Neuroblastoma
Treatment

Treatment options are related to the age of your child, tumor location, stage of disease, regional lymph node involvement and tumor biology. More than one method of treatment may be used, depending on your child's needs.

Surgery

Surgery is used when possible to remove as much of the cancer as possible. If the cancer can't be removed, surgery may be limited to a biopsy.

Radiation Therapy

Radiation therapy uses high-energy rays to damage or kill cancer cells and shrink tumors. Radiation usually comes from a machine outside the body called external beam radiation therapy.

Chemotherapy

Chemotherapy is the use of drugs to kill cancer cells and shrink tumors. Chemotherapy drugs may be taken orally or injected into a vein or a muscle. Chemotherapy is called a systemic treatment because the drug enters the bloodstream, travels through the body and can kill cancer cells throughout the body. Chemotherapy may be given after the tumor has been surgically removed to kill any remaining cancer cells. This is called adjuvant chemotherapy. Chemotherapy also can be used to shrink the tumor before surgery. This is called neoadjuvant chemotherapy.

Bone Marrow Transplant

Bone marrow transplantation is a procedure in which healthy bone marrow is given to replace bone marrow destroyed by anti-cancer drugs or radiation. At UCSF Benioff Children's Hospital, the Bone & Marrow Transplant Program offers autologous bone marrow stem cell transplants to treat children with high-risk neuroblastoma. In this type of transplant, high-dose chemotherapy is given to kill the tumor. Bone marrow function is restored by infusing the patient's own bone marrow cells, which were previously removed and stored.

MIBG Therapy

UCSF Benioff Children's Hospital is one of the few centers in the country to offer MIBG therapy for high-risk neuroblastomas that have not responded to chemotherapy, or have recurred after treatment. MIBG is a chemical that mimics certain natural compounds in the body that are concentrated in selective tissues, including neuroblastoma cells. MIBG can be attached to radioactive forms of iodine and given as an IV treatment.

Doctors at UCSF led a clinical trial of MIBG treatment for patients with resistant neuroblastoma. This treatment led to decreases in tumor size or number of tumor spots in approximately one-third of patients, making MIBG one of the most active drugs for these patients. In some patients, all of the tumor spots went away for a time.

MIBG therapy is only offered by entering a study or clinical trial because it is not yet FDA approved; UCSF conducts several MIBG therapy studies for the treatment of neuroblastoma and pheochromocytoma. The study a child enters will depend upon a number of factors, including open studies and their prior treatment. To search for a clinical trial, go to the UCSF Benioff Children's Hospital Clinical Trials page.

One of the major side effects of MIBG treatment is low blood counts, or bone marrow suppression. Giving a dose of the patient's own stem cells can help to improve blood counts after MIBG treatment.

Sometimes MIBG is combined with chemotherapy or other medications. This may make neuroblastoma cells more sensitive to MIBG treatment. Your doctor will decide whether your child should be treated with MIBG alone or in combination with other medications. To learn more about MIGB therapy, go to mibg.ucsf.edu.

Intraoperative Radiation Therapy (IORT)

Radiation oncologists at UCSF have been pioneering the use of intraoperative radiation therapy (IORT) for some of the most difficult cases of neuroblastoma. UCSF is one of the few medical centers nationwide at the forefront of advancing this mode of treatment.

IORT delivers a concentrated beam of radiation to cancerous tumors while they are exposed during surgery. This allows high doses of radiation to be administered to tumors without exposing nearby healthy organs to radiation, lessening the risk that healthy tissue will be damaged.

Another advantage of IORT is that patients do not have to return for successive rounds of therapy after surgery. Those who receive the most common standard alternative, external beam radiation therapy (EBRT), typically receive treatment five days a week for several weeks. A single dose of IORT may have as much effect on the tumor as 10 to 20 daily radiation treatments.

Prior to IORT, oncologists treated the most aggressive cases of neuroblastoma with surgery, high doses of chemotherapy and some form of radiation. These high-dose treatments kill tumor cells more effectively. But the treatments also result in toxicities in many normal tissues, such as the death of bone marrow cells that make blood and immune cells. Thanks to these high-dose combinations of radiotherapy and chemotherapy, survival among neuroblastoma patients has improved greatly over the past two decades. Even so, among the 45 percent with high-risk tumors, the worst prognosis, only about one in three lives free of cancer regrowth for three years or more.

Treatment by Category

As described in the diagnosis section, neuroblastoma is categorized as localized resected, localized unresected, regional, disseminated and special.

Localized Resectable Neuroblastoma

Your child's treatment may be one of the following:

  • Surgery to remove the cancer
  • Surgery plus adjuvant chemotherapy
  • Surgery plus radiation therapy

Localized Unresectable Neuroblastoma

Initial treatment generally consists of surgery to remove as much of the cancer as possible, followed by chemotherapy. A second surgery may be performed to remove any cancer that remains. Radiation therapy also may be prescribed.

Regional Neuroblastoma

Treatment depends on your child's age. If your child is younger than 1 year of age, treatment may include the following:

  • Surgery to remove the cancer
  • Chemotherapy

If your child is older than 1 year of age, treatment may be one of the following:

  • Surgery to remove the cancer
  • Surgery followed by chemotherapy
  • Chemotherapy with or without radiation therapy to reduce the tumor, followed by surgery
  • Multi-drug chemotherapy
  • Radiation therapy
  • New methods of treatment in a clinical trial or study

Disseminated Neuroblastoma

Your child's treatment may be one of the following:

  • Multi-drug chemotherapy with or without surgery and/or radiation therapy
  • Chemotherapy followed by peripheral stem cell transplantation and 13-cis retinoic acid
  • New methods of treatment in a clinical trial or study

Stage 4S Neuroblastoma

Children with this special type of neuroblastoma may not require therapy. You may want to have your child take part in a clinical trial that provides new methods of treatment.

Recurrent Neuroblastoma

The selection of treatment for recurrent or progressive neuroblastoma depends on the location and extent of the recurrence and previous therapy as well as individual circumstances. A clinical trial may be appropriate. UCSF Benioff Children's Hospital has available national and local studies for recurrent neuroblastoma, including new therapies with targeted radiation (I-131-MIBG), Phase I trials of the New Approaches to Neuroblastoma Therapy (NANT) consortium and Phase I and II trials of the Children's Oncology Group.

Reviewed by health care specialists at UCSF Benioff Children's Hospital.

Related Information

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