Bevacizumab and irinotecan in the treatment of children with recurrent/refractory medulloblastoma.

Relapsed/refractory medulloblastoma (MB) has a poor outcome regardless of the treatment employed. Novel therapies are needed in an effort to improve survivals. We present two children with recurrent/refractory MB treated with bevacizumab and irinotecan both given every 2 weeks. One patient also received temozolamide. The first patient had stable disease and remains without progression after 30 months. The second patient had a near complete response that was sustained for 18 months. The regimen was well tolerated with minimal toxicity and provided prolonged progression-free survival in these two patients. Prospective clinical trials are needed to evaluate the effectiveness of this strategy.