Reduced Chemotherapy Regimen May Be Effective in Neuroblastoma

According to results of a Phase III clinical trial, patients with intermediate-risk neuroblastoma may be effectively treated with reduced doses of chemotherapy given over a shorter period. These findings were published in the New England Journal of Medicine.

Neuroblastoma is a disease in which cancerous cells form in the nerve tissues of the adrenal gland, neck, chest, or spinal cord. Although neuroblastoma is rare (it affects roughly 650 children and adolescents in the United States each year), it is the most common malignancy diagnosed in infants. Based on the age of the patient and the specific characteristics of the cancer, neuroblastoma is classified as low-, intermediate-, or high-risk. Patients classified as low-risk have a significantly better outcome compared with those classified as high-risk.

Treatment of intermediate-risk neuroblastoma with dose-intensive chemotherapy is generally highly effective. It’s not known, however, whether excellent outcomes can still be achieved with decreased doses of chemotherapy that are administered for shorter periods.

To explore the survival rate among intermediate-risk neuroblastoma patients treated with reduced chemotherapy, researchers evaluated 479 patients. Specifically, they wanted to determine if a survival rate of more than 90% was possible with a reduced regimen.

Three-year overall survival for all patients was 96% percent. For patients with tumors with favorable biologic characteristics (factors associated with favorable outcomes), overall survival was 98%, and overall survival among those with unfavorable biologic characteristics was 93%.

The researchers concluded that the survival rate was high among patients with intermediate-risk neuroblastoma who received “substantially” reduced chemotherapy compared with regimens used in earlier trials. These findings may allow for reduction of chemotherapy among neuroblastoma patients in appropriate risk groups.

Reference: Baker DL, Schmidt ML, Cohn SL, et al. Outcome after reduced chemotherapy for intermediate-risk neuroblastoma. New England Journal of Medicine. 2010; 363:1313-1323. September 30, 2010.

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