International Journal of Hematology

, Volume 98, Issue 3, pp 323–330

Oral eltrombopag for up to three years is safe and well-tolerated in Japanese patients with previously treated chronic immune thrombocytopenia: an open-label, extension study


    • Hiroshima University
  • Yoshiaki Tomiyama
    • Osaka University Hospital
  • Akiro Kimura
    • Kure-kyosai Hospital
  • Yoshitaka Miyakawa
    • Keio University
  • Shinichiro Okamoto
    • Keio University
  • Yasushi Okoshi
    • University of Tsukuba
  • Haruhiko Ninomiya
    • University of Tsukuba
  • Hiroshi Kosugi
    • Ogaki Municipal Hospital
  • Kazuyoshi Ishii
    • Kishiwada City Hospital
  • Yasuo Ikeda
    • Waseda University Faculty of Science and Engineering
  • Toshihiro Hattori
    • GlaxoSmithKline
  • Koichi Katsura
    • GlaxoSmithKline
  • Yuzuru Kanakura
    • Osaka University Graduate School of Medicine
Original Article

DOI: 10.1007/s12185-013-1401-1

Cite this article as:
Katsutani, S., Tomiyama, Y., Kimura, A. et al. Int J Hematol (2013) 98: 323. doi:10.1007/s12185-013-1401-1


Eltrombopag is an oral, nonpeptide, thrombopoietin receptor agonist approved for treatment of chronic immune thrombocytopenia (ITP). The safety, tolerability, and efficacy of eltrombopag for up to 3 years were evaluated in 19 Japanese patients with chronic ITP who had completed a prior 6-month study. Patients received eltrombopag once daily at the last dosage received in the prior study (12.5, 25, or 50 mg). Dose adjustments and treatment interruptions were permitted to maintain platelet counts of 50,000–200,000/μL. Primary evaluations were safety and tolerability of long-term eltrombopag treatment. The median duration of exposure was 27.5 months (range, 9.9–32.3). Adverse events were similar to those reported with short-term use of eltrombopag, and none led to treatment discontinuation. Nine serious adverse events were reported. Median platelet counts began to increase after 1 week of treatment and remained above 50,000/μL for most assessments. Bleeding episodes decreased from 63 % at baseline to 21 % after 2 weeks of treatment and remained below baseline for all assessments. Of 15 patients receiving concomitant baseline ITP medications, 10 permanently discontinued or achieved a sustained reduction of at least one treatment without requiring rescue treatment. Long-term treatment with eltrombopag was safe, well tolerated, and effective in Japanese patients with chronic ITP.


BleedingPlateletsThrombopoietin receptor agonist

Copyright information

© The Japanese Society of Hematology 2013