Randomized trial of anti-thymocyte globulin plus low-dose post-transplant cyclophosphamide to prevent graft-versus-host disease in haploidentical transplantation

作者：Zhengli Xu, Tingting Han, Xiaolu Zhu, Jing Liu, Meng Lv, Yuqian Sun, Xiao‐Dong Mo, Yifei Cheng, Lan‐Ping Xu, Xiaohui Zhang, Xiao‐Jun Huang, Yu Wang · 发表于：Haematologica · 年份：2025 · DOI：10.3324/haematol.2025.287504 · 被引用次数：7 · 研究领域：Hematopoietic Stem Cell Transplantation、Renal Transplantation Outcomes and Treatments、Polyomavirus and related diseases

The combination of anti-thymocyte globulin (ATG) and posttransplant cyclophosphamide (PTCy) appears to be a potentially effective graft-versus-host disease (GVHD) prevention strategy for haploidentical transplantation. However, the majority of the evidence originated from retrospective studies without uniform protocols. Our previous findings indicated that 10 mg/kg ATG plus low-dose PTCy could decrease GVHD among high-risk populations transplanted from maternal or collateral relatives. We designed an open-label, phase III, randomized controlled trial to compare patients receiving granulocyte colony-stimulating factor (G-CSF)/ATG-based haploidentical transplantation with or without low-dose PTCy (14.5 mg/kg on days 3 and 4) in non-maternal, non-collateral haploidentical transplants from fathers, children or siblings. A total of 66 patients were randomly assigned to ATG-PTCy (N=44) or ATG (N=22) when the first interim analysis was performed. The interim analysis revealed that the 100-day cumulative incidences of grade 2-4 (18.2%, 95% confidence interval [CI]: 6.6-29.7 vs. 18.2%, 95% CI: 1.7-34.7; P=0.996) and 3-4 acute GVHD (2.3%, 95% CI: 0-6.7 vs. 0; P=0.480) were comparable between the ATG-PTCy and ATG cohorts, as was chronic GVHD at 1 year. The estimated 1-year disease-free survival (DFS) rates were also similar between ATG-PTCy and ATG cohorts (95.5%, 95% CI: 89.5-100 vs. 95.2%, 95% CI: 86.6-100; P=0.979). These results suggested that ATG/PTCy (low-dose) had no advantage ov...