Thrombopoietin receptor agonists (TPO-RAs) are currently indicated for continuous treatment of chronic primary immune thrombocytopenia (ITP). However, there is growing evidence that TPO-RAs can also trigger sustained response in 10–30% of cases after treatment tapering and discontinuation. Therefore, at least for selected responding patients, it might be rational to plan TPO-RA interruption to exploit off-treatment response. Intriguingly, complete or partial responses with TPO-RAs are frequently observed when treatments are initiated early, suggesting that unknown immune-related mechanisms may be involved in this phenomenon. The sustained responses observed after interruption of TPO-RAs may be interpreted as a recovery of immunological tolerance; thus, the re-establishment of immunological equilibrium might be primarily responsible for the observed off-treatment effect. Importantly, these findings may indicate that anticipated TPO-RA usage can lead to improved responses, and that optimized tapering and interruption in selected patients can furthermore improve prognoses. On the base of this rationale, a series of real-life considerations have been generated by a panel of Experts to elucidate possible novel criteria and modalities to identify subgroups of patients who can benefit from tapering and/or discontinuation of TPO-RAs. Towards this aim, the results of a survey of ITP experts are herein reported, reflecting a snapshot of current real-life experience on early discontinuation of TPO-RA-based therapy. The present manuscript also highlights the importance of future translational studies on novel prognostic and predictive biomarkers that can stratify patients and facilitate the clinical choice for second-line treatment of ITP.

目前表明血小板生成素受体激动剂（TPO-RA）可用于慢性原发性免疫性血小板减少症（ITP）的连续治疗。但是，越来越多的证据表明，在逐渐减少和终止治疗后的10％至30％的病例中，TPO-RAs也可以触发持续应答。因此，至少对于选定的反应患者，计划TPO-RA中断以利用非治疗反应可能是合理的。有趣的是，在早期开始治疗时，经常会观察到TPO-RA的完全或部分反应，这表明这种现象可能与未知的免疫相关机制有关。TPO-RAs中断后观察到的持续反应可以解释为免疫耐受的恢复；因此，免疫平衡的重新建立可能是造成观察到的治疗效果的主要原因。重要的是，这些发现可能表明，预期的TPO-RA用法可导致改善的反应，并且选定患者的最佳渐缩和中断可进一步改善预后。在此基础上，专家小组提出了一系列现实生活中的考虑因素，以阐明可能的新标准和方法，以识别可从TPO-RA的逐渐减量和/或停药中受益的患者亚组。为了实现这一目标，在此报告了ITP专家的调查结果，反映了当前在基于TPO-RA的疗法的早期停用方面的现实经验。