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Romiplostim in patients with refractory aplastic anaemia previously treated with immunosuppressive therapy: A dose-finding and long-term treatment phase 2 trial

The Lancet Haematology Sep 06, 2019

Lee JW, Lee SE, Jung CW, et al. - Researchers undertook this open-label, phase 2 study to evaluate romiplostim with respect to its activity and safety in patients with aplastic anaemia who had earlier received immunosuppressive therapy. The study was run in 2 clinical centres in Seoul, South Korea. The study comprised of a dose-finding part and an extension part. In the dose-finding part, they randomly allocated patients to fixed dose cohorts (1, 3, 6, or 10 μg/kg) of subcutaneous romiplostim once weekly for 8 weeks, based on a static allocation procedure following stratification by platelet count. The extension part involved continuation of taking romiplostim titrated every 4 weeks in single steps (1, 3, 6, 10, 13, 16, and 20 μg/kg once weekly), based on platelet response and safety up to 1 year (weeks 9–52). For all patients, the median duration of treatment was 53 weeks (IQR 35–155). Among patients with refractory aplastic anaemia, romiplostim appeared to be active. Also, it showed a favourable safety profile in these patients. In future investigations, the recommended starting dose might be 10 μg/kg once weekly.
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