Effectiveness and safety of belumosudil in 20 patients with chronic graft-versus-host disease
10.3760/cma.j.cn121090-20241017-00401
- VernacularTitle:贝舒地尔治疗重度慢性移植物抗宿主病20例的疗效与安全性
- Author:
Zhi WANG
1
;
Jianhua YOU
;
Wenting CHEN
;
Tingting XING
;
Yi LUO
;
Xiaodong MO
;
Jiong HU
Author Information
1. 上海交通大学医学院附属瑞金医院海南医院,琼海 571400
- Publication Type:Journal Article
- Keywords:
Belumosudil;
ROCK2 Inhibitor;
Chronic graft-versus-host disease;
Allo-HSCT
- From:
Chinese Journal of Hematology
2025;46(8):743-749
- CountryChina
- Language:Chinese
-
Abstract:
Objective:To evaluate the effectiveness and safety of belumosudil for the treatment of chronic graft-versus-host disease (cGVHD) .Methods:We retrospectively collected data on patients with cGVHD who received belumosudil at Ruijin Hospital Affiliated to Shanghai Jiaotong University School of Medicine from May 2023 to March 2024. The study endpoints were overall response rate (ORR), organ-specific response rates, time to response (TTR), changes in Lee Symptom Scale (LSS) scores, tapering or discontinuation of corticosteroid treatment, failure-free survival (FFS), and adverse events.Results:The study included 20 patients with cGVHD who received belumosudil, of whom 15 were men and 5 women. The median age was 34.5 (12-67) years, and three patients were under 18 years old. The median follow-up duration was 5.0 (1.4 - 9.8) months. All patients had severe cGVHD, and 18 (90.0%) showed involvement of at least four organs. The median number of prior treatment lines was 4, and 15 patients (75%) had previously received ruxolitinib. All patients received 200 mg of belumosudil once daily in combination with other cGVHD systemic therapies. The ORR was 90.0% (95% CI: 68.3%-98.8%), and all responses were partial responses. The median TTR was 1.6 (0.9 - 8.4) months. The LSS scores improved in a clinically meaningful way in 80.0% (16/20) of the patients within 3 months. The corticosteroid dose was reduced in 42.6% (6/14) of the patients. The 3-month FFS was 79.6% (95% CI: 61.4%-100.0%). Most adverse events were grade 1 or grade 2, and two patients (10.0%) experienced grade 3 or higher-grade adverse events. Conclusions:In the real-world setting, belumosudil demonstrated good effectiveness and safety in patients with cGVHD with a history of severe disease and multiorgan involvement.