Journal Article

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

USUKI Kensuke, MORI Shinichiro, LIZAMBRI Richard, TOMIYAMA Yoshiaki, WEI Helen, KIRITO Keita, OKAMOTO Shinichiro, HASHINO Satoshi, ANDO Kiyoshi, IWATO Koji, SHIRASUGI Yukari, MIYAZAKI Koji, KUROKAWA Mineo, YONEMURA Yuji

2011

Overview

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.

• Publisher: Springer Japan,Springer,Springer Nature B.V
• Subject: 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 / Efficacy / Female / Hematologic and hematopoietic diseases / Hematology / Humans / Idiopathic thrombocytopenic purpura / Japan / 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 / Safety / Thrombopoietin - administration & dosage / Thrombopoietin - adverse effects / Thrombopoietin - therapeutic use / TPO-receptor agonist / Treatment Outcome