Department of Clinical Hematology, Armed Forces Bone Marrow Transplant Center (AFBMTC), Rawalpindi, Pakistan.
J Coll Physicians Surg Pak. 2022 Sep;32(9):1225-1227. doi: 10.29271/jcpsp.2022.09.1225.
The objective of this study was to evaluate the role of Ruxolitinib in steroid-refractory graft versus host disease. This retrospective descriptive study was conducted from January 2018 to December 2021. A total of 157 patients underwent allogeneic stem cell transplants during the study period. Of these, 20 patients having steroid-refractory GVHD treated with Ruxolitinib were selected for the study. The primary endpoint was the overall response rate to Ruxolitinib measured at 4 weeks and 24 weeks for acute and chronic GVHD, respectively. The secondary endpoints were overall survival and failure-free survival. Of these 20 patients, 7 (35%) had acute GVHD, and 13 (65%) had chronic GVHD. Of acute GVHD, 2 (10%) had grade II, 4 (20%) had grade III, and 1 (5%) had grade IV acute GVHD. Of 13 patients with chronic GVHD, 7 (35%) had moderate and 6 (30%) had severe chronic GVHD. In steroid-refractory acute GVHD, the overall response rate to Ruxolitinib was 85.7%, and in chronic GVHD, it was 84.6%. The failure-free survival was 80% and overall survival was 85%. Adverse events of any grade occurred in 16 (80%) patients with grade III/IV adverse events in 4 (20%) patients only. The study showed that Ruxolitinib is a safe and effective second-line therapy for acute and chronic steroid-refractory GVHD. Key Words: Ruxolitinib, GVHD, Allogeneic stem cell transplant.
本研究旨在评估芦可替尼在激素难治性移植物抗宿主病（GVHD）中的作用。这是一项回顾性描述性研究，于 2018 年 1 月至 2021 年 12 月进行。在研究期间，共有 157 名患者接受了异基因干细胞移植。其中，20 名接受芦可替尼治疗的激素难治性 GVHD 患者被选入本研究。主要终点是分别在 4 周和 24 周时评估急性和慢性 GVHD 的芦可替尼总体缓解率。次要终点是总生存率和无失败生存率。在这 20 名患者中，7 名（35%）患有急性 GVHD，13 名（65%）患有慢性 GVHD。在急性 GVHD 中，2 名（10%）患者为 II 级，4 名（20%）患者为 III 级，1 名（5%）患者为 IV 级急性 GVHD。在 13 名患有慢性 GVHD 的患者中，7 名（35%）患者为中度，6 名（30%）患者为重度慢性 GVHD。在激素难治性急性 GVHD 中，芦可替尼的总体缓解率为 85.7%，在慢性 GVHD 中为 84.6%。无失败生存率为 80%，总生存率为 85%。16 名（80%）患者出现任何级别的不良反应，仅 4 名（20%）患者出现 3/4 级不良反应。该研究表明，芦可替尼是激素难治性急性和慢性 GVHD 的一种安全有效的二线治疗方法。关键词：芦可替尼；GVHD；异基因干细胞移植。
