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

• Published in: International journal of hematology 2011-07, Vol.94 (1), p.71-80
• Main Authors: Shirasugi, Yukari, Ando, Kiyoshi, Miyazaki, Koji, Tomiyama, Yoshiaki, Okamoto, Shinichiro, Kurokawa, Mineo, Kirito, Keita, Yonemura, Yuji, Mori, Shinichiro, Usuki, Kensuke, Iwato, Koji, Hashino, Satoshi, Wei, Helen, Lizambri, Richard
• Format: Article
• Language: English
• Subjects: Adult; Aged; Asian Continental Ancestry Group; Biological and medical sciences; Blood Platelets - drug effects; Double-Blind Method; Drug-Related Side Effects and Adverse Reactions; Edema - chemically induced; Female; Hematologic and hematopoietic diseases; Hematology; Humans; Male; Medical sciences; Medicine; Medicine & Public Health; Middle Aged; Nasopharyngitis - chemically induced; Oncology; Original Article; Pain - chemically induced; Platelet Count; Platelet diseases and coagulopathies; Purpura, Thrombocytopenic, Idiopathic - drug therapy; Receptors, Fc - administration & dosage; Receptors, Fc - therapeutic use; Recombinant Fusion Proteins - administration & dosage; Recombinant Fusion Proteins - adverse effects; Recombinant Fusion Proteins - therapeutic use; Thrombopoietin - administration & dosage; Thrombopoietin - adverse effects; Thrombopoietin - therapeutic use; Treatment Outcome
• ISSN: 0925-5710; 1865-3774
• DOI: 10.1007/s12185-011-0886-8

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  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.