Central nervous system (CNS) atypical teratoid/rhabdoid tumor (AT/RT) is a very rare, fast-growing tumor that begins in the brain and spinal cord. It usually occurs in children aged 3 years and younger, although it can occur in older children and adults.
About half of these tumors form in the cerebellum or brain stem. The cerebellum is the part of the brain that controls movement, balance, and posture. The brain stem controls breathing, heart rate, and the nerves and muscles used in seeing, hearing, walking, talking, and eating. AT/RT can also begin in other parts of the brain and spinal cord.
This summary describes the treatment of CNS AT/RT. Treatment of metastatic brain tumors, which are tumors formed by cancer cells that begin in other parts of the body and spread to the brain, is not covered in this summary.
Brain tumors can occur in both children and adults; however, treatment for children may be different than treatment for adults. To learn about treatment for adults, see Adult Central Nervous System Tumors Treatment.
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 AT/RT, and it will develop in some children who don't have a known risk factor. Talk with your child's doctor if you think your child may be at risk.
AT/RT may be linked to changes in the tumor suppressor genes SMARCB1 or SMARCA4. Tumor suppressor genes make a protein that helps control how and when cells grow. Changes in the DNA of tumor suppressor genes like SMARCB1 or SMARCA4 may lead to cancer.
The changes in the SMARCB1 or SMARCA4 genes may be inherited (passed on from parents to offspring). When this gene change is inherited, tumors may form in two parts of the body at the same time (for example, in the brain and the kidney). For patients with AT/RT, genetic counseling (a discussion with a trained professional about inherited diseases and a possible need for gene testing) may be recommended.
Signs and symptoms depend on the following:
Because AT/RT is fast growing, signs and symptoms may develop quickly and get worse over a period of days or weeks. Signs and symptoms may be caused by AT/RT or by other conditions. Check with your child's doctor if your child has any of the following:
In addition to asking about your child's personal and family health history and doing a physical exam, your child's doctor may perform the following tests and procedures:
If doctors think there might be a brain tumor, a biopsy may be done to remove a sample of tissue. For brain tumors, the biopsy can be done by removing part of the skull or making a small hole in the skull and using a needle or surgical device to remove a sample of tissue. Sometimes, when a needle is used, it is guided by a computer 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 pathologist checks the cancer cells to find out the type of brain tumor. It is often difficult to completely remove AT/RT because of where the tumor is in the brain and because it may already have spread at the time of diagnosis. The piece of skull is usually put back in place after the procedure.
The following test may be done on the sample of tissue that is removed:
The prognosis and treatment options depend on the following:
The process used to find out if cancer has spread to other parts of the body is called staging. There is no standard staging system for central nervous system (CNS) atypical teratoid/rhabdoid tumor (AT/RT).
For treatment, this tumor is grouped as newly diagnosed or recurrent. Treatment depends on the following:
Results from the following procedure are also used to plan treatment:
Different types of treatment are available for patients with central nervous system (CNS) atypical teratoid/rhabdoid tumor (AT/RT). Treatment for AT/RT is often within a clinical trial. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer.
Clinical trials are taking place in many parts of the country. Information about ongoing clinical trials is available from the NCI website. Choosing the most appropriate cancer treatment is a decision that ideally involves the patient, family, and health care team.
Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating children with CNS cancer and who specialize in certain areas of medicine. Other specialists may include:
Signs or symptoms caused by the tumor may begin before diagnosis. These signs or symptoms may continue for months or years. It is important to talk with your child's doctors about signs or symptoms caused by the tumor that may continue after treatment.
Surgery is used to diagnose and treat CNS AT/RT. To learn more about how this tumor is diagnosed, see the General Information section.
After the doctor removes all the cancer that can be seen at the time of the surgery, most patients will be given chemotherapy and possibly radiation therapy to try to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy.
Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach tumor cells throughout the body (systemic chemotherapy). Some oral chemotherapy drugs can cross the blood-brain barrier and reach the tumor. High doses of some chemotherapy drugs given into a vein can cross the blood-brain barrier and reach the tumor. When chemotherapy is placed directly into the cerebrospinal fluid, it is called intrathecal chemotherapy. Combination chemotherapy uses more than one anticancer drug.
Childhood CNS AT/RT is treated with systemic and intrathecal chemotherapy.
Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer.
External radiation therapy may be given to the brain and spinal cord.
Because radiation therapy can affect growth and brain development in young children, especially children who are 3 years old or younger, the dose of radiation therapy may be lower than in older children.
High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells.
This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website.
Targeted therapy uses drugs or other substances to block the action of specific enzymes, proteins, or molecules involved in the growth and spread of cancer cells. Alisertib is a type of targeted therapy being studied in the treatment of recurrent childhood CNS AT/RT.
Immunotherapy helps a person's immune system fight cancer. Biomarker tests can be used to help predict your response to certain immunotherapy drugs.
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 the following:
Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. To learn more, see Late Effects of Treatment for Childhood Cancer.
For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment.
Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment.
Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward.
Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment.
Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website.
Some of the tests that were done to diagnose the cancer may be repeated. Some tests will 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 child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check ups.
For information about the treatments listed below, see the Treatment Option Overview section.
There is no standard treatment for children with newly diagnosed central nervous system (CNS) atypical teratoid/rhabdoid tumor (AT/RT). Because AT/RT is fast-growing, a combination of treatments is usually given.
After surgery to remove the tumor, treatments for AT/RT may include combinations of the following:
Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available.
For information about the treatments listed below, see the Treatment Option Overview section.
There is no standard treatment for children with recurrent childhood central nervous system atypical teratoid/rhabdoid tumor. Treatment may include the following:
Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available.
For more information about childhood central nervous system atypical teratoid/rhabdoid tumor and other childhood brain tumors, see the following:
For more childhood cancer information and other general cancer resources, visit:
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