View source for Types/neuroblastoma/patient/neuroblastoma-treatment-pdq

<languages/>
<translate>
== Neuroblastoma Treatment (PDQ®)–Patient Version ==

=== General Information About Neuroblastoma ===

'''KEY POINTS'''

*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 is sometimes caused by a gene mutation (change) passed from the parent to the child.

*Signs and symptoms of neuroblastoma include a lump in the abdomen, neck, or chest or bone pain.

*Tests that examine many different body tissues and fluids are used to diagnose neuroblastoma.

*A biopsy is done to diagnose neuroblastoma.

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

'''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.

[[File:756627-750.jpg|600px|thumb|center|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:

*'''Abdominal ultrasound:''' A test in which high-energy sound waves (ultrasound) are bounced off the abdomen and make echoes. The echoes form a picture of the abdomen called a sonogram. The picture can be printed to be looked at later.

*'''Urine catecholamine studies:''' A test in which a urine sample is checked to measure the amount of certain substances, vanillylmandelic acid (VMA) and homovanillic acid (HVA), that are made when catecholamines break down and are released into the urine. A higher than normal amount of VMA or HVA can be a sign of neuroblastoma.

*'''Chest x-ray:''' An x-ray of the organs and bones inside the chest. An x-ray is a type of energy beam that can go through the body and onto film, making a picture of areas inside the body.

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:

*Lump in the abdomen, neck, or chest.

*Bone pain.

*Swollen stomach and trouble breathing (in infants).

*Bulging eyes.

*Dark circles around the eyes ("black eyes").

*Painless, bluish lumps under the skin (in infants).

*Weakness or paralysis (loss of ability to move a body part).

Less common signs and symptoms of neuroblastoma include the following:

*Fever.

*Shortness of breath.

*Feeling tired.

*Easy bruising or bleeding.

*Petechiae (flat, pinpoint spots under the skin caused by bleeding).

*High blood pressure.

*Severe watery diarrhea.

*Horner syndrome (droopy eyelid, smaller pupil, and less sweating on one side of the face).

*Jerky muscle movements.

*Uncontrolled eye movements.

'''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:

*'''Physical exam and health history:''' An exam of the body to check general signs of health, including checking for signs of disease, such as lumps or anything else that seems unusual. A history of the patient’s health habits and past illnesses and treatments will also be taken.

*'''Neurological exam:''' A series of questions and tests to check the brain, spinal cord, and nerve function. The exam checks a person’s mental status, coordination, ability to walk normally, and how well the muscles, senses, and reflexes work. This may also be called a neuro exam or a neurologic exam.

*'''Urine catecholamine studies:''' A test in which a urine sample is checked to measure the amount of certain substances, vanillylmandelic acid (VMA) and homovanillic acid (HVA), that are made when catecholamines break down and are released into the urine. A higher than normal amount of VMA or HVA can be a sign of neuroblastoma.

*'''Blood chemistry studies:''' A test in which a blood sample is checked to measure the amounts of certain substances released into the blood by organs and tissues in the body. A higher or lower than normal amount of a substance can be a sign of disease.

*'''MIBG scan:''' A procedure used to find neuroendocrine tumors, such as neuroblastoma. A very small amount of a substance called radioactive MIBG is injected into a vein and travels through the bloodstream. Neuroendocrine tumor cells take up the radioactive MIBG and are detected by a scanner. Scans may be taken over 1-3 days. An iodine solution may be given before or during the test to keep the thyroid gland from absorbing too much of the MIBG. This test is also used to find out how well the tumor is responding to treatment. MIBG is used in high doses to treat neuroblastoma.

*'''CT scan (CAT scan):''' A procedure that makes a series of detailed pictures of areas inside the body, taken from different angles. The pictures are made by a computer linked to an x-ray machine. A dye may be injected into a vein or swallowed to help the organs or tissues show up more clearly. This procedure is also called computed tomography, computerized tomography, or computerized axial tomography.

[[File:775531-750.jpg|600px|thumb|center|Computed tomography (CT) scan of the abdomen. The child lies on a table that slides through the CT scanner, which takes x-ray pictures of the inside of the abdomen.]]

*'''MRI (magnetic resonance imaging) with gadolinium:''' A procedure that uses a magnet, radio waves, and a computer to make a series of detailed pictures of areas inside the body. A substance called gadolinium is injected into a vein. The gadolinium collects around the cancer cells so they show up brighter in the picture. This procedure is also called nuclear magnetic resonance imaging (NMRI).

[[File:775532-750.jpg|600px|thumb|center|Magnetic resonance imaging (MRI) of the abdomen. The child lies on a table that slides into the MRI scanner, which takes pictures of the inside of the body. The pad on the child’s abdomen helps make the pictures clearer.]]

*'''PET scan (positron emission tomography scan):''' A procedure to find malignant tumor cells in the body. A small amount of radioactive glucose (sugar) is injected into a vein. The PET scanner rotates around the body and makes a picture of where glucose is being used in the body. Malignant tumor cells show up brighter in the picture because they are more active and take up more glucose than normal cells do.

*'''X-ray of the chest or bone:''' An x-ray is a type of energy beam that can go through the body and onto film, making a picture of areas inside the body.

*'''Ultrasound exam:''' A procedure in which high-energy sound waves (ultrasound) are bounced off internal tissues or organs and make echoes. The echoes form a picture of body tissues called a sonogram. The picture can be printed to be looked at later. An ultrasound exam is not done if a CT/MRI has been done.

[[File:775457-750.jpg|600px|thumb|center|Abdominal ultrasound. An ultrasound transducer connected to a computer is pressed against the skin of the abdomen. The transducer bounces sound waves off internal organs and tissues to make echoes that form a sonogram (computer picture).]]

'''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:

*'''Cytogenetic analysis:''' A laboratory test in which the chromosomes of cells in a sample of tissue are counted and checked for any changes, such as broken, missing, rearranged, or extra chromosomes. Changes in certain chromosomes may be a sign of cancer. Cytogenetic analysis is used to help diagnose cancer, plan treatment, or find out how well treatment is working.

*'''Light microscopy:''' A laboratory test in which cells in a sample of tissue are viewed under regular and high-powered microscopes to look for certain changes in the cells.

*'''Immunohistochemistry:''' A laboratory test that uses antibodies to check for certain antigens (markers) in a sample of a patient’s tissue. The antibodies are usually linked to an enzyme or a fluorescent dye. After the antibodies bind to a specific antigen in the tissue sample, the enzyme or dye is activated, and the antigen can then be seen under a microscope. This type of test is used to help diagnose cancer and to help tell one type of cancer from another type of cancer.

*'''MYCN amplification study:''' A laboratory study in which tumor or bone marrow cells are checked for the level of MYCN. MYCN is important for cell growth. A higher level of MYCN (more than 10 copies of the gene) is called MYCN amplification. Neuroblastoma with MYCN amplification is more likely to spread in the body and less likely to respond to treatment.

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:

*Age at the time of diagnosis.

*Tumor histology (the shape, function, and structure of the tumor cells).

*The child's risk group.

*Whether there are certain changes in the genes.

*Where in the body the tumor started.

*The stage of the cancer.

*How the tumor responds to treatment.

*How much time passed between diagnosis and when the cancer recurred (for recurrent cancer).

Prognosis and treatment options for neuroblastoma are also affected by tumor biology, which includes the following:

*The patterns of the tumor cells.

*How different the tumor cells are from normal cells.

*How fast the tumor cells are growing.

*Whether the tumor shows MYCN amplification.

*Whether the tumor has changes in the ALK gene.

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.

=== Stages of Neuroblastoma ===

'''KEY POINTS'''

*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.

*There are three ways that cancer spreads in the body.

*Cancer may spread from where it began to other parts of the body.

*The following stages are used for neuroblastoma:

:*Stage 1

:*Stage 2

:*Stage 3

:*Stage 4

*Treatment of neuroblastoma is based on risk groups.

*Sometimes neuroblastoma does not respond to treatment or comes back after treatment.

'''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:

*'''Bone marrow aspiration and biopsy:''' The removal of bone marrow, blood, and a small piece of bone by inserting a hollow needle into the hipbone or breastbone. A pathologist views the bone marrow, blood, and bone under a microscope to look for signs of cancer.

[[File:775458-750.jpg|600px|thumb|center|Bone marrow aspiration and biopsy. After a small area of skin is numbed, a bone marrow needle is inserted into the child’s hip bone. Samples of blood, bone, and bone marrow are removed for examination under a microscope.]]

*'''Lymph node biopsy:''' The removal of all or part of a lymph node. A pathologist views the lymph node tissue under a microscope to check for cancer cells. One of the following types of biopsies may be done:

:*'''Excisional biopsy:''' The removal of an entire lymph node.

:*'''Incisional biopsy:''' The removal of part of a lymph node.

:*'''Core biopsy:''' The removal of tissue from a lymph node using a wide needle.

:*'''Fine-needle aspiration (FNA) biopsy:''' The removal of tissue or fluid from a lymph node using a thin needle.

'''There are three ways that cancer spreads in the body.'''

Cancer can spread through tissue, the lymph system, and the blood:

*Tissue. The cancer spreads from where it began by growing into nearby areas.

*Lymph system. The cancer spreads from where it began by getting into the lymph system. The cancer travels through the lymph vessels to other parts of the body.

*Blood. The cancer spreads from where it began by getting into the blood. The cancer travels through the blood vessels to other parts of the body.

'''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.

*Lymph system. The cancer gets into the lymph system, travels through the lymph vessels, and forms a tumor (metastatic tumor) in another part of the body.

*Blood. The cancer gets into the blood, travels through the blood vessels, and forms a tumor (metastatic tumor) in another part of the body.

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:'''

'''Stage 1'''

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'''

Stage 2 is divided into stages 2A and 2B.

*Stage 2A: The cancer is in only one area and all of the cancer that can be seen is not completely removed during surgery.

*Stage 2B: The cancer is in only one area and all of the cancer that can be seen may or may not be completely removed during surgery. Cancer cells are found in the lymph nodes near the tumor.

'''Stage 3'''

In stage 3, one of the following is true:

*the cancer cannot be completely removed during surgery and has spread from one side of the body to the other side and may also have spread to nearby lymph nodes; or

*the cancer is on one side of the body and has spread to lymph nodes on the other side of the body; or

*the cancer is in the middle of the body and has spread to tissues or lymph nodes on both sides of the body, and the cancer cannot be removed by surgery.

'''Stage 4'''

Stage 4 is divided into stages 4 and 4S.

*In stage 4, the cancer has spread to distant lymph nodes or to other parts of the body.

*In stage 4S, the child is younger than 12 months, and:

:*the cancer has spread to the skin, liver, and/or bone marrow; or

:*the cancer is in only one area and all of the cancer that can be seen may or may not be completely removed during surgery; or

:*cancer cells may be found in the lymph nodes near the tumor.

'''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.

*Low-risk and intermediate-risk neuroblastoma have a good chance of being cured.

*High-risk neuroblastoma may be hard to cure.

'''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.

=== Treatment Option Overview ===

'''KEY POINTS'''

*There are different types of treatment for patients with neuroblastoma.

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

*Seven types of standard treatment are used:

:*Observation

:*Surgery

:*Radiation therapy

:*Iodine 131-MIBG therapy

:*Chemotherapy

:*High-dose chemotherapy and radiation therapy with stem cell rescue

:*Targeted therapy

*New types of treatment are being tested in clinical trials.

:*Immunotherapy

*Treatment for neuroblastoma causes side effects and late effects.

*Patients may want to think about taking part in a clinical trial.

*Patients can enter clinical trials before, during, or after starting their cancer treatment.

*Follow-up tests may be needed.

'''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:

*Pediatric surgeon.

*Pediatric radiation oncologist.

*Endocrinologist.

*Neurologist.

*Pediatric neuropathologist.

*Neuroradiologist.

*Pediatrician.

*Pediatric nurse specialist.

*Social worker.

*Child life professional.

*Psychologist.

'''Seven types of standard treatment are 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.

'''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.

'''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'''

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:

*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.

*Dinutuximab: A type of monoclonal antibody therapy that uses an antibody made in the laboratory from a single type of immune system cell. Dinutuximab identifies and attaches to a substance, called GD2, on the surface of neuroblastoma cells. Once dinutuximab attaches to the GD2, a signal is sent to the immune system that a foreign substance has been found and needs to be killed. Then the body's immune system kills the neuroblastoma cell. Dinutuximab is given by infusion. It is a type of targeted therapy.

*Granulocyte-macrophage colony-stimulating factor (GM-CSF): A cytokine that helps make more immune system cells, especially granulocytes and macrophages (white blood cells), which can attack and kill cancer cells.

*Interleukin-2 (IL-2): A type of immunotherapy that boosts the growth and activity of many immune cells, especially lymphocytes (a type of white blood cell). Lymphocytes can attack and kill cancer cells.
See Drugs Approved for Neuroblastoma for more information.

'''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 therapies:

*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.

Pembrolizumab and dinutuximab are monoclonal antibodies being studied to treat neuroblastoma that has come back after treatment or has not responded to treatment.

*Tyrosine kinase inhibitor therapy: These targeted therapy drugs block signals needed for tumors to grow.

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.

*Histone deacetylase inhibitor therapy: This treatment causes a chemical change that stops cancer cells from growing and dividing.

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.

*Ornithine decarboxylase inhibitor therapy: This treatment slows the growth and division of cancer cells.

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'''

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: 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.

[[File:774647-750.jpg|600px|thumb|center|CAR T-cell therapy. A type of treatment in which a patient’s T cells (a type of immune cell) are changed in the laboratory so they will bind to cancer cells and kill them. Blood from a vein in the patient’s arm flows through a tube to an apheresis machine (not shown), which removes the white blood cells, including the T cells, and sends the rest of the blood back to the patient. Then, the gene for a special receptor called a chimeric antigen receptor (CAR) is inserted into the T cells in the laboratory. Millions of the CAR T cells are grown in the laboratory and then given to the patient by infusion. The CAR T cells are able to bind to an antigen on the cancer cells and kill them.]]

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:

*Physical problems.

:*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).

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:

*Urine catecholamine studies.

*MIBG scan.

=== Treatment of Low-Risk Neuroblastoma ===

For information about the treatments listed below, see the Treatment Option Overview section.

Treatment of newly diagnosed low-risk neuroblastoma may include the following:

*Surgery followed by observation.

*Chemotherapy and surgery, for children with symptoms or children whose tumor has continued to grow and cannot be removed by surgery.

*Chemotherapy, for certain patients.

*Observation alone for infants younger than 6 months who have small adrenal tumors or for infants who do not have signs or symptoms of neuroblastoma.

*Radiation therapy to treat tumors that are causing serious problems and do not respond quickly to chemotherapy or surgery.

*A clinical trial of treatment based on the tumor's response to treatment and tumor biology.

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 Intermediate-Risk Neuroblastoma ===

For information about the treatments listed below, see the Treatment Option Overview section.

Treatment of newly diagnosed intermediate-risk neuroblastoma may include the following:

*Chemotherapy for children with symptoms or to shrink a tumor that cannot be removed by surgery. Surgery may be done after chemotherapy.

*Surgery alone for infants.

*Observation alone for infants.

*Radiation therapy to treat tumors that have continued to grow during treatment with chemotherapy or tumors that cannot be removed by surgery and have continued to grow after treatment with chemotherapy.

*A clinical trial of treatment based on the tumor's response to treatment and tumor biology.

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 High-Risk Neuroblastoma ===

For information about the treatments listed below, see the Treatment Option Overview section.

Treatment of newly diagnosed high-risk neuroblastoma may include the following:

*A regimen of the following treatments:

:*Combination chemotherapy.

:*Surgery.

:*Two courses of high-dose combination chemotherapy followed by stem cell rescue.

:*Radiation therapy.

:*Monoclonal antibody therapy (dinutuximab) with interleukin-2 (IL-2), granulocyte-macrophage colony-stimulating factor (GM-CSF), and isotretinoin.

*A clinical trial of iodine 131-MIBG therapy or targeted therapy (crizotinib) and other treatments.

*A clinical trial of monoclonal antibody therapy (dinutuximab), GM-CSF, and combination chemotherapy.

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 Stage 4S Neuroblastoma ===

For information about the treatments listed below, see the Treatment Option Overview section.

There is no standard treatment for newly diagnosed stage 4S neuroblastoma but treatment options include the following:

*Observation and supportive care for children who have favorable tumor biology and do not have signs or symptoms.

*Chemotherapy, for children who have signs or symptoms, for very young infants, or for children with unfavorable tumor biology.

*Radiation therapy for children with neuroblastoma that has spread to the liver.

*A clinical trial of treatment based on the tumor's response to treatment and tumor biology.

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 Recurrent Neuroblastoma ===

For information about the treatments listed below, see the Treatment Option Overview section.

'''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:

*Surgery followed by observation or chemotherapy.

*Chemotherapy that may be followed by surgery.

Treatment for recurrent neuroblastoma that comes back in other parts of the body or that has not responded to treatment may include the following:

*Observation.

*Chemotherapy.

*Surgery followed by chemotherapy.

*Treatment as for newly diagnosed high-risk neuroblastoma, for children older than 1 year.

'''Patients First Treated for Intermediate-Risk Neuroblastoma'''

Treatment for recurrent neuroblastoma that comes back in the area where the cancer first formed may include the following:

*Surgery that may be followed by chemotherapy.

*Radiation therapy for children whose disease has gotten worse after chemotherapy and second-look surgery.

Treatment for recurrent neuroblastoma that comes back in other parts of the body may include the following:

*Treatment as for newly diagnosed high-risk neuroblastoma, for children older than 1 year.

'''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:

*Chemotherapy.

*Combination chemotherapy with monoclonal antibody therapy (dinutuximab).

*Iodine 131-MIBG therapy to relieve symptoms and improve quality of life. It may be given alone or in combination with chemotherapy.

*Targeted therapy with crizotinib or other ALK inhibitors, for patients with changes in the ALK gene.

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:

*Surgery to remove the tumor in the CNS followed by radiation therapy.

*A clinical trial of a new therapy.

'''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:

*Chemotherapy and targeted therapy (dinutuximab with or without eflornithine).

*Checking 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.

*Targeted therapy (AZD1775) and chemotherapy.

*Targeted therapy (pembrolizumab or lorlatinib).

*Immunotherapy (CAR T-cell therapy).

*Iodine 131-MIBG therapy given alone or with other anticancer drugs.

*Iodine 131-MIBG therapy and targeted therapy (dinutuximab).

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.

=== To Learn More About Neuroblastoma ===

For more information from the National Cancer Institute about neuroblastoma, see the following:

*Neuroblastoma Home Page

*Neuroblastoma Screening

*Computed Tomography (CT) Scans and Cancer

*Drugs Approved for Neuroblastoma

*Targeted Cancer Therapies

*Immunotherapy to Treat Cancer

*New Approaches to Neuroblastoma Therapy (NANT)Exit Disclaimer

For more childhood cancer information and other general cancer resources, see the following:

*About Cancer

*Childhood Cancers

*CureSearch for Children's CancerExit Disclaimer

*Late Effects of Treatment for Childhood Cancer

*Adolescents and Young Adults with Cancer

*Children with Cancer: A Guide for Parents

*Cancer in Children and Adolescents

*Staging

*Coping with Cancer

*Questions to Ask Your Doctor about Cancer

*For Survivors and Caregivers

</translate>
<comments />