氯法拉滨论文.pdfVIP

Pediatr Blood Cancer 2014;61:479–487 Clofarabine Salvage Therapy in Refractory Multifocal Histiocytic Disorders, Including Langerhans Cell Histiocytosis, Juvenile Xanthogranuloma and Rosai–Dorfman Disease 1 2 2 1 3 Stephen J. Simko, MD, * Huy D. Tran, MD, Jeremy Jones, MD, Mrinalini Bilgi, MS, Lynda Kwon Beaupin, MD, Don Coulter, MD,4 Timothy Garrington, MD,5 Timothy L. McCavit, MD,6 Colin Moore, MD,5 Francisco Rivera-Ortego n, MD,7 Linda Shaffer, MD,8 Linda Stork, MD,9 Lucie Turcotte, MD,10 Esperanza C. Welsh, MD,11 M. John Hicks, MD, PhD, DDS,12 Kenneth L. McClain, MD, PhD,1 and Carl E. Allen, MD, PhD1 Background. Existing therapies for recurrent or refractory surviving patients showed demonstrable improvement after two to histiocytoses, including Langerhans cell histiocytosis (LCH), juvenile four cycles of therapy, with 11 (61%) complete responses, 4 (22%) xanthogranuloma (JXG), and Rosai–Dorfman disease (RDD), have partial responses, and 2 patients still receiving therapy. Five patients limited effectiveness. We report our experience with using experienced disease recurrence, but three of these subsequently clofarabine as therapy in children with recurrent or refractory achieved complete remission. All patients with JXG and RDD had histiocytic disorders, including LCH (11 patients), systemic JXG (4 complete or partial response at conclusion of therapy. Side effects patients), and RDD (3 patients). Methods. Patients treated with included neutropenia in all patients. Recurring but sporadic toxicities