Eltrombopag for the treatment of chronic idiopathic thrombocytopenic purpura.

@article{citeulike:2392314, abstract = {BACKGROUND: The pathogenesis of chronic idiopathic thrombocytopenic purpura (ITP) involves antibody-mediated platelet destruction and reduced platelet production. Stimulation of platelet production may be an effective treatment for this disorder. METHODS: We conducted a trial in which 118 adults with chronic ITP and platelet counts of less than 30,000 per cubic millimeter who had had relapses or whose platelet count was refractory to at least one standard treatment for ITP were randomly assigned to receive the oral thrombopoietin-receptor agonist eltrombopag (30, 50, or 75 mg daily) or placebo. The primary end point was a platelet count of 50,000 or more per cubic millimeter on day 43. RESULTS: In the eltrombopag groups receiving 30, 50, and 75 mg per day, the primary end point was achieved in 28\%, 70\%, and 81\% of patients, respectively. In the placebo group, the end point was achieved in 11\% of patients. The median platelet counts on day 43 for the groups receiving 30, 50, and 75 mg of eltrombopag were 26,000, 128,000, and 183,000 per cubic millimeter, respectively; for the placebo group the count was 16,000 per cubic millimeter. By day 15, more than 80\% of patients receiving 50 or 75 mg of eltrombopag daily had an increased platelet count. Bleeding also decreased during treatment in these two groups. The incidence and severity of adverse events were similar in the placebo and eltrombopag groups. CONCLUSIONS: Eltrombopag increased platelet counts in a dose-dependent manner in patients with relapsed or refractory ITP. (ClinicalTrials.gov number, NCT00102739.)}, address = {Weill Cornell Medical College of Cornell University, New York, NY 10065, USA. jbussel@med.cornell.edu}, author = {Bussel, J. B. and Cheng, G. and Saleh, M. N. and Psaila, B. and Kovaleva, L. and Meddeb, B. and Kloczko, J. and Hassani, H. and Mayer, B. and Stone, N. L. and Arning, M. and Provan, D. and Jenkins, J. M. }, citeulike-article-id = {2392314}, doi = {10.1056/NEJMoa073275}, issn = {1533-4406}, journal = {N Engl J Med}, month = {November}, number = {22}, pages = {2237--2247}, posted-at = {2008-02-18 03:39:34}, priority = {2}, title = {Eltrombopag for the treatment of chronic idiopathic thrombocytopenic purpura.}, url = {http://dx.doi.org/10.1056/NEJMoa073275}, volume = {357}, year = {2007} }

TY - JOUR ID - citeulike:2392314 L3 - citeulike-article-id:2392314 TI - Eltrombopag for the treatment of chronic idiopathic thrombocytopenic purpura. JF - N Engl J Med VL - 357 IS - 22 SP - 2237 EP - 2247 AD - Weill Cornell Medical College of Cornell University, New York, NY 10065, USA. jbussel@med.cornell.edu SN - 1533-4406 N2 - BACKGROUND: The pathogenesis of chronic idiopathic thrombocytopenic purpura (ITP) involves antibody-mediated platelet destruction and reduced platelet production. Stimulation of platelet production may be an effective treatment for this disorder. METHODS: We conducted a trial in which 118 adults with chronic ITP and platelet counts of less than 30,000 per cubic millimeter who had had relapses or whose platelet count was refractory to at least one standard treatment for ITP were randomly assigned to receive the oral thrombopoietin-receptor agonist eltrombopag (30, 50, or 75 mg daily) or placebo. The primary end point was a platelet count of 50,000 or more per cubic millimeter on day 43. RESULTS: In the eltrombopag groups receiving 30, 50, and 75 mg per day, the primary end point was achieved in 28%, 70%, and 81% of patients, respectively. In the placebo group, the end point was achieved in 11% of patients. The median platelet counts on day 43 for the groups receiving 30, 50, and 75 mg of eltrombopag were 26,000, 128,000, and 183,000 per cubic millimeter, respectively; for the placebo group the count was 16,000 per cubic millimeter. By day 15, more than 80% of patients receiving 50 or 75 mg of eltrombopag daily had an increased platelet count. Bleeding also decreased during treatment in these two groups. The incidence and severity of adverse events were similar in the placebo and eltrombopag groups. CONCLUSIONS: Eltrombopag increased platelet counts in a dose-dependent manner in patients with relapsed or refractory ITP. (ClinicalTrials.gov number, NCT00102739.) AU - Bussel, JB AU - Cheng, G AU - Saleh, MN AU - Psaila, B AU - Kovaleva, L AU - Meddeb, B AU - Kloczko, J AU - Hassani, H AU - Mayer, B AU - Stone, NL AU - Arning, M AU - Provan, D AU - Jenkins, JM PY - 2007/11/29/ UR - http://dx.doi.org/10.1056/NEJMoa073275 ER -