血小板生成素受体激动剂研发进展及安全性特征

Progress and safety characteristics in thrombopoietin receptor agonists against immune thrombocytopenia

原发免疫性血小板减少症(ITP)是一种免疫介导的出血性疾病,其特征是血小板计数减少和出血风险增加。传统的ITP治疗策略可通过减轻免疫介导的血小板破坏发挥治疗作用,如糖皮质激素、免疫球蛋白、免疫调节剂或脾切除术等。但许多患者对这些疗法响应性差,且与治疗相关的副作用和禁忌证也限制了其使用。近年来,促血小板生成素受体激动剂(TPO-RA)成为ITP治疗新的选择。本文着重讨论TPO-RA的研发进展及安全性特征,旨在为TPO-RA的研发和临床用药提供参考。

Primary immune thrombocytopenia(ITP)is an immune-mediated bleeding disorder characterized by decreased platelet counts and an increased risk of bleeding.Traditional ITP treatment strategies work through mitigating immune-mediated platelet destruction,such as glucocorticosteroids,immunoglobulins,immunomodulatory agents,or splenectomy.Although these strategies are usually effective,many patients have poor responsiveness to these therapies and their use is limited by treatment-related side effects and contraindications.In recent years,thrombopoietin receptor agonists(TPO-RA),by promoting platelet production,has become a new choice for the treatment of ITP.This article focuses on the progress and safety characteristics of TPO-RA,aiming to provide reference for the research and clinical use of TPO-RA.