Avatrombopag for the Treatment of Immune Thrombocytopenia.

Avatrombopag, a second-generation thrombopoietin receptor agonist (TPO-RA), represents a significant advancement in the treatment of chronic immune thrombocytopenic purpura (cITP) and a potential therapeutic option for other thrombocytopenic disorders. Approved in both the USA and Europe, avatrombopag offers a convenient oral dosing regimen, initiated at 20 mg daily with food, to achieve and maintain platelet counts ≥ 50 × 10/L. Its favorable safety profile, characterized by minimal hepatic toxicity and the absence of dietary restrictions, distinguishes it from older TPO-RAs such as eltrombopag and romiplostim. Clinical trials and real-world data support its efficacy, with over 90% of patients that fail to standard first- and second-line treatments or become unresponsive, achieving target platelet counts, and its hepatotoxicity-free profile makes it particularly advantageous for patients with liver disease or complex comorbidities. Economic evaluations, including a budget impact analysis for the Italian National Health Service, have projected significant healthcare cost savings associated with avatrombopag use, reinforcing its value as a cost-effective therapeutic option. However, challenges remain, including limited data on long-term safety. In this review, we aim to provide a comprehensive synthesis of clinical evidence and real-world data on avatrombopag's efficacy, safety, and pharmacological advantages, while exploring its current and potential therapeutic applications, such as chemotherapy-induced thrombocytopenia and aplastic anemia.

© 2025 The Author(s). European Journal of Haematology published by John Wiley & Sons Ltd.