Avatrombopag in immune thrombocytopenia and beyond: Current evidence and emerging perspectives

Abstract

Immune thrombocytopenia (ITP) is a rare autoimmune disorder characterized by reduced platelet counts, increased bleeding risk, and impaired quality of life. Thrombopoietin receptor agonists are established second-line therapies; among these, avatrombopag is an oral agent supported by its phase 3 clinical evidence base. In adults with ITP, avatrombopag has demonstrated a rapid onset of action, durable platelet response, and favorable safety profile. A subsequent trial in pediatric populations confirmed the efficacy and safety of avatrombopag was consistent with the adult data. Real-world studies further reinforce these findings, supporting avatrombopag's profile in routine clinical practice. Beyond ITP, emerging evidence suggests potential roles for avatrombopag in other thrombocytopenic settings, including chemotherapy-induced thrombocytopenia, aplastic anemia, and post–hematopoietic stem-cell transplantation, where early data appear promising.