不同异基因造血干细胞移植方式治疗167例中高危骨髓增生异常综合征疗效比较

Comparison of three different types of donor hematopoietic stem cell transplantation forintermediate and high-risk myelodysplastic syndrome

目的比较半相合移植（Haplo-HSCT）、无关供者移植（MUD-HSCT）及同胞相合移植（MSD-HSCT）治疗中高危骨髓增生异常综合征（MDS）的疗效及其预后影响因素。 方法回顾性分析2001年8月至2015年5月接受异基因造血干细胞移植（allo-HSCT）治疗的167例中高危MDS患者病例资料，比较不同移植方式下患者的预后特征及预后影响因素。 结果存活患者中位随访60（12-177）个月。5年实际累积无病生存（DFS）率为67.8%（95% CI 60.0%-75.6%），其中MSD-HSCT组为68.0%（95% CI 54.1%-81.9%），MUD-HSCT组为77.4%（95% CI 62.1%-92.7%），Haplo-HSCT组为64.0%（95% CI 52.4%-75.6%），三组比较差异无统计学意义（P=0.632）。单因素分析结果显示移植前病程〉12个月者累积DFS率明显低于≤12个月者（P=0.018）。5年累积复发死亡率和移植相关死亡率（TRM）与上面危险因素均无明显相关性。 结论Haplo-HSCT治疗MDS疗效显著，可以作为重要的替代供者，特别是在缺乏MSD而病情紧急没有时间去等待搜索MUD的情况下，可以主动选择；移植前病程是影响DFS的重要因素。

Objective To investigate three different types of donor hematopoietic stem cell transplantation （HSCT） for intermediate and high-risk myelodysplastic syndrome （MDS）. Methods Between August 2001 and May 2015, 167 consecutive patients with MDS in intermediate and high-risk who underwent allogeneic HSCT were analyzed retrospectively. Results With the median follow up of 60 （12-177） months, The total 5-year DFS was 67.8% （95% CI 60.0%-75.6% ）. Among three different types of donor, 5-year DFS rates were 68.0%（95%CI 54.1%-81.9%） in MSD-HSCT vs 77.4%（95%CI 62.1%- 92.7% ） in MUD- HSCT vs 64.0% （95% CI 52.4%- 75.6% ） in Haplo- HSCT （P=0.632）, respectively. Univariate analysis showed that median disease course before HSCT was the influencing factor of DFS （P=- 0.018）. Five-year relapse and TRM had no correlation with the above-mentioned factor. Conclusions Haplo-HSCT for intermediate and high-risk MDS achieved similar effect produced by MUD or MSD, Haplo-HSCT could be used as an important alternative donor, allo-HSCT must be performed on intermediate and high-risk MDS patients as early as possible after diagnosis.