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In up to one third of children with immune thrombocytopenia (ITP), the condition persists >12 months after diagnosis. Current therapies include corticosteroids, other immunosuppressive agents, or splenectomy. Eltrombopag, a thrombopoietin-receptor agonist, is approved for treatment of chronic ITP — but only in adults.
Now, in an industry-sponsored, multinational, two-part trial, researchers have evaluated this agent in children aged 1 to 17 years with chronic ITP (platelet counts <30×109/L). Nearly all of the 92 children, including 4 with splenectomy, had previously received treatment for the condition. Participants were randomized in a 2:1 ratio to receive eltrombopag or placebo during a 13-week double-blind period.
Forty percent of the eltr…