International Journal of Hematology

, Volume 96, Issue 2, pp 222–228

A multicenter randomized controlled trial of recombinant human thrombopoietin treatment in patients with primary immune thrombocytopenia

  • Shujie Wang
  • Renchi Yang
  • Ping Zou
  • Ming Hou
  • Depei Wu
  • Zhixiang Shen
  • Xijing Lu
  • Yan Li
  • Xiequn Chen
  • Ting Niu
  • Hui Sun
  • Li Yu
  • Zhao Wang
  • Yin Zhang
  • Naibai Chang
  • Gaokui Zhang
  • Yongqiang Zhao
Original Article

DOI: 10.1007/s12185-012-1124-8

Cite this article as:
Wang, S., Yang, R., Zou, P. et al. Int J Hematol (2012) 96: 222. doi:10.1007/s12185-012-1124-8

Abstract

This multicenter, randomized trial assessed the efficacy and safety of a recombinant human thrombopoietin (rhTPO) in patients with persistent primary immune thrombocytopenia (ITP) who had failed glucocorticosteroid treatment. A total of 140 eligible patients were randomized to receive rhTPO + danazol (rhTPO group, 73 patients) or danazol (control group, 67 patients) alone. During the first phase, the increase in the mean maximal platelet counts (101.2 × 109/L) and the area under curve (749.6) in the rhTPO group were significantly higher compared to control (33.3 × 109/L and 316.2; P = 0.0060 and 0.0000, respectively). The major response rate (MRR) and total response rate (TRR) in the rhTPO group were 38.4 and 60.3 %, respectively, significantly higher than in control (MRR 7.9 %, P = 0.0003; TRR 36.5 %, P = 0.0104). In the control group, 45 patients with platelet counts <20 × 109/L were given rhTPO during the second phase and achieved MRR 31.1 % and TRR 66.7 %. The mean platelet counts in the rhTPO group were still approximately 50 × 109/L on day 28 of the study. The overall incidence of rhTPO-related adverse events was 13.6 %. All the adverse events were generally mild. This study demonstrated that rhTPO was well tolerated, and it markedly increased platelet counts in chronic ITP patients. Stimulation of platelet production by rhTPO may provide a new therapeutic option for patients with ITP.

Keywords

Primary immune thrombocytopenia (ITP) Thrombopoietin (TPO) Recombinant human thrombopoietin (rhTPO) 

Supplementary material

12185_2012_1124_MOESM1_ESM.doc (42 kb)
Supplementary material 1 (DOC 41 kb)

Copyright information

© The Japanese Society of Hematology 2012

Authors and Affiliations

  • Shujie Wang
    • 1
  • Renchi Yang
    • 2
  • Ping Zou
    • 3
  • Ming Hou
    • 4
  • Depei Wu
    • 5
  • Zhixiang Shen
    • 6
  • Xijing Lu
    • 7
  • Yan Li
    • 8
  • Xiequn Chen
    • 9
  • Ting Niu
    • 10
  • Hui Sun
    • 11
  • Li Yu
    • 12
  • Zhao Wang
    • 13
  • Yin Zhang
    • 14
  • Naibai Chang
    • 15
  • Gaokui Zhang
    • 16
  • Yongqiang Zhao
    • 1
  1. 1.Peking Union Medical College HospitalChinese Academy of Medical Sciences and Peking Union Medical CollegeBeijingChina
  2. 2.Blood Diseases HospitalChinese Academy of Medical Sciences and Peking Union Medical CollegeBeijingChina
  3. 3.Affiliated Union Hospital of Huazhong University of Science and TechnologyWuhanChina
  4. 4.Qilu Hospital of Shandong UniversityJinanChina
  5. 5.The First Affiliated Hospital of Suzhou UniversitySuzhouChina
  6. 6.Ruijin HospitalShanghai Jiaotong University School of MedicineShanghaiChina
  7. 7.Institute of HematologyPeking University People’s HospitalBeijingChina
  8. 8.The First Affiliated HospitalChina Medical UniversityShenyangChina
  9. 9.Xijing HospitalFourth Military Medical UniversityXi’anChina
  10. 10.West China HospitalSichuan UniversityChengduChina
  11. 11.The First Affiliated HospitalZhengzhou UniversityZhengzhouChina
  12. 12.General Hospital of Chinese People’s Liberation ArmyBeijingChina
  13. 13.Beijing Friendship HospitalCapital Medical UniversityBeijingChina
  14. 14.Henan Province People’s HospitalZhengzhouChina
  15. 15.Beijing HospitalThe Ministry of HealthBeijingChina
  16. 16.Beijing DMS Pharma Ltd.BeijingChina