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

Abstract

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, has shown efficacy in clinical trials but lacks extensive real-world data, particularly in Central and Eastern Europe (CEE). This study aims to evaluate the effectiveness and safety of AVA in routine clinical practice for ITP patients across CEE countries.

Methods: A multicenter, noninterventional, 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 (W8).

Results: AVA treatment resulted in a 68.3% platelet response rate by W8, 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 favorable safety profile, supporting its use across different ITP stages and patient demographics.