{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,19]],"date-time":"2026-09-19T15:30:02Z","timestamp":1789831802046,"version":"4.0.1"},"reference-count":34,"publisher":"Frontiers Media SA","license":[{"start":{"date-parts":[[2025,7,14]],"date-time":"2025-07-14T00:00:00Z","timestamp":1752451200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["frontiersin.org"],"crossmark-restriction":true},"short-container-title":["Front. Immunol."],"abstract":"<jats:sec><jats:title>Introduction<\/jats:title><jats:p>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 steroid-refractory (SR)-GVHD, but data on its first-line use for aGVHD are limited.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>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.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>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\u20136.5, 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\u20137.8, 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\u20131.55, p=0.04), PFS (HR=0.37; 95% CI: 0.12\u20131.10, p=0.05) and FFS (HR=0.46; 95% CI: 0.19\u20131.11, 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.<\/jats:p><\/jats:sec><jats:sec><jats:title>Discussion<\/jats:title><jats:p>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.<\/jats:p><\/jats:sec>","DOI":"10.3389\/fimmu.2025.1621708","type":"journal-article","created":{"date-parts":[[2025,7,14]],"date-time":"2025-07-14T04:10:37Z","timestamp":1752466237000},"update-policy":"https:\/\/doi.org\/10.3389\/crossmark-policy","source":"Crossref","is-referenced-by-count":4,"title":["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, 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