Efficacy and safety of thrombopoietin receptor agonists in children and adults with persistent and chronic immune thrombocytopenia: a meta-analysis

Expert Opin Pharmacother. 2023 Apr;24(6):763-774. doi: 10.1080/14656566.2023.2198089. Epub 2023 Apr 6.

Abstract

Introduction: In this paper, we systematically review the efficacy and safety of thrombopoietin receptor agonists (TPORAs) for treatment of persistent and chronic immune thrombocytopenia (ITP) in children and adults.

Methods: We searched PubMed, MEDLINE, ScienceDirect, Scopus, EMbase and the Cochrane Library to collect randomized controlled trials (RCTs) of TPO-RAs which including avatrombopag hetrombopag eltrombopag and romiplostim treated persistent and chronic ITP from their earliest records to February 2022.

Results: We included 15 RCTs with a total of 1563 patients. There were ten trials of adults and five trials of children. The results of meta-analysis showed that in adult patients, patients treated with TPO-RAs had longer duration of platelet response, higher platelet response rate, lower use of rescue therapy, and lower incidence of bleeding events, and similar incidence of adverse events compared with placebo. Except for the incidence of any bleeding, the results in children were consistent with those in adults. The network meta-analysis of data on overall platelet response rates in adults showed that avatrombopag was more effective than eltrombopag and hetrombopag.

Conclusions: TPO-RAs has better efficacy and higher safety in the treatment of ITP. And the overall response rate of avatrombopag in adults was higher than that in eltrombopag and hetrombopag.

Keywords: Efficacy; Immune thrombocytopenia; Meta-analysis; Randomized controlled trial; Safety; Thrombopoietin receptor agonists.

Publication types

  • Systematic Review
  • Meta-Analysis

MeSH terms

  • Adult
  • Benzoates / adverse effects
  • Child
  • Hemorrhage / chemically induced
  • Humans
  • Hydrazines / adverse effects
  • Purpura, Thrombocytopenic, Idiopathic* / drug therapy
  • Receptors, Thrombopoietin / agonists
  • Recombinant Fusion Proteins / adverse effects

Substances

  • eltrombopag
  • hetrombopag
  • avatrombopag
  • Receptors, Thrombopoietin
  • Hydrazines
  • Benzoates
  • Recombinant Fusion Proteins