COVID-19: Vaccine | Testing | Self-assessment | Patient & Visitor Safety | Visitor Policy
Emergency Room Wait Times
Home > Living Well > Health Library > Neuroblastoma Treatment (PDQ®): Treatment - Patient Information [NCI]
This information is produced and provided by the National Cancer Institute (NCI). The information in this topic may have changed since it was written. For the most current information, contact the National Cancer Institute via the Internet web site at http://cancer.gov or call 1-800-4-CANCER.
Neuroblastoma is a disease in which malignant (cancer) cells form in neuroblasts (immature nerve tissue) in the adrenal glands, neck, chest, or spinal cord.
Neuroblastoma often begins in the nerve tissue of the adrenal glands. There are two adrenal glands, one on top of each kidney in the back of the upper abdomen. The adrenal glands make important hormones that help control heart rate, blood pressure, blood sugar, and the way the body reacts to stress. Neuroblastoma may also begin in nerve tissue in the neck, chest, abdomen, or pelvis. Neuroblastoma may be found in the adrenal glands and paraspinal nerve tissue from the neck to the pelvis.
Neuroblastoma most often begins in infancy. It is usually diagnosed between the first month of life and age five years. It is found when the tumor begins to grow and cause signs or symptoms. Sometimes it forms before birth and is found during an ultrasound of the baby.
By the time cancer is diagnosed, it has usually metastasized (spread). Neuroblastoma spreads most often to the lymph nodes, bones, bone marrow, liver, and skin in infants and children. Adolescents may also have metastasis to the lungs and brain.
Neuroblastoma is sometimes caused by a gene mutation (change) passed from the parent to the child.
Gene mutations that increase the risk of neuroblastoma are sometimes inherited (passed from the parent to the child). In children with a gene mutation, neuroblastoma usually occurs at a younger age and more than one tumor may form in the adrenal glands or in the nerve tissue in the neck, chest, abdomen, or pelvis.
Children with certain gene mutations or hereditary (inherited) syndromes should be checked for signs of neuroblastoma until they are 10 years old. The following tests may be used:
Talk to your child's doctor about how often these tests need to be done.
Signs and symptoms of neuroblastoma include a lump in the abdomen, neck, or chest or bone pain.
The most common signs and symptoms of neuroblastoma are caused by the tumor pressing on nearby tissues as it grows or by cancer spreading to the bone. These and other signs and symptoms may be caused by neuroblastoma or by other conditions.
Check with your child's doctor if your child has any of the following:
Less common signs and symptoms of neuroblastoma include the following:
Tests that examine many different body tissues and fluids are used to diagnose neuroblastoma.
The following tests and procedures may be used to diagnose neuroblastoma:
A biopsy is done to diagnose neuroblastoma.
Cells and tissues are removed during a biopsy so they can be viewed under a microscope by a pathologist to check for signs of cancer. The way the biopsy is done depends on where the tumor is in the body. Sometimes the whole tumor is removed at the same time the biopsy is done.
The following tests may be done on the tissue that is removed:
Children up to 6 months old may not need a biopsy or surgery to remove the tumor because the tumor may disappear without treatment.
Certain factors affect prognosis (chance of recovery) and treatment options.
The prognosis and treatment options depend on the following:
Prognosis and treatment options for neuroblastoma are also affected by tumor biology, which includes the following:
The tumor biology is said to be favorable or unfavorable, depending on these factors. A child with favorable tumor biology has a better chance of recovery.
In some children up to 6 months old, neuroblastoma may disappear without treatment. This is called spontaneous regression. The child is closely watched for signs or symptoms of neuroblastoma. If signs or symptoms occur, treatment may be needed.
After neuroblastoma has been diagnosed, tests are done to find out if cancer has spread from where it started to other parts of the body.
The process used to find out the extent or spread of cancer is called staging. The information gathered from the staging process helps determine the stage of the disease. For neuroblastoma, the stage of disease affects whether the cancer is low risk, intermediate risk, or high risk. It also affects the treatment plan. The results of some tests and procedures used to diagnose neuroblastoma may be used for staging. See the General Information section for a description of these tests and procedures.
The following tests and procedures also may be used to determine the stage:
There are three ways that cancer spreads in the body.
Cancer can spread through tissue, the lymph system, and the blood:
Cancer may spread from where it began to other parts of the body.
When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood.
The metastatic tumor is the same type of cancer as the primary tumor. For example, if neuroblastoma spreads to the liver, the cancer cells in the liver are actually neuroblastoma cells. The disease is metastatic neuroblastoma, not liver cancer.
The following stages are used for neuroblastoma:
In stage 1, the cancer is in only one area and all of the cancer that can be seen is completely removed during surgery.
Stage 2 is divided into stages 2A and 2B.
In stage 3, one of the following is true:
Stage 4 is divided into stages 4 and 4S.
Treatment of neuroblastoma is based on risk groups.
For many types of cancer, stages are used to plan treatment. For neuroblastoma, treatment depends on the patient's risk group. The stage of neuroblastoma is one factor used to determine risk group. Other factors are age, tumor histology, and tumor biology.
There are three risk groups: low risk, intermediate risk, and high risk.
Sometimes neuroblastoma does not respond to treatment or comes back after treatment.
Refractory neuroblastoma is a tumor that does not respond to treatment.
Recurrent neuroblastoma is cancer that has recurred (come back) after it has been treated. The tumor may come back in the site where it began or in the central nervous system.
There are different types of treatment for patients with neuroblastoma.
Different types of treatment are available for patients with neuroblastoma. 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 neuroblastoma should have their treatment planned by a team of doctors who are experts in treating childhood cancer, especially neuroblastoma.
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 neuroblastoma and who specialize in certain areas of medicine. These may include the following specialists:
Seven types of standard treatment are used:
Observation is closely monitoring a patient's condition without giving any treatment until signs or symptoms appear or change.
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.
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.
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.
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).
The use of two or more anticancer drugs is called combination chemotherapy.
See Drugs Approved for Neuroblastoma for more information.
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 patient completes 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.
Maintenance therapy is given after high-dose chemotherapy and radiation therapy with stem cell rescue for 6 months and includes the following treatments:
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 therapies:
Pembrolizumab and dinutuximab are monoclonal antibodies being studied to treat neuroblastoma that has come back after treatment or has not responded to treatment.
Crizotinib is a tyrosine kinase inhibitor used to treat neuroblastoma that has come back after treatment. AZD1775 and lorlatinib are tyrosine kinase inhibitors being studied to treat neuroblastoma that has come back after treatment or has not responded to treatment.
Vorinostat is a type of histone deacetylase inhibitor being studied to treat neuroblastoma that has come back after treatment or has not responded to treatment.
Eflornithine is a type of ornithine decarboxylase inhibitor being studied to treat neuroblastoma that has come back after treatment or has not responded to treatment.
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 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.
CAR T-cell therapy is being studied to treat neuroblastoma that has come back after treatment or has not responded to treatment.
Treatment for neuroblastoma causes side effects and late 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:
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. 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.
Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests.
Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups.
Follow-up tests for patients with neuroblastoma include the following:
For information about the treatments listed below, see the Treatment Option Overview section.
Treatment of newly diagnosed low-risk neuroblastoma 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.
Treatment of newly diagnosed intermediate-risk neuroblastoma may include the following:
Treatment of newly diagnosed high-risk neuroblastoma may include the following:
There is no standard treatment for newly diagnosed stage 4S neuroblastoma but treatment options include the following:
Patients First Treated for Low-Risk Neuroblastoma
Treatment for recurrent neuroblastoma that comes back in the area where the cancer first formed may include the following:
Treatment for recurrent neuroblastoma that comes back in other parts of the body or that has not responded to treatment may include the following:
Patients First Treated for Intermediate-Risk Neuroblastoma
Treatment for recurrent neuroblastoma that comes back in other parts of the body may include the following:
Patients First Treated for High-Risk Neuroblastoma
There is no standard treatment for recurrent neuroblastoma in patients first treated for high-risk neuroblastoma. Treatment may include the following:
Because there is no standard treatment, patients first treated for high-risk neuroblastoma may want to consider a clinical trial. For information about clinical trials, please see the NCI website.
Patients with Recurrent CNS Neuroblastoma
Treatment for neuroblastoma that recurs (comes back) in the central nervous system (CNS; brain and spinal cord) may include the following:
Treatments Being Studied for Progressive/Recurrent Neuroblastoma
Some of the treatments being studied in clinical trials for neuroblastoma that recurs (comes back) or progresses (grows, spreads, or does not respond to treatment) include the following:
For more information from the National Cancer Institute about neuroblastoma, see the following:
For more childhood cancer information and other general cancer resources, see the following:
Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish.
PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government's center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH.
Purpose of This Summary
This PDQ cancer information summary has current information about the treatment of neuroblastoma. 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 Neuroblastoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/neuroblastoma/patient/neuroblastoma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389278]
Images in this summary are used with permission of the author(s), artist, and/or publisher for use in the PDQ summaries only. If you want to use an image from a PDQ summary and you are not using the whole summary, you must get permission from the owner. It cannot be given by the National Cancer Institute. Information about using the images in this summary, along with many other images related to cancer can be found in Visuals Online. Visuals Online is a collection of more than 3,000 scientific images.
The information in these summaries should not be used to make decisions about insurance reimbursement. More information on insurance coverage is available on Cancer.gov on the Managing Cancer Care page.
More information about contacting us or receiving help with the Cancer.gov website can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the website's E-mail Us.
Last Revised: 2021-06-10
If you want to know more about cancer and how it is treated, or if you wish to know about clinical trials for your type of cancer, you can call the NCI's Cancer Information Service at 1-800-422-6237, toll free. A trained information specialist can talk with you and answer your questions.
Healthwise, Healthwise for every health decision, and the Healthwise logo are trademarks of Healthwise, Incorporated.
Disclaimer: The information contained in this website, and its associated websites, is provided as a benefit to the local community, and the Internet community in general; it does not constitute medical advice. We try to provide quality information, but we make no claims, promises or guarantees about the accuracy, completeness, or adequacy of the information contained in or linked to this website and its associated sites. As medical advice must be tailored to the specific circumstances of each patient and healthcare is constantly changing, nothing provided herein should be used as a substitute for the advice of a competent physician. Furthermore, in providing this service, Adventist HealthCare does not condone or support all of the content covered in this site. As an Adventist health care organization, Adventist HealthCare acts in accordance with the ethical and religious directives for Adventist health care services.
Find an Adventist HealthCare affiliated doctor by calling our FREE physician referral service at 800-642-0101 or by searching our online physician directory.
Set Your Location
Setting your location helps us to show you nearby providers and locations based on your healthcare needs.