造血干细胞移植在先天性溶血性贫血治疗中的应用

The application of hematopoietic stem cell transplantation in treatment of congenital hemolytic anemia

造血干细胞移植(HSCT)是目前惟一能治愈先天性溶血性贫血(如重型地中海贫血和鐮状细胞贫血)的治疗方法。HLA相合同胞供者移植治疗儿童患者的总生存(OS)率超过90%,无事件生存(EFS)率超过80%。无关供者移植使更多无HLA相合同胞供者的患者获得移植机会。

Summary ：Hematopoietic stem cell transplantation （HSCT） remains the only treatment method that can cure congenital he- molytic anemia such as severe thalassemia and sickle cell anemia （SCA） at present. The overall survival （OS） is over 90% in children treated with HLA-identical sibling donor transplantation, while event-free survival （EFS） is over 80%. The e- vent-free donor transplantation enables more patients without HLA-identical sibling donors to obtain transplantation chances.