重型再生障碍性贫血患者造血干细胞移植的临床研究进展  

Clinical research progress of hematopoietic stem cell transplantation in patients with severe aplastic anemia

作　　者：张东华[1] ZHANG Dong-hua(Department of Haematology,Tongji Hospital,Tongji Medical College,Huazhong University of Science and Technology,Wuhan 430030,China)

机构地区：[1]华中科技大学同济医学院附属同济医院血液科,湖北武汉430030

出　　处：《中国实用内科杂志》2021年第10期828-831,共4页Chinese Journal of Practical Internal Medicine

基　　金：卫生公益性行业科研专项经费项目(201202017)。

摘　　要：异基因造血干细胞移植(allo-HSCT)是治疗重型再生障碍性贫血(SAA)最有效的方法之一。然而,在同胞兄弟姐妹中,只有不到30%的全相合供者可行allo-HSCT。近年来,替代供者(包括无血缘、脐带血和单倍体等供者)的HSCT进展迅速,特别是在预处理方案的不断探索与完善[如北京方案,或后置环磷酰胺(PT-CY)方案],以及强有力的支持治疗等措施,使allo-HSCT技术明显提高,供者来源更容易。这些为SAA患者作为二线甚至一线治疗选择allo-HSCT提供了可能,对年轻患者获益更多。有研究显示同胞供者HSCT或单倍体HSCT更有益于SAA分型程度更严重的患者。此外,以TPO激动剂——艾曲波帕(Eltrombopag,EPAG)为代表的新药,不仅与强化免疫抑制治疗(IST)联合用于SAA,促进造血恢复;也有益于HSCT疗效的提高:初步的对比研究提示,HSCT似乎有益于SAA患者。HSCT研究的进展极大地改善SAA的临床疗效及预后。Allogenic hematopoietic stem cell transplantation(allo-HSCT)is one of the most effective treatments for severe aplastic anemia(SAA).However,among siblings,less than 30%of all are suitable matched sibling donors(MSD)for alloHSCT.In recent years,HSCT of alternative donors(including HLA-matched unrelated donors,cord blood,and haploidentical donors)has greatly progressed.In particular,the continuous exploration and improvement of conditioning regimens[such as Beijing regimen or post-transplant cyclophosphamide(PT-Cy)regimen],as well as strong supportive treatment and other measures,have significantly improved the technology of allo-HSCT and made donor sources easier.These offer the possibility of allo-HSCT as a second-line or even first-line treatment option for SAA patients,which can benefit younger patients more.The studies have shown that sibling donor HSCT or haploidentical donor HSCT were more beneficial to patients with a more severe type of VSAA(very SAA).In addition,new drugs represented by eltrombopag(EPAG),the agonist of TPO,can not only be used in combination with immunosuppressant(IST)in SAA to promote hematopoietic recovery but also beneficial to improve the efficacy of HSCT.Preliminary control study suggest that HSCT seems to benefit SAA patients.Advances in HSCT studies have greatly improved the clinical efficacy and prognosis of SAA.

关 键 词：重型再生障碍性贫血 造血干细胞移植 进展 

分 类 号：R551.3[医药卫生—血液循环系统疾病]