Bussel James B
Weill Cornell Medical College, 525 East 68th Street, P695, New York, NY 10065, USA.
Best Pract Res Clin Haematol. 2014 Sep-Dec;27(3-4):288-92. doi: 10.1016/j.beha.2014.10.012. Epub 2014 Oct 15.
The two generations of thrombopoietin (TPO) receptor (R) agonists have had utility in a number of hematologic conditions. However their use has often been surprisingly complex and drawbacks have been revealed in certain conditions more than in others. The first-generation megakaryocyte growth and development factor (MGDF) was discontinued due to the production of antibodies against it that cross-reacted with native TPO. Nonetheless it was tested in a wide variety of thrombocytopenic conditions and showed unequivocal efficacy in increasing the number of platelets in certain ones. As a result of lessons learned with MGDF, second-generation TPO-R agonists romiplostim and eltrombopag were initially tested and have been approved for the treatment of chronic immune thrombocytopenia (ITP), thrombocytopenia in hepatitis C, and recently aplastic anemia. These agents have had more mixed outcomes in diseases such as acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). Results of several studies will be discussed.
两代血小板生成素（TPO）受体（R）激动剂已在多种血液系统疾病中发挥作用。然而，它们的使用往往出人意料地复杂，且某些情况下的缺点比其他情况更为明显。第一代巨核细胞生长和发育因子（MGDF）因产生与天然TPO发生交叉反应的抗体而停用。尽管如此，它仍在多种血小板减少症中进行了测试，并在某些情况下明确显示出增加血小板数量的疗效。鉴于从MGDF中吸取的经验教训，第二代TPO-R激动剂罗米司亭和艾曲泊帕最初进行了测试，并已获批用于治疗慢性免疫性血小板减少症（ITP）、丙型肝炎相关性血小板减少症，以及最近的再生障碍性贫血。在急性髓系白血病（AML）和骨髓增生异常综合征（MDS）等疾病中，这些药物的疗效更为参差不齐。将讨论几项研究的结果。
