Hôpitaux Universitaires de Genève and Faculty of Medicine, University of Geneva, Geneva, Switzerland.
EBMT Leiden Statistical Unit, Leiden, The Netherlands.
Blood Adv. 2024 Sep 24;8(18):4792-4802. doi: 10.1182/bloodadvances.2024013468.
It has been reported in prospective randomized trials that antithymocyte globulin (ATG)-based graft-versus-host disease (GVHD) prophylaxis has benefits in the setting of allogeneic hematopoietic stem cell transplantation (allo-HSCT) with unrelated donors (UDs). However, the optimal GVHD prophylaxis strategy has been challenged recently by the increasing use of posttransplant cyclophosphamide (PTCY). We report from the European Society for Blood and Marrow Transplantation registry the outcomes of 960 patients with myelodysplastic neoplasms who underwent allo-HSCT from UD with PTCY or ATG as GVHD prophylaxis. The primary outcomes were overall survival (OS) and progression-free survival (PFS). The disease characteristics were similar in both groups. Day 28 neutrophil engraftment was significantly better with ATG (93% vs 85%). Over a median follow-up of 4.4 years, the 5-year OS was 58% with PTCY, and 49% in the ATG group. The 5-year PFS was higher for PTCY at 53% vs 44% for ATG. Grade 2 to 4 acute GVHD incidence was lower when PTCY was used (23%), whereas there was no difference in the incidence of chronic GVHD at 5 years. Multivariable analyses confirmed better OS and PFS with PTCY with a hazard ratio (HR) for ATG of 1.32 (1-1.74) and a better PFS for PTCY with a HR for ATG of 1.33. 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.
据前瞻性随机试验报道，抗胸腺细胞球蛋白（ATG）为基础的移植物抗宿主病（GVHD）预防在异基因造血干细胞移植（allo-HSCT）联合无关供者（UD）的情况下具有益处。然而，最近越来越多地使用移植后环磷酰胺（PTCY）对最佳 GVHD 预防策略提出了挑战。我们从欧洲血液和骨髓移植学会登记处报告了 960 例接受 PTCY 或 ATG 作为 GVHD 预防的 UD 来源异基因造血干细胞移植的骨髓增生异常综合征患者的结局。主要结局是总生存（OS）和无进展生存（PFS）。两组的疾病特征相似。ATG 组第 28 天中性粒细胞植入明显更好（93% vs 85%）。在中位随访 4.4 年后，PTCY 的 5 年 OS 为 58%，ATG 组为 49%。PTCY 的 5 年 PFS 更高，为 53%，而 ATG 组为 44%。当使用 PTCY 时，2 级至 4 级急性 GVHD 的发生率较低（23%），而 5 年慢性 GVHD 的发生率无差异。多变量分析证实，与 ATG 相比，PTCY 具有更好的 OS 和 PFS，ATG 的危险比（HR）为 1.32（1-1.74），PTCY 具有更好的 PFS，ATG 的 HR 为 1.33。这项研究表明，在这种情况下，使用 PTCY 而不是 ATG 进行 GVHD 预防仍然是一种有效的选择。需要进一步的前瞻性随机研究来证实这些结果。
