同种异体造血干细胞移植（HCT）仍然是治疗晚期骨髓增生异常综合征（MDS）的唯一可能的治愈性治疗方法。在HCT中，脐血移植（CBT）程序的进展导致CBT作为MDS供体来源的大幅扩展。然而，对于晚期MDS（在HCT时根据世界卫生组织分类被定义为难治性贫血伴有过多幼稚细胞（RAEB）-1和RAEB-2）而言，尚未探讨与匹配的同胞供体（MSDs）HCT和CBT之间的数据比较。我们回顾性比较了2011年至2020年在日本接受同种异体HCT的999名成年MDS患者的生存率和其他移植后结果，使用的供体来源分别为MSD（n = 331）和单个无关脐血（UCB）（n = 668）。在多变量分析中，MSD受者和UCB受者之间的总生存率（风险比[HR]，1.10；95%可信区间[CI]，0.90-1.34；P = 0.347）、无病生存（HR，1.01；95% CI，0.84-1.23；P = 0.845）、复发（HR，0.88；95% CI，0.68-1.15；P = 0.370）或非复发性死亡（HR，1.15；95% CI，0.87-1.50；P = 0.310）没有显著差异。与MSD相比，UCB与中性粒细胞（HR，0.28；95% CI，0.24-0.33；P < 0.001）和血小板（HR，0.29；95% CI，0.23-0.36；P < 0.001）恢复较差显著相关。与MSD相比，UCB与慢性移植物抗宿主病（GVHD）发生率较低（HR，0.57；95% CI，0.44-0.75；P < 0.001）和广泛慢性GVHD发生率较低（HR，0.46；95% CI，0.32-0.67；P < 0.001）显著相关。通过倾向评分匹配分析调整MSD和UCB受体之间的差异后，观察到类似的结果。我们的研究表明，对于晚期MDS成年患者，单一CBT和MSD HCT的存活结果相似，尽管CBT受者的造血恢复较低，而MSD受者的慢性GVHD较高。©2023 The Authors. Hematological Oncology 由John Wiley & Sons Ltd.发布。

Allogeneic hematopoietic stem cell transplantation (HCT) remains the only potential curative therapeutic modality for advanced myelodysplastic syndrome (MDS). Within HCT, the advancement of cord blood transplantation (CBT) procedures has resulted in a drastic expansion of CBT as a donor source for MDS. However, data comparing matched sibling donors (MSDs) HCT with CBT for advanced MDS, which was defined as refractory anemia with an excess of blasts (RAEB)-1 and RAEB-2 according to the World Health Organization classification at the time of HCT, have not been explored. We retrospectively compared survival and other posttransplant outcomes in 999 adult patients with advanced MDS after receiving allogeneic HCT in Japan between 2011 and 2020, using either MSD (n = 331) or single-unit unrelated cord blood (UCB) (n = 668). In the multivariate analysis, there were no significant differences in overall survival (hazard ratio [HR], 1.10; 95% confidence interval [CI], 0.90-1.34; P = 0.347), disease-free survival (HR, 1.01; 95% CI, 0.84-1.23; P = 0.845), relapse (HR, 0.88; 95% CI, 0.68-1.15; P = 0.370), or non-relapse mortality (HR, 1.15; 95% CI, 0.87-1.50; P = 0.310) between MSD recipients and UCB recipients. UCB was significantly associated with lower neutrophil (HR, 0.28; 95% CI, 0.24-0.33; P < 0.001) and lower platelet (HR, 0.29; 95% CI, 0.23-0.36; P < 0.001) recovery compared to MSD. UCB was significantly associated with a lower incidence of chronic graft-versus-host disease (GVHD) (HR, 0.57; 95% CI, 0.44-0.75; P < 0.001) and extensive chronic GVHD (HR, 0.46; 95% CI, 0.32-0.67; P < 0.001) compared to MSD. Similar results were observed after adjusting for differences between MSD and UCB recipients by propensity score matching analysis. Our study demonstrated that single CBT and MSD HCT had similar survival outcomes for adult patients with advanced MDS despite the lower hematopoietic recovery in CBT recipients and higher chronic GVHD in MSD recipients.© 2023 The Authors. Hematological Oncology published by John Wiley & Sons Ltd.