Department of Hematology, Zhujiang Hospital, Southern Medical University, Guangzhou, 510280, People's Republic of China.
Department of Hematology, Shanghai Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, 200011, People's Republic of China.
Drug Des Devel Ther. 2021 Nov 30;15:4875-4883. doi: 10.2147/DDDT.S338752. eCollection 2021.
Graft-versus-host disease (GVHD) is a main complication following allogeneic hematopoietic stem cell transplantation and is a leading cause of non-relapse-related death. Unsatisfactory response to standard first-line therapy with glucocorticoids is a predictor of a poor prognosis in patients with GVHD. Ruxolitinib is a selective Janus kinases 1/2 inhibitor which has been shown to control acute (a) and chronic (c) GVHD while maintaining graft-versus-tumor effects.
This study aims to evaluate the efficacy and safety of ruxolitinib in the treatment of steroid-refractory GVHD (SR-GVHD) in a population of Chinese patients.
We report the results of 55 patients, including 23 patients with aGVHD and 32 patients with cGVHD, who were treated with ruxolitinib as salvage therapy between August, 2017 and December, 2020.
In patients with aGVHD, the overall response rate (ORR) was 86.9%, and the 1-year overall survival (OS) was 82.6% (95% CI, 67.1-98.1%). The 1-year OS was significantly improved in responders than in non-responders (90.0% vs 33.3%, P=0.004). In patients with cGVHD, the ORR was 78.1%, and the 1-year OS was 81.3% (95% CI, 67.8-94.8%). There was no significant difference in the 1-year OS between responders and non-responders (84.0% vs 71.4%, P=0.327). Cytopenia, cytomegalovirus-reactivation and infections were common adverse events, particularly in patients with aGVHD.
Our real-world data from Chinese patients further confirm that ruxolitinib is a safe and effective treatment for SR-GVHD.
移植物抗宿主病（GVHD）是异基因造血干细胞移植后的主要并发症，也是非复发相关死亡的主要原因。对糖皮质激素标准一线治疗反应不佳是 GVHD 患者预后不良的预测因素。鲁索利替尼是一种选择性的 Janus 激酶 1/2 抑制剂，已被证明可控制急性（a）和慢性（c）GVHD，同时保持移植物抗肿瘤效应。
本研究旨在评估鲁索利替尼治疗中国患者类固醇难治性 GVHD（SR-GVHD）的疗效和安全性。
我们报告了 55 例患者的结果，其中 23 例为急性 GVHD，32 例为慢性 GVHD，他们在 2017 年 8 月至 2020 年 12 月期间接受了鲁索利替尼作为挽救治疗。
在急性 GVHD 患者中，总体缓解率（ORR）为 86.9%，1 年总生存率（OS）为 82.6%（95%CI，67.1-98.1%）。与无反应者相比，反应者的 1 年 OS 显著提高（90.0%vs33.3%，P=0.004）。在慢性 GVHD 患者中，ORR 为 78.1%，1 年 OS 为 81.3%（95%CI，67.8-94.8%）。反应者与无反应者的 1 年 OS 无显著差异（84.0%vs71.4%，P=0.327）。血细胞减少症、巨细胞病毒再激活和感染是常见的不良事件，尤其是在急性 GVHD 患者中。
我们来自中国患者的真实世界数据进一步证实，鲁索利替尼是治疗 SR-GVHD 的一种安全有效的治疗方法。
