title: Romiplostim for the treatment of chronic immune thrombocytopenia in adult Japanese patients: a double-blind, randomized Phase III clinical trial.
abstract: 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.
authors.affiliation: Department of Hematology and Oncology, Tokai University Hospital, 143 Shimokasuya, Isehara, Kanagawa, 259-1193, Japan. whiteced@is.icc.u-tokai.ac.jp.
authors.forename: Yukari
authors.initials: Y
authors.lastname: Shirasugi
authors.affiliation: Department of Hematology and Oncology, Tokai University Hospital, 143 Shimokasuya, Isehara, Kanagawa, 259-1193, Japan.
authors.forename: Kiyoshi
authors.initials: K
authors.lastname: Ando
authors.affiliation: Department of Hematology, Kitasato University Hospital, Kanagawa, Japan.
authors.forename: Koji
authors.initials: K
authors.lastname: Miyazaki
authors.affiliation: Department of Blood Transfusion, Osaka University Hospital, Osaka, Japan.
authors.forename: Yoshiaki
authors.initials: Y
authors.lastname: Tomiyama
authors.affiliation: Division of Hematology, Keio University Hospital, Tokyo, Japan.
authors.forename: Shinichiro
authors.initials: S
authors.lastname: Okamoto
authors.affiliation: Department of Hematology and Oncology, The University of Tokyo Hospital, Tokyo, Japan.
authors.forename: Mineo
authors.initials: M
authors.lastname: Kurokawa
authors.affiliation: Department of Hematology and Oncology, University of Yamanashi Hospital, Yamanashi, Japan.
authors.forename: Keita
authors.initials: K
authors.lastname: Kirito
authors.affiliation: Department of Transfusion Medicine and Cell Therapy, Kumamoto University Hospital, Kumamoto, Japan.
authors.forename: Yuji
authors.initials: Y
authors.lastname: Yonemura
authors.affiliation: Department of Hematology and Oncology, Kansai Medical University Hirakata Hospital, Osaka, Japan.
authors.forename: Shinichiro
authors.initials: S
authors.lastname: Mori
authors.affiliation: Division of Hematology, NTT Kanto Medical Center, Tokyo, Japan.
authors.forename: Kensuke
authors.initials: K
authors.lastname: Usuki
authors.affiliation: Department of Blood Transfusion, Hiroshima Red Cross Hospital and Atomic-bomb Survivors Hospital, Hiroshima, Japan.
authors.forename: Koji
authors.initials: K
authors.lastname: Iwato
authors.affiliation: Department of Gastroenterology and Hematology, Hokkaido University Hospital, Hokkaido, Japan.
authors.forename: Satoshi
authors.initials: S
authors.lastname: Hashino
authors.affiliation: Biostatistics, Amgen Inc., Thousand Oaks, CA, USA.
authors.forename: Helen
authors.initials: H
authors.lastname: Wei
authors.affiliation: Clinical Development, Amgen Inc., Thousand Oaks, CA, USA.
authors.forename: Richard
authors.initials: R
authors.lastname: Lizambri
mesh_terms: D000328:Adult; D000368:Aged; D044466:Asian People; D001792:Blood Platelets / Q000187:drug effects; D004311:Double-Blind Method; D064420:Drug-Related Side Effects and Adverse Reactions; D004487:Edema / Q000139:chemically induced; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D009304:Nasopharyngitis / Q000139:chemically induced; D010146:Pain / Q000139:chemically induced; D010976:Platelet Count; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000188:drug therapy*; D011961:Receptors, Fc / Q000008:administration & dosage / Q000627:therapeutic use*; D011993:Recombinant Fusion Proteins / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D013926:Thrombopoietin / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D016896:Treatment Outcome
chemical_list: D011961:Receptors, Fc; D011993:Recombinant Fusion Proteins; D013926:Thrombopoietin; C488777:romiplostim