• neurofibromatosis type 1
• Turcot syndrome
• Li-Fraumeni syndrome
• tuberous sclerosis

KEY POINTS

• Childhood astrocytomas are tumors that start in the star-shaped brain cells called astrocytes.
• Astrocytomas may be low-grade or high-grade.
• The central nervous system controls many important body functions.
• The cause of most childhood brain tumors is not known.
• The signs and symptoms of astrocytomas are not the same in every child.
• Tests that examine the brain and spinal cord are used to detect (find) childhood astrocytomas.
• Childhood astrocytomas are usually diagnosed and removed in surgery.
• Certain factors affect prognosis (chance of recovery) and treatment options.

There are different types of treatment for patients with childhood astrocytoma.

Different types of treatment are available for children with astrocytomas. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment.

Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment.

Children with astrocytomas should have their treatment planned by a team of health care providers who are experts in treating childhood brain tumors.

Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other healthcare providers who are experts in treating children with brain tumors and who specialize in certain areas of medicine. These may include the following specialists:

• Pediatrician.
• Pediatric neurosurgeon.
• Neurologist.
• Neuropathologist.
• Neuroradiologist.
• Rehabilitation specialist.
• Radiation oncologist.
• Endocrinologist.
• Psychologist.

Childhood brain tumors may cause signs or symptoms that begin before the cancer is diagnosed and continue for months or years.

Signs or symptoms caused by the tumor may begin before 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.

Six types of treatment are used:

Surgery

Surgery is used to diagnose and treat childhood astrocytoma, as discussed in the General Information section of this summary. After surgery an MRI (magnetic resonance imaging) is done to see if any cancer cells remain. If cancer cells are found, further treatment depends on:

• Where the remaining cancer cells are.
• The grade of the tumor.
• The age of the child.

After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy.

Observation

Observation is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. Observation may be used:

• If the patient has no symptoms, such as patients with neurofibromatosis type1.
• If the tumor is small and is found when a different health problem is being diagnosed or treated.
• After the tumor is removed by surgery until signs or symptoms appear or change.

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.

Certain ways of giving radiation therapy can help keep radiation from damaging nearby healthy tissue. These types of radiation therapy include the following:

• Conformal radiation therapy: Conformal radiation therapy is a type of external radiation therapy that uses a computer to make a 3-dimensional (3-D) picture of the tumor and shapes the radiation beams to fit the tumor.
• Intensity-modulated radiation therapy (IMRT): IMRT is a type of 3-dimensional (3-D) external 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 radiation therapy: Stereotactic radiation therapy is a type of external radiation therapy. A rigid head frame is attached to the skull to keep the head still during the radiation treatment. A machine aims radiation directly at the tumor. The total dose of radiation is divided into several smaller doses given over several days. This procedure is also called stereotactic external-beam radiation therapy and stereotaxic radiation therapy.
• Proton beam radiation therapy: Proton beam radiation therapy is a type of high-energy, external radiation therapy that uses streams of protons (tiny particles with a positive charge) to kill tumor cells. This type of treatment can lower the amount of radiation damage to healthy tissue near a tumor.

The way the radiation therapy is given depends on the type of tumor and where the tumor formed in the brain or spinal cord. External radiation therapy is used to treat childhood astrocytomas.

Radiation therapy to the brain can affect growth and development, especially in young children. For children younger than 3 years, chemotherapy may be given instead, to delay or reduce the need for radiation therapy.

Chemotherapy

Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Combination chemotherapy is the use of more than one anticancer drug.

The way the chemotherapy is given depends on the type of tumor and where the tumor formed in the brain or spinal cord. Systemic combination chemotherapy is used in the treatment of children with astrocytoma. High-dose chemotherapy may be used in the treatment of children with newly diagnosed high-grade astrocytoma.

High-dose chemotherapy with stem cell transplant

High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells.

For high-grade astrocytoma that has come back after treatment, high-dose chemotherapy with stem cell transplant is used if there is only a small amount of tumor.

Targeted therapy

Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do.

There are different types of targeted therapy:

• Monoclonal antibodies: Monoclonal antibodies are immune system proteins made in the laboratory to treat many diseases, including cancer. As a cancer treatment, these antibodies can attach to a specific target on cancer cells or other cells that may help cancer cells grow. The antibodies are able to then kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. Bevacizumab is a monoclonal antibody and vascular endothelial growth factor inhibitor that binds to a protein called vascular endothelial growth factor (VEGF) and may prevent the growth of new blood vessels that tumors need to grow. Bevacizumab is used to treat childhood astrocytoma.
• Protein kinase inhibitors work in different ways. There are several kinds of protein kinase inhibitors.
  • mTOR inhibitors: This treatment stops the protein that helps cells divide and survive. Everolimus and sirolimus are mTOR inhibitors used to treat childhood subependymal giant cell astrocytomas.
  • BRAF inhibitors: This treatment blocks the activity of proteins needed for cell growth and may kill cancer cells. The BRAF gene is found in a mutated (changed) form in some gliomas and blocking it may help keep cancer cells from growing. The combination of the BRAF inhibitors dabrafenib and trametinib are being studied to treat newly diagnosed low-grade astrocytomas or high-grade astrocytomas that have recurred or stopped responding to treatment. Dabrafenib and trametinib given after radiation are also being studied to treat newly diagnosed high-grade gliomas.
  • MEK inhibitors: This treatment blocks proteins needed for cell growth and may kill cancer cells. The MEK inhibitor is being studied to treat low-grade astrocytoma that has recurred or stopped responding to treatment. It is also being studied in combination with chemotherapy to treat newly diagnosed NF1-associated low-grade gliomas.
  • PARP inhibitors: This treatment blocks DNA repair and may cause cancer cells to die. Veliparib is a PARP inhibitor that is being studied in combination with radiation therapy and chemotherapy to treat newly diagnosed malignant glioma that does not have mutations (changes) in the BRAF gene.

See Drugs Approved for Brain Tumors for more information.

New types of treatment are being tested in clinical trials.

This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website.

Immunotherapy

Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This cancer treatment is a type of biologic therapy.

• Immune checkpoint inhibitor therapy: Some types of immune cells, such as T cells, and some cancer cells have certain proteins, called checkpoint proteins, on their surface that keep immune responses in check. When cancer cells have large amounts of these proteins, they will not be attacked and killed by T cells. Immune checkpoint inhibitors block these proteins and the ability of T cells to kill cancer cells is increased.
  • PD-1 and PD-L1 inhibitor therapy: PD-1 is a protein on the surface of T cells that helps keep the body’s immune responses in check. PD-L1 is a protein found on some types of cancer cells. When PD-1 attaches to PD-L1, it stops the T cell from killing the cancer cell. PD-1 and PD-L1 inhibitors keep PD-1 and PD-L1 proteins from attaching to each other. This allows the T cells to kill cancer cells. PD-1 inhibitors are being studied to treat high-grade astrocytoma that has recurred.

If fluid builds up around the brain and spinal cord, a cerebrospinal fluid diversion procedure may be done.

Cerebrospinal fluid diversion is a method used to drain fluid that has built up around the brain and spinal cord. A shunt (long, thin tube) is placed in a ventricle (fluid-filled space) of the brain and threaded under the skin to another part of the body, usually the abdomen. The shunt carries extra fluid away from the brain so it may be absorbed elsewhere in the body.

Treatment for childhood astrocytomas may cause side effects.

For information about side effects that begin during treatment for cancer, see our Side Effects page.

Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following:

• Physical problems that affect the following:
  • Vision problems, including blindness.
  • Blood vessel problems.
  • Endocrine function.
• Changes in mood, feelings, thinking, learning, or memory.
• Second cancers (new types of cancer).

Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information.)

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.

Follow-up tests may be needed.

Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests.

Regular MRIs will continue to be done after treatment has ended. The results of the MRI can show if your child's condition has changed or if the astrocytoma has recurred (come back). If the results of the MRI show a mass in the brain, a biopsy may be done to find out if it is made up of dead tumor cells or if new cancer cells are growing.

Treatment of Childhood Low-Grade Astrocytomas

When the tumor is first diagnosed, treatment for childhood low-grade astrocytoma depends on where the tumor is, and is usually surgery. An MRI is done after surgery to see if there is tumor remaining.

If the tumor was completely removed by surgery, more treatment may not be needed and the child is closely watched to see if signs or symptoms appear or change. This is called observation.

If there is tumor remaining after surgery, treatment may include the following:

• Observation.
• Combination chemotherapy with or without radiation therapy.
• Radiation therapy, which may include conformal radiation therapy, intensity-modulated radiation therapy, proton beam radiation therapy, or stereotactic radiation therapy, when the tumor begins to grow again.
• Targeted therapy with mTOR inhibitors (everolimus and sirolimus) to treat subependymal giant cell astrocytoma.
• A clinical trial of targeted therapy with a MEK inhibitor (selumetinib).

In some cases, observation is used for children who have a visual pathway glioma. In other cases, treatment may include surgery to remove the tumor, radiation therapy, or chemotherapy. A goal of treatment is to save as much vision as possible. In most patients who receive radiation therapy, the tumor is controlled, but changes in vision may develop. These changes are most likely to occur within 2 years after radiation therapy. The effect of tumor growth on the child's vision will be closely followed during and after treatment.

Children with neurofibromatosis type 1 (NF1) may not need treatment unless the tumor grows or signs or symptoms, such as vision problems, appear. When the tumor grows or signs or symptoms appear, treatment may include surgery to remove the tumor, radiation therapy, and/or chemotherapy.

Children with tuberous sclerosis may develop low-grade tumors in the brain called subependymal giant cell astrocytoma (SEGAs). Targeted therapy with everolimus or sirolimus may be used instead of surgery, to shrink the tumors.

Treatment of Progressive or Recurrent Childhood Low-Grade Astrocytomas

When low-grade astrocytoma recurs after treatment, it usually comes back where the tumor first formed. Before more cancer treatment is given, imaging tests, biopsy, or surgery are done to find out if there is cancer and how much there is.

Treatment of progressive or recurrent childhood low-grade astrocytoma may include the following:

• A second surgery to remove the tumor, if surgery was the only treatment given when the tumor was first diagnosed.
• Radiation therapy to the tumor only, if radiation therapy was not used when the tumor was first diagnosed. Conformal radiation therapy may be given.
• Chemotherapy, if the tumor recurred where it cannot be removed by surgery.
• Targeted therapy with a monoclonal antibody (bevacizumab) with or without chemotherapy.
• A clinical trial that checks a sample of the patient's tumor for certain gene changes. The type of targeted therapy that will be given to the patient depends on the type of gene change.
• A clinical trial of targeted therapy with a BRAF inhibitor (dabrafenib) or a MEK inhibitor (selumetinib).
• A clinical trial of targeted therapy with an mTOR inhibitor (everolimus).

Treatment of Childhood High-Grade Astrocytomas

Treatment of newly diagnosed childhood high-grade astrocytoma may include the following:

• Surgery to remove the tumor, followed by chemotherapy and/or radiation therapy.
• A clinical trial of targeted therapy with a PARP inhibitor (veliparib) combined with radiation therapy and chemotherapy to treat newly diagnosed malignant glioma that does not have mutations (changes) in the BRAF gene.
• A clinical trial of targeted therapy with a combination of BRAF inhibitors (dabrafenib and trametinib) after radiation therapy to treat newly diagnosed high-grade glioma that has mutations in the BRAF gene.

Treatment of Recurrent Childhood High-Grade Astrocytomas

When high-grade astrocytoma recurs after treatment, it usually comes back where the tumor first formed. Before more cancer treatment is given, imaging tests, biopsy, or surgery are done to find out if there is cancer and how much there is.

Treatment of recurrent childhood high-grade astrocytoma may include the following:

• Surgery to remove the tumor.
• High-dose chemotherapy with stem cell transplant.
• Radiation therapy.
• Targeted therapy with a BRAF inhibitor (vemurafenib or dabrafenib).
• A clinical trial of immunotherapy with an immune checkpoint inhibitor.
• A clinical trial that checks a sample of the patient's tumor for certain gene changes. The type of targeted therapy that will be given to the patient depends on the type of gene change.