Objective: To compare the differences between hematopoietic reconstitution and longterm prognosis of patients with severe aplastic anemia （SAA） after HLA- matched sibling donor hematopoietic stem cell transplantation（MSD-HSCT）, Haploidentical HSCT（Haplo-HSCT）, unrelated donor allogeneic HSCT（UD-HSCT）and umbilical cord blood HSCT（UCB-HSCT）.
Methods: In this retrospective study, 63 patients with SAA who received HSCT in the First Affiliated Hospital of Soochow University between May 2008 and December 2013 were enrolled. The subjects were divided into 4 groups according to the transplantation types. The hematopoietic reconstitution, the incidence of acute graft-versushost disease（aGVHD）and 5- year survival rate after transplantation were compared.
Results: All 53 subjects who received MSD-HSCT, Haplo-HSCT and UD-HSCT achieved hematopoietic reconstitution. Of them, the recovery of neutrophil and platelet were not significantly different（P<0.05）. Patients receiving UCB-HSCT had delayed recovery of hematopoiesis, and a significantly reduced reconstruction rate, when compared with those in the other 3 groups （P<0.01）. However, 4 patients undergoing UCB- HSCT presented with autologous hematopoiesis, a period of time after the failure of hematopoietic reconstitution. The expected 5- year survival rates after MSD- HSCT, Haplo- HSCT, UD- HSCT and UCB- HSCT were 70.0%, 81.0%, 88.9% and 77.8%, respectively（P>0.05）.
Conclusion: MSD-HSCT, Haplo-HSCT and UD-HSCT had no statistically significance in terms of hematopoietic reconstitution or prognosis. Although hematopoietic reconstitution of UCB-HSCT was lower than other transplantation types, but no significant difference in overall prognosis. So if HLA-matched sibling donor is not available, SAA patients can choose Haplo- HSCT, UD - HSCT or UCB- HSCT with comparable efficacy to MSD- HSCT, as an alternative therapy.
目的: 比较HLA相合同胞供者造血干细胞移植(MSD-HSCT)、亲缘半相合HSCT(Haplo-HSCT)、无关供者HSCT(UD-HSCT)和脐血HSCT(UCB-HSCT)四种不同移植方式治疗重型再生障碍性贫血(SAA)造血重建以及预后的差异。
方法: 回顾性分析2008年5月8日至2013年12月13日在苏州大学附属第一医院接受HSCT治疗的63例SAA患者临床资料，分别比较不同移植方式对移植后造血重建、移植物抗宿主病(GVHD)发生及5年总生存(OS)率的影响。
结果: 53例行MSD-HSCT、Haplo-HSCT和UD-HSCT的患者全部获得造血重建，其中性粒细胞和血小板植入时间比较，差异无统计学意义(P=0.111和0.577)。UCB-HSCT中性粒细胞和血小板植入时间分别为19(18~29) d及32(24~70) d，明显长于其他移植方式(P值均<0.01)；植入率分别为42.0％及42.0%，明显低于其他移植方式(P值均<0.01)。但是造血重建失败的4例UCB-HSCT患者在一段时间后均出现自体造血重建。MSD-HSCT、Haplo-HSCT、UD-HSCT和UCB-HSCT 5年预期生存率分别为70.0%、81.0%、88.9%和77.8%，差异均无统计学意义(P>0.05)。
结论: MSD-HSCT、Haplo-HSCT和UD-HSCT 3种移植方式造血重建及预后方面差异均无统计学意义。UCB-HSCT虽然造血重建率明显低于其他移植方式，但总体预后无明显差异，因此对于无HLA相合同胞供者的SAA患者，Haplo-HSCT、UD-HSCT及UCB-HSCT作为替代治疗疗效与MSD-HSCT相当。