İstinye University School of Medicine, MedicalPark Antalya Hospital, Department of Pediatric Bone Marrow Transplantation Unit, Antalya, Turkey.
İstinye University School of Medicine, MedicalPark Göztepe Hospital, Department of Pediatric Bone Marrow Transplantation Unit, İstanbul, Turkey.
Pediatr Blood Cancer. 2020 Apr;67(4):e28190. doi: 10.1002/pbc.28190. Epub 2020 Jan 25.
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.
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.
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.
Steroid-refractory acute and chronic pediatric GVHD patients treated with ruxolitinib had a high overall response rate, with the additional benefit of steroid sparing.
尽管过去几十年来同种异体造血细胞移植的疗效不断提高，但移植物抗宿主病（GVHD）仍然是发病率和死亡率的主要原因。鲁索利替尼治疗 GVHD 的疗效已在成人研究中得到证实；然而，在儿童中进行的研究很少。
本研究旨在评估鲁索利替尼在 29 例类固醇难治性急性或慢性 GVHD 儿童中的疗效。在开始使用鲁索利替尼之前，25 名（87%）患者接受了至少三种不同的免疫调节剂药物治疗，包括甲基强的松龙。
所有 2 级急性 GVHD 患者均对鲁索利替尼治疗完全有反应；82%的 3-4 级急性 GVHD 患者和 80%的慢性 GVHD（中重度）患者至少有部分反应。在 7 例闭塞性细支气管炎患者中，有 5 例在鲁索利替尼使用期间的急性 GVHD 第一个月或慢性 GVHD 的前三个月内出现部分反应。在 29 例患者中，有 22 例在急性 GVHD 的第一个月或慢性 GVHD 的前三个月内的任何时间使用类固醇，在观察期结束时类固醇显著减少。
用鲁索利替尼治疗的类固醇难治性急性和慢性儿科 GVHD 患者总体反应率高，并有类固醇节约的额外益处。
