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SYSTEMATIC REVIEW article

Front. Med.

Sec. Hematology

Volume 12 - 2025 | doi: 10.3389/fmed.2025.1531824

This article is part of the Research Topic Immune Thrombocytopenia (ITP) - Diagnosis and Treatment - Volume II View all 4 articles

Efficacy and safety and analysis of thrombopoietin receptor agonists for the treatment of immune thrombocytopenia in adults: analysis of a systematic review and network meta-analysis of randomized controlled trials and results of real-world safety data

Provisionally accepted
Yao Liu Yao Liu 1*Yumeng Liu Yumeng Liu 1Ni Zhang Ni Zhang 1Yu Xiong Yu Xiong 2Tingting Jiang Tingting Jiang 1Yanping Li Yanping Li 1
  • 1 Department of Pharmacy, Daping Hospital, Chongqing, China
  • 2 Department of Pharmacy, Mianyang Central Hospital, Mianyang, Sichuan Province, China

The final, formatted version of the article will be published soon.

    This study aimed to compare the efficacy and safety of thrombopoietin receptor agonists (TPO-RA) in the treatment of immune thrombocytopenia (ITP) in adults. A systematic review was conducted using network meta-analysis and a disproportionality analysis based on the FDA Adverse Event Reporting System (FAERS) database to evaluate hemorrhagic and thrombotic events of clinical concern. Our network metaanalysis included 14 randomized controlled trials (RCTs) involving 1,454 patients. The results indicated that, in terms of efficacy, romiplostim (odds ratio [OR], 0.04; 95% confidence interval [CI], 0 to 0.68) was the most effective, followed by avatrombopag, hetrombopag, and eltrombopag. Regarding safety, there were no significant differences in the safety profiles of the four TPO-RA compared to placebo. According to the surface under the cumulative ranking curve (SUCRA), avatrombopag exhibited the highest safety ranking at 23.8%. Within the FAERS database, we identified 982 cases of TPO-RA-related hemorrhagic and thrombotic events. The highest number of preferred terms (PTs) associated with romiplostim was 26, followed by 18 for eltrombopag and 7 for avatrombopag. The findings of this study suggest that romiplostim exhibits significant efficacy, whereas avatrombopag presents a superior safety profile. In the context of clinical second-line treatment, the selection of the most suitable TPO-RA should be guided by the specific circumstances of each patient.

    Keywords: thrombopoietin receptor agonists, immune thrombocytopenia, adults, Network meta-analysis, Disproportionality analysis

    Received: 21 Nov 2024; Accepted: 17 Feb 2025.

    Copyright: © 2025 Liu, Liu, Zhang, Xiong, Jiang and Li. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

    * Correspondence: Yao Liu, Department of Pharmacy, Daping Hospital, Chongqing, China

    Disclaimer: All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.

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