Amgen announced published results from a Phase 3 study of Nplate (romiplostim) for the treatment of symptomatic immune thrombocytopenia (ITP) in children. Study data were published in The Lancet.

The Phase 3 study was a randomized (2:1), double-blind, placebo-controlled trial of 62 children who have had ITP for more than 6 months. Patients received either Nplate or placebo weekly for 24 weeks, and were permitted to use the same standard-of-care therapy, dose and schedule from when screening platelet counts were measured. The primary endpoint was durable platelet response, defined as achieving weekly platelet responses (increased platelets) without rescue medication in ≥6 weeks of the final 8 weeks. Secondary endpoints included evaluation of overall platelet response, total number of weekly platelet responses, use of ITP rescue medications, composite bleeding episodes and the overall safety of Nplate.

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Results from the study showed that by the final 8 weeks, 52% of children treated with Nplate achieved a durable platelet response vs. 10% of children treated with placebo (OR 9.1, 95% CI: 1.9, 43.2; P=0.002), meeting the study’s primary endpoint.Rates of overall platelet response with Nplate were 71% vs. 20% with placebo (OR 9, 95% CI: 2.5, 32.3; P=0.0002), and rates of any platelet response with Nplate were 81% vs. 55% with placebo (P=0.0313).

Nplate is a thrombopoietin receptor agonist. It is currently indicated for the treatment of low blood platelet counts in adults with chronic ITP, the first FDA-approved therapy for this indication.

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