• Neuroblastoma

    Dr. Suzanne Shusterman leads the Neuroblastoma ProgramNeuroblastoma expert Suzanne Shusterman,
    MD, leads the Neuroblastoma Program

    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).

    Neuroblastoma Treatment at Dana-Farber/Boston Children's

    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.

    What causes neuroblastoma?

    In the United States, about 650 children are diagnosed with neuroblastoma each year.

    • Neuroblastoma usually occurs before a child is 5 years of age.
    • It is the most common tumor found in children younger than 1 year old.
    • Neuroblastoma is rare in children older than 10 years; however, it does occur occasionally in adults.

    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.

    What are the symptoms of neuroblastoma?

    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:

    • an abdominal mass, either felt during an examination or seen as a swollen abdomen
    • enlarged lymph nodes in the neck
    • swelling and bruising of the area around the eyes
    • unexplained fevers, bone pain or limping caused by bone or bone marrow involvement
    • weakness or paralysis caused by compression of the spinal cord by tumor
    • weight loss or poor appetite
    • uncontrolled eye or leg movements caused by an autoimmune response to the tumor
    • high blood pressure, due to compression of the kidney by the tumor
    • high blood pressure, increased heart rate and flushing due to a substance produced by the tumor
    • diarrhea caused by a substance produced by the tumor

    Keep in mind that neuroblastoma symptoms may resemble other, more common conditions or medical problems. Always consult your child's physician for a diagnosis.

    How is neuroblastoma diagnosed?

    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:

    • imaging studies—to evaluate the primary tumor and determine the extent and location of metastases, including:
      • computerized tomography scan (CT or CAT scan)—A CT scan shows detailed images of any part of the body, including the bones, muscles and fat.
      • magnetic resonance imaging (MRI)—A test that uses a combination of large magnets, radiofrequencies and a computer to produce detailed images of organs and structures within the body.
      • bone scans—Pictures or x-rays taken of the bone after a dye is injected into a vein and is absorbed by bone tissue. These are used to detect tumors and bone abnormalities. This test is used to detect spread of neuroblastoma to the bones.
      • Metaiodobenzylguanidine (MIBG) scan—MIBG is a radioactive chemical that is absorbed by most neuroblastomas. A specialized scanner is then used to detect the chemical and locate places the presence of the neuroblastoma. This test is used to detect spread of neuroblastoma to the bones and other tissues and is more sensitive than a bone scan.
    • bone marrow biopsy and/or aspiration—a procedure that involves taking a small amount of bone marrow fluid and tissue, usually from part of the hip bones, to determine whether neuroblastoma cells have spread to the bone marrow.
    • urine test—to measure specific chemicals made by neuroblastoma cells.
    • biopsy—Surgeons or interventional radiologists will remove either a piece of the tumor or the whole tumor depending on tumor location and size. Pediatric pathologists will analyze the tumor and other important tests will be done to determine the tumor biology, such as tumor histology and genetic studies.
    • blood tests—including a complete blood count, blood chemistries, as well askidney and liver function 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:

    • Stage 1—Localized tumor that is completely surgically removed at diagnosis.
    • Stage 2—Localized tumor that cannot be completely surgically removed at diagnosis. Lymph nodes on the same side of the tumor may have tumor cells present.
    • Stage 3—Large tumor that has spread across the middle of the body and cannot be surgically removed at diagnosis.
    • Stage 4—Tumor of any size that has metastasized (spread) to distant lymph nodes, bone marrow, bone or liver.
    • Stage 4s—Special stage that applies only to a child less than 1-year-old. Small, localized tumor that has metastasized (spread) to liver, skin and/or bone marrow.

    How is neuroblastoma treated?

    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:

    • Surgery: Surgery is used to remove the tumor either at diagnosis or after using chemotherapy or radiation to make the tumor smaller.
    • Chemotherapy: Several chemotherapy medications are known to be effective in killing neuroblastoma cells. Chemotherapy is used to decrease the size of the tumor, kill tumor that has spread and prevent further tumor spread.
    • Radiation: Radiation is used to kill neuroblastoma cells left behind after chemotherapy and/or surgery. It is also sometimes used for patients who need immediate shrinkage of the tumor to prevent or treat a complication (such as the tumor pressing on an important nerve or the spinal cord).
    • Stem cell transplant: This involves temporarily removing some of a child’s healthy blood cells and giving them very high doses of chemotherapy and, in some cases, radiation therapy to overwhelm and destroy the neuroblastoma. The child is then given back his or her own stored healthy blood cells to promote normal body and organ function once treatment is done.
    • Biologic therapy: These therapies include antibody therapy and a medicine called isotretinoin, which take advantage of what we have learned about the biologic characteristics of neuroblastoma cells. The objective of biologic therapy is to kill any remaining microscopic tumor cells.
    • MIBG Therapy: If your child has high-risk neuroblastoma (including relapsed or refractory neuroblastoma), she may receive a specialized treatment known as metaiodobenzylguanidine (MIBG) therapy. MIBG therapy, which uses a radioactive isotope that is readily absorbed by most neuroblastomas, can be used to detect neuroblastoma in the body or to deliver radiation in order to kill the neuroblastoma cells.
    • Immunotherapy: Used only for treatment of high-risk neuroblastoma, immunotherapy generally is given in combination with chemotherapy, using Unituxin (dinutuximab), an antibody that binds to the surface of neuroblastoma cells

    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:

    Low-risk neuroblastoma

    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.

    Intermediate-risk neuroblastoma

    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

    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.

    What is the long-term outlook for children with neuroblastoma?

    Your child’s prognosis (chance of recovery) and treatment options depend on a number of different factors:

    • the extent of the disease
    • the size and location of the tumor
    • the tumor's characteristics when examined under a microscope
    • the presence or absence of metastasis
    • the tumor's response to therapy
    • the age and overall health of your child
    • your child's tolerance of specific medications, procedures or therapies
    • new developments in treatment

    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.

    What follow-up neuroblastoma survivor care will my child receive?

    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.

    • Our childhood cancer survivorship clinic is held weekly.
    • In addition to meeting with your pediatric oncologists, your child may see one of our endocrinologists, cardiologists, neurologists, neuro-psychologists or alternative/complementary therapy specialists.

    We also offer the following services:

    Your child should visit a survivorship clinic every year to:

    • manage disease complications
    • screen for early recurrence of cancer
    • manage late effects of treatment

    A typical follow-up visit may include some or all of the following:

    • a physical exam
    • laboratory testing
    • imaging scans (such as MRI or CT)

    Learn more about our patient and family support services.

    What is the latest research for neuroblastoma?

    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.

    In addition:

    • Our scientists are currently studying how to use high-dose therapy in combination with stem cell transplantation to treat neuroblastoma.
    • Dana-Farber/Boston Children’s Pediatric Stem Cell Transplant Center is one of only eight institutions around the U.S. investigating the use of umbilical cord transplantation.
    • Also under study is a novel method for preventing graft-versus-host disease, a serious complication that occurs when transplanted cells don’t recognize the tissues and organs of the recipient's body and react against the recipient's tissue. This treatment approach may dramatically increase the pool of potential donors for each patient.
    • Another promising area of research is developing treatments that allow doctors to use a patient's own stem cells to overcome neuroblastoma.

    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.

  • Clinical Trials

    Search our open clinical trials for neuroblastoma.callout bg
  • Taking a sideswipe at high-risk neuroblastoma

    neuroblastoma illustration

    Researchers led by Kimberly Stegmaier, MD, have found a new pathway that could combat high-risk neuroblastoma in children.

  • MIBG Therapy

    MIBG Reading 

    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.