Efficacy and safety of ruxolitinib for graft-versus-host disease prophylaxis in patients with aplastic anemia undergoing PBSC-only allogeneic stem cell transplantation: a prospective phase II study

作者：Xiaoyu Zhang, Lulu Pan, Yanhong Zhao, Runzhi Ma, Lining Zhang, YING ZHANG, Gang Li, Weihua Zhai, Qiaoling Ma, Aiming Pang, Donglin Yang, S. feng, Ping Zhang, Yi He, Guoyou Qin, Erlie Jiang, Mingzhe Han · 发表于：Experimental Hematology and Oncology · 年份：2026 · DOI：10.1186/s40164-026-00753-9 · 被引用次数：1 · 研究领域：Hematopoietic Stem Cell Transplantation、Platelet Disorders and Treatments、Blood transfusion and management

BACKGROUND: While peripheral blood hematopoietic stem cell transplantation (PB-HSCT) supports rapid engraftment and reduces graft failure risk in aplastic anemia (AA) patients, it compromised higher risk of acute graft-versus-host disease (aGVHD), highlighting the need for more effective prophylactic strategies. This phase II clinical trial was designed to assess the efficacy and safety of ruxolitinib, a JAK1/2 inhibitor, as part of GVHD prophylaxis regimen following PB-HSCT. METHODS: This open-label, single-arm, Phase II clinical trial (ClinicalTrials.gov: NCT05914714) enrolled patients with AA between June 2023 and December 2024. Ruxolitinib was initiated at the start of conditioning and continued for 3 months post-transplant at a dose of 5 mg twice daily. A historical control cohort receiving standard GVHD prophylaxis between January 2019 and May 2023 was included for comparison. To address baseline imbalances, propensity score-based inverse probability of treatment weighting (IPTW) was applied. The primary objective was the incidence of aGVHD six months after HSCT. Secondary endpoints included one-year overall survival (OS) and GVHD-free, failure-free survival (GFFS). Immune reconstitution-including T cells, B cells, NK cells-and levels of pro-inflammatory cytokines were also evaluated to explore potential mechanisms. RESULTS: A total of 82 patients were enrolled (ruxolitinib group n = 46, historical control cohort n = 36). Comparative analysis showed that ruxolitinib sig...