University of Pennsylvania Cancer Center
Last Modified: May 18, 1998
Neuroblastoma, a malignant solid tumor which arises from the peripheral nervous system, is one of the most aggressive of childhood cancers. Five hundred new neuroblastoma cases are diagnosed each year in the U.S.
539 high-risk neuroblastoma patients (85% stage IV) were treated in this study, the first randomized study to show that 13-cis-retinoic acid, a vitamin A derivative, is effective in treating neuroblastoma in patients with a poor survival prognosis. When used after either chemotherapy or intensive chemotherapy supported by autologous bone marrow transplantation, the drug significantly increased disease-free survival in patients with this difficult to treat cancer, and had low toxicity. For patients treated with bone marrow transplantation and 13-cis-retinoic acid, the three-year disease-free survival from time of starting therapy wit the vitamin A derivative was 55%, compared to only 13% for patients receiving chemotherapy alone.