Supplementary Material for: Real-World Insights into Avatrombopag’s Effectiveness and Safety in Adults with Primary Immune Thrombocytopenia: A Retrospective Analysis from Central and Eastern Europe

Introduction: Primary immune thrombocytopenia (ITP) is an autoimmune condition marked by low platelet counts, leading to symptoms such as bleeding. Avatrombopag (AVA), a second-generation thrombopoietin receptor agonist (TPO-RA), has shown efficacy in clinical trials but lacks extensive real-world data, particularly in Central and Eastern Europe (CEE). Objective: This study aims to evaluate the effectiveness and safety of AVA in routine clinical practice for ITP patients across CEE countries. Methods: A multicentre, non-interventional, retrospective analysis was conducted in Slovenia, Croatia and the Czech Republic, involving 41 patients treated with AVA for primary ITP. The primary endpoint was achieving a platelet response at Week 8. Results: AVA treatment resulted in a 68.3% platelet response rate by Week 8, with a significant increase in median platelet counts. Rescue medication use decreased post-AVA initiation, and many patients reduced or discontinued steroids. AVA was well-tolerated, with only one adverse event reported and no significant bleeding events. Conclusion: AVA is effective in improving platelet counts and reducing medication dependency in ITP patients in CEE countries. It offers a viable treatment option with a favourable safety profile, supporting its use across different ITP stages and patient demographics.

引言：原发免疫性血小板减少症（Primary immune thrombocytopenia, ITP）是一种以血小板计数降低为特征的自身免疫性疾病，可引发出血等相关症状。阿伐曲泊帕（Avatrombopag, AVA）作为第二代血小板生成素受体激动剂（thrombopoietin receptor agonist, TPO-RA），已在多项临床试验中证实其疗效，但目前缺乏大规模真实世界数据，尤其是在中东欧地区（Central and Eastern Europe, CEE）。
研究目的：本研究旨在评估阿伐曲泊帕在中东欧国家的常规临床实践中，用于原发免疫性血小板减少症患者的有效性与安全性。
研究方法：本研究在斯洛文尼亚、克罗地亚与捷克共和国开展多中心、非干预性回顾性分析，共纳入41例接受阿伐曲泊帕治疗的原发免疫性血小板减少症患者，其主要研究终点为第8周时达到血小板应答。
研究结果：接受阿伐曲泊帕治疗后，第8周的血小板应答率达68.3%，患者中位血小板计数显著升高。阿伐曲泊帕启动治疗后，急救药物的使用量明显减少，多数患者可减少或停用糖皮质激素。阿伐曲泊帕整体耐受性良好，仅报告1例不良事件，未出现显著出血事件。
研究结论：在中东欧地区的原发免疫性血小板减少症患者中，阿伐曲泊帕可有效提升血小板计数并降低药物依赖程度。该药物安全性良好，是一项可行的治疗选择，支持其在不同疾病阶段与患者人群中的应用。