Gómez-Almaguer David
Hematology Service, Facultad de Medicina y Hospital Universitario Dr José Eleuterio González, Francisco I. Madero and José E. González, 64460 Monterrey, Mexico.
Ther Adv Hematol. 2018 Oct 4;9(10):309-317. doi: 10.1177/2040620718798798. eCollection 2018 Oct.
Immune thrombocytopenia (ITP) is a bleeding disorder caused by a decrease in platelet count resulting from increased destruction and insufficient production of platelets. Although impaired regulatory T-lymphocyte activity plays a critical role in platelet destruction, many other immunologic abnormalities are also likely to be involved. Importantly, patients with ITP appear to have defects in a thrombopoietin-mediated physiological mechanism that compensates for a decrease in platelet count by increasing platelet production. Thus, simultaneous treatment of multiple pathogenic pathways involved in ITP could potentially result in synergistic efficacy. While conventional treatments for ITP suppress or modulate the immune system to reduce platelet destruction, a unique class of ITP therapy, namely thrombopoietin receptor agonists (TPO-RAs), improves platelet production by activating the thrombopoietin pathway. As hypothesized, preliminary studies show that combinations of eltrombopag, an oral TPO-RA, with conventional treatments improve outcomes in both newly diagnosed and refractory patients. In this review, the clinical experience with eltrombopag-based combinations in patients with ITP is summarized and the implications of the available data are discussed.
免疫性血小板减少症（ITP）是一种出血性疾病，由血小板计数减少引起，其原因是血小板破坏增加和生成不足。尽管调节性T淋巴细胞活性受损在血小板破坏中起关键作用，但许多其他免疫异常也可能参与其中。重要的是，ITP患者似乎在血小板生成素介导的生理机制中存在缺陷，该机制通过增加血小板生成来补偿血小板计数的减少。因此，同时治疗ITP涉及的多种致病途径可能会产生协同疗效。虽然ITP的传统治疗方法是抑制或调节免疫系统以减少血小板破坏，但一类独特的ITP治疗药物，即血小板生成素受体激动剂（TPO-RAs），通过激活血小板生成素途径来改善血小板生成。正如所假设的，初步研究表明，口服TPO-RA艾曲泊帕与传统治疗方法联合使用可改善新诊断患者和难治性患者的治疗效果。在这篇综述中，总结了基于艾曲泊帕的联合治疗在ITP患者中的临床经验，并讨论了现有数据的意义。
