匹配的同种异体造血干细胞移植后儿童急性白血病移植后环磷酰胺（PTCy）的数据仅限于病例系列。本研究旨在评估一大群急性白血病儿童在匹配的同种异体造血干细胞移植后基于 PTCy 的 GVHD 预防的结果。对 2008 年至 2020 年间接受第一次同种异体移植的 190 名急性白血病儿童患者进行回顾性分析。进行了匹配的兄弟姐妹供体（MSD）或匹配的无关供体（MUD）。在 MSD 环境中，GVHD 预防包括研究组的单独 PTCy (n = 28) 和对照组的钙调神经磷酸酶抑制剂 (CNI) ± 抗代谢药 (n = 30)。在 MUD 环境中，研究组中的大多数患者接受了 PTCy CNI 吗替麦考酚酯（n = 42，66.7%）或 PTCy CNI 西罗莫司（n = 12，19%）预防 GVHD。对照组所有患者（n = 69）均接受 ATG CNI 抗代谢药物治疗。 MUD allo-HSCT 后，PTCy 组急性 GVHD III-IV 级和中/重度慢性 GVHD 的发生率显着低于对照组（6.6%）。分别对比 35.0% 和 12.7% 对比 47.1%，p < .0001）。与对照组相比，PTCy 组 MUD allo-HSCT 后的五年无 GVHD、无复发生存率 (GRFS) 较高（35.1% vs. 7.3%，p < .0001）。同时，两组之间MSD​​ allo-HSCT后无显着差异。对于小儿急性白血病，基于PTCy的MUD allo-HSCT GVHD预防是一种可行且有效的选择，GVHD发生率较低。与基于 ATG 的方法相比，PTCy 可以更好地控制儿童 GVHD。在默沙东的儿科异基因造血干细胞移植中，PTCy 表现出与传统 GVHD 预防相当的有效性。© 2023 John Wiley

The data on post-transplant cyclophosphamide (PTCy) in pediatric acute leukemia after matched allo-HSCT are limited to case series. The present study aimed to assess the results of PTCy-based GVHD prophylaxis in a large cohort of children with acute leukemia after matched allo-HSCT.A retrospective analysis of 190 pediatric patients with acute leukemia who had a first allograft between 2008 and 2020 from a matched sibling donor (MSD) or matched unrelated donor (MUD) was carried out. In the MSD setting, GVHD prophylaxis consisted of PTCy alone (n = 28) for the study group, and calcineurin inhibitor (CNI) ± antimetabolite (n = 30) for the control group. In MUD setting, most patients in the study group received GVHD prophylaxis with PTCy+CNI+mycophenolate mofetil (n = 42, 66.7%) or PTCy+CNI+sirolimus (n = 12, 19%). All patients (n = 69) in the control group received ATG+CNI+antimetabolite.After MUD allo-HSCT, the incidences of acute GVHD grade III-IV and moderate/severe chronic GVHD were significantly lower in the PTCy group compared to control (6.6% vs. 35.0% and 12.7% vs. 47.1%, respectively, p < .0001). Five-year GVHD-free, relapse-free survival (GRFS) after MUD allo-HSCT was higher in the PTCy group compared to control (35.1% vs. 7.3%, p < .0001). At the same time, there was no significant difference between both groups after MSD allo-HSCT.In pediatric acute leukemia, PTCy-based GVHD prophylaxis for MUD allo-HSCT is a feasible and effective option that results in a low incidence of GVHD. Compared to the ATG-based approach, PTCy provides better control of GVHD in children. In pediatric allo-HSCT from MSD, PTCy demonstrates comparable effectiveness to conventional GVHD prophylaxis.© 2023 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd.