Safety and Efficacy of Romiplostim in Patients With Lower-Risk Myelodysplastic Syndrome and Thrombocytopenia

Abstract

Romiplostim appeared well-tolerated in this study and may be a useful treatment for patients with MDS and thrombocytopenia.

Subcutaneous or intravenous administration of romiplostim in thrombocytopenic patients with lower risk myelodysplastic syndromes

“…Patients who experienced durable platelet responses had fewer clinically relevant bleeding events and fewer platelet transfusions, as reported in Part A of the study. 12 Although most patients had at least 1 adverse event; the most common and serious adverse events were consistent with those in a similar populations of patients with MDS who received treatment with romiplostim or other agents. 6,12,15 Serious adverse events included those that are anticipated in an MDS population and that we did not believe were related to study drug.…”

“…Patients were assigned in the order 1) weekly subcutaneous, 2) biweekly subcutaneous, and 3) weekly intravenous to receive a dose recommended by the Safety Review Panel based on results from Part A of the study. 12 The protocol was reviewed by an institutional ethics committee or review board at each center and over the course of the study by all primary investigators through scheduled teleconferences. The study was conducted in accordance with applicable regulations and the International Conference on Harmonization for Good Clinical Practice guidelines.…”

“…Reduced platelet transfusions and bleeding events were observed in patients who achieved durable platelet responses. 12 These responses were observed in patients independent of their baseline platelet count. Patients who experienced durable platelet responses had fewer clinically relevant bleeding events and fewer platelet transfusions, as reported in Part A of the study.…”

“…At weekly subcutaneous doses of 300 to 1500 lg, romiplostim produced platelet responses in 40% to 50% of patients with lower risk MDS and severe thrombocytopenia who were not receiving other therapy in a phase 1/2 study in which response was defined using modified International Working Group (IWG) criteria. 12,13 In the current study, we evaluated the safety, efficacy, and pharmacokinetics (PK) of romiplostim (Nplate; Amgen, Thousand Oaks, Calif) in thrombocytopenic patients with lower risk MDS. The study comprised a 4-week dose-finding phase (Part A), an 8-week schedulefinding phase (Part B), and a treatment extension phase.…”

“…Data from the patients treated in Part A (including both the 4-week dose-finding phase and the treatment extension phase) have been reported previously. 12 This article reports for the first time on the 8-week schedule-finding phase and the corresponding treatment extension phase data for the Part B patients only, including duration of platelet responses, long-term safety data, and on the PK profiles of intravenous and subcutaneous dosing routes. This trial is registered with the National Institutes of Health as a National Clinical Trial (NCT00303472; http:// www.clinicaltrials.gov; accessed September 28, 2010).…”