Introduction 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. Methods 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. Results A total of 12 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 ( I 2 = 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. Discussion Ruxolitinib as GVHD prophylaxis significantly reduces acute GVHD severity and maintains favorable survival outcomes, likely due to Janus kinase and signal transducer and activator (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.
Xie et al. (2025) studied this question.