{"title":"Bu/Cy/ATG与Flu/Cy/ATG调理方案在非亲属供体造血干细胞移植治疗重度再生障碍性贫血中的比较研究","authors":"Liangliang Wu, Xiaowei Chen, Ming Zhou, Wenjian Mo, Ruiqing Zhou, Yumiao Li, Shilin Xu, Caixia Wang, Shiyi Pan, Wei Zhou, Tingfen Deng, Yuling Zhang, Yuping Zhang, Shunqing Wang","doi":"10.1016/j.jtct.2025.06.014","DOIUrl":null,"url":null,"abstract":"<p><p>Unrelated donor hematopoietic stem cell transplantation (URD-HSCT) is a curative option for severe aplastic anemia (SAA), but the optimal conditioning regimen remains unclear. This retrospective study compares Busulfan/Cyclophosphamide/Anti-thymocyte globulin (Bu/Cy/ATG) and Fludarabine/Cyclophosphamide/Anti-thymocyte globulin (Flu/Cy/ATG) protocols to identify the best regimen for SAA patients. We retrospectively analyzed the clinical outcomes of 107 SAA patients who underwent URD-HSCT with Flu/Cy/ATG (n=63) or Bu/Cy/ATG (n=44) between November 2012 and December 2022. No significant differences were observed in the cumulative incidence of neutrophil/platelet engraftment, graft failure, graft-versus-host disease (GVHD), or CMV viremia. Overall survival (OS) at 7 years was 95.5% (95% CI: 89.5-100) with Bu/Cy/ATG vs. 85.5% (95% CI: 77.1-94.7) with Flu/Cy/ATG, and failure-free survival (FFS) at 7 years was 95.5% (95% CI: 89.5-100) versus 83.9% (95% CI: 75.2-93.6). Multivariate analysis identified Bu/Cy/ATG protocol as favorable for OS (Hazard ratio, HR 0.122, 95% CI: 0.021-0.715, P = 0.020) and FFS (HR 0.090, 95% CI: 0.015-0.538, P = 0.008). Moreover, multivariate analysis confirmed that the Bu/Cy/ATG regimen significantly reduced the risk of EBV viremia (Relative risk, RR 0.175, 95% CI: 0.026-0.717, P = 0.032) and post-transplant lymphoproliferative disorder (RR 0.031, 95% CI: 0-0.536, P = 0.012). Subgroup analysis through multivariate modeling further demonstrated that the Bu/Cy/ATG regimen demonstrated superior OS, FFS and EBV infection outcomes in patients older than 30 years. The Bu/Cy/ATG regimen, compared to Flu/Cy/ATG protocol, offers superior outcomes, including improved OS/FFS and reduced EBV infection, suggesting it may be the preferred choice for SAA patients undergoing URD-HSCT, especially for patients older than 30 years. Larger cohorts and prospective trials are needed to validate these findings.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6000,"publicationDate":"2025-06-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Comparative study of Bu/Cy/ATG and Flu/Cy/ATG conditioning regimens for unrelated donor hematopoietic stem cell transplantation in severe aplastic anemia.\",\"authors\":\"Liangliang Wu, Xiaowei Chen, Ming Zhou, Wenjian Mo, Ruiqing Zhou, Yumiao Li, Shilin Xu, Caixia Wang, Shiyi Pan, Wei Zhou, Tingfen Deng, Yuling Zhang, Yuping Zhang, Shunqing Wang\",\"doi\":\"10.1016/j.jtct.2025.06.014\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Unrelated donor hematopoietic stem cell transplantation (URD-HSCT) is a curative option for severe aplastic anemia (SAA), but the optimal conditioning regimen remains unclear. This retrospective study compares Busulfan/Cyclophosphamide/Anti-thymocyte globulin (Bu/Cy/ATG) and Fludarabine/Cyclophosphamide/Anti-thymocyte globulin (Flu/Cy/ATG) protocols to identify the best regimen for SAA patients. We retrospectively analyzed the clinical outcomes of 107 SAA patients who underwent URD-HSCT with Flu/Cy/ATG (n=63) or Bu/Cy/ATG (n=44) between November 2012 and December 2022. No significant differences were observed in the cumulative incidence of neutrophil/platelet engraftment, graft failure, graft-versus-host disease (GVHD), or CMV viremia. Overall survival (OS) at 7 years was 95.5% (95% CI: 89.5-100) with Bu/Cy/ATG vs. 85.5% (95% CI: 77.1-94.7) with Flu/Cy/ATG, and failure-free survival (FFS) at 7 years was 95.5% (95% CI: 89.5-100) versus 83.9% (95% CI: 75.2-93.6). Multivariate analysis identified Bu/Cy/ATG protocol as favorable for OS (Hazard ratio, HR 0.122, 95% CI: 0.021-0.715, P = 0.020) and FFS (HR 0.090, 95% CI: 0.015-0.538, P = 0.008). Moreover, multivariate analysis confirmed that the Bu/Cy/ATG regimen significantly reduced the risk of EBV viremia (Relative risk, RR 0.175, 95% CI: 0.026-0.717, P = 0.032) and post-transplant lymphoproliferative disorder (RR 0.031, 95% CI: 0-0.536, P = 0.012). Subgroup analysis through multivariate modeling further demonstrated that the Bu/Cy/ATG regimen demonstrated superior OS, FFS and EBV infection outcomes in patients older than 30 years. The Bu/Cy/ATG regimen, compared to Flu/Cy/ATG protocol, offers superior outcomes, including improved OS/FFS and reduced EBV infection, suggesting it may be the preferred choice for SAA patients undergoing URD-HSCT, especially for patients older than 30 years. Larger cohorts and prospective trials are needed to validate these findings.</p>\",\"PeriodicalId\":23283,\"journal\":{\"name\":\"Transplantation and Cellular Therapy\",\"volume\":\" \",\"pages\":\"\"},\"PeriodicalIF\":3.6000,\"publicationDate\":\"2025-06-12\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Transplantation and Cellular Therapy\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1016/j.jtct.2025.06.014\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"HEMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Transplantation and Cellular Therapy","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1016/j.jtct.2025.06.014","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"HEMATOLOGY","Score":null,"Total":0}
Comparative study of Bu/Cy/ATG and Flu/Cy/ATG conditioning regimens for unrelated donor hematopoietic stem cell transplantation in severe aplastic anemia.
Unrelated donor hematopoietic stem cell transplantation (URD-HSCT) is a curative option for severe aplastic anemia (SAA), but the optimal conditioning regimen remains unclear. This retrospective study compares Busulfan/Cyclophosphamide/Anti-thymocyte globulin (Bu/Cy/ATG) and Fludarabine/Cyclophosphamide/Anti-thymocyte globulin (Flu/Cy/ATG) protocols to identify the best regimen for SAA patients. We retrospectively analyzed the clinical outcomes of 107 SAA patients who underwent URD-HSCT with Flu/Cy/ATG (n=63) or Bu/Cy/ATG (n=44) between November 2012 and December 2022. No significant differences were observed in the cumulative incidence of neutrophil/platelet engraftment, graft failure, graft-versus-host disease (GVHD), or CMV viremia. Overall survival (OS) at 7 years was 95.5% (95% CI: 89.5-100) with Bu/Cy/ATG vs. 85.5% (95% CI: 77.1-94.7) with Flu/Cy/ATG, and failure-free survival (FFS) at 7 years was 95.5% (95% CI: 89.5-100) versus 83.9% (95% CI: 75.2-93.6). Multivariate analysis identified Bu/Cy/ATG protocol as favorable for OS (Hazard ratio, HR 0.122, 95% CI: 0.021-0.715, P = 0.020) and FFS (HR 0.090, 95% CI: 0.015-0.538, P = 0.008). Moreover, multivariate analysis confirmed that the Bu/Cy/ATG regimen significantly reduced the risk of EBV viremia (Relative risk, RR 0.175, 95% CI: 0.026-0.717, P = 0.032) and post-transplant lymphoproliferative disorder (RR 0.031, 95% CI: 0-0.536, P = 0.012). Subgroup analysis through multivariate modeling further demonstrated that the Bu/Cy/ATG regimen demonstrated superior OS, FFS and EBV infection outcomes in patients older than 30 years. The Bu/Cy/ATG regimen, compared to Flu/Cy/ATG protocol, offers superior outcomes, including improved OS/FFS and reduced EBV infection, suggesting it may be the preferred choice for SAA patients undergoing URD-HSCT, especially for patients older than 30 years. Larger cohorts and prospective trials are needed to validate these findings.
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