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

However, the majority of the evidence originated from retrospective studies without uniform protocols.

然而，大多数证据来源于没有统一方案的回顾性研究。

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.

我们之前的研究发现，10 mg/kg ATG联合低剂量PTCy可以降低来自母亲或旁系亲属的高风险人群移植中的移植物抗宿主病（GVHD）。

We designed an open-label, phase III, randomized controlled trialto 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.

我们设计了一项开放标签、III期、随机对照试验，以比较接受粒细胞集落刺激因子（G-CSF）/ATG为基础的半相合移植的患者，在非母亲、非旁系亲属的半相合移植中，是否使用低剂量PTCy（第3天和第4天各14.5 mg/kg），供体为父亲、子女或兄弟姐妹。

A total of 66 patients were randomly assigned to ATG-PTCy (N=44) or ATG (N=22) when the first interim analysis was performed.

在进行第一次中期分析时，共有66名患者被随机分配到ATG-PTCy组（N=44）或ATG组（N=22）。

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.

中期分析显示，ATG-PTCy组和ATG组在100天时2-4级（18.2%，95%置信区间[CI]：6.6-29.7 vs. 18.2%，95% CI：1.7-34.7；P=0.996）和3-4级急性移植物抗宿主病（GVHD）（2.3%，95% CI：0-6.7 vs. 0；P=0.480）的发生率相当，1年时的慢性GVHD也是如此。

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).

ATG-PTCy 和 ATG 组的估计 1 年无病生存（DFS）率也相似（95.5%，95% 置信区间：89.5-100 对比 95.2%，95% 置信区间：86.6-100；P=0.979）。

These results suggested that ATG/PTCy (low-dose) had no advantage over 10 mg/kg ATG-based prophylaxis in patients with haploidentical transplantation other than that of maternal donors or collateral relatives.

这些结果表明，除了母亲供体或旁系亲属的患者外，低剂量的 ATG/PTCy 在异基因移植患者中并没有比基于 10 mg/kg ATG 的预防治疗有优势。

Future work needs to focus on identifying which populations might benefit from the combined strategy in the context of G-CSF/ATG-based protocols (clinicaltrials gov.

未来的工作需要集中在识别哪些人群可能从基于G-CSF/ATG方案的联合策略中受益（clinicaltrials gov.）

Identifier: NCT06108739).

注册号：NCT06108739）。