再生障碍性贫血中血小板生成素受体激动剂的应用

Thrombopoietin receptor agonists for aplastic anemia

再生障碍性贫血(aplastic anemia,AA)是一种由T细胞介导的造血功能衰竭性疾病。重型AA(severe AA,SAA)一线治疗方案为人类白细胞抗原相合同胞供者造血干细胞移植或强化免疫抑制疗法(intensive immunosuppressive therapy,IST)联合血小板生成素受体激动剂(thrombopoietin receptor agonists,TPO-RAs)。TPORAs作为一种小分子化合物对初发及难治性SAA、输血依赖非严重型AA疗效及耐受性较好,IST联合小分子TPORAs治疗SAA疗效更佳,严重不良反应及克隆演变发生率未见显著增加,可以做为一线标准治疗方案。

Aplastic anemia(AA)is a kind of hematopoietic failure mediated by T cells.The first-line treatment for sever aplastic anemia(SAA)is human leukocyte antigen matched sibling donor hematopoietic stem cell transplantation(MSDHSCT),or intensive immunosuppressive therapy(IST)combined with thrombopoietin receptor agonists(TPO-RAs).TPORAs,a small molecule compound,is effective and well tolerated in the treatment of primary and refractory SAA,and transfusion dependent non-severe aplastic anemia.IST combined with TPO-RAs has better efficacy,with no significant increase in the incidence of serious adverse reactions and clonal evolution,which can be used as the first-line standard treatment for adult SAA patients who are not suitable for allogeneic hematopoietic stem cell transplantation.