Lozano Maria L, Godeau Bertrand, Grainger John, Matzdorff Axel, Rodeghiero Francesco, Hippenmeyer Jane, Kuter David J
Hospital JM Morales Meseguer, Universidad de Murcia, IMIB-Arrixaca , Murcia, Spain.
Grupo de investigación CB15/00055 del Centro de Investigación Biomedica en Red de Enfermedades Raras (CIBERER), Instituto de Salud Carlos III (ISCIII) , Madrid, Spain.
Expert Rev Hematol. 2020 Dec;13(12):1319-1332. doi: 10.1080/17474086.2020.1850253. Epub 2020 Nov 30.
: Three distinct phases are recognized in immune thrombocytopenia (ITP): newly diagnosed (≤3 months after diagnosis), persistent (>3-12 months after diagnosis), and chronic (>12 months). Several international guidelines/expert recommendations have been released in the past 2 years regarding the treatment of newly diagnosed/persistent ITP. : Across the guidelines/expert recommendations, thrombopoietin receptor agonists (TPO-RAs), including romiplostim (the focus of this review), are recommended in newly diagnosed or persistent ITP for patients who fail to respond to corticosteroids or intravenous immunoglobulin (or where these are contraindicated). To identify data relating to romiplostim in adults with newly diagnosed or persistent ITP, we conducted a search of PubMed (with no time limit applied) and abstracts from 2019 EHA/ASH meetings using the term 'romiplostim.' : The findings from nine clinical trials, six real-world studies and ten case reports provide insight into the early use of romiplostim, which could help to reduce exposure to the adverse effects associated with prolonged corticosteroid use, as well as reduce the risk of severe bleeding. Additionally, given the durable responses observed in patients with newly diagnosed/persistent ITP, as well as the potential for treatment-free responses following discontinuation, romiplostim might help to avoid the need for subsequent treatment.
免疫性血小板减少症(ITP)可分为三个不同阶段:新诊断阶段(诊断后≤3个月)、持续性阶段(诊断后>3 - 12个月)和慢性阶段(诊断后>12个月)。在过去两年中,已经发布了几项关于新诊断/持续性ITP治疗的国际指南/专家建议。在这些指南/专家建议中,对于对皮质类固醇或静脉注射免疫球蛋白无反应(或这些药物有禁忌)的新诊断或持续性ITP患者,推荐使用血小板生成素受体激动剂(TPO - RAs),包括罗米司亭(本综述的重点)。为了识别与新诊断或持续性ITP成年患者中罗米司亭相关的数据,我们使用“罗米司亭”一词在PubMed(无时间限制)以及2019年欧洲血液学协会/美国血液学会会议摘要中进行了检索。九项临床试验、六项真实世界研究和十项病例报告的结果为罗米司亭的早期使用提供了见解,这有助于减少长期使用皮质类固醇相关的不良反应暴露,以及降低严重出血的风险。此外,鉴于在新诊断/持续性ITP患者中观察到的持久反应,以及停药后出现无需治疗的反应的可能性,罗米司亭可能有助于避免后续治疗的需要。