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Neuroblastoma - Childhood - Overview

This section has been reviewed and approved by the Cancer.Net Editorial Board, 03/2015

ON THIS PAGE: You will find some basic information about this disease and the parts of the body it may affect. This is the first page of Cancer.Net’s Guide to Childhood Neuroblastoma. To see other pages, use the menu on the side of your screen. Think of that menu as a roadmap to this full guide.

Cancer begins when healthy cells change and grow uncontrollably, forming a mass called a tumor. A tumor can be cancerous or benign. A cancerous tumor is malignant, meaning it can grow and spread to other parts of the body. A benign tumor means the tumor can grow but will not spread.

Neuroblastoma is a solid cancerous tumor that begins in the nerve cells outside the brain of infants and young children. It can start in the nerve tissue near the spine in the neck, chest, abdomen, or pelvis, but it most often begins in the adrenal glands. The adrenal glands are located on top of both kidneys and make hormones that help control body functions, such as heart rate and blood pressure.

Neuroblasts are immature nerve cells found in unborn babies. Normal neuroblasts mature into nerve cells or adrenal medulla cells, which are cells found in the center of the adrenal gland. Neuroblastoma forms when neuroblasts don’t mature properly.

Sometimes, babies are born with small clusters of neuroblasts that eventually mature into nerve cells and do not become cancer. A neuroblast that does not mature can continue to grow, forming a mass called a tumor.

Neuroblastoma develops most often in infants and children younger than five. It can form before the baby is born and can sometimes be found during a prenatal (before birth) ultrasound. Most often, however, neuroblastoma is found after the cancer has spread to other parts of the body, such as the lymph nodes, which are tiny, bean-shaped organs that help fight infection, liver, lungs, bones, and bone marrow, the spongy, red tissue in the inner part of large bones.

Looking for More of an Overview?

If you would like additional introductory information, explore these related items. Please note these links will take you to other sections on Cancer.Net:

  • ASCO Answers Fact Sheet: Read a one-page fact sheet (available as a PDF) that offers an easy-to-print introduction for this type of cancer.

  • Cancer.Net Patient Education Video: View a short video led by an ASCO expert in childhood cancer that provides basic information and areas of research.

The next section in this guide is Statistics and it helps explain how many people are diagnosed with this disease and general survival rates. Or, use the menu on the side of your screen to choose another section to continue reading this guide.  

Neuroblastoma - Childhood - Statistics

This section has been reviewed and approved by the Cancer.Net Editorial Board, 03/2015

ON THIS PAGE: You will find information about how many children are diagnosed with neuroblastoma each year. You will also learn some general information on surviving the disease. Remember, survival rates depend on several factors. To see other pages, use the menu on the side of your screen.

Each year, about 700 people are diagnosed with neuroblastoma in the United States. It accounts for 6% of all childhood cancers in the United States. Neuroblastoma is found most often in children under age 5. It is the third most common type of cancer in children in general and the most common cancer in babies younger than 1 year old. It is rare in people over age 10.

The 5-year survival rate tells you what percent of children live at least 5 years after the cancer is found. Percent means how many out of 100. The 5-year survival rate for neuroblastoma depends on many factors, particularly the risk grouping of the tumor.

For children with low-risk and intermediate-risk neuroblastoma, the 5-year survival rate is higher than 95%. For children in the immediate-risk group, the 5-year survival rate is 90% to 95%. For children with high-risk neuroblastoma, the 5-year survival rate is 40% to 50%. See Stages and Groups for information on risk groupings.

It is important to remember that statistics on how many children survive this type of cancer are an estimate. The estimate comes from data based on children with this cancer in the United States each year. So, your own child’s risk may be different. Doctors cannot say for sure how long any child will live with neuroblastoma. Also, experts measure the survival statistics every 5 years. This means that the estimate may not show the results of better diagnosis or treatment available for less than 5 years. Learn more about understanding statistics.

Statistics adapted from the American Cancer Society's (ACS) publication, Cancer Facts & Figures 2016, and the ACS website.

The next section in this guide is Medical Illustrations. It offers drawings of body parts often affected by this disease. Or, use the menu on the left side of your screen to choose another section to continue reading this guide.

Neuroblastoma - Childhood - Medical Illustrations

This section has been reviewed and approved by the Cancer.Net Editorial Board, 03/2015

ON THIS PAGE: You will find a basic drawing about the main body parts affected by this disease. To see other pages, use the menu on the side of your screen.

Childhood Central Nervous System Anatomy

Larger image

The next section in this guide is Risk Factors and it explains what factors may increase the chance of developing this disease. Or, use the menu on the side of your screen to choose another section to continue reading this guide.

Neuroblastoma - Childhood - Risk Factors

This section has been reviewed and approved by the Cancer.Net Editorial Board, 03/2015

ON THIS PAGE: You will find out more about the factors that increase the chance of developing this type of tumor. To see other pages, use the menu on the side of your screen

For most types of cancer, a risk factor is anything that increases a person’s chance of developing cancer. Although risk factors can influence the development of cancer, most do not directly cause cancer. Some people with several risk factors never develop cancer, while others with no known risk factors do.

For neuroblastoma, the term “risk factor” is more commonly used to describe the factors that are used to predict how the tumor will grow and how well treatment will work (see Stages and Groups).

Neuroblastoma occurs more often in boys than in girls. So far, no environmental factors have been shown to increase the risk of developing neuroblastoma. Rarely, more than one member of a family is diagnosed with neuroblastoma.

Researchers have found inherited gene mutations (changes) that play a role in the development of neuroblastoma for children with a family history of the disease. Other genetic changes, called single-nucleotide polymorphisms (SNPs), may contribute to the development of neuroblastoma in children who do not have a family history.

Family history and genetic predisposition

Approximately 1% to 2% of children with neuroblastoma have a family history of the disease. Children with an inherited likelihood of neuroblastoma tend to develop the disease, on average, nine to thirteen months earlier than other children with neuroblastoma. In children who have a family history of neuroblastoma, the disease may occur in two or more organs.

Neuroblastoma tumors have been diagnosed in patients with congenital central hypoventilation syndrome (CCHS), a unique disorder of breathing control associated with Hirschsprung disease (HSCR). CCHS results from germline mutations, which is a mutation passed directly from parent to child, in the paired-like homeobox (PHOX) 2B gene. Germline PHOX2B alterations are present in the tumors of people with neuroblastoma and with CCHS and/or HSCR. Rare germline PHOX2B mutations have also been found in patients who are genetically predisposed to neuroblastoma.

Most of the time, when multiple members of a family have neuroblastoma, they have germline mutations in the anaplastic lymphoma kinase (ALK) gene. These mutations can be found in a blood sample. However, ALK mutations have also been found in DNA from tumors in some patients without a family history.

Neuroblastoma has also been diagnosed in several patients who are missing portions of chromosomes 1p and 11q that are thought to prevent tumor growth.

Genetic factors without a family history

The genetic factors that have a role in the development of neuroblastoma in patients who do not have a family history are not well understood. Common copy number variation of NBPF23 has been shown to be associated with neuroblastoma. This involves looking at the genes and how they differ between individuals. In addition, genome-wide association studies have identified a number of germline neuroblastoma susceptibility SNPs. These include SNPs within or upstream of these genes:

  • CASC15

  • CASC14

  • BARD1

  • LMO1

  • DUSP12

  • HSD17B12

  • DDX4/IL31RA

  • HACE1

  • LIN28B genes

  • Rare variants in TP53

  • NEFL 

However, these variants account for only a small portion of the risk for developing neuroblastoma. Read more about the genetics of cancer. Learn more about genetic studies here (please note this link takes you out of Cancer.Net). 

The next section in this guide is Symptoms and Signs and it explains what body changes or medical problems this disease can cause. Or, use the menu on the side of your screen to choose another section to continue reading this guide.  

Neuroblastoma - Childhood - Symptoms and Signs

This section has been reviewed and approved by the Cancer.Net Editorial Board, 03/2015

ON THIS PAGE: You will find out more about body changes and other things that can signal a problem that may need medical care. To see other pages, use the menu on the side of your screen.

Many symptoms of neuroblastoma are caused by pressure from the tumor or bone pain if the cancer has spread to the bones. Pain may cause the child to limp, refuse to walk, or become unable to walk. Other symptoms may include:

  • A lump or mass in the abdomen, chest, neck, or pelvis, often found by a parent when bathing the child

  • Skin lesions or nodules under the skin with blue or purple patches

  • Eyes that bulge out and dark circles under the eyes, if the cancer has spread behind the eyes

  • Changes in the eyes, such as black eyes, a droopy eyelid, a pupil that is constricted, vision problems, or changes in the color of the iris

  • Pain in the chest, difficulty breathing, or a persistent cough

  • Pain in the arms, legs, or other bones

  • Pain in the back, or weakness, numbness, or paralysis of the legs if the tumor has spread to the spinal cord

  • Fever and anemia, which is a low level of red blood cells

  • Constant diarrhea or high blood pressure caused by hormones released by the tumor

  • Rotating movements of the eyes and sudden muscle jerks, likely from immune system problems caused by the disease

Sometimes, children with neuroblastoma do not show any of these symptoms. Or, these symptoms may be caused by a medical condition that is not cancer.          

If you are concerned about one or more of the symptoms or signs on this list, please talk with your child’s doctor. Your doctor will ask how long and how often your child has been experiencing the symptom(s), in addition to other questions. This is to help find out the cause of the problem, called a diagnosis.

If neuroblastoma is diagnosed, relieving symptoms remains an important part of care and treatment. This may also be called symptom management, palliative care, or supportive care. Be sure to talk with the health care team about symptoms your child experiences, including any new symptoms or a change in symptoms.

The next section in this guide is Diagnosis and it explains what tests may be needed to learn more about the cause of the symptoms. Or, use the menu on the side of your screen to choose another section to continue reading this guide.   

Neuroblastoma - Childhood - Diagnosis

This section has been reviewed and approved by the Cancer.Net Editorial Board, 03/2015

ON THIS PAGE: You will find a list of the common tests, procedures, and scans that doctors can use to find out what’s wrong and identify the cause of the problem. To see other pages, use the menu on the side of your screen.

Doctors use many tests to diagnose cancer and find out if it has spread to another part of the body, called metastasis. Some tests may also determine which treatments may be the most effective.

For most tumors, a biopsy is the only way to make a definitive diagnosis of cancer.

If a biopsy is not possible, the doctor may suggest other tests that will help make a diagnosis. Imaging tests may be used to find out whether the cancer has spread.

Your child’s doctor may consider these factors when choosing a diagnostic test:

  • Age and medical condition

  • Type of cancer suspected

  • Signs and symptoms

  • Previous test results

According to international criteria developed by an International Neuroblastoma Risk Group task force, a diagnosis of neuroblastoma can be made if:

  • Neuroblastoma cells are detected in the bone marrow and higher than normal levels of one of the main chemicals produced by the nervous system, called catecholamine are present in the urine, or

  • By a tumor biopsy showing neuroblastoma cells

In addition to a physical examination, the following tests may be used to evaluate patients with neuroblastoma. Not all tests listed will be used for every person.

  • Blood tests and urine tests. Complete blood counts (CBC) are tested to find out if the child has signs of anemia, which is having low levels of red blood cells in the blood. To evaluate liver and kidney function, additional blood tests will be done.  In addition, blood clotting tests may also be recommended.

    Urine is collected to test for tumor markers produced by a neuroblastoma tumor. A tumor marker is a substance found in higher than normal amounts in the blood, urine, or body tissues of people with certain kinds of cancer. Urinary catecholamine metabolites are detected in more the 85% of patients with neuroblastoma.

    Catecholamines are organic compounds that include the hormones epinephrine (adrenaline), norepinephrine (noradrenaline) and dopamine. Release of the hormones epinephrine and norepinephrine from the adrenal medulla of the adrenal glands is part of the fight-or-flight response. Eventually the body breaks down the catecholamine molecules into smaller pieces, called metabolites, which are then passed out of the body in the urine.

    The two catecholamine metabolites most often measured are homovanillic acid (HVA) or vanillylmandelic acid (VMA).

  • Biopsy. A biopsy is the removal of a small amount of tissue for examination under a microscope. A pathologist then analyzes the sample(s).  A pathologist is a doctor who specializes in interpreting laboratory tests and evaluating cells, tissues, and organs to diagnose disease. The type of biopsy performed depends on the location of the tumor. If the surgeon determines that the entire tumor can be removed, the whole tumor is commonly removed instead of doing a separate biopsy.

  • Genetic studies. Tests of neuroblastoma cell DNA are used to find a change in the oncogene MYCN, a gene responsible for cell growth. More than 10 copies of the gene, called amplification, is associated with a tumor that grows and spreads quickly, making it more difficult to treat. A non-amplified MYCN gene is linked to less aggressive tumors, which grow and spread more slowly.

    Additional tests are done to find out if the tumor has changes in the numbers of whole chromosome or parts of chromosomes. A series of studies have shown that segmental chromosomal aberrations are associated with more aggressive disease, while whole chromosome gains are seen in tumors with more favorable outcome. DNA sequencing tests to find out if mutations of ALK are also commonly done. Sequencing studies have shown that a small subset of neuroblastoma tumors have gene mutations, and some of these mutations, like ALK, can be targeted with new drugs.

  • Bone marrow aspiration and biopsy. These two procedures are similar and often done at the same time to examine the bone marrow. Bone marrow has both a solid and a liquid part. A bone marrow aspiration removes a sample of the fluid with a needle. A bone marrow biopsy is the removal of a small amount of solid tissue using a needle.

    A pathologist then analyzes the samples. A common site for a bone marrow aspiration and biopsy is the pelvic bone, which is located in the lower back by the hip. The skin in that area is usually numbed with medication beforehand. Other types of anesthesia, which is medication to block the awareness of pain, may also be used.

  • Computed tomography (CT or CAT) scan. A CT scan creates a three-dimensional picture of the inside of the body with an x-ray machine. A computer then combines these images into a detailed, cross-sectional view that shows any abnormalities or tumors. A CT scan can also be used to measure the tumor’s size. A special dye called a contrast medium is usually given before the scan to provide better detail on the image. This dye is injected into a patient’s vein.

  • Magnetic resonance imaging (MRI). An MRI uses magnetic fields, not x-rays, to produce detailed images of the brain and spinal column. MRI can also be used to measure the tumor’s size. A special dye called a contrast medium is given before the scan to create a clearer picture. This dye is injected into a patient’s vein. An MRI is better at showing tumors around the spine, and it is essential to look at a tumor that is near where nerves leave the spinal column, which can press on the spinal cord.

  • MIBG scan. MIBG stands for meta-iodobenzylguanidine. Neuroblastoma cells absorb this protein. When the protein is linked to a small amount of radioactive iodine, it can be used to find neuroblastoma in the bone, bone marrow and other parts of the body. Because the child’s thyroid gland will also absorb radioactive iodine, regular iodine is taken by mouth for several days before the scan to protect the thyroid.

  • Positron emission tomography (PET) scan. A PET scan is a way to create pictures of organs and tissues inside the body. A small amount of a radioactive substance is injected into a patient’s body. This substance is absorbed mainly by organs and tissues that use the most energy. Because cancer tends to use energy actively, it absorbs more of the radioactive substance. A scanner then detects this substance to produce images of the inside of the body. This test is usually performed for patients who have tumors that do not take up MIBG (see above).

After diagnostic tests are done, your child’s doctor will review all of the results with you. If the diagnosis is neuroblastoma, these results also help the doctor describe the cancer; this is called stages and risk grouping

The next section in this guide is Stages and Groups, and it explains the system doctors use to describe the extent of the disease. Or, use the menu on the side of your screen to choose another section to continue reading this guide.  

Neuroblastoma - Childhood - Stages and Groups

This section has been reviewed and approved by the Cancer.Net Editorial Board, 03/2015

ON THIS PAGE: You will learn about how doctors describe neuroblastoma’s growth or spread. This is called the stage. To see other pages, use the menu on the side of your screen.

Staging is a way of describing a cancer, such as where it is located, if or where it has spread, and whether it is affecting other parts of the body. Doctors use diagnostic tests to find out the cancer's stage, so staging may not be complete until all of the tests are finished.

Knowing the stage helps the doctor to decide what kind of treatment is best and can help predict a patient's prognosis, which is the chance of recovery. There are different stage descriptions for different types of cancer. Once a diagnosis of neuroblastoma is confirmed, how much the tumor has grown and spread is evaluated or staged. The tumor’s stage helps your child’s doctor plan treatment.

There are two staging systems for neuroblastoma: INSS and INRGSS. Each is described below, followed by an explanation of risk groupings.

International Neuroblastoma Staging System Committee (INSS) system

The following is a brief summary of each INSS stage:

Stage 1: The tumor can be removed completely during surgery. Lymph nodes attached to the tumor removed during surgery may or may not contain cancer, but other lymph nodes near the tumor do not.

Stage 2A: The tumor is located only in the area it started and cannot be completely removed during surgery. Nearby lymph nodes do not contain cancer.

Stage 2B: The tumor is located only in the area where it started and may or may not be completely removed during surgery, but nearby lymph nodes do contain cancer.

Stage 3: The tumor cannot be removed with surgery. It has spread to regional lymph nodes (lymph nodes near the tumor) or other areas near the tumor, but not to other parts of the body.

Stage 4: The original tumor has spread to distant lymph nodes (lymph nodes in other parts of the body), bones, bone marrow, liver, skin, and/or other organs, except for those listed in stage 4S, below.

Stage 4S: The original tumor is located only where it started (as in stage 1, 2A, or 2B), and it has spread only to the skin, liver, and/or bone marrow, in infants younger than one. The spread to the bone marrow is minimal, usually less than 10% of cells examined show cancer.

The International Neuroblastoma Risk Group Staging System (INRGSS)

The INRGSS was designed specifically for the International Neuroblastoma Risk Group (INRG) pre-treatment classification system (see Risk groups, below). Unlike the INSS explained above, the INRGSS uses only the results of imaging tests taken before surgery. It does not include surgical results or spread to lymph nodes to determine the stage.  Knowledge regarding the presence or absence of image defined risk factors (IDRF) are required for this staging system.

Stage L1: The tumor is located only in the area where it started; no IDRFs are found on imaging scans, such as CT or MRI.

Stage L2: The tumor has not spread beyond the area where it started and the nearby tissue; IDRFs are found on imaging scans, such as CT or MRI.

Stage M: The tumor has spread to other parts of the body (except stage MS, see below)

Stage MS: The tumor has spread to only the skin, liver, and/or bone marrow (less than 10% marrow involvement) in patients less than 18 months.

Risk groups

Some children are cured with surgery alone or surgery with chemotherapy (see Treatment Options). Others have a very aggressive disease that is difficult to treat. By using a combination of factors, doctors can usually predict how well treatment will work for a neuroblastoma tumor, or if it will come back after treatment.

In the INRG classification system, a combination of clinical, pathologic and genetic markers that predict whether the tumor will grow and how it will respond to treatment. These markers are used to define risk. Using the following factors, neuroblastoma is classified as very low-risk, low-risk, intermediate-risk, or high-risk:

  • The stage of the disease according to the INRG staging system

  • Age at the time of diagnosis

  • Histologic category, such as maturing ganglioneuroma versus ganglioneuroblastoma, intermixed versus ganglioneuroblastoma or nodular versus neuroblastoma

  • Grade or how cells of the tumor are differentiated

  • MYCN gene status

  • Chromosome 11q status

  • Tumor cell ploidy, which is the DNA content of tumor cells

The Children’s Oncology Group (COG) uses the following factors to determine risk:

  • The stage of the disease according to the INSS system

  • Age at the time of diagnosis

  • MYCN gene status

  • Tumor ploidy; this is only important for children younger than 18 months

  • Tumor histopathology according to the International Neuroblastoma Pathologic Classification (INPC) system

Descriptions of low-risk, intermediate-risk, or high-risk neuroblastoma are listed below.

Low-risk neuroblastoma

  • Stage 1 disease

  • Stage 2A or 2B disease in which more than 50% of the tumor was surgically removed, except for a child with MYCN amplification

  • Stage 4S disease, no MYCN amplification, favorable histopathology, and hyperdiploidy, meaning having extra chromosomes

Intermediate-risk neuroblastoma

  • Stage 2A or 2B disease with no MYCN amplification in which less than 50% of the tumor was removed with surgery

  • Stage 3 disease in children younger than 18 months, no MYCN amplification

  • Stage 3 disease in children older than 18 months, no MYCN amplification, and favorable histopathology

  • Stage 4 disease in children younger than 12 months, no MYCN amplification.

  • Stage 4 disease in children age 12 months to 18 months, no MYCN amplification, hyperdiploidy, and favorable histology.

  • Stage 4S disease, no MYCN amplification, unfavorable histopathology and/or diploidy

High-risk neuroblastoma

  • Stage 2A or 2B disease and MYCN amplification

  • Stage 3 disease and MYCN amplification

  • Stage 3 disease in children age 18 months or older, no MYCN amplification, and unfavorable histopathology

  • Stage 4 disease in children younger than 12 months and MYCN amplification

  • Stage 4 disease in children between 12 months and 18 months old with MYCN amplification, and/or diploidy, and/or unfavorable histology

  • Stage 4 disease in children 18 months or older

  • Stage 4S disease and MYCN amplification

Treatment is then tailored according to the risk assigned to the disease. Most patients with low-risk disease receive surgery alone. Sometimes, infants with small localized tumor have been successfully watched closely without any surgery, called observation.

Patients with intermediate-risk disease receive surgery and chemotherapy. In the Children’s Oncology Group, the recently completed clinical trial for the intermediate-risk group varied the length of treatment such as the number of cycles of chemotherapy. This resulted due to the presence or absence of tumor genetic changes in chromosomes 1p and 11q, tumor histology, tumor ploidy, stage, and age.

A very intensive approach, often using several types of treatments, is used for patients with high-risk neuroblastoma. More information can be found in the Treatment Options section. 

Information about the cancer’s stage and risk group will help the doctor recommend a specific treatment plan. The next section in this guide is Treatment Options. Or, use the menu on the side of your screen to choose another section to continue reading this guide.  

Neuroblastoma - Childhood - Treatment Options

This section has been reviewed and approved by the Cancer.Net Editorial Board, 03/2015

ON THIS PAGE: You will learn about the different ways doctors use to treat children with this type of cancer. To see other pages, use the menu on the side of your screen.

In general, cancer in children is uncommon, so it can be hard for doctors to plan treatments unless they know what has been most effective in other children. That’s why more than 60% of children with cancer are treated as part of a clinical trial.

Clinical trials are research studies that compare standard treatments (the best known treatments available) with newer approaches to treatments that may be more effective. Clinical trials may test such approaches as a new drug, a new combination of standard treatments, or new doses of current therapies. Studying new treatments involves careful monitoring using scientific methods, and all participants are followed closely to track their health and progress.

To take advantage of these newer treatments, children with cancer should be treated at a specialized cancer center. Doctors at these centers have extensive experience in treating children with cancer and have access to the latest research. A doctor who specializes in treating children with cancer is called a pediatric oncologist. If a pediatric cancer center is not nearby, general cancer centers sometimes have pediatric specialists who are able to be part of your child’s care.

 In many cases, a team of doctors treats a child with cancer; this is called a multidisciplinary team. Pediatric cancer centers often have extra support services for children and their families, such as child life specialists, dietitians, physical and occupational therapists, social workers, and counselors. Special activities and programs to help your child and family cope may also be available.

Making treatment decisions

Treatment options and recommendations depend on several factors, including:

  • The size and location of the tumor

  • Whether the cancer has spread

  • The risk classification

  • Possible side effects

  • Family preferences

  • The child’s overall health

The treatment is tailored according to the assigned risk group.  Descriptions of the most common treatment options for neuroblastoma are listed below.

Your child’s care plan may also include treatment for symptoms and side effects, an important part of cancer care. Take time to learn about all of your child’s treatment options and be sure to ask questions about things that are unclear. Also, talk about the goals of each treatment with your child’s doctor and what your child can expect while receiving the treatment.

Learn more about making treatment decisions.

Surgery

Surgery is the removal of the tumor and some surrounding healthy tissue during an operation. A surgical oncologist is a doctor who specializes in treating cancer using surgery. If the cancer has not spread, surgery can sometimes be used to remove the entire tumor. However, most neuroblastoma is not found until after the cancer has spread. In that situation, the doctor removes as much of the tumor as possible during surgery.

If the tumor cannot be completely removed, the child may receive radiation therapy and chemotherapy (see below) to destroy the remaining cancer cells. In addition, the doctor may take a biopsy of the liver to find out if the disease has spread to the liver.

Talk with your child’s surgeon about the possible side effects of the surgery and how they will be relieved or managed. Learn more about the basics of cancer surgery.  

Even if a tumor cannot be removed because of its location, a surgical biopsy (see Diagnosis) may still be done to determine the type of tumor.

Chemotherapy

Chemotherapy is the use of drugs to destroy cancer cells, usually by stopping the cancer cells’ ability to grow and divide. Chemotherapy is given by a pediatric oncologist or a medical oncologist, a doctor who specializes in treating cancer with medication.

Systemic chemotherapy gets into the bloodstream to reach cancer cells throughout the body. Common ways to give chemotherapy include an intravenous (IV) tube placed into a vein or muscle using a needle or in a pill or capsule that is swallowed.

A chemotherapy regimen (schedule) usually consists of a specific number of cycles given over a set period of time. Most children with neuroblastoma will need to have chemotherapy. Chemotherapy may be used as the primary treatment for neuroblastoma. Or, it may be given before surgery to shrink the tumor or after surgery to destroy any remaining cancer cells.

Children with intermediate-risk neuroblastoma often receive the following drugs:

  • Carboplatin (Paraplatin)

  • Cyclophosphamide (Neosar)

  • Doxorubicin (Adriamycin)

  • Etoposide

Children with high-risk neuroblastoma often receive the following drugs:

  • Busulfan (Busulfex, Myleran)

  • Carboplatin (Paraplatin)

  • Chimeric 14.18 antibody

  • Cisplatin (Platinol)

  • Cyclophosphamide

  • Cytokines (GM-CSF and IL2)

  • Doxorubicin

  • Etoposide (VePesid, Toposar)

  • Ifosfamide (Ifex)

  • Isotretinoin

  • Melphalan (Alkeran)

  • Topotecan (Hycamtin)

  • Vincristine (Vincasar)

The side effects of chemotherapy depend on the individual and the dose used, but they can include fatigue, risk of infection, nausea and vomiting, hair loss, loss of appetite, and diarrhea. These side effects usually go away once treatment is finished. The severity of the side effects depends on the type and amount of the drug being given and the length of time the child receives the drug.

Learn more about the basics of chemotherapy and preparing for treatment. The medications used to treat cancer are continually being evaluated. Talking with your child’s doctor is often the best way to learn about the medications prescribed for you, their purpose, and their potential side effects or interactions with other medications. Learn more about your prescriptions by using searchable drug databases.

Radiation therapy

Radiation therapy is the use of high-energy x-rays or other particles to destroy cancer cells. A doctor who specializes in giving radiation therapy to treat cancer is called a radiation oncologist. The most common type of radiation treatment is called external-beam radiation therapy, which is radiation given from a machine outside the body.

A radiation therapy regimen (schedule) usually consists of a specific number of treatments given over a set period of time. Side effects from radiation therapy may include fatigue, mild skin reactions, upset stomach, and loose bowel movements. Most side effects go away soon after treatment is finished.

Because radiation therapy can sometimes cause problems with the normal growth and development of a child’s brain and the ovaries in girls or testicles in boys, the doctor may choose to treat the cancer in another way.

Learn more about the basics of radiation therapy.

Stem cell transplantation/bone marrow transplantation

A stem cell transplant is a medical procedure in which diseased bone marrow is replaced by highly specialized cells, called hematopoietic stem cells. Hematopoietic stem cells are blood-forming cells found both in the blood stream and in the bone marrow.

Today, this procedure is more commonly called a stem cell transplant, rather than a bone marrow transplant. That’s because it is the blood stem cells that are typically being transplanted, not the actual bone marrow tissue.

Before recommending transplantation, doctors will talk with the patient about the risks of this treatment and consider several other factors, such as the type of cancer, results of any previous treatment, and the patient’s age and general health.

There are two types of stem cell transplantation depending on the source of the replacement blood stem cells: allogeneic (ALLO) and autologous (AUTO). AUTO transplants are most often used for children with high-risk neuroblastoma.

In an AUTO transplant, the patient’s own stem cells are used. The stem cells are obtained from the patient when he or she is in remission from previous treatment. The stem cells are then frozen until they are needed, usually after the high-dose treatment (explained below) is completed.

The goal of transplantation is to destroy cancer cells in the marrow, blood, and other parts of the body and allow replacement blood stem cells create healthy bone marrow. In most stem cell transplants, the patient is treated with high doses of chemotherapy and/or radiation therapy to destroy as many cancer cells as possible. This also destroys the patient’s bone marrow tissue and suppresses the patient’s immune system. After the high-dose treatment is given, blood stem cells are infused into the patient’s vein to replace the bone marrow and restore normal blood counts.

Different combinations of high-dose chemotherapy have been used before the stem cells are infused. The best combination has not yet been determined. Recent research suggests that busulfan (Busulfex, Myleran) and melphalan may cause fewer side effects and may also be more effective than carboplatin, etoposide, and melphalan. The Children’s Oncology Group is currently testing the safety and efficiency of busulfan and melphalan in a clinical trial.

In stem cell transplantation, the replacement cells engraft, or begin to make new blood cells, and turn into healthy, blood-producing tissue in 10 days to three weeks. Destroying the patient’s own marrow reduces the body’s natural defenses, temporarily leaving the patient at an increased risk of infection. Until the patient’s immune system is back to normal, patients may need antibiotics and blood transfusions. They also need to follow certain safety restrictions provided by the health care team to help avoid infections.

In an AUTO transplant, there is little risk of tissue rejection because the replacement stem cells are the patient’s own cells. However, there is a risk in an autologous transplant that some of the cells that are put back into the patient could still be cancerous.

Learn more about the basics of stem cell and bone marrow transplantation.

Retinoid therapy

Retinoids are substances that are similar to vitamin A. They are thought to help some cells mature into normal cells. When chemotherapy no longer works for some tumor cells, 13-cis-retinoic acid (RA) or isotretinoin has been given to children with high-risk neuroblastoma after completion of consolidation therapy, which is treatment given after the initial treatment. A Children’s Oncology Group study showed that when children who were in remission following a stem cell transplant were given RA, their risk of recurrence was reduced.

Immunotherapy

Immunotherapy is designed to boost the body’s natural defenses to fight the cancer. It uses compounds made either by the body or in a laboratory to bolster, target, or restore immune system function.

GD2 is a compound on the surface of cells, called a disialoganglioside, that is found in large amounts in most neuroblastomas. A variety of monoclonal antibodies directed against GD2 have been used to treat patients with neuroblastoma. A monoclonal antibody is a substance made in a laboratory that acts like the antibodies the body’s immune system naturally makes to fight diseases such as a tumor.

The Children's Oncology Group has used antibody therapy combined with cytokines, which are proteins that stimulate the immune system, and retinoid therapy (see above) for patients with high-risk neuroblastoma that:

  • Responded to induction (first) chemotherapy, and

  • Who have had a stem cell transplantation without the neuroblastoma growing or spreading.

In a Children’s Oncology Group clinical trial, children without evidence of active disease following stem cell transplantation and radiation received either RA alone, or RA and anti-GD2 monoclonal antibody ch14:18 and cytokines. The patients who received the immunotherapy and RA lived longer and had fewer recurrences than those who were treated with RA alone. Immunotherapy and RA is now a part of the standard treatment for patients with high-risk neuroblastoma.  Based on the results of this study, the U.S. Food and Drug Administration (FDA) approved dinutuximab (Unituxin) as part of first-line therapy for  children patients with high-risk neuroblastoma. 

Learn more about the basics of immunotherapy.

Targeted delivery of radionuclides

A radionuclide called 131I MIBG is being researched to treat neuroblastoma. 131I MIBG has led to responses in about 30% of children with recurrent neuroblastoma. Based on how efficient this drug is when there is a recurrence, a new clinical trial has been opened by the Children’s Oncology Group. The clinical trial tests 131I MIBG combined with high-dose chemotherapy and stem cell transplantation in patients newly diagnosed with high-risk disease.

Remission and the chance of recurrence

A remission is when a tumor cannot be detected in the body and there are no symptoms. This may also be called having “no evidence of disease” or NED. 

A remission may be temporary or permanent. This uncertainty causes many people to worry that their cancer will come back. While many remissions are permanent, it’s important to talk with your doctor about the possibility of the tumor returning. Understanding your child’s risk of recurrence and the treatment options may help you and your family feel more prepared if the cancer does return. Learn more about coping with the fear of recurrence.

If the cancer does return after the original treatment, it is called a recurrent or relapsed cancer. It may come back in the same place (called a local recurrence), nearby (regional recurrence), or in another place (distant recurrence). If there is a recurrence, the cancer may need to be staged again using the system described in stages and groups

When this occurs, a cycle of testing will begin again to learn as much as possible about the recurrence. After testing is done, you and your child’s doctor will talk about the treatment options. Often the treatment plan will include the treatments described above, such as surgery, chemotherapy, and radiation therapy, but they may be used in a different combination or given at a different pace. Your child’s doctor may also suggest clinical trials that are studying new ways to treat this type of recurrent tumor.

Treatment of recurrent neuroblastoma

The treatment of recurrent neuroblastoma depends on where the tumor recurred and how aggressive the new tumor is. While there are treatments that work well for patients with low-risk and intermediate-risk disease who have a recurrence where the original tumor started, treating recurrent high-risk neuroblastoma is difficult. Neuroblastoma comes back in more than 50% of children with high-risk disease.

Over the past several years, more new treatments and combinations have been developed for these patients:

  • Chemotherapy. The drugs irinotecan (Camptosar) and topotecan are often used early when there is a recurrence. The combination of irinotecan and temozolomide (Methazolastone, Temodar) has few side effects. This chemotherapy treatment regimen has been evaluated in a Children’s Oncology Group study. Some recurrent tumors respond to this treatment. Other chemotherapy regimens have used in the setting of recurrence including topotecan combined with low-dose cyclophosphamide. A clinical trial evaluating the activity of the combination of chemotherapy (irinotecan and temozolomide) plus ch14:18 antibody or an inhibitor of the mTOR pathway is ongoing in the Children’s Oncology Group for patients with recurrent neuroblastoma.

  • Targeted delivery of radionuclides. As explained above, radionuclides have been attached to MIBG, as well as somatostatin analogs, which are substances similar to a specific hormone produced by cells, and anti-GD2 antibodies. Early studies looking at combinations of radio-labeled MIBG and radiation sensitizers like irinotecan or histone deacetylase inhibitors like vorinostat (Zolinza) are ongoing. Radiosensitizers are drugs that make tumor cells more sensitive to radiation therapy, making radiation therapy more effective.

  • Immunotherapy. A humanized antibody directed against the GD2 linked with interleukin-2 (IL2), which is a substance that can be used to boost the immune system, is being evaluated for patients with recurrent neuroblastoma. Other monoclonal antibodies directed against the GD2 antigen are also being evaluated. The combination of the immune-modulating agent lenolinomide and chimeric anti- GD2 is currently being tested in an ongoing Phase I clinical trial. Vaccines against tumor cell DNA, function, and structure, as well as use of immune cells created in the laboratory that can destroy tumor cells, are also being researched.

  • Retinoids. Fenretinide works against neuroblastoma in a laboratory, even when other retinoids do not. Research shows that the drug has few side effects. Newer versions of this drug are being created to make it easier to give this medication to young children.

  • Angiogenesis inhibitors. Anti-angiogenesis therapy is focused on stopping angiogenesis, which is the process of making new blood vessels from existing blood vessels. Because a tumor needs the nutrients delivered by blood vessels to grow and spread, the goal of anti-angiogenesis therapies is to “starve” the tumor.

  • Tyrosine kinase inhibitors. Tyrosine kinase inhibitors are drugs that block cell communication and can stop tumor growth. Drugs that inhibit ALK are being tested in clinical trials. Other tyrosine kinase inhibitors that are being tested in clinical trials include inhibitors of the epidermal growth factor receptor (EGFR) and the insulin growth factor 1 receptor (IGR1R). These receptors help tumor cells grow and blocking them may slow or stop neuroblastoma growth. 

  • Aurora kinase inhibitors. Aurora A kinase helps cells divide early on and is found in all cells that are dividing. Aurora kinase inhibitors are drugs that block this protein, stopping or slowing the cells from dividing. Inhibition of Aurora A kinase also destabilizes the MYCN protein. An Aurora A kinase inhibitor is currently being evaluated in combination with irinotecan and temozolomide in an early phase clinical trial for patients with recurrent neuroblastoma.

  • Other treatment options. Demethylating drugs such as decitabine (Dacogen) are currently being studied. Histone deacetylase inhibitors, substances that can prevent a tumor from growing and spreading, have also shown some promise as a treatment for neuroblastoma. At least three histone deacetylase inhibitors are now being studied in clinical trials for patients with refractory solid tumors, which are tumors that remain after the initial treatment.

An inhibitor of a key downstream target of MYCN, ornithine decarboxylase (ODC), is also being tested in early phase neuroblastoma clinical trials.

The New Approaches to Neuroblastoma Therapy (NANT) Phase I Consortium has a number of open clinical trials testing new treatments for children with recurrent or refractory neuroblastoma. Several studies are testing combination therapy that includes biologic agents plus chemotherapy.

Whichever treatment plan you choose, palliative care will be important for relieving symptoms and side effects.

When a child has a recurrent tumor, family members often experience emotions such as disbelief or fear. Families are encouraged to talk with the health care team about these feelings and ask about support services to help them cope. Learn more about dealing with cancer recurrence.

If treatment fails

Although treatment is successful for the majority of children with cancer, sometimes it is not. If a child’s cancer cannot be cured or controlled, this is called an advanced or terminal cancer. This diagnosis is stressful, and advanced cancer may be difficult to discuss. However, it is important to have open and honest conversations with your child’s doctor and health care team to express your family’s feelings, preferences, and concerns. The health care team is there to help, and many team members have special skills, experience, and knowledge to support patients and their families.

Parents or guardians are encouraged to think about where the child would be most comfortable: at home, in a home-like setting elsewhere, in the hospital, or in a hospice environment. 

Hospice care is a type of palliative care for people who are expected to live less than six months. It is designed to provide the best possible quality of life for people who are near the end of life. Nursing care and special equipment can make staying at home a workable alternative for many families.

Some children may be happier if they can arrange to attend school part-time or keep up other activities and social connections. The child’s health care team can help parents or guardians decide on an appropriate level of activity. Making sure a child is physically comfortable and free from pain is extremely important as part of end-of-life care. Learn more about caring for a terminally ill child and advanced cancer care planning.

The death of a child is an enormous tragedy, and families may need support to help them cope with the loss. Pediatric cancer centers often have professional staff and support groups to help with the process of grieving. Learn more on grieving the loss of a child.

The next section in this guide is About Clinical Trials and it offers more information about research studies that are focused on finding better ways to care for people with cancer. Or, use the menu on the side of your screen to choose another section to continue reading this guide.  

Neuroblastoma - Childhood - About Clinical Trials

This section has been reviewed and approved by the Cancer.Net Editorial Board, 03/2015

ON THIS PAGE: You will learn more about clinical trials, which are the main way that new medical approaches are tested to see how well they work. To see other pages, use the menu on the side of your screen.

What are clinical trials?

Doctors and scientists are always looking for better ways to care for children with neuroblastoma. To make scientific advances, doctors create research studies involving volunteers, called clinical trials. In fact, every drug that is now approved by the FDA was previously tested in clinical trials. Clinical trials for newly diagnosed patients and for patients with refractory or recurrent disease are ongoing in the Children’s Oncology Group.

Many clinical trials are focused on new treatments, evaluating whether a new treatment is safe, effective, and possibly better than the current (standard) treatment. These types of studies evaluate new drugs, different combinations of existing treatments, new approaches to radiation therapy or surgery, and new methods of treatment.

Children who participate in clinical trials are often among the first to receive new treatments before they are widely available. However, there is no guarantee that the new treatment will be safe, effective, or better than a standard treatment.

There are also clinical trials that study new ways to ease symptoms and side effects during treatment and manage the late effects that may occur after treatment. Talk with your child’s doctor about clinical trials regarding side effects. In addition, there are ongoing studies about ways to prevent the disease.

Deciding to join a clinical trial

Patients decide to participate in clinical trials for many reasons. For some patients, a clinical trial is the best treatment option available. Because standard treatments are not perfect, patients are often willing to face the added uncertainty of a clinical trial in the hope of a better result. Other patients volunteer for clinical trials because they know that these studies are the only way to make progress in treating neuroblastoma. Even if they do not benefit directly from the clinical trial, their participation may benefit future patients with neuroblastoma.

Sometimes people have concerns that, in a clinical trial, their child may receive no treatment by being given a placebo or a “sugar pill.” The use of placebos in cancer clinical trials in this way is rare overall and not done at all in childhood cancer research. Find out more about placebos in cancer clinical trials.

Patient safety and informed consent

To join a clinical trial, parents and children must participate in a process known as informed consent. During informed consent, the doctor should list all of the patient’s options so that the person understands how the new treatment differs from the standard treatment. The doctor must also list all of the risks of the new treatment, which may or may not be different from the risks of standard treatment. Finally, the doctor must explain what will be required of each patient in order to participate in the clinical trial, including the number of doctor visits, tests, and the schedule of treatment.

People who participate in a clinical trial may stop participating at any time for any personal or medical reason. This may include that the new treatment is not working or there are serious side effects. It is important that parents of a child participating in a clinical trial talk with the doctor and researchers about who will be providing their child’s treatment and care during the clinical trial, after the clinical trials ends, and/or if the person chooses to leave the clinical trial before it ends. 

Finding a clinical trial

Research through clinical trials is ongoing for all types of cancer. For specific topics being studied for neuroblastoma, learn more in the Latest Research section.

Cancer.Net offers a lot of information about cancer clinical trials in other areas of the website, including a complete section on clinical trials and places to search for clinical trials for a specific type of cancer.

In addition, this website offers free access to a video-based educational program about cancer clinical trials, located outside of this guide.

The next section in this guide is Latest Research and it explains areas of scientific research currently going on for this type of cancer. Or, use the menu on the side of your screen to choose another section to continue reading this guide.    

Neuroblastoma - Childhood - Latest Research

This section has been reviewed and approved by the Cancer.Net Editorial Board, 03/2015

ON THIS PAGE: You will read about the scientific research being done now to learn more about this type of tumor and how to treat it. To see other pages, use the menu on the side of your screen.

Doctors are working to learn more about neuroblastoma, ways to prevent it, how to best treat it, and how to provide the best care to people diagnosed with this disease. The following areas of research may include new options for patients through clinical trials. Always talk with your child’s doctor about the diagnostic and treatment options best for your child.

Many of the items below are explained earlier in the Treatment Options section. Studies are underway to improve their use and effectiveness.

  • New drug combinations. Clinical trials are underway to study the use of a drug called topotecan in combination with cyclophosphamide. Researchers hope that this drug combination will increase the effectiveness and decrease the side effects of initial treatment.

  • Stem cell transplantation.  A clinical trial comparing two cycles of high-dose chemotherapy to one cycle of high-dose chemotherapy and stem cell transplantation has recently been completed. The results of this trial should be available within the next two years. Previous Children’s Oncology Group studies used carboplatin, etoposide, and melphan as the conditioning regimen before a stem cell transplant.

    The Children’s Oncology Group is now testing a new consolidation regimen consisting of busulfan and melphalan before a stem cell transplant. A European study compared outcome for children who received conditioning prior to a stem cell transplant with carboplatin, etoposide, melphan vs. busulfan and melphalan. The study suggested that the outcome was better for children treated with busulfan and melphalan.

  • New radiation therapy techniques. A radioactive MIBG delivers radiation therapy to neuroblastoma cells for recurrent or refractory disease, with or without stem cell transplantation. By using a high-energy form of radioactive iodine with the MIBG, enough radiation therapy is given off to kill the neuroblastoma cells. This treatment is currently available at about a dozen centers in the United States. Clinical trials involving radioactive MIBG when there is a recurrence and for newly diagnosed children with high-risk neuroblastoma are ongoing.

  • Other treatment options. Research on the use of small molecules to target the cell functions that are abnormal in neuroblastoma cells are ongoing. Crizotinib and other drugs that inhibit ALK, a tyrosine kinase that is mutated in a small subset some types of neuroblastomas, as well as other tyrosine kinase inhibitors are being tested in early phase clinical trials. Studies testing crizotinib in newly diagnosed patients with neuroblastoma tumors with ALK mutations are planned in the Children’s Oncology Group.

  • Palliative care. Clinical trials are underway to find better ways of reducing symptoms and side effects of current neuroblastoma treatments in order to improve patients’ comfort and quality of life.

Looking for More About the Latest Research?

If you would like additional information about the latest areas of research regarding neuroblastoma, explore these related items that take you outside of this guide:

The next section in this guide is Coping with Side Effects and it offers some guidance in how to cope with the physical, emotional, and social changes that cancer and its treatment can bring. Or, use the menu on the side of your screen to choose another section to continue reading this guide.  

Neuroblastoma - Childhood - Coping with Side Effects

This section has been reviewed and approved by the Cancer.Net Editorial Board, 03/2015

ON THIS PAGE: You will find out more about steps to take to help cope with physical, social, and emotional side effects. This page includes several links outside of this guide to other sections of this website. To see other pages, use the menu on the side of your screen.

Fear of treatment side effects is common after a diagnosis of a tumor, but it may be helpful to know that preventing and controlling side effects is a major focus of your child’s health care team. This is called palliative care, and it is an important part of the overall treatment plan, regardless of the stage of the disease.

There are possible side effects for every cancer treatment, but patients don’t experience the same side effects when given the same treatments for many reasons. That can make it hard to predict exactly how your child will feel during treatment.

Common side effects for each treatment option are described in detail within the Treatment Options section.  Learn more about the most common side effects of cancer and different treatments, along with ways to prevent or control them.

The specific side effects that can occur depend on a variety of factors, including:

  • The type of cancer

  • Its location

  • The individual treatment plan, including the length and dosage of treatment

  • Your child’s overall health

Talking with your health care team about side effects

Before treatment begins, talk with your child’s doctor about possible side effects of the specific treatments your child will receive. Ask which side effects are most likely to happen and which are not, when side effects are likely to occur, and what can be done to prevent or relieve them.

And, ask about the level of caregiving your child may need during treatment and recovery, as family members and friends often play an important role in the care of a child with neuroblastoma. Learn more about caregiving.

In addition to physical side effects, there may be emotional and social effects as well. Patients and their families are encouraged to share their feelings with a member of their health care team who can help with coping strategies, including concerns about managing the cost of your child’s cancer care

During and after treatment, be sure to tell the health care team about the side effects your child experiences, even if you feel they are not serious. Sometimes, side effects can last beyond the treatment period, called a long-term side effect. A side effect that occurs months or years after treatment is called a late effect. Treatment of both types of effects is an important part of survivorship care. Learn more by reading the Follow-up Care section of this guide or talking with your child’s doctor.

The next section in this guide is Follow-up Care and it explains the importance of check-ups after cancer treatment is finished. Or, use the menu on the side of your screen to choose another section to continue reading this guide.  

Neuroblastoma - Childhood - Follow-Up Care

This section has been reviewed and approved by the Cancer.Net Editorial Board, 03/2015

ON THIS PAGE: You will read about your child’s medical care after treatment is finished and why this follow-up care is important. To see other pages, use the menu on the side of your screen.

Care for children diagnosed with cancer doesn’t end when active treatment has finished. Your child’s health care team will continue to check to make sure the cancer has not returned, manage any side effects, and monitor your child’s overall health. This is called follow-up care. All children treated for cancer, including neuroblastoma, should have life-long follow-up care.

A plan may include regular physical examinations and/or medical tests to monitor your child’s recovery for the coming months and years. Follow-up treatment care children treated for neuroblastoma depends on the risk grouping:

  • Low-risk or intermediate-risk neuroblastoma. The child is evaluated every three to six months until 24 months after treatment ends, depending on the treatment given, the patient’s age, and other factors. Then, the child is evaluated at least once a year.

  • High-risk, advanced neuroblastoma. Follow-up care is decided on an individual basis. Tests are performed every few months for two to three years after treatment ends to find out whether the disease has recurred or gotten worse.

Watching for recurrence

One goal of follow-up care is to check for a recurrence. Cancer recurs because small areas of cancer cells may remain undetected in the body. Over time, these cells may increase in number until they show up on test results or cause signs or symptoms.

During follow-up care, a doctor familiar with your child’s medical history can give you personalized information about the risk of recurrence. Your doctor will also ask specific questions about your child’s health. Some children may have blood tests or imaging tests as part of regular follow-up care. However, testing recommendations depend on several factors, including the type and stage of cancer originally diagnosed and the types of treatment given.

Managing long-term and late side effects of childhood cancer

Sometimes, side effects may linger beyond the active treatment period. These are called long-term side effects. In addition, other side effects called late effects may develop months or even years afterwards. Late effects can occur almost anywhere in the body and include physical problems, such as heart and lung problems and second cancers, and emotional and cognitive (memory, thinking, and attention) problems, such as anxiety, depression, and learning difficulties.

Based on the type of treatment the child received, the doctor will determine what examinations and tests are needed to check for long-term side effects and the possibility of secondary cancers. Your child’s doctor can recommend the necessary screening tests. Follow-up care should also address the child’s quality of life, including any developmental or emotional concerns. Learn more about the childhood cancer survivorship.

Possible long-term side effects or late effects include:

  • Cardiovascular problems. If your child received doxorubicin during chemotherapy, he or she may be at risk for heart problems, including weakening of the heart muscle. The doctor may recommend imaging of the heart with echocardiograms (echo) or other tests, as well as electrocardiograms (ECG or EKG) and blood pressure monitoring. The risk of these problems is related to the total dose of doxorubicin, but is also increased if the child received radiation therapy to the chest.

  • Hearing problems. If your child has taken cisplatin/carboplatin, hearing loss is a possible side effect. Hearing tests are recommended at the end of treatment, and then once a year if the test results indicate a hearing problem.

  • Kidney problems. If your child has taken cisplatin or had a bone marrow/stem cell transplant the doctor will monitor kidney function by doing specific blood and urine tests as a part of a yearly visit. More tests may be needed if test results indicate a problem.

  • Hormonal changes. If your child received radiation therapy, his or her primary care doctor will monitor growth and development yearly and evaluate your child for delayed puberty at age 12 (girls) or 14 (boys) through hormone blood tests.

  • Other cancers.  Children diagnosed with neuroblastoma are at increased for developing other cancers. The doctor will monitor your child for subsequent cancers using blood tests and physical exams.  More tests may be needed if the test results indicate a problem.

Follow-up care after radiation therapy

Children who have had radiation may be at risk for other cancers, including:

  • Breast cancer. If your child received total body radiation therapy or radiation therapy to the chest, your child should learn how to do a breast self-examination once he or she reaches puberty and perform them monthly. Regular mammograms may begin in early adulthood, rather than waiting until later.

  • Skin cancer. You should learn to inspect your child’s skin and ask the doctor to inspect any unusual skin findings at each yearly physical examination.

  • Other cancers. It is important for children with cancer to receive regular primary medical care. Talk with your child’s doctor if you are concerned about any symptoms, especially if your child has ongoing pain or a lump in an area that received radiation therapy.

Follow-up care after stem cell transplantation/bone marrow transplantation

Children who have had a bone marrow or stem cell transplantation may have late effects. Possible late effects include:

  • Problems with the way the thyroid gland, kidneys, lungs, and heart work

  • Problems with growth

  • Problems handling infections

  • Increased risk of other cancers

  • Problems with fertility, which is the ability to have a child, and hormones

  • Hearing loss

Because of these possible problems, it is very important for children treated with stem cell transplantation to have certain tests and immunizations once each year. These may include the following evaluations:

  • Heart tests, such as an echocardiogram and EKG

  • Lung tests, such as pulmonary function testing (PFT)

  • Blood tests that check the function of the kidneys, liver, and thyroid

  • Immunoglobulin levels, such as IgG

  • Hearing tests, if needed

  • Eye examination, if the child received radiation therapy to the head or total body radiation therapy

  • Dental examination

  • Blood tests to evaluate hormone levels, such as testosterone, estrogen, and growth hormones

  • If needed, an examination by an endocrinologist, a doctor who specializes in problems with glands and hormones

  • Gynecologic examination for girls

  • Immunizations as directed by the health care team

Recommendations for long-term follow-up care

The Children's Oncology Group (COG) has studied the physical and psychological effects that childhood cancer survivors face. Based on these studies, COG has created recommendations for long-term follow-up care for childhood, adolescent, and young adult cancer survivors that can be found on a separate website: www.survivorshipguidelines.org.

Keeping a child’s personal health record

You are encouraged to organize and keep a personal record of the child’s medical information. The doctor will help you create this. That way, as the child enters adulthood, he or she has a clear, written history of the diagnosis, the treatment given, and the doctor’s recommendations about the schedule for follow-up care. ASCO offers forms to help create a treatment summary to keep track of the cancer treatment your child received and develop a survivorship care plan once treatment is completed.

Some children continue to see their oncologist, while others transition back to the general care of their family doctor or another health care professional. This decision depends on several factors, including the type and stage of cancer, side effects, health insurance rules, and your family’s personal preferences. Talk with your health care team about your child’s ongoing medical care and any concerns you have about his or her future health.

If a doctor who was not directly involved in your child’s cancer care will lead the follow-up care, be sure to share the cancer treatment summary and survivorship care plan forms with him or her, as well as all future health care providers. Details about the specific cancer treatment given are very valuable to the health care professionals who will care for your child throughout his or her lifetime.

The next section in this guide is Survivorship and it describes how to cope with challenges in everyday life after a cancer diagnosis. Or, use the menu on the side of your screen to choose another section to continue reading this guide.

Neuroblastoma - Childhood - Survivorship

This section has been reviewed and approved by the Cancer.Net Editorial Board, 03/2015

ON THIS PAGE: You will read about how to cope with challenges in everyday life after your child’s cancer diagnosis. To see other pages, use the menu on the side of your screen.

What is survivorship?

The word survivorship means different things to different people, but it often describes the process of living with, through, and beyond cancer. In some ways, survivorship is one of the most complex aspects of the cancer experience because it is different for every patient and his or her family.

After active cancer treatment ends, children and their families may experience a mixture of strong feelings, including joy, concern, relief, guilt, and fear. Some people say they appreciate life more after a cancer diagnosis. Other families stay very anxious about their child’s health and become uncertain of how to cope with everyday life.

One source of stress may occur when frequent visits to the health care team end following treatment. Often, relationships built with the cancer care team provide a sense of security during treatment, and children and their families miss this source of support. This may be especially true as new worries and challenges surface over time, such as any late effects of treatment, educational issues, emotional challenges, sexual development and fertility concerns, and/or financial issues.

Every family faces different concerns and challenges. With any challenge, a good first step is being able to recognize each fear and talk about it. Effective coping requires:

  • Understanding the challenge your family is facing,

  • Thinking through solutions,

  • Asking for and allowing the support of others, and

  • Feeling comfortable with the course of action your family chooses.

It may be helpful to join an in-person support group or online community of childhood cancer survivors. Support groups also exist for parents of children diagnosed with cancer. This allows you to talk with people who have had similar first-hand experiences. Other options for finding support include talking with a friend or member of your health care team, individual counseling, or asking for assistance at the learning resource center of the center where your child received treatment.

Changing role of caregivers

Parents, other family members, and friends may also go through periods of transition. A caregiver plays a very important role in supporting a child diagnosed with cancer, providing physical, emotional, and practical care on a daily or as-needed basis. Many caregivers become focused on providing this support, especially if the treatment period lasts for many months or longer.

However, as treatment is completed, the caregiver's role often changes. Eventually, the need for caregiving related to a child’s cancer diagnosis will become much less or come to an end as your child gets older. Family counselors at pediatric cancer centers can help with this transition. You can also learn more about adjusting to life after caregiving in this article.

Healthy living after cancer

Survivorship often serves as a strong motivator to make positive lifestyle changes, often for the family as a whole. Children who have had cancer can enhance the quality of their future by following established guidelines for good health into and through adulthood, including not smoking, maintaining a healthy weight, eating well, managing stress, and participating in regular physical activity. Talk with the doctor about developing a plan that is best for your child’s needs. Learn more about making healthy lifestyle choices.

In addition, it is important that your child has recommended medical check-ups and tests (see Follow-up Care) to take care of his or her health. Cancer rehabilitation may be recommended, and this could mean any of a wide range of services such as physical therapy, family or individual counseling, nutritional planning, and/or educational assistance. The goal of rehabilitation is to help survivors and their families regain control over many aspects of their lives and remain as independent and productive as possible.

Talk with your doctor to develop a survivorship care plan that is best for your child’s needs.

Looking for More Survivorship Resources?

For more information about cancer survivorship, explore these related items. Please note these links will take you to other sections of Cancer.Net:

  • Survivorship Resources: Cancer.Net offers a lot of information and resources to help survivors cope, including specific sections for children, teens, and young adults. There is also a main section on survivorship for people of all ages.

  • ASCO Answers Cancer Survivorship Guide: This 44-page booklet (available as a PDF) can help with the transition into life after treatment. It includes blank treatment summary and survivorship care plan forms.

  • Cancer.Net Patient Education Video: View a short video led by an ASCO expert that provides information about childhood cancer survivorship.

The next section offers Questions to Ask the Doctor to help start conversations with your child’s cancer care team. Or, use the menu on the side of your screen to choose another section to continue reading this guide.  

Neuroblastoma - Childhood - Questions to Ask the Doctor

This section has been reviewed and approved by the Cancer.Net Editorial Board, 03/2015

ON THIS PAGE: You will find some questions to ask your child’s doctor or other members of the health care team, to help you better understand your child’s diagnosis, treatment plan, and overall care. To see other pages, use the menu on the side of your screen.

Talking often with the doctor is important to make informed decisions about your child’s health care. These suggested questions are a starting point to help you learn more about your child’s cancer care and treatment. You are also encouraged to ask additional questions that are important to you.

You may want to print this list and bring it to your child’s next appointment, or download Cancer.Net’s free mobile app for an e-list and other interactive tools to manage your child’s care.

Questions to ask after getting a diagnosis

  • Where is the cancer located?

  • Can you explain my child’s pathology report (laboratory test results) to me?

  • What is the stage of the cancer? What does this mean?

  • What risk group has my child’s neuroblastoma been classified as? What does this mean?

  • Does my child’s tumor secrete any tumor markers?

Questions to ask about choosing a treatment and managing side effects

  • What are my child’s treatment options?

  • What clinical trials are open to my child? Where are they located, and how do I find out more about them?

  • Does this hospital participate in clinical trials for children with neuroblastoma?

  • What treatment plan do you recommend? Why?

  • What is the goal of each treatment? Is it to eliminate the cancer, help my child feel better, or both?

  • What is my child’s prognosis?

  • How many children with neuroblastoma are seen and treated at this hospital?

  • Who will be part of my child’s health care team, and what does each member do?

  • Who will be coordinating my child’s overall treatment?

  • What are the possible side effects of this treatment, both in the short term and the long term?

  • How will this treatment affect my child’s daily life? Will he or she be able to attend school or perform his or her usual activities?

  • Could this treatment affect my child’s ability to become pregnant or have children? If so, should my family talk with a fertility specialist before cancer treatment begins?

  • If I’m worried about managing the costs related to my child’s medical care, who can help me with these concerns?

  • What follow-up tests are needed, and how often are they needed?

  • What support services are available to my child? To my family?

  • Whom should I call for questions or problems?

  • Is there anything else I should be asking?

Questions to ask about having surgery

  • What type of surgery will my child have? Will lymph nodes be removed?

  • How long will the operation take?

  • How long will my child be in the hospital?

  • Can you describe what my child’s recovery from surgery will be like?

  • What are the possible long-term effects of having this surgery?

Questions to ask about having multiple types of treatments for high-risk neuroblastoma, such as radiation therapy, chemotherapy, stem cell transplantation/bone marrow transplantation, immunotherapy, and other therapies.

  • Why are multiple types of treatment recommended?

  • What is the goal of each treatment?

  • How long will it take to give each treatment?

  • What side effects can my child expect during each treatment?

  • What are the possible long-term effects of having each treatment?

  • What can be done to relieve the side effects?

Questions to ask about planning follow-up care

  • What is the risk of the cancer returning? Are there signs and symptoms we should watch for?

  • What long-term side effects or late effects are possible based on the cancer treatment my child received?

  • What follow-up tests will my child need, and how often will he or she need them?

  • How do I get a treatment summary and survivorship care plan to keep in my child’s personal records? 

  • Who will be coordinating my child’s follow-up care?

  • What survivorship support services are available to my child? To my family?

The next section in this guide is Additional Resources, and it offers some more resources on this website beyond this guide that may be helpful to you. Or, use the menu on the side of your screen to choose another section to continue reading this guide.  

Neuroblastoma - Childhood - Additional Resources

This section has been reviewed and approved by the Cancer.Net Editorial Board, 03/2015

ON THIS PAGE: You will find some helpful links to other areas of Cancer.Net that provide information about cancer care and treatment. This is the final page of Cancer.Net’s Guide to Childhood Neuroblastoma. To go back and review other pages, use the menu on the side of your screen.

Cancer.Net includes many other sections about the medical and emotional aspects of cancer, both for the person diagnosed and their family members and friends. This website is meant to be a resource for you and your loved ones from the time of diagnosis, through treatment, and beyond.

Beyond this guide, here are a few links to help you explore other parts of Cancer.Net:

This is the end of Cancer.Net’s Guide to Childhood Neuroblastoma. Use the menu on the side of to select another section to continue reading this guide.