Ogura Toru, Shiraishi Chihiro, Urawa Aiko
Clinical Research Support Center, Mie University Hospital, Tsu, Japan.
Department of Pharmacy, Mie University Hospital, Tsu, Japan.
Clin Transplant Res. 2025 Jun 30;39(2):131-141. doi: 10.4285/ctr.24.0049. Epub 2025 Apr 15.
Graft-versus-host disease (GVHD) is a severe complication for transplant patients, particularly those undergoing allogeneic hematopoietic stem cell transplantation. Although various GVHD prophylaxis drug combinations are administered in clinical settings, previous studies have primarily focused on monotherapies or limited drug combinations.
We analyzed data from the U.S. Food and Drug Administration Adverse Event Reporting System for patients receiving GVHD prophylaxis drugs between January 2004 and March 2024. Efficacy was evaluated based on the recorded occurrence or nonoccurrence of GVHD following the administration of prophylactic drugs. Drug combinations were compared using the reporting odds ratio (ROR) and adjusted ROR (aROR), which were calculated through univariate and multivariate binomial logistic regression analyses, respectively. The aROR controlled for differences in patient backgrounds.
This study identified 10 GVHD prophylaxis drug combinations with aROR values significantly less than 1, indicating high effectiveness, and 13 combinations with aROR values significantly greater than 1, representing low effectiveness.
The results demonstrated that certain GVHD prophylaxis drug combinations, particularly those including cyclosporine, may be relatively ineffective. However, avoiding cyclosporine is not always feasible in clinical settings, where treatment plans must be tailored to each patient. To address this issue, the study also identified cyclosporine-containing drug combinations that exhibit high efficacy. These findings could help inform the development of personalized treatment strategies for GVHD prophylaxis and thus improve outcomes in patients undergoing allogeneic hematopoietic stem cell transplantation.
移植物抗宿主病（GVHD）是移植患者尤其是接受异基因造血干细胞移植患者的一种严重并发症。尽管临床中使用了各种GVHD预防药物组合，但以往研究主要集中在单一疗法或有限的药物组合上。
我们分析了美国食品药品监督管理局不良事件报告系统中2004年1月至2024年3月接受GVHD预防药物患者的数据。根据预防性药物给药后记录的GVHD发生或未发生情况评估疗效。使用报告比值比（ROR）和调整后的ROR（aROR）比较药物组合，分别通过单变量和多变量二项逻辑回归分析计算得出。aROR控制了患者背景的差异。
本研究确定了10种aROR值显著小于1的GVHD预防药物组合，表明有效性高，以及13种aROR值显著大于1的组合，代表有效性低。
结果表明，某些GVHD预防药物组合，特别是那些包含环孢素的组合，可能相对无效。然而，在临床环境中避免使用环孢素并不总是可行的，治疗方案必须根据每个患者量身定制。为解决这一问题，该研究还确定了具有高疗效的含环孢素药物组合。这些发现有助于为GVHD预防的个性化治疗策略制定提供参考，从而改善异基因造血干细胞移植患者的预后。
