Objective: To evaluate the efficacy of HLA- haploidentical donor hematopoietic transplantation (Haplo- HSCT)for severe aplastic anemia (SAA)by compared with the same period of unrelated donor transplantation (UD- HSCT).
Methods: Of a cohort of 50 SAA patients between September 2012 and July 2014, 26 patients underwent UD- HSCT and 24 patients Haplo- HSCT.
Results: OS rate was 91.3% with a median follow-up of 9 (2-26)months. According to transplant type, there was no significant difference between UD- and Haplo-HSCT (96.1%vs 86.0%,P=0.30). 3 of 50 (6%)patients had primary engraft failure. Haplo- HSCT developed higher significantly incidence of Ⅱ- Ⅳ aGVHD (37.5%vs 3.83%,P=0.003)and cGVHD (37.5%vs 15.3%,P=0.030)than UD-HSCT. Haplo-HSCT also had significantly higher incidences of CMV viremia (78.2%vs 46.1%,P=0.005)and EBV viremia (43.1%vs 16.0%,P=0.040), respectively than UD-HSCT. But the incidences of hemorrhagic cystitis were similar between two transplant types (39.1%vs 23.0%,P=0.120).
Conclusion: This study showed favorable outcome of Haplo-HSCT for SAA, which was comparable with UD-HSCT.
目的: 评价亲缘半相合造血干细胞移植（Haplo-HSCT）治疗重型再生障碍性贫血（SAA）的疗效，并且与同期进行的无关供者造血干细胞移植（UD-HSCT）进行比较。
方法: 回顾性分析2012年9月至2014年7月接受替代供者造血干细胞移植的50例SAA患者临床资料；其中24例接受Haplo-HSCT, 26例接受UD-HSCT。
结果: 中位随访时间9(2~26)个月，总生存（OS）率为91.3%。UD-HSCT组和Haplo-HSCT组OS率分别为96.1%和86.0%（P=0.30）。3例（6%）患者原发植入失败。Haplo-HSCT组患者Ⅱ~Ⅳ度急性移植物抗宿主病、慢性移植物抗宿主病、CMV血症、EBV血症发生率分别为37.5%、37.5%、78.2％及43.1%，均明显高于UD-HSCT组（3.83%、15.3%、46.1％及16.0%）（P值分别为0.003、0.030、0.005、0.040）；出血性膀胱炎发生率差异无统计学意义（39.1％对23.0%，P=0.120）。
结论: Haplo-HSCT治疗SAA OS率较高，疗效与UD-HSCT相近。