Eltrombopag (ELTR), a thrombopoietin receptor agonist, combined with immunosuppressive therapy (IST) yields a 54.9% overall response rate in pediatric severe aplastic anemia (SAA) at 26 weeks.
Patients with relapsed/refractory SAA demonstrate higher response rates (71.4%) compared to treatment-naive cohorts (48.6%).
Significant transfusion independence is achieved in transfusion-dependent patients: approximately two-thirds for red blood cells and over three-quarters for platelets.
Safety profiles remain manageable, primarily hepatic enzyme elevations without new safety signals.
