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 therapy: If 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 https://www.dana-farber.org/pediatric-survivorship-programs/ 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.