TOPLINE:

Eltrombopag combined with immunosuppressive therapy in pediatric patients with severe aplastic anemia achieved a 54.9% overall response rate at 26 weeks, with 71.4% response in relapsed/refractory patients and 48.6% in treatment-naive patients. Among transfusion-dependent patients, 66.7% and 76.7% achieved red blood cell and platelet transfusion independence, respectively.

METHODOLOGY:

A phase 2, open-label, noncontrolled, interpatient dose-escalation study evaluated eltrombopag combined with immunosuppressive therapy in pediatric patients aged 1 to < 18 years with relapsed/refractory or treatment-naive severe aplastic anemia.

Initial eltrombopag dosing started at 25 mg/d for patients aged 1 to < 6 years and at 50 mg/d for patients aged 6 to < 18 years, with allowed dose modif

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