Avatrombopag: más allá de la ficha técnica. Experiencia de un centro

Introduction: Avatrombopag, a thrombopoietin receptor agonist, is approved for chronic immune thrombocytopenia (ITP) in adults and in patients with chronic liver disease prior to surgery. However, its “off-label” use has shown promising results in various clinical scenarios where standard options are limited or contraindicated. Objectives: Report a single center’s experience with avatrombopag used outside its approved indications, exploring its application in different situations. Material and method: A retrospective data collection from electronic medical records, following informed consent. Five detailed clinical cases illustrating the different usage scenarios are presented and analyzed. Results: The presented cases demonstrate avatrombopag’s efficacy and good tolerability in “off-label” situations. Rapid and sustained platelet responses were observed in newly diagnosed ITP, maintenance of safe platelet counts in advanced liver disease, successful surgical preparation, and control of thrombocytopenia in clonal cytopenias of undetermined significance, allowing anticoagulation. Conclusions: Our experience suggests that avatrombopag may be a promising therapeutic option in complex clinical scenarios where conventional options are limited. Its favorable tolerability and efficacy profile in “off-label” uses warrant further investigation through controlled clinical trials to formally validate these emerging indications.