Abstract:

Allogeneic-hematopoietic stem cell transplantation（HSCT） is the most important or even the only radical cure for some primary immunodeficiency diseases. The long-term survival is 90% or even higher with HLA matched sibling donor transplantation. In total，the overall survival for the patients with severe combined immunodeficiency disease，Wiskott-Aldrich sydrome and chronic granulomatous disease is 70%，80% and 90%，respectively. Proper conditioning regimen promotes the success of transplantation，while severe graft versus host disease and reactivation of cytomegalovirus in patients negetively affect the overall survival and life quality after transplantation. This review summerized the rencent advances in allogeneic-HSCT for primary immunodeficiency patients with respect to alternative donor transplantation，conditiong regimen consideration，prevention and treatment of graft versus host disease and reactivation of cytomegalovirus.

Key words: hematopoietic stem cell transplantation, primary immunodeficiency disease, graft versus host disease, cytomegalovirus

摘要：

异基因造血干细胞移植（HSCT）是根治部分原发性免疫缺陷病（PID）的重要甚至惟一手段。人类白细胞抗原（HLA）全相合的同胞供者移植后PID患者的长期存活率可达90%以上。国际上重症联合免疫缺陷病（SCID）移植后的长期存活率在70%以上，Wiskott-Aldrich综合征患者移植后的总体存活率在80%以上，慢性肉芽肿病患者接受HLA全相合移植后长期存活率可达90%以上。预处理方案是决定HSCT成功与否的重要因素，移植物抗宿主病及巨细胞病毒的复燃严重影响患者干细胞移植后的存活率及生活质量。文章对近年HSCT治疗PID患者在非HLA全相合同胞供者移植、预处理方案、移植物抗宿主病的防治、巨细胞病毒复燃的防治方面的进展进行介绍。

关键词: 造血干细胞移植, 原发性免疫缺陷病, 移植物抗宿主病, 巨细胞病毒