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.