无关供者移植后使用环磷酰胺与 ATG 治疗骨髓增生异常肿瘤。
Unrelated donor transplantation with post-transplant cyclophosphamide versus ATG for myelodysplastic neoplasms.
发表日期：2024 Jul 15
作者：
Yves Chalandon, Diderik-Jan Eikema, Ivan Sergeevich Moiseev, Fabio Ciceri, Linda Koster, Jan Vydra, Jakob R Passweg, Montserrat Rovira, Tulay Ozcelik, Tobias Gedde-Dahl, Nicolaus Kröger, Victoria Potter, Ibrahim Yakoub-Agha, Alessandro Rambaldi, Maija Itälä-Remes, Alina D Tanase, Francesco Onida, Carmelo Gurnari, Christof Scheid, Joanna Drozd-Sokolowska, Kavita Raj, Donal P McLornan, Marie Robin
来源：
Cellular & Molecular Immunology
摘要：
前瞻性随机试验报告了在异体造血干细胞移植（Allo-HSCT）与无关供者（UD）的情况下基于抗胸腺细胞球蛋白（ATG）的移植物抗宿主病（GvHD）预防的益处。然而，最佳 GvHD 预防策略最近因移植后环磷酰胺 (PTCY) 的使用增加而受到挑战。我们从 EBMT 登记处报告了 960 名骨髓增生异常肿瘤 (MDS) 患者接受 UD 异基因造血干细胞移植并使用 PTCY 或 ATG 作为 GvHD 预防的结果。主要结局是总生存期（OS）和无进展生存期（PFS）。两组的疾病特征相似。第 28 天，ATG 的中性粒细胞植入明显更好（93% vs 85%，p<0.001）。中位随访时间为 4.4 年（95% 置信区间 [CI] 4.2 - 4.8），PTCY 组的 5 年 OS 为 58% (95% CI 50-65)，PTCY 组的 5 年 OS 为 49% (95% CI 46-53%) ）在 ATG 组中，p=0.07。 PTCY 的 5 年 PFS 较高，为 53% (95% CI 45-60)，而 ATG 为 44% (95% CI 40-48)，p=0.043。使用PTCY时，II-IV级aGvHD发生率较低（23% [95% CI 17-29%] vs 30% [95% CI 27-33%]），p=0.044，而5时cGvHD发生率没有差异年。多变量分析证实，PTCY 具有更好的 OS 和 PFS，ATG 的 HR 为 1.32 (1 - 1.74)，p=0.05；PTCY 的 PFS 更好，ATG 的 HR 为 1.33 (1.03 - 1.73)，p=0.03。这项研究表明，在这种情况下，使用 PTCY 代替 ATG 进行 GvHD 预防仍然是一个有效的选择。进一步的前瞻性随机研究对于证实这些结果至关重要。版权所有 © 2024 美国血液学会。
Prospective randomized trials have reported a benefit for anti-thymocyte globulin (ATG)-based graft-versus-host disease (GvHD) prophylaxis in the setting of allogeneic hematopoietic stem cell transplantation (Allo-HSCT) with unrelated donors (UD). However, the optimal GvHD prophylaxis strategy has been recently challenged by the increasing use of post-transplant cyclophosphamide (PTCY). We report from the EBMT registry the outcomes of 960 patients with myelodysplastic neoplasms (MDS) undergoing allo-HSCT from UD with PTCY or ATG as GvHD prophylaxis. Primary outcomes were overall survival (OS) and progression-free survival (PFS). Disease characteristics were similar in both groups. Day 28 neutrophil engraftment was significantly better with ATG (93% vs 85%, p<0.001). With a median follow-up of 4.4 years (95% confidence interval [CI] 4.2 - 4.8), 5-year OS was 58% (95% CI 50-65) with PTCY and 49% (95% CI 46-53%) in the ATG group, p=0.07. 5-year PFS was higher for PTCY with 53% (95% CI 45-60) vs 44% (95% CI 40-48) for ATG, p=0.043. Grade II-IV aGvHD incidence was lower using PTCY (23% [95% CI 17-29%] vs 30% [95% CI 27-33%]), p=0.044 while there was no difference in incidence of cGvHD at 5 years. Multivariable analyses confirmed better OS and PFS with PTCY, with a HR for ATG of 1.32 (1 - 1.74), p=0.05, and a better PFS for PTCY with a HR for ATG of 1.33 (1.03 - 1.73), p=0.03. This study suggests that GvHD prophylaxis using PTCY instead of ATG in this setting remains a valid option. Further prospective randomized studies would be essential to confirm these results.Copyright © 2024 American Society of Hematology.