Pediatric Neuroblastoma: Treatment with Oral Irinotecan and Temozolomide

Abstract

New strategies are needed to improve outcomes for children with high-risk neuroblastoma, as fewer than half of these patients are expected to survive with current therapies. The combination of temozolomide and irinotecan has recently been evaluated in cooperative group studies for patients with relapsed high-risk neuroblastoma. This combination is attractive given the reported single-agent activity of each drug, their non-overlapping toxicity profiles, and the preclinical evidence of synergy in mouse models of neuroblastoma. Clinical trials involving 120 patients with relapsed or refractory neuroblastoma have shown the combination to produce responses or sustained disease stabilization in a significant subset of patients. In addition, the tolerability of the regimen may allow it to serve as a therapeutic backbone on which to add targeted agents. Finally, use of orally administered irinotecan given on shorter treatment schedules can reduce costs and improve patient convenience.