{"title":"Xenopax用于治疗类固醇难治性急性移植物抗宿主病：RELAX研究。","authors":"Le-Qing Cao, Wen-Xuan Huo, Er-Lie Jiang, Yue-Wen Fu, Xiao-Jun Xu, Ping-Chong Lei, Ming-Feng Zhao, Zhi Chen, Shu-Xia Guo, Xiao-Bing Huang, Yan-Ming Zhang, Xian-Jing Wang, Guan-Chen Bai, Feng-Bo Jin, Qing-Sheng Li, Ming-Yang Deng, Hao Zhang, Xin-Feng Wang, Xiao-Jun Huang, Xiao-Dong Mo","doi":"10.1186/s40779-025-00640-0","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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%), 24.2% (95% CI 18.0-30.9%), and 19.0% (95% CI 12.8-25.2%), respectively. The ORR and survival were similar between patients with and without prior second-line treatments. The conditioning regimen and human leukocyte antigen disparity did not impact the efficacy of xenopax treatment. According to the multivariate analysis, the presence of grade III-IV aGVHD did not adversely affect the therapeutic response or survival. Xenopax also showed some superiority over BATs in historical cohorts.</p><p><strong>Conclusions: </strong>Our real-world findings suggest that xenopax is an effective and safe treatment for SR-aGVHD.</p>","PeriodicalId":18581,"journal":{"name":"Military Medical Research","volume":"12 1","pages":"63"},"PeriodicalIF":22.9000,"publicationDate":"2025-09-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12477784/pdf/","citationCount":"0","resultStr":"{\"title\":\"Xenopax for the treatment of steroid-refractory acute graft-versus-host disease: the RELAX study.\",\"authors\":\"Le-Qing Cao, Wen-Xuan Huo, Er-Lie Jiang, Yue-Wen Fu, Xiao-Jun Xu, Ping-Chong Lei, Ming-Feng Zhao, Zhi Chen, Shu-Xia Guo, Xiao-Bing Huang, Yan-Ming Zhang, Xian-Jing Wang, Guan-Chen Bai, Feng-Bo Jin, Qing-Sheng Li, Ming-Yang Deng, Hao Zhang, Xin-Feng Wang, Xiao-Jun Huang, Xiao-Dong Mo\",\"doi\":\"10.1186/s40779-025-00640-0\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background: </strong>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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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%), 24.2% (95% CI 18.0-30.9%), and 19.0% (95% CI 12.8-25.2%), respectively. The ORR and survival were similar between patients with and without prior second-line treatments. The conditioning regimen and human leukocyte antigen disparity did not impact the efficacy of xenopax treatment. According to the multivariate analysis, the presence of grade III-IV aGVHD did not adversely affect the therapeutic response or survival. Xenopax also showed some superiority over BATs in historical cohorts.</p><p><strong>Conclusions: </strong>Our real-world findings suggest that xenopax is an effective and safe treatment for SR-aGVHD.</p>\",\"PeriodicalId\":18581,\"journal\":{\"name\":\"Military Medical Research\",\"volume\":\"12 1\",\"pages\":\"63\"},\"PeriodicalIF\":22.9000,\"publicationDate\":\"2025-09-29\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12477784/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Military Medical Research\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1186/s40779-025-00640-0\",\"RegionNum\":2,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"MEDICINE, GENERAL & INTERNAL\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Military Medical Research","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1186/s40779-025-00640-0","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"MEDICINE, GENERAL & INTERNAL","Score":null,"Total":0}
引用次数: 0
摘要
背景：类固醇难治性（SR）急性移植物抗宿主病（aGVHD）是异体造血干细胞移植（alloo - hsct）术后早期死亡的主要原因。Xenopax是一种新型且目前唯一的人源化白细胞介素-2受体拮抗剂，已被国家药品监督管理局批准为二类生物制品。本研究旨在评估现实环境下xenopax治疗SR-aGVHD的疗效、安全性和预后因素。方法：这是一项多中心、回顾性分析，包括在中国17家医院接受xenopax治疗的SR-aGVHD患者。数据收集自移植数据库中的电子病历。主要终点是28天总缓解率（ORR），包括部分缓解和完全缓解。本研究还纳入了接受最佳可用治疗（BATs, n = 1009）的独立历史SR-aGVHD队列作为对照。结果：本研究共纳入172例SR-aGVHD患者。Xenopax作为单一治疗（n = 60）或与其他二线治疗（n = 112）联合使用。在第28天，ORR为64.5%[95%可信区间（CI） 57.3-71.7%]，在xenopax治疗后的任何时间，ORR为82.6% （95% CI 76.9-88.3%）。xenopax治疗后的2年无病生存率、总生存率、非复发死亡率（NRM）和复发概率分别为57.0% （95% CI 49.9-65.0%）、68.0% （95% CI 61.4-75.4%）、24.2% （95% CI 18.0-30.9%）和19.0% （95% CI 12.8-25.2%）。接受和未接受过二线治疗的患者的ORR和生存率相似。调理方案和人白细胞抗原差异不影响异氧派治疗的疗效。根据多变量分析，III-IV级aGVHD的存在并未对治疗反应或生存产生不利影响。Xenopax在历史队列中也比BATs有一定的优势。结论：我们的现实研究结果表明，xenopax是一种有效且安全的治疗SR-aGVHD的方法。
Xenopax for the treatment of steroid-refractory acute graft-versus-host disease: the RELAX study.
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%), 24.2% (95% CI 18.0-30.9%), and 19.0% (95% CI 12.8-25.2%), respectively. The ORR and survival were similar between patients with and without prior second-line treatments. The conditioning regimen and human leukocyte antigen disparity did not impact the efficacy of xenopax treatment. According to the multivariate analysis, the presence of grade III-IV aGVHD did not adversely affect the therapeutic response or survival. Xenopax also showed some superiority over BATs in historical cohorts.
Conclusions: Our real-world findings suggest that xenopax is an effective and safe treatment for SR-aGVHD.
期刊介绍：
Military Medical Research is an open-access, peer-reviewed journal that aims to share the most up-to-date evidence and innovative discoveries in a wide range of fields, including basic and clinical sciences, translational research, precision medicine, emerging interdisciplinary subjects, and advanced technologies. Our primary focus is on modern military medicine; however, we also encourage submissions from other related areas. This includes, but is not limited to, basic medical research with the potential for translation into practice, as well as clinical research that could impact medical care both in times of warfare and during peacetime military operations.
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