重型再生障碍性贫血的治疗与造血干细胞移植  

Treatment of severe aplastic anemia and hematopoietic stem cell transplantation

作　　者：李燕娟 张连生[1] 李莉娟[1] Li Yanjuan;Zhang Liansheng;Li Lijuan(Department of Hematology,Lanzhou University Second Hospital,the Second Clinical Medical College of Lanzhou University,Lanzhou 730000,China)

机构地区：[1]兰州大学第二医院血液科兰州大学第二临床医学院,兰州730000

出　　处：《器官移植》2023年第3期442-448,共7页Organ Transplantation

基　　金：国家血液系统疾病临床医学研究中心委托课题(2021WWA01);甘肃省科技计划项目(21JR11RA104);兰州市科技计划项目(2020-ZD-99);兰州大学第二医院萃英科技创新项目(CY2017-ZD04、CY2019-MS14)。

摘　　要：重型再生障碍性贫血(SAA)是由多种病因导致的重度骨髓造血功能衰竭综合征,临床以严重的贫血、感染、出血为主要特征。SAA发病机制复杂,至今尚未完全明了。SAA起病急,病情重,病情进展快,目前随着对SAA的深入研究以及诊疗水平的提高,对于其治疗策略也发生了改变。从经典的免疫抑制治疗,即主要以抗胸腺细胞球蛋白和环孢素为基础的治疗方案,到血小板生成素受体激动剂的应用及以异基因造血干细胞移植等为基础的联合治疗方案,均不同程度促进SAA患者的造血功能重建,极大改善其生存及预后,成为当下SAA治疗的研究热点。本文结合国内外文献对SAA治疗的新进展进行综述。Severe aplastic anemia(SAA)is a severe bone marrow failure syndrome caused by multiple causes,which is clinically manifested with severe anemia,infection and bleeding.The complex pathogenesis of SAA has not been fully understood.SAA is characterized with acute onset,severe disease condition and rapid progression.At present,with the in-depth study of SAA and the improvement of diagnosis and treatment,the therapeutic strategy for SAA has been evolved from classical immunosuppressive therapy based on antithymocyte globulin and cyclosporine to the application of thrombopoietin receptor agonist and combined treatment based on allogeneic hematopoietic stem cell transplantation,which may promote the reconstruction of hematopoietic function of SAA patients to varying degree and significantly improve survival and clinical prognosis,becoming the research hotspot of SAA treatment.In this article,new advances in the treatment of SAA at home and abroad were reviewed.

关 键 词：重型再生障碍性贫血 免疫抑制疗法 异基因造血干细胞移植 单倍体造血干细胞移植 脐血造血干细胞移植 抗胸腺细胞球蛋白 艾曲波帕 西罗莫司 阿仑单抗 环磷酰胺 

分 类 号：R617[医药卫生—外科学] R551[医药卫生—临床医学] R556.5