a Department of Hematology , Jagiellonian University , Krakow , Poland.
Expert Rev Clin Immunol. 2019 Apr;15(4):327-339. doi: 10.1080/1744666X.2019.1587294. Epub 2019 Mar 8.
Thrombopoietin-receptor agonists (TPO-RAs) are the only American Society of Hematology (ASH) guideline-advocated, second-line treatment for immune thrombocytopenia (ITP) that have been validated by randomized, controlled trials with a placebo comparator. Avatrombopag is a new candidate in this class that has been investigated as a treatment option for the treatment of ITP. Areas covered: In this Drug Profile, we provide a review of the clinical data of avatrombopag, which was approved in May 2018 by the United States Food and Drug Administration (FDA) for the treatment of thrombocytopenia in patients with chronic liver disease undergoing an invasive procedure, and an opinion of its potential place in the current evidence-based ITP treatment landscape. Expert commentary: Avatrombopag induces doubling of platelet counts, increasing them to above 50 X 10/L, and prevents the need for platelet transfusions while minimizing the need for rescue medications. Treatment-emergent adverse events (TEAEs) are comparable to placebo. Oral delivery, a 5-day dosing schedule and good tolerability (<1% discontinuation rate) with no clinically significant hepatoxicity make it a promising entrant as a potential second-line treatment for ITP. Further, data from a phase 3 study in patients with ITP supports its utility in the treatment of patients with ITP.
血小板生成素受体激动剂（TPO-RAs）是唯一经美国血液学会（ASH）指南推荐的、经随机对照试验与安慰剂对照验证的二线治疗免疫性血小板减少症（ITP）的药物。avatrombopag 是该类药物中的一种新药，已被研究作为 ITP 的治疗选择。
在这篇药物简介中，我们回顾了 avatrombopag 的临床数据，该药于 2018 年 5 月获得美国食品和药物管理局（FDA）批准，用于接受有创性操作的慢性肝病患者的血小板减少症的治疗，并且我们对其在当前基于证据的 ITP 治疗领域中的潜在地位提出了看法。
avatrombopag 可使血小板计数增加一倍，将血小板计数增加到 50×10/L 以上，并可预防血小板输注的需要，同时最大限度地减少对抢救药物的需求。治疗中出现的不良事件（TEAEs）与安慰剂相当。口服给药、5 天的给药方案以及良好的耐受性（<1%的停药率），无明显的肝毒性，使其成为治疗 ITP 的二线药物的一个有前途的候选药物。此外，一项 3 期研究在 ITP 患者中的数据支持其在 ITP 患者治疗中的效用。
