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

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.