Al-Samkari Hanny, Kuter David J
Division of Hematology, Massachusetts General Hospital, Harvard Medical School, Suite 118, Room 112, Zero Emerson Place, Boston, MA 02114, USA.
Division of Hematology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.
Ther Adv Hematol. 2019 Apr 11;10:2040620719841735. doi: 10.1177/2040620719841735. eCollection 2019.
The thrombopoietin receptor agonists (TPO-RAs) are a class of platelet growth factors commonly used to treat immune thrombocytopenia (ITP). There are three agents that have been investigated for the treatment of chronic ITP: the peptide agent romiplostim and the small molecule agents eltrombopag and avatrombopag. These agents offer a higher clinical response rate than most other ITP therapies but may require indefinite use. This review is a critical appraisal of the TPO-RAs in adult ITP, defining the optimal patient groups to receive these agents and assisting the hematologist with agent choice, goals of treatment, dosing strategies, and toxicity management. Use of endogenous thrombopoietin levels to predict response to eltrombopag and romiplostim treatment is discussed and alternative dosing protocols suited for certain patient subgroups are described. Finally, indications for discontinuation and combination therapy with other agents are considered.
血小板生成素受体激动剂（TPO-RAs）是一类常用于治疗免疫性血小板减少症（ITP）的血小板生长因子。有三种药物已被研究用于治疗慢性ITP：肽类药物罗米司亭以及小分子药物艾曲泊帕和阿伐曲泊帕。这些药物比大多数其他ITP治疗方法具有更高的临床缓解率，但可能需要长期使用。本综述对成人ITP中TPO-RAs进行了批判性评估，确定了接受这些药物的最佳患者群体，并协助血液科医生进行药物选择、治疗目标、给药策略和毒性管理。讨论了使用内源性血小板生成素水平预测对艾曲泊帕和罗米司亭治疗反应的情况，并描述了适用于某些患者亚组的替代给药方案。最后，考虑了停药指征以及与其他药物的联合治疗。
