埃尔特罗姆博帕格
血小板生成素
罗米普洛斯蒂姆
医学
再生障碍性贫血
血小板生成素受体
骨髓增生异常综合症
免疫学
药理学
肿瘤科
内科学
血小板生成素
血小板
免疫性血小板减少症
造血
生物
骨髓
干细胞
遗传学
作者
James B. Bussel,Austin Kulasekararaj,Nichola Cooper,Amit Verma,Ulrich Steidl,John W. Semple,Britta Will
标识
DOI:10.1053/j.seminhematol.2019.09.001
摘要
The second-generation thrombopoietin (TPO) receptor agonists eltrombopag and romiplostim are potent activators of megakaryopoiesis and represent a growing treatment option for patients with thrombocytopenic hematological disorders. Both TPO receptor agonists have been approved worldwide for the treatment of children and adults with chronic immune thrombocytopenia. In the EU and USA, eltrombopag is approved for the treatment of patients with severe aplastic anemia who have had an insufficient response to immunosuppressive therapy and in the USA for the first-line treatment of severe aplastic anemia in combination with immunosuppressive therapy. Eltrombopag has also shown efficacy in several other disease settings, for example, chemotherapy-induced thrombocytopenia, selected inherited thrombocytopenias, and myelodysplastic syndromes. While both TPO receptor agonists stimulate TPO receptor signaling and enhance megakaryopoiesis, their vastly different biochemical structures bestow upon them markedly different molecular and functional properties. Here, we review and discuss results from preclinical and clinical studies on the functional and molecular mechanisms of action of this new class of drug.
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