Medical Clinic, Department for Haematology, Oncology and Tumorimmunology, Charité Universitätsmedizin Berlin, Berlin, Germany.
EBMT Transplant Complications Working Party, Paris, France.
Leukemia. 2024 May;38(5):1156-1163. doi: 10.1038/s41375-024-02225-7. Epub 2024 Mar 27.
There is a high risk of GVHD and non-relapse mortality (NRM) after allogeneic stem cell transplantations (alloSCT) from unrelated donors. Prophylaxis with rabbit anti-thymocyte globulin (rATG) is standard in Europe but post-transplantation Cyclophosphamide (PTCy) is an emerging alternative. We analyzed outcomes of rATG (n = 7725) vs. PTCy (n = 1039) prophylaxis in adult patients with hematologic malignancies undergoing peripheral blood alloSCT from 10/10 antigen-matched unrelated donors (MUD) between January 2018 and June 2021 in the EBMT database. The provided P-values and hazard ratios (HR) are derived from multivariate analysis. Two years after alloSCT, NRM in the PTCy group was 12.1% vs. 16.4% in the rATG group; p = 0.016; HR 0.72. Relapse was less frequent after PTCy vs. rATG (22.8% vs. 26.6%; p = 0.046; HR 0.87). Overall survival after PTCy was higher (73.1% vs. 65.9%; p = 0.001, HR 0.82). Progression free survival was better after PTCy vs. rATG (64.9% vs. 57.2%; p < 0.001, HR 0.83). The incidence of chronic GVHD was lower after PTCy (28.4% vs. rATG 31.4%; p = 0.012; HR 0.77), whereas the incidence and severity of acute GVHD were not significantly different. GVHD-free relapse-free survival was significantly higher in the PTCy arm compared to the rATG arm (2 y incidence: 51% vs. 45%; HR: 0.86 [95% CI 0.75-0.99], p = 0.035). In the absence of evidence from randomized controlled trials, our findings support a preference for the use of PTCy in adult recipients of peripheral blood alloSCTs from MUD.
在异基因造血干细胞移植（alloSCT）后，移植物抗宿主病（GVHD）和非复发死亡率（NRM）的风险很高。在欧洲，使用兔抗胸腺细胞球蛋白（rATG）预防是标准的，但在移植后环磷酰胺（PTCy）是一种新兴的替代方法。我们分析了 2018 年 10 月至 2021 年 6 月期间，在 EBMT 数据库中，10/10 抗原匹配的无关供者（MUD）外周血 alloSCT 后，接受血液系统恶性肿瘤治疗的成年患者中 rATG（n=7725）与 PTCy（n=1039）预防的结果。提供的 P 值和风险比（HR）是从多变量分析中得出的。alloSCT 后 2 年，PTCy 组的 NRM 为 12.1%，rATG 组为 16.4%；p=0.016；HR 0.72。与 rATG 相比，PTCy 后复发率较低（22.8% vs. 26.6%；p=0.046；HR 0.87）。PTCy 后总生存率较高（73.1% vs. 65.9%；p=0.001，HR 0.82）。与 rATG 相比，PTCy 后无进展生存率较好（64.9% vs. 57.2%；p<0.001，HR 0.83）。PTCy 后慢性 GVHD 的发生率较低（28.4% vs. rATG 31.4%；p=0.012；HR 0.77），而急性 GVHD 的发生率和严重程度无显著差异。与 rATG 组相比，PTCy 组的 GVHD 无复发无进展生存率显著提高（2 年发生率：51% vs. 45%；HR：0.86[95%CI 0.75-0.99]，p=0.035）。在没有随机对照试验证据的情况下，我们的研究结果支持在 MUD 外周血 alloSCT 受者中使用 PTCy。
