ORIGINAL RESEARCH article
Front. Immunol.
Sec. Alloimmunity and Transplantation
Volume 16 - 2025 | doi: 10.3389/fimmu.2025.1621708
Efficacy and safety of ruxolitinib combined with steroids for first-line treatment of acute graft-versus-host disease after hematopoietic stem cell transplantation: A single-center, real-world experience
Provisionally accepted
- 1Department of Hematology, Second Affiliated Hospital of Dalian Medical University, Dalian, China
- 2Department of Pharmacy, Second Affiliated Hospital of Dalian Medical University, Dalian, China
- 3Department of Hematology, Shanghai Ninth People’s Hospital Affiliated to Shanghai Jiaotong University School of Medicine, Shanghai, China
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Introduction:Despite the increasing use of allogeneic hematopoietic stem cell transplantation (allo-HSCT), graft-versus-host disease (GVHD) remains the main cause of morbidity and mortality, significantly impacting HSCT outcomes. Steroids are the standard first-line treatment for acute GVHD (aGVHD); however, standardized treatment algorithms for patients who do not respond to steroid therapy are lacking. Ruxolitinib is the most promising second-line therapy for steroidrefractory (SR)-GVHD, but data on its first-line use for aGVHD are limited.Methods:In this retrospective study, we analyzed the data of 133 patients with aGVHD who underwent transplantation at our institution. Eighty-three patients received ruxolitinib combined with methylprednisolone, while 50 received methylprednisolone alone as the initial treatment.The ruxolitinib/steroids group had a significantly higher overall response rate (ORR) on day 7 (86%) compared to the steroid-only group (68%; odds ratio [OR]=2.8, 95% confidence interval [CI]: 1.2-6.5, p=0.019). Similarly, ORR on day 14 was higher in the ruxolitinib/steroids group (92% vs. 79%; OR=2.7, 95% CI: 0.9-7.8, p=0.05). Although no statistical differences were observed in overall survival (OS), progression-free survival (PFS), and failure-free survival (FFS) between the two groups, patients who achieved early ORR on days 7 and 14 had better OS, PFS, and FFS. Additionally, in subgroup analysis of patients who underwent peripheral blood stem cell transplantations, the ruxolitinib/steroids cohort had significantly better OS (Hazard Ratio [HR]=0.34, 95% CI: 0.11-1.55, p=0.04), PFS (HR=0.37; 95% CI: 0.12-1.10, p=0.05) and FFS (HR=0.46; 95% CI: 0.19-1.11, p=0.05) compared to the steroid-only cohort. Adverse event (AEs) frequencies were comparable between groups, with the exception of neutropenia (32.5% vs. 12%, p=0.008) and CMV infection (34.9% vs. 18%, p=0.036), which were more frequent in the ruxolitinib/steroid group.Discussion：To the best of our knowledge, this is the first real-world study to demonstrate that adding ruxolitinib to a standard methylprednisolone regimen provides an effective and safe first-line treatment for aGVHD.
Keywords: graft-versus-host disease, Hematopoietic Stem Cell Transplantation, Ruxolitinib, Steroids, Leukemia
Received: 01 May 2025; Accepted: 27 Jun 2025.
Copyright: © 2025 Yan, Yang, Huo, Zhou, Zhijie, Huang, Wang, Fan and Ma. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence: Jinsong Yan, Department of Hematology, Second Affiliated Hospital of Dalian Medical University, Dalian, China
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