Nov 11, 2010 - Romiplostim, a thrombopoietin mimetic, increases platelet counts in ... occurred in 23% of patients (35 of 154) receiving romiplostim and 37% of ...
new england journal of medicine The
established in 1812
november 11, 2010
vol. 363 no. 20
Romiplostim or Standard of Care in Patients with Immune Thrombocytopenia David J. Kuter, M.D., D.Phil., Mathias Rummel, M.D., Ralph Boccia, M.D., B. Gail Macik, M.D., Ingrid Pabinger, M.D., Dominik Selleslag, M.D., Francesco Rodeghiero, M.D., Beng H. Chong, M.D., Xuena Wang, Ph.D., and Dietmar P. Berger, M.D., Ph.D.*
A BS T R AC T 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; P65 years of age and 18% >75 years). The median duration of treatment for immune thrombocytopenia at the time of study entry was approximately 2 years, but 36% of patients (85 of 234)
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entered the study after having immune thrombocytopenia for a year or less (median duration in this subgroup, 0.25 years). Treatments
Among the various types of treatments for immune thrombocytopenia in the two study groups (Table 2), glucocorticoids were the most commonly administered. Overall, treatments were given in a much smaller proportion of patients in the romiplostim group (44%) than in the standard-of-care group (79%). Platelet Counts
The mean platelet count was higher in the romi plostim group than in the standard-of-care group throughout the treatment period (Fig. 1A). Between weeks 2 and 52, the percentage of patients with a platelet response (platelet count >50×109 per liter at any scheduled visit) ranged from 71% (108 of 152 patients) to 92% (127 of 138 patients) in the romiplostim group (median platelet count, 108×109 to 176×109 per liter) and from 26% (16 of 62 patients) to 51% (26 of 51 patients) in the standard-of-care group (median platelet count, 35×109 to 52×109 per liter). Patients in the romi plostim group were 2.3 times as likely to have a platelet response as those in the standard-of-care group (95% confidence interval, 2.0 to 2.6; P
