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Efficacy and Safety of Eltrombopag for Aplastic Anemia: A Systematic Review and Meta-analysis

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Abstract

Background and Objectives

Eltrombopag seems to be effective in treating patients with aplastic anemia in several clinical trials. This paper aims to perform the first meta-analysis analyzing the efficacy and safety of eltrombopag for aplastic anemia.

Methods

Literatures were retrieved from PubMed, EMBASE, OVID, Web of Science, Cochrane, Wanfang, http://clinicaltrials.gov and World Health Organization International Clinical Trials Registry Platform search portal from establishment to July 2018. Using Stata statistical software version 12.0, subgroup analyses and sensitivity analyses were conducted.

Results

The overall hematologic response rate is 88% (95% CI 83–94%) for patients treated with eltrombopag plus immunosuppressive therapy, and 47% (95% CI 38–56%) for patients with refractory aplastic anemia using eltrombopag alone. Karyotype abnormality rates include an overall rate of 10% (95% CI 7–14%), a subtotal rate of 8% (95% CI 3–13%) for patients who are treated with eltrombopag plus immunosuppressive therapy without using antithymocyte globulin before, and a subtotal rate of 17% (95% CI 10–24%) for patients with refractory aplastic anemia treated with eltrombopag alone.

Conclusions

With different treatments and in different conditions eltrombopag showed a distinctive effect for aplastic anemia. However, clone evolution and adverse events were associated with treatment.

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Author notes

  1. Yaqun Hong and Xiaofan Li contribute equally to this article.

Authors and Affiliations

  1. Fujian Provincial Key Laboratory on Hematology, Department of Hematology, Fujian Institute of Hematology, Fujian Medical University Union Hospital, No. 29 Xinquan Street, Gulou District, Fuzhou, 350001, China

    Yaqun Hong, Xiaofan Li, Nainong Li & Yuanzhong Chen

  2. Union Clinical Medical Colleges, Fujian Medical University, No. 1 Xuefubei Street, Fuzhou, 350108, Minhou County, China

    Yaqun Hong

  3. INSERM U1160, Hopital Saint Louis, Université Paris Diderot, Sorbonne Paris Cité, 75007, Paris, France

    Xiaofan Li

  4. Faculty of Life Sciences and Medicine, King’s College London, London, WC2R 2LS, UK

    Bo Wan

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Correspondence to Xiaofan Li.

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Funding

This work was supported by Youth Project of National Natural Science Foundation (Grant no. 81200400), top-notch innovative talents project and Fujian project (Grants 2016Y9025 & 2016J06018 & 2017I0004); Fujian Medical University teaching reform project (Y17005) and Fujian Provincial Health and Family planning Commission Youth Research Project (2017-1-6).

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Yaqun Hong, Xiaofan Li, Bo Wan, Nainong Li, and Yuanzhong Chen declare that they have no conflict of interest.

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Hong, Y., Li, X., Wan, B. et al. Efficacy and Safety of Eltrombopag for Aplastic Anemia: A Systematic Review and Meta-analysis. Clin Drug Investig 39, 141–156 (2019). https://doi.org/10.1007/s40261-018-0725-2

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