• Vedat Uygun, Gülsün Karasu, Hayriye Daloğlu, Seda Öztürkmen, Suar Çakı Kılıç, Koray Yalçın, Safiye Suna Çelen, Volkan Hazar, and Akif Yeşilipek.
• İstinye University School of Medicine, MedicalPark Antalya Hospital, Department of Pediatric Bone Marrow Transplantation Unit, Antalya, Turkey.
• Pediatr Blood Cancer. 2020 Apr 1; 67 (4): e28190.

BackgroundDespite the increasing performance of allogeneic hematopoietic cell transplantation over the last decades, graft-versus-host disease (GVHD) remains the main cause of morbidity and mortality. The efficacy of ruxolitinib against GVHD has been demonstrated in adult studies; however, very few studies have been conducted in children.ProcedureThis study aimed to evaluate the efficacy of ruxolitinib in 29 children with steroid-refractory acute or chronic GVHD. Twenty-five (87%) patients received at least three different immune modulator agents, including methylprednisolone, before initiating ruxolitinib.ResultsAll grade 2 acute GVHD patients completely responded to ruxolitinib treatment; 82% of high-grade (3-4) acute GVHD patients and 80% of chronic GVHD (moderate-severe) patients had at least a partial response. Of seven patients with bronchiolitis obliterans, five had a partial response after ruxolitinib. Of 29 patients, 22 were administered steroids at any time in the first month of acute GVHD or the first three months of chronic GVHD during ruxolitinib usage, which was significantly tapered by the end of the observation period.ConclusionSteroid-refractory acute and chronic pediatric GVHD patients treated with ruxolitinib had a high overall response rate, with the additional benefit of steroid sparing.© 2020 Wiley Periodicals, Inc.

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