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The thrombopoietin receptor agonist romiplostim is effective and safe for the treatment of adult patients with immune thrombocytopenia (IT; NEJM JW Oncol Hematol Jan 2011, and N Engl J Med 2010 Nov 11; 363:1889). IT in children is often short-lived and does not require therapeutic interventions, but if the thrombocytopenia becomes chronic, treatment with corticosteroids, intravenous immunoglobulin, or rituximab is usually considered.
To determine whether romiplostim is safe and effective in children with chronic IT, investigators conducted an industry-supported, phase III, randomized, double-blind, placebo-controlled study, in which 62 patients with platelet counts ≤30,000/µL and disease of >6 months duration received weekly romiplostim (42 …