Neuroblastoma is a cancerous tumor that begins in nerve tissue of young children. Abnormal nerve cells may be present before birth, but the diagnosis isn’t made until the cells begin to multiply, forming a tumor. Neuroblastoma is most commonly diagnosed in children less than 5 years of age and is very rare after the age of 10. A neuroblastoma tumor usually begins in the abdomen, especially in the tissues of the adrenal glands, which sit on top of the kidneys but also may begin in nerve tissue in the neck, chest or pelvis. Neuroblastoma cancer cells can spread (metastasize) to other areas of the body (including the lymph nodes, liver, bones and bone marrow).
Because neuroblastoma is rarely seen in adults, it is important that your child receive care from an experienced team of pediatric specialists who focus exclusively on treating childhood cancers. Our neuroblastoma specialists and surgeons are known for treating children with the most complex cases (including relapsed or refractory neuroblastoma), as well as for their expertise in delivering specialized treatments, including MIBG therapy and stem cell transplantation. Continue reading to learn more about neuroblastoma, or visit our Neuroblastoma Program page to learn about our expertise and treatment options for neuroblastoma.
In the United States, about 650 children are diagnosed with neuroblastoma each year.
The cause of neuroblastoma is not completely understood. It is likely that neuroblastoma develops from an early form of a nerve cell (called a neuroblast), found in the fetus, which grows abnormally due to a genetic mutation inside the cell. In most cases of neuroblastoma, scientists still are trying to identify this genetic abnormality and what causes it.
In rare cases, neuroblastoma is caused by an inherited alteration in a specific gene. The gene most commonly affected is the ALK gene, a protein coding gene. Hereditary neuroblastoma accounts for only 1 to 2 percent of cases of neuroblastoma.
Because neuroblastoma often occurs very early in childhood, research has been done to find any links between maternal environmental exposures and tumor development. No such associations have been found.
Neuroblastoma usually begins in the abdomen, especially in the tissues of the adrenal glands, which sit on top of the kidneys, but may also begin in nerves tissues in the neck, chest or pelvis. These tumors often spread (metastasize) to other areas of the body, including the lymph nodes, liver, bones and bone marrow. Nearly 70 percent of children with neuroblastoma will have metastatic disease at diagnosis.
Neuroblastoma symptoms can vary greatly, depending on the size and location of the tumor and whether or not the tumor has spread.
Common signs and symptoms of neuroblastoma include:
Keep in mind that neuroblastoma symptoms may resemble other, more common conditions or medical problems. Always consult your child's physician for a diagnosis.
Your child’s doctor will perform a complete medical and physical examination. In addition, your child’s physician may order one or more of the following tests:
Neuroblastoma tumor risk groups
Diagnosing neuroblastoma also involves determining the neuroblastoma risk group, which will help your child’s doctor determine neuroblastoma treatment options and prognosis. Children with neuroblastoma are divided into three risk groups depending on their age, the stage of the tumor and certain biological factors of the tumor. The three risk groups are low, intermediate and high.
Staging the tumor
"Tumor stage" describes the size of the tumor and whether or not it has spread. The most commonly used staging system is the International Neuroblastoma Staging System:
Your child’s physician will determine a specific course of neuroblastoma treatment for your child based on the tumor risk group and other factors. Neuroblastoma treatment may include:
These neuroblastoma treatments can be used alone or in combination depending on the neuroblastoma risk group. In general, recommended treatment for the three neuroblastoma risk groups is:
Patients with low-risk neuroblastoma usually require only surgery and/or observation. Occasionally, a patient with a low-risk tumor will need chemotherapy (or radiation treatment) to shrink a tumor before it is removed.
Patients with intermediate-risk disease are treated with chemotherapy to shrink the tumor (and/or its sites of spread), before the tumor can be surgically removed. The actual medications used are chosen to try to minimize side effects, both during treatment and in the future.
High-risk neuroblastoma is an aggressive disease that is treated with a combination of therapies including chemotherapy, surgery, radiation, stem cell transplant, biologic therapy, MIBG therapy and immunotherapy.
Your child’s prognosis (chance of recovery) and treatment options depend on a number of different factors:
In general, low- and intermediate-risk neuroblastomas tend to be more treatable. High-risk neuroblastomas are more difficult to treat and require more aggressive therapy. Prompt medical attention and appropriate therapy are important for the best prognosis.
Many children diagnosed with neuroblastoma will survive into adulthood. However, chemotherapy and radiation therapy can cause damage to healthy tissues. As a result, survivors may face physical, psychological, social and intellectual challenges related to their treatment and require ongoing assessment and specialized care.
Through our cancer survivorship program at the David B. Perini, Jr. Quality of Life Clinic at Dana-Farber/Boston Children’s, childhood cancer survivors receive a comprehensive follow-up evaluation from their cancer care team.
We also offer the following services:
Your child should visit a survivorship clinic every year to:
A typical follow-up visit may include some or all of the following:
Learn more about our patient and family support services.
Research is a top priority at Dana-Farber/Boston Children's, and our physicians work continuously to translate laboratory findings into clinical therapies.
It’s possible that your child will be eligible to participate in one of the Neuroblastoma Program’s current clinical trials. In addition to launching our own clinical trials, we also offer the most Phase I studies in New England for children whose disease has recurred through the Children's Oncology Group and the New Approaches to Neuroblastoma Therapy (NANT) consortium.
Our current research efforts focus on improving established neuroblastoma therapies, studying the genetic causes of the disease and developing novel therapies.
Reducing late effects
Children with low- and intermediate-risk neuroblastoma often can be cured with surgery alone, or surgery and chemotherapy. Because of the risk of physical, psychological, social, intellectual and other late effects, we are constantly working to decrease the intensity of treatment as much as possible while still providing the best cure rates.
Recent neuroblastoma treatment developments
Recent developments in the treatment of neuroblastoma have included identification of several genetic findings in the tumors that can predict either a favorable or unfavorable prognosis. These tests have provided valuable information indicating which tumors need to be treated aggressively and which tumors require little treatment beyond surgical removal.
Attacking the genetic cause
Researchers at Dana-Farber/Boston Children’s have published some of the early work on the association between neuroblastoma and the ALK gene. Together with researchers at Children’s Hospital of Philadelphia, we are working on therapies that target this gene and may improve treatment for neuroblastoma.
Advanced radiation treatment for neuroblastoma
Nuclear medicine physicians and scientists are an integral part of the Dana-Farber/Boston Children’s team's MIBG treatment for children with relapsed neuroblastoma (cancerous tumors that originate in the nerve tissues). The treatment, which uses a radioactive compound to kill tumor cells, is available in only a handful of major medical centers in North America. Dana-Farber/Boston Children’s is the only center in New England to offer this therapy.
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We were the first hospital in New England to offer MIBG therapy to treat neuroblastoma and currently one of only a handful of hospitals in the country to provide this therapy.