -18) or a three-shot placebo injection regimen. Of the 4,065 patients, 602 reported having sex with male partners in the year before enrollment. Heterosexual participants were between the ages of 16 years and 23 years and had no more than five lifetime female partners. Patients who reported sex with male partners were between the ages of 16 years and 26 years and had no more than five lifetime male or female partners. Persistent infection was defined as detection of the same HPV type in anogenital swabs or biopsy specimens collected on two or more consecutive visits, with an interval of 6 months between visits. In the intent-to-treat analysis, which included participants regardless of their baseline HPV status, the efficacy against persistent HPV-6, -11, -16, and -18 infection was 48% (95% confidence interval [CI], 36.0%–57.6%). Among those who were negative for the four HPV strains of interest at baseline (per the protocol analysis, which included 1,397 intervention-arm and 1,408 control-arm participants), vaccine efficacy against persistent HPV-6, -11, -16, and -18 infection was 90% (95% CI, 69.2%–98.1%).[1]

A nonrandomized, phase II, open-label trial (AMC 072 [NCT01209325]) was conducted in 149 men who have sex with men living with HIV. This study did not show a statistically significant difference between incident-persistent infections in the naïve, per-protocol, and previously exposed per-protocol groups. However, there was a statistically significant reduction in incident HPV-16–associated histological high-grade squamous intraepithelial lesions (HSIL) in the naïve group when compared with the previously exposed group (P = .014). The authors conducted a secondary analysis that compared the per-protocol quadrivalent HPV (qHPV)-type naïve participants in the AMC 072 trial to the original, per-protocol placebo group in the Merck 020 trial. This analysis demonstrated that vaccinated qHPV-naïve AMC-072 participants had significantly reduced disease in a combined analysis of all four qHPV types (5.8 per 100 person-years vs. 0 per 100 person-years; P = .008).[2]

Among the 602 patients who had sex with men, the vaccine efficacy against persistent HPV-6, -11, -16, and -18 infection was 59% (95% CI, 43.0%–71.4%) in the intent-to-treat analysis and 95% (95% CI, 80.4%–99.4%) in the per-protocol analysis. Efficacy against HPV-6, -11, -16 or -18–associated anal intraepithelial neoplasia (AIN) was 50% (95% CI, 25.7%–67.2%) in the intention-to-treat analysis and 77.5% (95% CI, 39.6%–93.9%) in the per-protocol analysis (275 intervention-arm and 276 control-arm participants). Efficacy against HPV-6, -11, -16, or -18–associated high-grade AIN was 54.2% (95% CI, 18.0%–75.3%) in the intent-to-treat analysis and 74.9% (95% CI, 8.8%–95.4%) in the per-protocol analysis (194 intervention-arm and 208 control-arm participants).[3]

Efficacy of the bivalent (HPV-16 and HPV-18) vaccine against anal infection was evaluated in the context of a randomized controlled trial of cervical cancer prevention. Conducted in 6,300 Costa Rican women aged 18 to 25 years at enrollment, the trial compared the efficacy of the three-dose bivalent vaccine with that of a control vaccine. Four years after vaccination, most women were offered the option of providing an anal specimen. Among the 2,103 intervention-arm and 2,107 control-arm participants who provided specimens, vaccine efficacy (i.e., absence of HPV-16 or -18 in the specimen) was 62% (95% CI, 47.1%–73.1%). Among the 1,003 intervention-arm and 986 control-arm participants who provided anal specimens, received the three doses, had no evidence of cervical HPV-16 or -18 infection before vaccination, and were seronegative before vaccination, vaccine efficacy was 84% (95% CI, 66.7%–92.8%).[4]

These data strongly suggest that vaccination against oncogenic HPV strains will lead to reductions in anal cancer. They also suggest that vaccination before exposure will provide the most benefit.

Treatment of Anal HSIL

Until the Anal Cancer–HSIL Outcomes Research (ANCHOR) trial was published, there had not been confirmation that treating anal HSIL decreased an individual’s risk for invasive cancer.[5] However, the findings from the ANCHOR trial provided solid evidence that treatment of anal HSIL prevented the incidence of anal cancer. This trial, which was a phase III trial conducted at 25 U.S. sites, included individuals living with HIV who were aged 35 years or older and had biopsy-proven anal HSIL. Participants were randomly assigned in a 1:1 ratio to receive either HSIL treatment or active monitoring without treatment. Nine cases were diagnosed in the treatment group (173 per 100,000 person-years; 95% CI, 90.0–332.0), and 21 cases were diagnosed in the active-monitoring group (402 per 100,000 person-years; 95% CI, 262.0–616.0) after a median follow-up of 25.8 months. The rate of progression to anal cancer was 57% lower in the treatment group than it was in the active-monitoring group (95% CI, 6.0–80.0; P = .03 by log-rank test).

In a recent cohort study of 28,175 individuals being treated for HIV (59.7%, men who have sex with men [MSM]), 227 primary anal cancer cases were diagnosed.[6] Despite the increasing average age of the cohort, crude incidence rates of anal cancer in MSM declined slowly over time from 107.0 per 100,000 person-years (95% CI, 75.7–147.0) in 1996 to 2005 to 93.7 per 100,000 person-years (95% CI, 75.3–115.0) in 2013 to 2020 (P = .49). Crude incidence rates in men who do not have sex with men (non-MSM) and women were generally lower than those in MSM. However, crude incidence rates increased slightly over time from 51.08 per 100,000 person-years (95% CI, 20.54–105.25) to 67.82 per 100,000 person-years (95% CI, 40.83–105.91; P = .52) in non-MSM and from 8.09 per 100,000 person-years (95% CI, 0.20–45.06) to 24.95 per 100,000 person-years (95% CI, 10.03–51.40; P = .29) in women. In addition, the authors compared a subcohort of 3,866 MSM who received high-resolution anoscopy (HRA) screening at least once and treatment for HSIL. Furthermore, if screened individuals had low-grade squamous intraepithelial lesions (LSIL), they continued HRA screening once yearly, and those who had HSIL treatment received HRA screening every 6 months. TNM tumor staging was more favorable (Cochrane-Armitage test for trend, P = .033) in individuals diagnosed with anal cancer during screening. Crude anal cancer–associated 5-year mortality in people living with HIV decreased from 30.4% (1996–2005) to 18.3% (2013–2020; odds ratio, 0.48; P = .070). Anal cancer–related mortality was 3.7% (95% CI, 0.5–23.5) in all men who had been screened and 24.0% (95% CI, 18.1–31.3) in men who had not been screened (P = .023). In men, screening participation (hazard ratio [HR], 0.31; P = .051) and cumulative exposure to CD4 counts of less than 200 cells per µL (HR, 1.11 per year; P = .0022) were independently associated with anal cancer–related mortality.

Even though people living with HIV represent a high-risk population, anal cancer is still rare, and there were few anal cancers in this cohort, rendering conclusions difficult. In particular, there were only 37 anal cancers in non-MSM and only 10 among women. As a result, CIs were wide, and conclusions were suggestive at best. Of note, there are no known randomized controlled trials that provide evidence to support the conclusion of this study.

Data do not support the conclusion that men had improved survival when they were diagnosed with anal cancer after screening or that it is important to screen those who are at high risk of developing anal cancer. The study purported to show reduced mortality after an anal cancer diagnosis among screened men, but the curves in the study’s results did not make adjustments for lead-time bias, selection bias, nor the possibility of overdiagnosis bias. The study also suggested that there was a larger proportion of early-stage cases among screened individuals, but the proportional stage shift caused by anal cancer screening is known to be influenced by lead-time and overdiagnosis biases. There was no mention of adjustment for these biases in the study’s results. In addition, there was a high proportion of unknown stages, further suggesting that the conclusions in this study were not supported by the data. This is an additional rationale for why the data did not support the conclusion of the study.

Condom Use

Because human papillomavirus (HPV) can be transmitted through microabrasions, as well as through more pronounced exposures such as exchange of certain bodily fluids,[1] restriction of condom use to penetrative activity will not protect against transmission that occurs as part of other sexual contact. Nevertheless, condom use would be expected to reduce some risk of transmission and thus anal cancer risk. Few data that explore these hypotheses exist, and those that do suggest a very modest effect, if any. Of note, the ability of condom use to reduce cervical cancer risk is still uncertain and the subject of debate.[2]

In an Italian cohort of 258 HIV-negative men who have sex with men (MSM), the odds ratio (OR) for infection with high-risk HPV strains was 1.7 (95% confidence interval [CI], 0.52–6.3) for inconsistent or no use of condoms in receptive anal sex, compared with consistent condom use.[3] In a Brazilian cohort that included 176 MSM, the OR for oncogenic HPV infection was 1.8 (95% CI, 0.77–4.35) for men who sometimes used condoms for anal sex and 1.8 (95% CI, 0.58–5.68) for men who never used condoms, compared with men who always used condoms.[4]

Purpose of This Summary

This PDQ cancer information summary for health professionals provides comprehensive, peer-reviewed, evidence-based information about anal cancer prevention. It is intended as a resource to inform and assist clinicians in the care of their patients. It does not provide formal guidelines or recommendations for making health care decisions.

Reviewers and Updates

This summary is reviewed regularly and updated as necessary by the PDQ Screening and Prevention Editorial Board, which is editorially independent of the National Cancer Institute (NCI). The summary reflects an independent review of the literature and does not represent a policy statement of NCI or the National Institutes of Health (NIH).

Board members review recently published articles each month to determine whether an article should:

Changes to the summaries are made through a consensus process in which Board members evaluate the strength of the evidence in the published articles and determine how the article should be included in the summary.

Any comments or questions about the summary content should be submitted to Cancer.gov through the NCI website’s Email Us. Do not contact the individual Board Members with questions or comments about the summaries. Board members will not respond to individual inquiries.

Levels of Evidence

Some of the reference citations in this summary are accompanied by a level-of-evidence designation. These designations are intended to help readers assess the strength of the evidence supporting the use of specific interventions or approaches. The PDQ Screening and Prevention Editorial Board uses a formal evidence ranking system in developing its level-of-evidence designations.

Permission to Use This Summary

PDQ is a registered trademark. Although the content of PDQ documents can be used freely as text, it cannot be identified as an NCI PDQ cancer information summary unless it is presented in its entirety and is regularly updated. However, an author would be permitted to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks succinctly: [include excerpt from the summary].”

The preferred citation for this PDQ summary is:

PDQ® Screening and Prevention Editorial Board. PDQ Anal Cancer Prevention. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: /types/anal/hp/anal-prevention-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389511]

Images in this summary are used with permission of the author(s), artist, and/or publisher for use within the PDQ summaries only. Permission to use images outside the context of PDQ information must be obtained from the owner(s) and cannot be granted by the National Cancer Institute. Information about using the illustrations in this summary, along with many other cancer-related images, is available in Visuals Online, a collection of over 2,000 scientific images.

Disclaimer

The information in these summaries should not be used as a basis for insurance reimbursement determinations. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page.

Contact Us

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 Email Us.

Incidence and Mortality

Estimated new cases and deaths from anal, anal canal, and anorectal cancer in the United States in 2025:[1]

Prognosis and Survival

The two major prognostic factors for anal cancer are tumor size and nodal status. Primary tumors smaller than 2 cm have a better prognosis.[2] Nodal drainage of the anus follows the inguinal vein. The initial evaluation of a patient with anal cancer will include a careful clinical examination of the inguinal region and biopsy of any palpable lymph nodes. For more information, see the American Joint Committee on Cancer Stage Groupings and TNM Definitions section.

Anal cancer is usually curable. At presentation, most patients have T1 or T2 disease (≤5 cm), and fewer than 20% of patients have node-positive disease. The 5-year survival rate for these early-stage patients exceeds 85%.[3,4] Even in patients with node-positive disease, 5-year survival rates exceed 50% in the absence of invasion into adjacent organs or distant metastases.[5]

Risk Factors

Overall, the risk of anal cancer is rising due to increased incidence of human papillomavirus (HPV) infection.[6,7] Ninety-five percent of anal cancers are HPV related, with the highest risk for serotypes 16 and 18. Involvement of HPV can be pathologically correlated with P16+ staining.[8] Patients with HIV have a higher risk of HPV coinfection, and consequently have a higher risk of anal cancer.

Data suggest that certain sexual practices, such as receptive anal intercourse or a high lifetime number of sexual partners, portend an increased risk of anal cancer. These practices may have led to an increase in the number of individuals at risk of infection with HPV.[6]

American Joint Committee on Cancer (AJCC) Stage Groupings and TNM Definitions

The following is a staging system for anal canal cancer that has been described by the AJCC and the International Union Against Cancer.[1] The AJCC has designated staging by TNM (tumor, node, metastasis) classification to define anal cancer.

Capecitabine and Fluorouracil Dosing

Treatment Options for Stage 0 Anal Cancer

Stage 0 anal cancer is carcinoma in situ. Rarely diagnosed, it is a very early cancer that has not spread below the limiting membrane of the first layer of anal tissue.

Treatment options for stage 0 anal cancer include:

Current Clinical Trials

Use our advanced clinical trial search to find NCI-supported cancer clinical trials that are now enrolling patients. The search can be narrowed by location of the trial, type of treatment, name of the drug, and other criteria. General information about clinical trials is also available.

Treatment Options for Stages I, II, and III Anal Cancer

Current sphincter-sparing therapies include wide local excision for small tumors of the perianal skin or anal margin, or definitive chemoradiation therapy (fluorouracil [5-FU] and mitomycin) for cancers of the anal canal. Radical resection is reserved for patients with incomplete responses or recurrent disease.

Continued surveillance with rectal examination every 3 months for the first 2 years and endoscopy with biopsy when indicated after completion of sphincter-preserving therapy is important to monitor for recurrence.

Treatment options for stage I, stage II, and stage III anal cancer include:

Current Clinical Trials

Use our advanced clinical trial search to find NCI-supported cancer clinical trials that are now enrolling patients. The search can be narrowed by location of the trial, type of treatment, name of the drug, and other criteria. General information about clinical trials is also available.

Treatment Options for Stage IV Anal Cancer

Treatment options for stage IV anal cancer include:

Current Clinical Trials

Use our advanced clinical trial search to find NCI-supported cancer clinical trials that are now enrolling patients. The search can be narrowed by location of the trial, type of treatment, name of the drug, and other criteria. General information about clinical trials is also available.

Current Clinical Trials

Use our advanced clinical trial search to find NCI-supported cancer clinical trials that are now enrolling patients. The search can be narrowed by location of the trial, type of treatment, name of the drug, and other criteria. General information about clinical trials is also available.

Current Clinical Trials

Use our advanced clinical trial search to find NCI-supported cancer clinical trials that are now enrolling patients. The search can be narrowed by location of the trial, type of treatment, name of the drug, and other criteria. General information about clinical trials is also available.

Purpose of This Summary

This PDQ cancer information summary for health professionals provides comprehensive, peer-reviewed, evidence-based information about the treatment of anal cancer. It is intended as a resource to inform and assist clinicians in the care of their patients. It does not provide formal guidelines or recommendations for making health care decisions.

Reviewers and Updates

This summary is reviewed regularly and updated as necessary by the PDQ Adult Treatment Editorial Board, which is editorially independent of the National Cancer Institute (NCI). The summary reflects an independent review of the literature and does not represent a policy statement of NCI or the National Institutes of Health (NIH).

Board members review recently published articles each month to determine whether an article should:

Changes to the summaries are made through a consensus process in which Board members evaluate the strength of the evidence in the published articles and determine how the article should be included in the summary.

The lead reviewers for Anal Cancer Treatment are:

Any comments or questions about the summary content should be submitted to Cancer.gov through the NCI website’s Email Us. Do not contact the individual Board Members with questions or comments about the summaries. Board members will not respond to individual inquiries.

Levels of Evidence

Some of the reference citations in this summary are accompanied by a level-of-evidence designation. These designations are intended to help readers assess the strength of the evidence supporting the use of specific interventions or approaches. The PDQ Adult Treatment Editorial Board uses a formal evidence ranking system in developing its level-of-evidence designations.

Permission to Use This Summary

PDQ is a registered trademark. Although the content of PDQ documents can be used freely as text, it cannot be identified as an NCI PDQ cancer information summary unless it is presented in its entirety and is regularly updated. However, an author would be permitted to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks succinctly: [include excerpt from the summary].”

The preferred citation for this PDQ summary is:

PDQ® Adult Treatment Editorial Board. PDQ Anal Cancer Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: /types/anal/hp/anal-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389221]

Images in this summary are used with permission of the author(s), artist, and/or publisher for use within the PDQ summaries only. Permission to use images outside the context of PDQ information must be obtained from the owner(s) and cannot be granted by the National Cancer Institute. Information about using the illustrations in this summary, along with many other cancer-related images, is available in Visuals Online, a collection of over 2,000 scientific images.

Disclaimer

Based on the strength of the available evidence, treatment options may be described as either “standard” or “under clinical evaluation.” These classifications should not be used as a basis for insurance reimbursement determinations. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page.

Contact Us

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 Email Us.