International Journal of Hematology

, Volume 94, Issue 1, pp 71–80 | Cite as

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

  • Yukari Shirasugi
  • Kiyoshi Ando
  • Koji Miyazaki
  • Yoshiaki Tomiyama
  • Shinichiro Okamoto
  • Mineo Kurokawa
  • Keita Kirito
  • Yuji Yonemura
  • Shinichiro Mori
  • Kensuke Usuki
  • Koji Iwato
  • Satoshi Hashino
  • Helen Wei
  • Richard Lizambri
Original Article

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 × 109/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.

Keywords

Idiopathic thrombocytopenic purpura TPO-receptor agonist Japan Efficacy Safety 

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Copyright information

© The Japanese Society of Hematology 2011

Authors and Affiliations

  • Yukari Shirasugi
    • 1
  • Kiyoshi Ando
    • 1
  • Koji Miyazaki
    • 2
  • Yoshiaki Tomiyama
    • 3
  • Shinichiro Okamoto
    • 4
  • Mineo Kurokawa
    • 5
  • Keita Kirito
    • 6
  • Yuji Yonemura
    • 7
  • Shinichiro Mori
    • 8
  • Kensuke Usuki
    • 9
  • Koji Iwato
    • 10
  • Satoshi Hashino
    • 11
  • Helen Wei
    • 12
  • Richard Lizambri
    • 13
  1. 1.Department of Hematology and OncologyTokai University HospitalIseharaJapan
  2. 2.Department of HematologyKitasato University HospitalKanagawaJapan
  3. 3.Department of Blood TransfusionOsaka University HospitalOsakaJapan
  4. 4.Division of HematologyKeio University HospitalTokyoJapan
  5. 5.Department of Hematology and OncologyThe University of Tokyo HospitalTokyoJapan
  6. 6.Department of Hematology and OncologyUniversity of Yamanashi HospitalYamanashiJapan
  7. 7.Department of Transfusion Medicine and Cell TherapyKumamoto University HospitalKumamotoJapan
  8. 8.Department of Hematology and OncologyKansai Medical University Hirakata HospitalOsakaJapan
  9. 9.Division of HematologyNTT Kanto Medical CenterTokyoJapan
  10. 10.Department of Blood TransfusionHiroshima Red Cross Hospital and Atomic-bomb Survivors HospitalHiroshimaJapan
  11. 11.Department of Gastroenterology and HematologyHokkaido University HospitalHokkaidoJapan
  12. 12.Biostatistics, Amgen Inc.Thousand OaksUSA
  13. 13.Clinical Development, Amgen Inc.Thousand OaksUSA

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