慢性特发性血小板减少性紫癜治疗药物罗米司亭和艾曲波帕

The drugs in therapy of chronic idiopathic thrombocytopenic purpura:romiplostim and eltrombopag

在慢性特发性血小板减少性紫癜的治疗中,当患者不能耐受或对传统治疗比如皮质激素、免疫球蛋白以及脾切除等疗效不佳时,能刺激血小板生成增多的药物为患者提供了另外一条治疗途径。第一代血小板刺激药物由于使用后在患者体内产生内源性TPO中和抗体,使患者发生继发性血小板减少而被停止临床试验,第二代药物罗米司亭和艾曲波帕在临床试验中能够安全增加和维持对传统治疗抵抗患者的血小板数量,并且没有内源性中和抗体产生。文中详细介绍了两药分子结构特点、作用机制、药动学、疗效评价以及安全性。

In the treatment of idiopathic thrombocytopenic purpura,newly developed agents that can stimulate platelet production offer an alternative treatment strategy when patients could not tolerate or have a insufficient response to traditional therapies such as corticosteroids,immunoglobulin and splenectomy.The first generation of thrombopoietic agents was discontinued development owing to neutralizing auto-antibodies cross-reacting with endogenous TPO,which can cause secondary thrombocytopenia in patients.In clinical trials,second-generation thrombopoietin-receptor agonists,eltrombopag and romiplostim,safely increase and maintain platelet production in patients with refractory disease,without developing auto-antibodies against TPO.This article introduced the molecule construction features,mechanism of action,pharmacokinetics,clinical efficacy and safety of the two drugs.