This retrospective study aimed to investigate the outcomes of allogeneic hematopoietic stem cell transplantation (HSCT) for childhood myelodysplastic syndrome (MDS). Thirty-six patients (low-grade MDS, 24; advanced MDS, 12) received HSCT at the Asan Medical Center over two decades (early period, 1997–2007; recent period, 2008–2017). The transplantation outcomes were analyzed according to disease status, conditioning regimen, various donor types, and period of HSCT. During a median follow-up of 5.6 (range, 1.4–21.1) years, the probability of overall survival (OS) and failure-free survival was 77% and 69%, respectively. The cumulative incidence of transplantation-related mortality (TRM) was 12%. Significantly reduced TRM and improved OS were observed in patients who received HSCT during the recent period vs. the early period (TRM, 4% vs. 30%, P = 0.021; OS, 87% vs. 50%, P = 0.006). Comparable outcomes were observed for HSCT from haploidentical family donors vs. HLA-identical donors (TRM, 10% vs. 14%, P = 0.837; OS, 86% vs. 79%, P = 0.625). This study identified the improved outcomes of allogeneic HSCT for childhood MDS over time, in addition, the feasible outcomes of haploidentical HSCT suggested its use as an attractive alternative in the future procedures.

这项回顾性研究旨在调查同种异体造血干细胞移植 (HSCT) 治疗儿童骨髓增生异常综合征 (MDS) 的结果。20 多年来（早期，1997-2007 年；近期，2008-2017 年），36 名患者（低级别 MDS，24 名；晚期 MDS，12 名）在牙山医疗中心接受了 HSCT。根据疾病状态、预处理方案、各种供体类型和HSCT时间分析移植结果。在 5.6（范围，1.4-21.1）年的中位随访期间，总生存（OS）和无失败生存的概率分别为 77% 和 69%。移植相关死亡率（TRM）的累积发生率为 12%。在近期与早期接受 HSCT 的患者中观察到 TRM 显着降低和 OS 改善（TRM，4% 与 30%，P  = 0.021；OS，87% 对 50%，P  = 0.006）。单倍体家族供体与 HLA 相同供体的 HSCT 观察到可比较的结果（TRM，10% 对 14%，P  =  0.837；OS，86% 对 79%，P  = 0.625）。该研究确定了同种异体 HSCT 对儿童 MDS 的改善结果，此外，半相合 HSCT 的可行结果表明其在未来程序中用作有吸引力的替代方案。