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Eltrombopag treatment promotes platelet recovery and reduces platelet transfusion for patients with post-transplantation thrombocytopenia

Annals of Hematology volume 99, pages 2679–2687 (2020)Cite this article

Abstract

Post-transplantation thrombocytopenia (PT) is a common and severe complication which usually leads to poor prognosis. Eltrombopag (EPAG), a novel oral thrombopoietin (TPO) receptor agonist, has shown promising effects in thrombocytopenia due to immune thrombocytopenic purpura (ITP) and refractory severe aplastic anemia (rSAA), while the effectiveness of EPAG for PT patients still needs to be evaluated. A total of 32 PT patients receiving EPAG were retrospectively analyzed between September 2017 and July 2019, including 15 patients with poor graft function (PGF) and 17 patients with secondary failure of platelet recovery (SFPR). To date, 21 (65.6%) patients achieved overall recovery (OR) and 14 (43.8%) patients achieved complete recovery (CR). Among responders, 18 (85.7%) patients discontinued or tapered the drug and 16 (76.2%) patients successfully maintained their best response. During the EPAG treatment, responders received much lower median platelet transfusion units than non-responders (11 vs. 95, P < 0.001). After a median follow-up time of 364 days (range, 24–842), the overall survival in these patients was 78.1% (100% for responders and 36.4% for non-responders, P < 0.001). In the univariate and multivariate analysis, PGF was identified as the independent risk factor for OR (P = 0.041, HR = 5.333). Megakaryocyte (Megk) amounts (P = 0.025, HR = 14.638) and splenomegaly (P = 0.042, HR = 11.278) were identified as independent risk factors for CR. Besides, PGF patients tended to take a longer time to achieve PR and CR than SFPR patients. In conclusion, our data suggest that EPAG can promote platelet recovery and reduce platelet transfusion in PT patients.

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Funding

This work was supported by the National Natural Science Foundation of China (grant no. 81670148) and Key R&D Program of Zhejiang Province (grant no. 2019C03016).

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Authors and Affiliations

  1. Bone Marrow Transplantation Center, the First Affiliated Hospital, School of Medicine, Zhejiang University, 79 Qingchun Road, Hangzhou, 310003, Zhejiang, People’s Republic of China

    Fei Gao, Xiaoyu Zhou, Jimin Shi, Yi Luo, Yamin Tan, Huarui Fu, Xiaoyu Lai, Jian Yu, He Huang & Yanmin Zhao

  2. Institute of Hematology, Zhejiang University, Hangzhou, Zhejiang, People’s Republic of China

    Fei Gao, Xiaoyu Zhou, Jimin Shi, Yi Luo, Yamin Tan, Huarui Fu, Xiaoyu Lai, Jian Yu, He Huang & Yanmin Zhao

  3. Zhejiang Engineering Laboratory for Stem Cell and Immunotherapy, Hangzhou, Zhejiang, People’s Republic of China

    Fei Gao, Xiaoyu Zhou, Jimin Shi, Yi Luo, Yamin Tan, Huarui Fu, Xiaoyu Lai, Jian Yu, He Huang & Yanmin Zhao

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  1. Fei Gao
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  2. Xiaoyu Zhou
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  10. Yanmin Zhao
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Contributions

Yanmin Zhao and He Huang designed the study and supervised the analyses and manuscript preparation. Fei Gao collected and analyzed the data, and wrote the manuscript. All authors discussed and interpreted the results. All authors approved the final version of the manuscript.

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Correspondence to He Huang or Yanmin Zhao.

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The study protocol was in compliance with the Declaration of Helsinki and was approved by the Ethics Review Committee of the First Affiliated Hospital of Zhejiang University. Informed consent was obtained from each patient in the study.

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Gao, F., Zhou, X., Shi, J. et al. Eltrombopag treatment promotes platelet recovery and reduces platelet transfusion for patients with post-transplantation thrombocytopenia. Ann Hematol 99, 2679–2687 (2020). https://doi.org/10.1007/s00277-020-04106-2

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Keywords

  • Eltrombopag
  • Post-transplantation thrombocytopenia
  • Platelet recovery