Childhood Neuroblastoma

Expert Care and Treatment for Childhood Solid Tumors

The Childhood Solid Tumor Center at Dana-Farber/Boston Children's Cancer and Blood Disorders Center treats children and teens with a variety of solid tumors. Our specialty programs are led by pediatric clinicians with deep expertise in diagnosis and treatment of rare and complex solid tumors. Learn more about solid tumor types that we treat.

Childhood Solid Tumor Center

What Is Neuroblastoma?

Neuroblastomas start in the nerve tissue of infants and young children, typically before birth. Most neuroblastomas develop in the abdomen, especially in the adrenal glands. They may also form in the neck, chest, or pelvis.

  • Neuroblastoma occurs most often in children under five years of age.
  • It is the most common tumor in children younger than one year of age.
  • It is rare in children older than 10 years of age.
  • In the United States, doctors diagnose approximately 650 children with neuroblastoma each year.
  • These tumors often metastasize (spread) to other body areas, such as the lymph nodes, liver, bones, and bone marrow.

At Dana-Farber/Boston Children’s Cancer and Blood Disorder Center, we have the experience, expertise, and compassion necessary to treat very young children with complex conditions. The pediatric specialists in our Childhood Neuroblastoma Program use advanced therapies, including MIBG therapy, to provide the most effective treatment possible.

Causes and Symptoms of Childhood Neuroblastoma

Neuroblastoma likely develops from an early form of a nerve cell (called a neuroblast) found in the fetus. A genetic mutation inside the cell causes it to grow abnormally. Scientists continue to research the genetic abnormality to understand what causes the cancer.

In rare cases, an inherited alteration in a specific gene causes neuroblastoma. The gene most commonly affected is the ALK gene, a protein-coding gene. Hereditary neuroblastoma accounts for 1 to 2 percent of cases.

Neuroblastoma symptoms can vary greatly, depending on the tumor's size, location, and spread.

Symptoms of neuroblastoma may include:

  • 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.

How We Diagnose and Classify Childhood Neuroblastoma

Your child's doctor will perform several tests to diagnose and stage neuroblastoma, including a metaiodobenzylguanidine (MIBG) scan. This specialized diagnostic screening tool involves injecting a particular chemical that most neuroblastomas will absorb to show up on a scan. We may also use other advanced imaging studies.

After completing all diagnostic tests, our specialists meet to review and examine what they have learned about your child's condition. Your doctor will discuss their findings and recommended a treatment plan with you and your family.

Doctors use the International Neuroblastoma Staging System:

  • Stage I: Localized tumor that is entirely surgically removed at diagnosis.
  • Stage II: Localized tumor that cannot be entirely surgically removed at diagnosis. Lymph nodes on the same side of the tumor may have tumor cells present.
  • Stage III: Large tumor spread across the middle of the body, and surgical removal at diagnosis isn't possible.
  • Stage IV: Tumor of any size metastasized (spread) to distant lymph nodes, bone marrow, bone, or liver.
  • Stage IVs: Special stage that applies only to a child less than 1-year-old; small, localized tumor metastasized (spread) to liver, skin, or bone marrow.

During diagnosis, doctors also determine a child's neuroblastoma risk group, including:

  • Low-risk neuroblastoma: Patients with low-risk neuroblastoma usually require only surgery or observation. Occasionally, a patient with a low-risk tumor will need chemotherapy (or radiation treatment) to shrink a tumor before surgical removal.
  • Intermediate-risk neuroblastoma: Patients with intermediate-risk disease are treated with chemotherapy to shrink the tumor before surgical removal. Oncologists choose the medicines to minimize side effects, both during treatment and in the future.
  • High-risk neuroblastoma: High-risk neuroblastoma is an aggressive disease treated with a combination of therapies, including chemotherapy, surgery, radiation therapy, stem cell transplant, biologic therapy, MIBG therapy, and immunotherapy.

Together, this information helps your child’s medical team determine a specific treatment course for your child.

How We Treat Childhood Neuroblastoma

Our pediatric cancer experts treat neuroblastomas with the most advanced, effective therapies available.

Neuroblastoma treatment options include:

  • Surgery: Surgeons may remove the tumor either at diagnosis or after using chemotherapy or radiation to shrink the tumor.
  • Chemotherapy: Several chemotherapy medications are known to be effective in destroying neuroblastoma cells. We use chemotherapy to decrease the tumor's size, eliminate the cancer, and prevent further tumor spread.
  • Radiation therapy: We use radiation therapy to eliminate neuroblastoma cells left behind after chemotherapy 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: A procedure that temporarily removes 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. We infuse the child's stored healthy blood cells back into their body to promote normal body and organ function.
  • Biologic therapy: These therapies include antibody therapy and a medicine called isotretinoin, which takes advantage of what we have learned about neuroblastoma cells' biologic characteristics. The objective of biologic therapy is to eliminate any remaining microscopic tumor cells.
  • MIBG therapyIf your child has high-risk neuroblastoma (including relapsed or refractory neuroblastoma), he or she may receive a specialized treatment known as metaiodobenzylguanidine (MIBG) therapy. MIBG therapy, which uses a radioactive isotope readily absorbed by most neuroblastomas, can detect neuroblastoma in the body or deliver radiation to eliminate the neuroblastoma cells.
  • Immunotherapy: We use immunotherapy only for the treatment of high-risk neuroblastoma. Patients generally receive it combined with chemotherapy, using Unituxin (dinutuximab), an antibody that binds to the surface of neuroblastoma cells.

Beyond treatment, we continue to care for your child through our pediatric cancer survivorship programs. Services include annual visits to our clinic to manage disease complications and screen for recurrence, and counseling services ranging from psychological to nutritional support.

How We Treat Relapsed or Refractory Neuroblastoma

Approximately 50 percent of children with high-risk neuroblastoma will experience initial remission followed by cancer relapse.

Another 15 percent of children with high-risk neuroblastoma won’t respond to initial treatment. These children are said to have refractory neuroblastoma.

The treatment approach for children with refractory neuroblastoma is similar to that for children with relapsed neuroblastoma. In each of these cases, treatment is personalized for each patient, and may include participation in a clinical trial.

Childhood Cancer Clinical Trials

Find answers to common questions about clinical trials for childhood cancer, including whether or not a clinical trial may be the right choice for your child. Contact us and we can help you navigate your options.

Clinical Trials for Pediatric Patients

Research and Clinical Trials for Childhood Neuroblastoma

At Dana-Farber/Boston Children's, our physicians work continuously to translate laboratory findings into clinical therapies. We offer innovative clinical trials for children with all stages of neuroblastoma, including relapsed and refractory.

Our current research efforts focus on improving established neuroblastoma therapies, studying the disease's genetic causes, and developing novel therapies. Our research programs include:

  • Attacking the genetic cause: Our researchers have published some 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 improve neuroblastoma treatment.
  • Reducing late effects: Children with low- and intermediate-risk neuroblastoma often can be cured with surgery alone or surgery and chemotherapy combined. Because of the risk of physical, psychological, social, intellectual, and other late effects, we are continually working to decrease the intensity of treatment as much as possible while still providing the best cure rates.
  • Advancing neuroblastoma treatment: Recent developments in the treatment of neuroblastoma have included identifying 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 aggressive treatment and which tumors require little treatment beyond surgical removal.
  • Innovating radiation treatment for neuroblastoma: Nuclear medicine physicians and scientists are an integral part of the MIBG treatment for children with relapsed neuroblastoma. MIBG therapy is only available at a few major medical centers in North America. Dana-Farber/Boston Children's is the only center in New England to offer this therapy.
  • Examining stem cell transplantation: We are researching various uses of stem cells, including high-dose therapy combined with stem cell transplantation to treat neuroblastoma, and umbilical cord transplantation.

Long-term Outcomes for Children with Neuroblastoma

Your child's prognosis will depend on several different factors:

  • The extent of the disease
  • Size and location of the tumor
  • Tumor's characteristics when examined under a microscope
  • Presence or absence of metastasis
  • Tumor’s response to therapy
  • 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 challenging to treat and require more aggressive therapy. Prompt medical attention and appropriate treatment are essential for the best prognosis.

Childhood Neuroblastoma Treatment Team

The Dana-Farber/Boston Children's solid tumor treatment team includes some of the top pediatric cancer specialists worldwide. See a complete list of the specialists in our Childhood Solid Tumor Center.