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

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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.

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Acknowledgments

This study was partially supported by Special Research Projects 200802031 (to Renchi Yang), Ministry of Health People’s Republic of China.

Conflict of interest

All the authors declare no actual or potential conflict of interest in relation to this article.

Author information

Authors and Affiliations

  1. Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, 100730, China

    Shujie Wang & Yongqiang Zhao

  2. Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China

    Renchi Yang

  3. Affiliated Union Hospital of Huazhong University of Science and Technology, Wuhan, China

    Ping Zou

  4. Qilu Hospital of Shandong University, Jinan, China

    Ming Hou

  5. The First Affiliated Hospital of Suzhou University, Suzhou, China

    Depei Wu

  6. Ruijin Hospital, Shanghai Jiaotong University School of Medicine, Shanghai, China

    Zhixiang Shen

  7. Institute of Hematology, Peking University People’s Hospital, Beijing, China

    Xijing Lu

  8. The First Affiliated Hospital, China Medical University, Shenyang, China

    Yan Li

  9. Xijing Hospital, Fourth Military Medical University, Xi’an, China

    Xiequn Chen

  10. West China Hospital, Sichuan University, Chengdu, China

    Ting Niu

  11. The First Affiliated Hospital, Zhengzhou University, Zhengzhou, China

    Hui Sun

  12. General Hospital of Chinese People’s Liberation Army, Beijing, China

    Li Yu

  13. Beijing Friendship Hospital, Capital Medical University, Beijing, China

    Zhao Wang

  14. Henan Province People’s Hospital, Zhengzhou, China

    Yin Zhang

  15. Beijing Hospital, The Ministry of Health, Beijing, China

    Naibai Chang

  16. Beijing DMS Pharma Ltd., Beijing, China

    Gaokui Zhang

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  1. Shujie Wang
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  13. Zhao Wang
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Correspondence to Yongqiang Zhao.

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Wang, S., Yang, R., Zou, P. et al. A multicenter randomized controlled trial of recombinant human thrombopoietin treatment in patients with primary immune thrombocytopenia. Int J Hematol 96, 222–228 (2012). https://doi.org/10.1007/s12185-012-1124-8

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  • DOI: https://doi.org/10.1007/s12185-012-1124-8

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