An open-label extension study evaluating the safety and efficacy of romiplostim for up to 3.5 years in thrombocytopenic Japanese patients with immune thrombocytopenic purpura (ITP)

摘要

Long-term use of the thrombopoietin mimetic romiplostim was examined in Japanese patients with chronic immune thrombocytopenic purpura (ITP) in this open-label extension. The starting dose of romiplostim was the previous trial dose or 3 μg/kg/week, which was titrated up to 10 μg/kg/week to maintain platelet counts between 50 and 200 × 10 9/L. As of April 2010, 44 patients had enrolled; 71 % women, median age 55.5 years, with five patients discontinuing romiplostim due to patient request (2), administrative decision (2), or not achieving studydefined platelet response (1). Median treatment duration was 100 weeks; median average weekly dose was 3.8 μg/kg. Twenty-eight patients (64 %) self-injected romiplostim. The most frequent adverse events were nasopharyngitis and headache. Nine patients (20 %) had a total of 14 serious adverse events (0.31/100 patient-weeks); of these, only oral hemorrhage was considered treatment related. Fifty hemorrhagic adverse events were reported in 20 patients (46 %) (1.12/100 patient-weeks). Ninety-six percent of patients had a platelet response (doubling of baseline platelet count and platelet count ≥50 × 10 9/L). Of the 25 patients receiving concurrent ITP therapy at baseline, all reduced or discontinued the therapy. Eight patients (18 %) received rescue medications. Administration of up to 3.5 years of romiplostim increased platelet counts and was well tolerated in Japanese patients with chronic ITP.