Supplementary Material for: Tapering and Discontinuation of Thrombopoietin Receptor Agonist Therapy in Patients with Immune Thrombocytopenia: Results from a Modified Delphi Panel

<b><i>Background:</i></b> Recent evidence suggests that in patients with immune thrombocytopenia (ITP) with a stable response on thrombopoietin receptor agonists, treatment may be tapered and/or discontinued. <b><i>Objectives:</i></b> The objective of this study was to provide a guide for tapering and discontinuation of TPO-RA therapy in patients with ITP, based on hematologist survey results, existing evidence, and expert consensus. <b><i>Patients/Methods:</i></b> UK hematologists completed a survey to characterize self-reported practice patterns related to TPO-RA tapering and discontinuation in patients with ITP. Using a modified Delphi panel approach, ITP experts developed consensus statements regarding the use of TPO-RA tapering and discontinuation. <b><i>Results:</i></b> Survey respondents estimated that 30–34% of their patients were suitable for tapering or discontinuation and that 29–35% of these patients required treatment re-initiation after an average treatment-free interval of 86–106 days. No clear predictors of patient suitability or response to tapering or discontinuation were identified. The ITP expert consensus was that approximately 30% of patients are eligible for tapering and discontinuation, which may be considered after 6–12 months for patients demonstrating an adequate treatment response (platelet count &gt;50,000/µL at ≥75% of assessments in the preceding 6 months). Treatment re-initiation may be considered if the platelet count decreases or if the patient becomes symptomatic. Individual differences need to be taken into account when considering TPO-RA tapering or discontinuation. <b><i>Conclusions:</i></b> Tapering and discontinuation of TPO-RA therapy may be considered for certain patients with ITP. Further study is needed to better predict patients likely to achieve sustained off-treatment responses after tapering and discontinuation.**背景**：现有研究证据表明，对于接受血小板生成素受体激动剂（thrombopoietin receptor agonists, TPO-RA）治疗后获得稳定应答的免疫性血小板减少症（immune thrombocytopenia, ITP）患者，可对其治疗方案进行减量乃至停药。**目标**：本研究旨在基于血液科医师的调研结果、现有研究证据及专家共识，为免疫性血小板减少症患者的TPO-RA治疗减量与停药操作提供指导规范。**患者与方法**：英国血液科医师参与了一项调研，以明确其报告的、与免疫性血小板减少症患者TPO-RA治疗减量及停药相关的临床实践模式。本研究采用改良德尔菲法（modified Delphi panel），由免疫性血小板减少症领域专家就TPO-RA减量与停药的临床应用制定共识声明。**结果**：调研受访者估算，其接诊的免疫性血小板减少症患者中，30%~34%适合接受TPO-RA治疗减量或停药；其中29%~35%的患者会在平均86~106天的无治疗间歇期后需要重新启动治疗。本研究未明确识别出可预测患者是否适合减量/停药、或对减量/停药产生应答的明确因素。免疫性血小板减少症领域专家达成的共识为：约30%的患者符合TPO-RA减量与停药的指征，对于在既往6个月内≥75%的评估中血小板计数＞50000/µL且获得充分治疗应答的患者，可在治疗6~12个月后考虑减量或停药。若患者血小板计数下降或出现相关症状，则可考虑重新启动TPO-RA治疗。在考虑对患者实施TPO-RA减量或停药时，需兼顾个体差异。**结论**：对于部分免疫性血小板减少症患者，可考虑实施TPO-RA治疗的减量与停药操作。未来仍需开展进一步研究，以更好地预测哪些患者在减量停药后可获得持续的无治疗应答。