Coinfusion of unrelated cord blood (UCB) units in haploidentical hematopoietic cell transplantation (haplo-HCT) (haplo-cord HCT) for hematopoietic malignancies showed promising results in previous reports, but the efficiency of haplo-cord HCT in acute myeloid leukemia (AML) still lacks sufficient evidence. This multicenter, randomized, phase 3 trial (ClinicalTrials.gov NCT03719534) aimed to assess the efficacy and safety of haplo-cord HCT in AML patients. A total of 268 eligible patients aged 18–60 years, diagnosed with measurable residual disease in AML (excluding acute promyelocytic leukemia), with available haploidentical donors and suitable for allotransplantation, were randomly allocated (1:1) to receive haplo-cord HCT (n = 134) or haplo-HCT (n = 134). The 3-year overall survival (OS) was the primary endpoint in this study. Overall median follow-up was 36.50 months (IQR 24.75–46.50). The 3-year OS of Haplo-cord HCT group was better than haplo-HCT group (80.5%, 95% confidence interval [CI]: 73.7–87.9 vs. 67.8% 95% CI 60.0–76.5, p = 0.013). Favorable progression-free survival (70.3%, 95% CI 62.6–78.8 vs. 57.6%, 95% CI 49.6–67.0, p = 0.012) and cumulative incidence of relapse (12.1%, 95% CI 12.0–12.2 vs. 30.3%, 95% CI 30.1–30.4, p = 0.024) were observed in haplo-cord HCT group. Grade 3–4 adverse events (AEs) within two years posttransplantation in the two groups were similar. Haplo-cord HCT patients exhibited a faster cumulative incidence of neutrophil recovery (p = 0.026) and increased T-cell reconstitution in the early period posttransplantation. Haplo-cord HCT can improve OS in AML patients without excessive AEs, which may exert additional benefits for recipients of haplo-HCT.

在之前的报道中，在单倍体相合造血细胞移植（haplo-HCT）（haplo-cord HCT）中混合不相关的脐带血（UCB）单位治疗造血系统恶性肿瘤显示出有希望的结果，但单倍体脐带HCT在急性髓系白血病（AML）中的效率仍缺乏足够的证据。这项多中心、随机、3 期试验 (ClinicalTrials.gov NCT03719534) 旨在评估单倍索 HCT 在 AML 患者中的疗效和安全性。共有 268 名年龄在 18-60 岁、诊断为可测量的 AML 残留病灶（不包括急性早幼粒细胞白血病）、具有可用的单倍体供体且适合同种异体移植的患者，被随机分配（1:1）接受单倍体 HCT（n  = 134) 或单倍体 HCT ( n  = 134)。 3 年总生存期 (OS) 是本研究的主要终点。总体中位随访时间为 36.50 个月 (IQR 24.75–46.50)。 Haplo-cord HCT 组的 3 年 OS 优于 haplo-HCT 组（80.5%，95% 置信区间 [CI]：73.7–87.9 vs. 67.8% 95% CI 60.0–76.5，p  = 0.013）。良好的无进展生存率（70.3%，95% CI 62.6–78.8 vs. 57.6%，95% CI 49.6–67.0，p  = 0.012）和累积复发率（12.1%，95% CI 12.0–12.2 vs. 30.3%） 在单倍索 HCT 组中观察到，95% CI 30.1–30.4，p = 0.024）。两组移植后两年内发生的 3-4 级不良事件 (AE) 相似。 Haplo-cord HCT 患者 在移植后早期表现出中性粒细胞恢复的累积发生率更快 ( p = 0.026) 和 T 细胞重建增加。 Haplo-cord HCT 可以改善 AML 患者的 OS，而不会出现过多的 AE，这可能为 haplo-HCT 的接受者带来额外的好处。