使用移植后环磷酰胺 (PTCy)、他克莫司 (TAC) 和吗替麦考酚酯 (MMF) 预防移植物抗宿主病 (GVHD) 用于同种异体单倍体供体 (haplo) 造血细胞移植 (HCT)，其结果与匹配的非相关供体相当移植。莫菲特癌症中心的一项 II 期研究用西罗莫司 (SIRO) 代替 TAC，报告了 II-IV 级急性 GVHD 的发生率相当。尽管比较数据有限，但在这种情况下，许多中心已根据首选副作用情况用 SIRO 代替 TAC。本研究的目的是比较 haplo HCT 与 PTCy/SIRO/MMF 与 PTCy/TAC/MMF 的结果。我们回顾性比较了 PTCy/SIRO/MMF 与 PTCy/TAC/MMF 的单倍体 HCT 结局。包括 2014 年至 2019 年间在莫菲特癌症中心或希望之城国家医疗中心接受单倍体供体 T 细胞充满外周血干细胞移植 HCT 治疗血液恶性肿瘤的所有连续患者。总共包括 423 名患者，其中 84 名（20 名） %）收到了 SIRO，339（80%）收到了 TAC。所有患者的中位年龄为 54 岁（范围为 18 至 78 岁），整个研究队列的中位随访时间为 30 个月。 SIRO组≥60岁的患者比例较高（58% vs 34%，P=<.01），并且各组在诊断类型、预处理方案和巨细胞病毒血清状态方面也存在差异。第 100 天的 II-IV 级急性 GVHD 发生率（45% 对比 47%，P=.6）或术后 2 年的慢性 GVHD 发生率（47% 对比 54%，P=.79）没有显着差异。血细胞CT。在多变量分析中，TAC 在第 30 天的中性粒细胞植入明显更好（OR = 0.30，CI 0.1 至 0.83，P = 0.02），中位植入时间为 17 天，而 SIRO 为 18 天，但血小板植入与 SIRO 相似。两组。另外，在多变量分析中，GVHD 预防类型对急性或慢性 GVHD、非复发死亡率、复发、无 GVHD 无复发生存期、无病生存期或外周血单倍体 HCT 后的总生存期没有显着影响。这些结果表明 SIRO与 TAC 结合 PTCy/MMF 预防 GVHD 相比，它是一种可比的替代方案，尽管外周血单倍体 HCT 后植入延迟，但总体临床结果相似。虽然 TAC 仍然是护理标准，但可以根据这些药物的副作用情况并考虑 HCT 中患者的医疗合并症来替代 SIRO。版权所有 © 2023。由 Elsevier Inc. 出版。

Graft-versus-host disease (GVHD) prophylaxis with post-transplant cyclophosphamide (PTCy), tacrolimus (TAC) and mycophenolate mofetil (MMF) for allogeneic haploidentical donor (haplo) hematopoietic cell transplantation (HCT) results in comparable outcomes to matched unrelated donor transplantation. A phase II study from Moffitt Cancer Center substituting sirolimus (SIRO) in place of TAC reported comparable rates of grade II-IV acute GVHD. Many centers have substituted SIRO for TAC in this setting based on a preferred side effect profile, though comparative data is limited.The objective of this study is to compare outcomes of haplo HCT with PTCy/SIRO/MMF versus PTCy/TAC/MMF.We retrospectively compared haplo HCT outcomes with PTCy/SIRO/MMF versus PTCy/TAC/MMF. Included were all consecutive patients receiving haplo donor T cell replete peripheral blood stem cell graft HCT for hematologic malignancies at Moffitt Cancer Center or the City of Hope National Medical Center between 2014 and 2019.A total of 423 patients were included, of which 84 (20%) received SIRO and 339 (80%) received TAC. Median age for all patients was 54 (range 18 to 78) years, and median follow-up for entire study cohort was 30 months. SIRO group had a higher proportion of patients ≥60 years (58% versus 34%, P = <.01), and the groups also differed in diagnosis type, conditioning regimen, and cytomegalovirus serostatus. There were no significant differences in the rates of grade II-IV acute GVHD (45% versus 47%, P = .6) at day +100 or chronic GVHD (47% versus 54%, P = .79) at 2 years post-HCT. In multivariate analysis, neutrophil engraftment at day +30 was significantly better with TAC (OR = 0.30, CI 0.1 to 0.83, P = .02) with a median time to engraftment of 17 days versus 18 days for SIRO, but platelet engraftment was similar in both groups. Otherwise, in multivariate analysis, GVHD prophylaxis type had no significant influence on acute or chronic GVHD, nonrelapse mortality, relapse, GVHD-free relapse-free survival, disease free survival, or overall survival after peripheral blood haplo HCT.These findings suggest that SIRO is a comparable alternative to TAC in combination with PTCy/MMF for GVHD prophylaxis, resulting in overall similar clinical outcomes despite delay in engraftment after peripheral blood haplo HCT. While TAC remains the standard of care, SIRO may be substituted based on the side effect profile of these medications with consideration of patient medical comorbidities at HCT.Copyright © 2023. Published by Elsevier Inc.