High-Risk Neuroblastoma Treatment Options

What is high-risk neuroblastoma?

Neuroblastoma is an aggressive solid tumor that starts in immature nerve cells. It is one of the most common types of solid tumors in children. This condition is categorized into three risk levels – low, intermediate and high. The category a tumor is placed into is based on factors like tumor size and spread, and the status of the MYCN gene, a gene that plays a role in the development and growth of cells.

Children with low- and intermediate-risk neuroblastoma can often be cured with such treatments as surgery, chemotherapy and radiation therapy. High-risk neuroblastoma is harder to cure and children usually require more intensive treatments to increase their chances of a successful cure.

Treatment for high-risk neuroblastoma

If surgery isn't successful, or if it isn't an option, UCSF Benioff Children's Hospitals offers other forms of treatment for patients with high-risk neuroblastoma. These treatments include autologous bone marrow transplant (BMT) and MIBG therapy.

Autologous bone marrow transplant (BMT)

This treatment involves collecting healthy bone marrow cells from a patient before they receive high-dose chemotherapy or radiation therapy. After these treatments, the healthy cells are returned to their body to help restore function in their bone marrow and immune system. Transplanting the child's own stem cells is less risky than transplanting stem cells from a donor.

Because most children with high-risk neuroblastoma have cancer cells in their bone marrow, we use special techniques to remove the cancer cells from the healthy cells before the transplant. The purified cells are safely stored until the day of the transplant.

Before the treatment, patients need to go through a conditioning regimen designed to help the transplant succeed.

We also offer experimental procedures for children who didn't respond to initial chemotherapy treatments or who have relapsed after an initial response. These patients aren't candidates for the standard autologous transplant but may be eligible for targeted radiation therapy followed by an autologous BMT.

MIBG therapy

This treatment is aimed at high-risk neuroblastomas that haven't responded to chemotherapy or have returned after treatment. UCSF Benioff Children's Hospitals is one of the few centers in the U.S. that offers MIBG therapy. It's only offered to patients through a clinical trial -- one of our many clinical trials for neuroblastoma.

MIBG is a compound that mimics certain natural substances in the body found in specific tissues, including neuroblastoma cells. It is a clear liquid given through an IV tube in the arm or hand. The IV treatment itself takes around two hours, but your child will stay in the hospital for about six to eight days.

Most patients don't experience significant side effects, but they are considered radioactive for four to five days and must be partially isolated in a special room outfitted with lead shields. Parents are allowed daily contact with their child for some amount of time each day, depending on the amount of radiation detected.

The most difficult part of the process for many kids is the boredom that goes with lying in bed for several days. Video games and other electronic devices are allowed, and the nurses and other staff will work with you to make sure your child is as comfortable as possible.

One of the main side effects of MIBG treatment is low blood counts or bone marrow suppression. To manage this, your child may receive a dose of their own stem cells using an analogous BMT to help improve their blood counts after treatment.

At times, MIBG is paired with chemotherapy or other medications to help fight the neuroblastoma cells. If your child is eligible for MIBG therapy, you doctor will decide whether your child should undergo it alone or in combination with other treatments.