血小板生成素受体激动剂治疗免疫性血小板减少症的新药研发进展

Progress in new drug research and development of thrombopoietin receptor agonists against immune thrombocytopenia

免疫性血小板减少症(immune thrombocytopenia,ITP)是一种免疫介导的血小板过度破坏或生成不足导致的常见出血性疾病,以血小板减少和出血为主要症状。临床上包括以糖皮质激素、免疫抑制剂的药物治疗及脾脏切除术的外科手术治疗,从而减少血小板的破坏。随着对疾病机制的深入了解,针对巨核细胞增殖分化以增加血小板生成的一类药物血小板生成素受体激动剂(thrombopoietin receptor agonists,TPORAs,TRAs)成为ITP治疗的新途径。本文对TRAs类药物治疗ITP的新药研发现状进行综述。

Immune thrombocytopenia （ITP） is an acquired common hemorrhagic condition characterized by a low platelet count and hemorrhage,which is caused by excessive platelet destruction and thrombopoiesis deficiency.Clinical therapy includes pharmacotherapy with corticosteroids and immunosuppressants and surgical treatment of splenectomy in order to reduce the destruction of platelets.With the deep understanding of the pathogenesis and etiology of ITP,thrombopoietin receptor agonists （TPO-RAs,TRAs） become a new way of ITP treatment,which increases platelet production by stimulating megakaryocytes.In this article,the status of research and development of TRAs for the treatment of ITP is reviewed.