药物诱导胎儿血红蛋白(HbF)治疗β地中海贫血研究进展  

Reaserch Advances on Induction of Fetal Hemoglobin(HbF)by Drugs in the Treatment of β-Thalassemia——Review

作　　者：李丽 孙志强[1,2] LI Li;SUN Zhi-Qiang(Department of Hematology,Shenzhen Hospital,Southern Medical University,Shenzhen 518110,Guangdong Province,China;The Third School of Clinical Medicine,Southern Medical University,Guangzhou 510515,Guangdong Province,China)

机构地区：[1]南方医科大学深圳医院血液科,广东深圳518110 [2]南方医科大学第三临床医学院,广东广州510515

出　　处：《中国实验血液学杂志》2020年第4期1424-1428,共5页Journal of Experimental Hematology

摘　　要：地中海贫血是β珠蛋白基因缺陷所致的遗传性血红蛋白病,近年的研究重新提及了药物诱导胎儿血红蛋白(HbF)的治疗意义,主要通过增加γ链的表达,减轻α、β链的不平衡,从而改善贫血严重程度。药物试验,如羟基脲、沙利度胺和地西他滨,均显示治疗后血红蛋白升高,输血依赖降低,贫血以外的症状减轻。另外,体外实验则提示,其他药物亦具有诱导HbF的潜能,这为安全有效的HbF诱导剂提供了重要线索。本文就目前的研究进展作一综述,以期有助于临床治疗。β-thalassaemias are inherited hemoglobin disorders caused by defects in theβ-globin gene.In recent years,researches have re-mentioned the therapeutic significance of drug-induced fetal hemoglobin(HbF),which can reduce the imbalance ofαandβchains and improve the severity of anemia by increasing the expression ofγchain.Drug trials,such as hydroxyurea,thalidomide and desitabine have shown elevated hemoglobin,decreased blood transfusion dependence,and reduced symptoms other than anemia after treatment.In addition,in vitro experiments suggested that HbF can also induce by other drugs,which providing important clues for safe and effective HbF inducers.Therefore,this article reviews the current research progress so as to expect beneficial to clinical treatment.

关 键 词：Β地中海贫血 胎儿血红蛋白 药物治疗 羟基脲 沙利度胺 地西他滨 

分 类 号：R556.61[医药卫生—血液循环系统疾病]