Department of Pediatrics, Columbia University Medical Center, New York, New York, United States.
Hamostaseologie. 2019 Aug;39(3):272-278. doi: 10.1055/s-0038-1676129. Epub 2019 Jan 15.
Management of patients with corticosteroid-refractory immune thrombocytopaenia (ITP) possesses a significant challenge to practitioners. Until recently, options included splenectomy and immunosuppression. With improved knowledge of both thrombopoiesis and the pathophysiology of ITP, novel drug development with thrombopoietin-receptor agonists (TPO-RAs) was undertaken. Two agents, romiplostim and eltrombopag, are currently approved for use in patients with chronic ITP. Both agents have been shown to increase the platelet count, improve health-related quality of life and reduce bleeding symptoms and concomitant medication use. This review will highlight the discovery of TPO-RA agents, appraise key clinical trials and explore future directions.
管理皮质类固醇难治性免疫性血小板减少症（ITP）患者对临床医生来说是一个重大挑战。直到最近，治疗方案还包括脾切除术和免疫抑制治疗。随着对血小板生成和 ITP 病理生理学的认识不断提高，新型药物开发也采用了促血小板生成素受体激动剂（TPO-RAs）。目前有两种药物，罗米司亭和艾曲泊帕，被批准用于慢性 ITP 患者。这两种药物都已被证明能增加血小板计数，改善与健康相关的生活质量，并减少出血症状和同时使用的药物。这篇综述将重点介绍 TPO-RA 药物的发现，评估关键临床试验，并探讨未来的方向。
