Xenopax for the treatment of steroid‑refractory acute graft‑versus‑host disease: the RELAX study

作者：Leqing Cao, Wen‐Xuan Huo, Erlie Jiang, Yuewen Fu, Xiaojun Xu, Ping‑Chong Lei, Mingfeng Zhao, Zhi Chen, Shuxia Guo, Xiaobing Huang, Yanming Zhang, Xianjing Wang, Guan‑Chen Bai, Jin F, Qingsheng Li, Mingyang Deng, Hao Zhang, Xinfeng Wang, Xiao‐Jun Huang, Xiao‐Dong Mo · 发表于：Military Medical Research · 年份：2025 · DOI：10.1186/s40779-025-00640-0 · 被引用次数：7 · 研究领域：Hematopoietic Stem Cell Transplantation、T-cell and B-cell Immunology、Mesenchymal stem cell research

BACKGROUND: Steroid-refractory (SR) acute graft-versus-host disease (aGVHD) is the major cause of early mortality after allogeneic hematopoietic stem cell transplantation (allo-HSCT). Xenopax, a novel and the only available humanized interleukin-2 (IL-2) receptor antagonist, has been approved as a category 2 biological product by the National Medical Products Administration. This study aims to evaluate the efficacy, safety, and prognostic factors of xenopax treatment for SR-aGVHD in real-world settings. METHODS: This was a multicenter, retrospective analysis that included SR-aGVHD patients who received xenopax at 17 hospitals across China. The data were collected from the electronic medical records in transplant databases. The primary endpoint was the 28-day overall response rate (ORR), encompassing both partial and complete responses. This study also included independent historical SR-aGVHD cohorts treated with best available treatments (BATs, n = 1009) as controls. RESULTS: In total, 172 SR-aGVHD patients were included in this study. Xenopax was administered either as monotherapy (n = 60) or in combination with other second-line treatments (n = 112). The ORR was 64.5% [95% confidence interval (CI) 57.3-71.7%] on day 28 and 82.6% (95% CI 76.9-88.3%) at any time after xenopax treatment. The 2-year probabilities of disease-free survival, overall survival, non-relapse mortality (NRM), and relapse after xenopax treatment were 57.0% (95% CI 49.9-65.0%), 68.0% (95% CI 61.4-75.4%),...