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Int J Hematol. 2011 Jul;94(1):71-80. doi: 10.1007/s12185-011-0886-8. Epub 2011 Jun 25.

Romiplostim for the treatment of chronic immune thrombocytopenia in adult Japanese patients: a double-blind, randomized Phase III clinical trial.

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Department of Hematology and Oncology, Tokai University Hospital, 143 Shimokasuya, Isehara, Kanagawa, 259-1193, Japan.
Department of Hematology and Oncology, Tokai University Hospital, 143 Shimokasuya, Isehara, Kanagawa, 259-1193, Japan.
Department of Hematology, Kitasato University Hospital, Kanagawa, Japan.
Department of Blood Transfusion, Osaka University Hospital, Osaka, Japan.
Division of Hematology, Keio University Hospital, Tokyo, Japan.
Department of Hematology and Oncology, The University of Tokyo Hospital, Tokyo, Japan.
Department of Hematology and Oncology, University of Yamanashi Hospital, Yamanashi, Japan.
Department of Transfusion Medicine and Cell Therapy, Kumamoto University Hospital, Kumamoto, Japan.
Department of Hematology and Oncology, Kansai Medical University Hirakata Hospital, Osaka, Japan.
Division of Hematology, NTT Kanto Medical Center, Tokyo, Japan.
Department of Blood Transfusion, Hiroshima Red Cross Hospital and Atomic-bomb Survivors Hospital, Hiroshima, Japan.
Department of Gastroenterology and Hematology, Hokkaido University Hospital, Hokkaido, Japan.
Biostatistics, Amgen Inc., Thousand Oaks, CA, USA.
Clinical Development, Amgen Inc., Thousand Oaks, CA, USA.


The efficacy and safety of romiplostim, a thrombopoietin-mimetic peptibody, were evaluated in a double-blind, placebo-controlled, randomized trial of Japanese patients with chronic immune thrombocytopenia (ITP). Thirty-four ITP patients received romiplostim (n = 22) or placebo (n = 12) for 12 weeks, with a starting romiplostim dose of 3 μg/kg weekly. The primary end point was the number of weeks with platelet response, defined as a platelet count ≥50 × 10(9)/L (not including the 4 weeks after rescue medication administration). Patients received a median of 4 (range 1-19) prior ITP therapies including splenectomy in 44%. On study, 68% also received concomitant ITP therapy. Weekly responses occurred for a median of 11 weeks with romiplostim as compared to 0 weeks with placebo (p < 0.0001). Most romiplostim-treated patients (95%) achieved platelet responses; two showed extended responses after the treatment period. The use of rescue medication was required in 9% of romiplostim-treated patients as compared with 17% of placebo-treated patients. Both treatment groups had similar incidences of adverse events (91% romiplostim, 92% placebo). Adverse events that occurred more frequently (>10%) in romiplostim-treated patients included nasopharyngitis, headache, peripheral edema, back pain, and extremity pain. In conclusion, romiplostim significantly increased and maintained platelet counts and was well tolerated in Japanese patients with ITP.

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