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The thrombopoietin receptor agonist eltrombopag, which has been approved to treat adults with chronic immune thrombocytopenia, is safe and effective in children, according to the phase 3 PETIT2 trial conducted at 38 centers in 12 countries (Grainger JD et al. Lancet. doi:10.1016/S0140-6736(15)61107-2 [published online July 28, 2015]). Immune thrombocytopenia, an autoimmune disease that destroys platelets, can lead to excessive bruising or bleeding. Approximately 5 in 100 000 children have the disease, with 13% to 36% developing chronic immune thrombocytopenia. The most common treatments include immunosuppression with corticosteroids, rituximab, and splenectomy.
Slomski A. Eltrombopag Benefits Children With Chronic Immune Thrombocytopenia. JAMA. 2015;314(20):2122. doi:10.1001/jama.2015.15714