What is neuroblastoma?

Neuroblastoma is a tumor of nerve tissue that develops in infants and children and can occur in many areas of the body. It develops from the tissues that form the sympathetic nervous system (which controls body functions such as heart rate and blood pressure, digestion, and levels of certain hormones). It most commonly begins in the abdomen in the tissues of the adrenal gland, but it may also occur in other areas. Neuroblastoma can spread to the lymph nodes, liver, bones and bone marrow.

Doctors at Children's Hospital of Philadelphia (CHOP) have discovered many of the causes of neuroblastomas. These discoveries will help in the development of new and better neuroblastoma treatments. Genetic testing for children with neuroblastoma is also available at CHOP, where there appears to be a family history of the disease.

Treatment for neuroblastoma

Treatment for newly diagnosed neuroblastoma

When deciding on your child's neuroblastoma treatment plan, we start by determining whether the disease risk is low, intermediate or high.

• Low-risk disease. If your child is younger than 1 year old at diagnosis, or has small, easily removed tumors, the child may only need a simple surgical procedure to treat neuroblastoma or careful follow-up.
• Intermediate-risk disease. If your child has a larger tumor that has not spread, or is a baby with disease that has spread (but does not have the MYCN abnormality), we will probably treat the child's neuroblastoma with surgery and four to eight months of chemotherapy. Often, patients with intermediate-risk disease do not need radiation treatments.
• High-risk disease. About half of neuroblastoma patients have high-risk disease, either because the tumor has spread to other parts of the body or because test results show high-risk features, such as MYCN amplification. If your child has high-risk neuroblastoma, treatment includes five months of chemotherapy, surgery, radiation therapy including proton therapy, high-dose chemotherapy followed by a stem cell rescue, and immunotherapy combined with biological therapy. These very intense treatments have improved the cure rate of this complex disease and Children's Hospital has led the efforts to develop these modern treatment approaches.

Newly diagnosed patients are generally treated according to Children's Oncology Group protocols, many of which were developed by CHOP doctors. However, every child will receive individualized treatment planning and other treatments may be recommended.

Surgery

For most children with neuroblastoma, treatment includes surgery, sometimes just for a biopsy at first, then eventually for removal of the tumor. Although some neuroblastomas can be safely removed early in the course of therapy — which in very rare cases might be the only treatment necessary — the vast majority of neuroblastomas have a very rich blood supply and tend to wrap around vital structures (such as major blood vessels), making it unsafe to try to remove them early in the course of treatment.

After a course of chemotherapy — and depending on the results of the MRI or CT scan — most patients with neuroblastoma will then proceed to surgery. The goal of the operation is to remove as much of the tumor as possible but also to avoid injury to all important organs and anatomic structures.

Because these tumors tend to wrap themselves around delicate anatomic structures, neuroblastoma resection can be a long and difficult operation. At CHOP, you can be assured that your child’s surgeon will be an expert at performing these very challenging operations, and they will always do everything possible to keep your child safe from harm. It is also important to remember that not all neuroblastomas can or need to be removed completely to achieve a potential cure. Removing most of the tumor (debulking) is sometimes all that can be safely accomplished, but may also be all the patient needs.

Treating relapsed or refractory neuroblastoma

Unfortunately, high-risk neuroblastoma still has a very high rate of non-responsiveness, or recurring/relapsing during or after treatment. We offer many innovative options to treat children whose disease has relapsed or is refractory, through the Refractory Neuroblastoma Program at Children's Hospital of Philadelphia.