Neuroblastoma Treatment Option Overview

There are different types of treatment for children with neuroblastoma.

There are different types of treatment for children and adolescents with neuroblastoma. You and your child's cancer team will work together to decide treatment. Many factors will be considered, such as your child's overall health and whether the cancer is newly diagnosed or has come back.

Children with neuroblastoma should have their treatment planned by a team of doctors who are experts in treating childhood cancer, especially neuroblastoma.

A pediatric oncologist, a doctor who specializes in treating children with cancer, will oversee treatment. The pediatric oncologist works with other pediatric health care professionals who are experts in treating children with cancer and who specialize in certain areas of medicine. These may include the following specialists and others:

• pediatric surgeon
• pediatric radiation oncologist
• endocrinologist
• neurologist
• pediatric neuropathologist
• neuroradiologist
• pediatrician
• pediatric nurse specialist
• social worker
• child life professional
• psychologist

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:

Observation

Observation is closely monitoring a patient's condition without giving any treatment until signs or symptoms appear or change.

Surgery

Surgery is used to treat neuroblastoma that has not spread to other parts of the body. As much of the tumor as is safely possible is removed. Lymph nodes are also removed and checked for signs of cancer.

If the tumor cannot be removed, a biopsy may be done instead.

Learn more about Surgery to Treat Cancer.

Chemotherapy

Chemotherapy (also called chemo) 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).

The use of two or more anticancer drugs is called combination chemotherapy.

See Drugs Approved for Neuroblastoma for more information.

Learn more about Chemotherapy to Treat Cancer.

Radiation therapy

Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are different types of radiation therapy:

• External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. This treatment is used for patients with high-risk neuroblastoma or for patients whose tumor grew while being treated with chemotherapy.
• Internal radiation therapy uses a radioactive substance sealed in needles, seeds, wires, or catheters that are placed directly into or near the cancer.
• Radioimmunotherapy is a type of radiation therapy in which a radioactive substance is linked to a monoclonal antibody and injected into the body. The monoclonal antibody can bind to substances in the body, including cancer cells. The radioactive substance gives off radiation, which may help kill cancer cells.

Learn more about Radiation Therapy to Treat Cancer.

High-dose chemotherapy and radiation therapy with stem cell rescue

High-dose chemotherapy and radiation therapy are given to kill any cancer cells that may regrow and cause the cancer to come back. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell rescue 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 completion of chemotherapy and radiation therapy, 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.

Iodine 131-MIBG therapy

Iodine 131-MIBG therapy is a treatment with radioactive iodine. The radioactive iodine is given through an intravenous (IV) line and enters the bloodstream, which carries radiation directly to tumor cells. Radioactive iodine collects in neuroblastoma cells and kills them with the radiation that is given off. Iodine 131-MIBG therapy is sometimes used to treat high-risk neuroblastoma that comes back after initial treatment.

Targeted therapy

Targeted therapy uses drugs or other substances to block the action of specific enzymes, proteins, or other molecules involved in the growth and spread of cancer cells.

• Monoclonal antibody therapy: 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.
  • Dinutuximab is used to treat patients with high-risk neuroblastoma and neuroblastoma that has come back after treatment or has not responded to treatment.
• Ornithine decarboxylase inhibitor therapy slows the growth and division of cancer cells.
  • Eflornithine may be given for two years after maintenance therapy for high-risk neuroblastoma.

Learn more about Targeted Therapy to Treat Cancer.

Other drug therapy

Other drugs used in combination to treat neuroblastoma include:

• Isotretinoin: A vitamin-like drug that slows the cancer's ability to make more cancer cells and changes how these cells look and act. This drug is taken by mouth.

Immunotherapy

Immunotherapy helps a child's immune system fight cancer.

• Granulocyte-macrophage colony-stimulating factor (GM-CSF): a cytokine that helps the body make more white blood cells, especially granulocytes and macrophages, which can attack and kill cancer cells. GM-CSF can also enhance the immune system response against cancer by increasing the number of cancer-fighting T cells.
• CAR T-cell therapy: The patient's T cells (a type of immune system cell) are changed so that they will attack certain proteins on the surface of cancer cells. T cells are taken from the patient, and special receptors are added to their surface in the laboratory. The changed cells are called chimeric antigen receptor (CAR) T cells. The CAR T cells are grown in the laboratory and given to the patient by infusion. The CAR T cells multiply in the patient's blood and attack cancer cells.

  CAR T-cell therapy is being studied to treat neuroblastoma that has come back after treatment or has not responded to treatment.

Learn more about T-Cell Transfer Therapy.

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 people with cancer. For some patients, taking part in a clinical trial may be an option. Because cancer in children is rare, taking part in a clinical trial should be considered.

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. Some clinical trials are open only to patients who have not started treatment. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website.

Learn more at Clinical Trials Information for Patients and Caregivers.

Treatment for neuroblastoma causes side effects and late effects.

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

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:

• physical problems, such as:
  • tooth development
  • intestinal blockage (obstruction)
  • bone and cartilage growth
  • hearing function
  • metabolic syndrome (elevated blood pressure, elevated triglycerides, elevated cholesterol, increased body fat percentage)
• changes in mood, feelings, thinking, learning, or memory
• second cancers (new types of cancer), such as myelodysplastic syndrome, acute myeloid leukemia, thyroid cancer, or kidney 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. Learn more about Late Effects of Treatment for Childhood Cancer.

Follow-up care may be needed.

As your child goes through treatment, they 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 child's condition has changed or if the cancer has recurred (come back).

Follow-up care for children with neuroblastoma may include:

• urine catecholamine studies
• MIBG scan