SYSTEMATIC REVIEW article
Front. Med.
Sec. Hematology
Efficacy and Safety of Ruxolitinib for Graft Versus Host Disease Prophylaxis in Allogeneic Hematopoietic Stem Cell Transplantation: A Systematic Review and Meta-Analysis
Provisionally accepted
- First Affiliated Hospital, Guangxi Medical University, Nanning, China
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Graft-versus-host disease (GVHD) remains a major complication after allogeneic hematopoietic stem cell transplantation (allo-HSCT). Despite standard prophylaxis, acute and chronic GVHD incidence remains high. Ruxolitinib, a Janus kinase inhibitor (JAK1/2), shows promise in treating steroid-refractory GVHD. However, its efficacy and safety as an adjunct to standard prophylactic regimens remain subjects of debate. This meta-analysis aims to evaluate the efficacy and safety of ruxolitinib when used as an adjunct to GVHD prophylaxis following allo-HSCT. Comprehensive studies were searched in PubMed, Web of Science, Embase, Cochrane Library, and ClinicalTrials.gov. Outcomes measures included the incidence rates of acute/chronic GVHD, overall survival (OS), cytomegalovirus (CMV) and Epstein-Barr virus (EBV) reactivation events. Pooled proportions with 95% confidence intervals (CIs) were calculated using random/fixed-effects models. Twelve studies including 406 patients were analyzed. Most of these studies were non-randomized. The pooled incidence of grade II–IV and III–IV acute GVHD was 10.4% (95% CI: 7.3–13.5%) and 2.9% (0.6–5.2%), respectively, with no heterogeneity (I²=0%). Chronic GVHD occurred in 26.8% (19.2–34.4%). One- and two-year OS rates were 86.6% (78.8–94.5%) and 81.2% (68.2–94.2%). CMV and EBV reactivation rates were 30.6% (14.6–46.6%) and 19.0% (0.4–37.7%), respectively. Ruxolitinib as GVHD prophylaxis significantly reduces acute GVHD severity and maintains favorable survival outcomes, likely due to JAK-STAT pathway inhibition. However, elevated CMV/EBV reactivation rates the need for vigilant monitoring. These findings support ruxolitinib’s role as a promising adjunct in GVHD prevention, warranting further randomized trials to confirm long-term safety and efficacy.
Keywords: Ruxolitinib, GvHD, prophylaxis, Allo-HSCT, Meta-analysis
Received: 07 Aug 2025; Accepted: 28 Oct 2025.
Copyright: © 2025 Xie, Shi, Wei, Lai and Liu. 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: Rongrong Liu, rong2liu@hotmail.com
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