Clofarabine salvage therapy for refractory high-risk langerhans cell histiocytosis

Abstract Pediatric patients with refractory multisystem Langerhans cell histiocytosis (LCH) have a poor prognosis despite aggressive chemotherapy. Salvage therapy with cytarabine and cladribine has shown promise as an effective treatment but is associated with significant toxicity. A previous report described two patients with refractory LCH who had a rapid response to single‐agent clofarabine with minimal toxicity. In this report, we describe four children with refractory, risk‐organ‐positive LCH who were treated with clofarabine and provide follow‐up for the two previously reported cases. The results support development of a formal trial evaluating clofarabine as front‐line salvage for refractory LCH. Pediatr Blood Cancer 2013; 60: E19–E22. © 2012 Wiley Periodicals, Inc.