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Deprecated: Implicit conversion from float 300.79999999999995 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Pediatr+Blood+Cancer 2014 ; 61 (3): 479-87 Nephropedia Template TP
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Clofarabine salvage therapy in refractory multifocal histiocytic disorders, including Langerhans cell histiocytosis, juvenile xanthogranuloma and Rosai-Dorfman disease #MMPMID24106153
Simko SJ; Tran HD; Jones J; Bilgi M; Beaupin LK; Coulter D; Garrington T; McCavit TL; Moore C; Rivera-Ortegón F; Shaffer L; Stork L; Turcotte L; Welsh EC; Hicks MJ; McClain KL; Allen CE
Pediatr Blood Cancer 2014[Mar]; 61 (3): 479-87 PMID24106153show ga
Background: Existing therapies for recurrent or refractory histiocytoses, including Langerhans cell histiocytosis (LCH), juvenile xanthogranuloma (JXG), and Rosai-Dorfman disease (RDD), have limited effectiveness. We report our experience with using clofarabine as therapy in children with recurrent or refractory histiocytic disorders, including LCH (11 patients), systemic JXG (4 patients), and RDD (3 patients). Methods: Patients treated with clofarabine for LCH, JXG, or RDD by Texas Children?s Hospital physicians or collaborators between May 2011 and January 2013 were reviewed for response and toxicity. Results: Patients were treated with a median of 3 chemotherapeutic regimens prior to clofarabine. Clofarabine was typically administered at 25 mg/m2/day for five days. Cycles were administered every 28 days for a median of six cycles (range: 2?8 cycles). Seventeen of eighteen patients are alive. All surviving patients showed demonstrable improvement after 2?4 cycles of therapy, with eleven (61%) complete responses, four (22%) partial responses, and two patients still receiving therapy. Five patients experienced disease recurrence, but three of these subsequently achieved complete remission. All patients with JXG and RDD had complete or partial response at conclusion of therapy. Side effects included neutropenia in all patients. Recurring but sporadic toxicities included prolonged neutropenia, severe vomiting, and bacterial infections. Conclusion: Clofarabine has activity against LCH, JXG, and RDD in heavily pretreated patients, but prospective multi-center trials are warranted to determine long-term efficacy, optimal dosing, and late toxicity of clofarabine in this population.