Friday, December 03, 2010
romiplostin thrombocytopenia
Original Article
Romiplostim or Standard of Care in Patients with Immune Thrombocytopenia
N Engl J Med 2010; 363:1889-1899November 11, 2010
Background
Romiplostim, a thrombopoietin mimetic, increases platelet counts in patients with immune thrombocytopenia, with few adverse effects.
Methods
In this open-label, 52-week study, we randomly assigned 234 adult patients with immune thrombocytopenia, who had not undergone splenectomy, to receive the standard of care (77 patients) or weekly subcutaneous injections of romiplostim (157 patients). Primary end points were incidences of treatment failure and splenectomy. Secondary end points included the rate of a platelet response (a platelet count >50×109 per liter at any scheduled visit), safety outcomes, and the quality of life.
Results
The rate of a platelet response in the romiplostim group was 2.3 times that in the standard-of-care group (95% confidence interval [CI], 2.0 to 2.6; P<0.001).>
Conclusions
Patients treated with romiplostim had a higher rate of a platelet response, lower incidence of treatment failure and splenectomy, less bleeding and fewer blood transfusions, and a higher quality of life than patients treated with the standard of care. (Funded by Amgen; ClinicalTrials.gov number, NCT00415532.)
Dr. Kuter reports receiving consulting fees from Amgen, GlaxoSmithKline, Ligand, Pfizer, Eisai, MGI Pharma, Shionogi, and Ono Pharmaceutical and research support from Amgen, GlaxoSmithKline, Eisai, and Shionogi; Dr. Boccia reports receiving speaker's fees, consulting fees, and research support from Amgen; speaker's fees from GlaxoSmithKline; and holding equity in Amgen and Johnson and Johnson; Dr. Pabinger reports receiving consulting fees from Amgen and GlaxoSmithKline and helping to develop speakers' bureau programs for Amgen; Dr. Selleslag reports receiving consulting fees and speaker's fees from Amgen; Dr. Rodeghiero reports receiving research support from and serving on the boards of Amgen, GlaxoSmithKline, and Shionogi, as well as receiving speaker's fees from Amgen and GlaxoSmithKline; Dr. Chong reports serving on the board, receiving honoraria, and receiving research support from Amgen, GlaxoSmithKline, and Commonwealth Serum Lab; and Drs. Wang and Berger report being employees of, and holding equity in, Amgen.
Disclosure forms provided by the authors are available with the full text of this article at NEJM.org.
