Vedat Uygun1, Gülsün Karasu2, Hayriye Daloğlu3, Seda Öztürkmen3, Suar Çakı Kılıç4, Koray Yalçın4, Safiye Suna Çelen4, Volkan Hazar4, Akif Yeşilipek3. 1. İstinye University School of Medicine, MedicalPark Antalya Hospital, Department of Pediatric Bone Marrow Transplantation Unit, Antalya, Turkey. 2. İstinye University School of Medicine, MedicalPark Göztepe Hospital, Department of Pediatric Bone Marrow Transplantation Unit, İstanbul, Turkey. 3. MedicalPark Antalya Hospital, Department of Pediatric Bone Marrow Transplantation Unit, Antalya, Turkey. 4. MedicalPark Göztepe Hospital, Department of Pediatric Bone Marrow Transplantation Unit, İstanbul, Turkey.
Abstract
BACKGROUND: Despite 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. PROCEDURE: This 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. RESULTS: All 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. CONCLUSION: Steroid-refractory acute and chronic pediatric GVHD patients treated with ruxolitinib had a high overall response rate, with the additional benefit of steroid sparing.
BACKGROUND: Despite 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. PROCEDURE: This 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. RESULTS: All grade 2 acute GVHDpatients completely responded to ruxolitinib treatment; 82% of high-grade (3-4) acute GVHDpatients 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. CONCLUSION:Steroid-refractory acute and chronic pediatric GVHDpatients treated with ruxolitinib had a high overall response rate, with the additional benefit of steroid sparing.
Authors: Cong Wei; Xiaoting Zhang; Dan Liang; Jilong Yang; Jingwen Du; Chunyan Yue; Lan Deng Journal: Drug Des Devel Ther Date: 2021-11-30 Impact factor: 4.162
Authors: Allan R Glanville; Christian Benden; Anne Bergeron; Guang-Shing Cheng; Jens Gottlieb; Erika D Lease; Michael Perch; Jamie L Todd; Kirsten M Williams; Geert M Verleden Journal: ERJ Open Res Date: 2022-07-25