Eltrombopag versus romiplostim in treatment of children with persistent or chronic immune thrombocytopenia: a systematic review incorporating an indirect-comparison meta-analysis

Sci Rep. 2018 Jan 12;8(1):576. doi: 10.1038/s41598-017-19099-8.


In absence of direct comparison, we conducted an indirect-comparison meta-analysis to evaluate the efficacy and safety of thrombopoietin-receptor agonists(TPO-RAs) in treatment of pediatric persistent or chronic immune thrombocytopenia(ITP). PubMed, Embase, Cochrane Library, Clinical Trials.gov, China National Knowledge Infrastructure, and Chinese Biomedical Literature Database were searched from their earliest records to May 2017. Randomized controlled trials comparing the TPO-RAs with placebo in pediatric ITP were included. Outcomes included overall response rate(primary), durable response, overall or clinically significant bleeding, the proportion of patients receiving rescue medication, and safety. Five randomized placebo-controlled studies(N = 261) were analyzed. The overall response[Risk Ratio(RR) 0.57, 95% confidence interval(CI) 0.21-1.56], the incidence of adverse events (RR 0.96, 95%CI 0.66-1.39), durable response(RR 2.48, 95%CI 0.31-19.97), and the proportion of patients receiving rescue treatment(RR 0.73, 95%CI 0.20-2.73) were similar between eltrombopag and romiplostim group. Nevertheless, eltrombopag might have lower risk of overall bleeding(RR 0.43, 95%CI 0.23-0.80) and clinically significant bleeding(RR 0.33, 95%CI 0.12-0.89) than romiplostim. This meta-analysis suggests that eltrombopag might be similar to romiplostim in efficacy and safety, but seems to reduce the risk of bleeding compared to romiplostim. Furthermore, cost of the treatment, comorbidity of patients and drug compliance should also be considered in clinical decision making.

Publication types

  • Comparative Study
  • Multicenter Study
  • Review
  • Systematic Review

MeSH terms

  • Adolescent
  • Benzoates / adverse effects
  • Benzoates / therapeutic use*
  • Child
  • Child, Preschool
  • Female
  • Humans
  • Hydrazines / adverse effects
  • Hydrazines / therapeutic use*
  • Infant
  • Male
  • Pyrazoles / adverse effects
  • Pyrazoles / therapeutic use*
  • Randomized Controlled Trials as Topic
  • Receptors, Fc / therapeutic use*
  • Recombinant Fusion Proteins / adverse effects
  • Recombinant Fusion Proteins / therapeutic use*
  • Thrombocytopenia / drug therapy*
  • Thrombopoietin / adverse effects
  • Thrombopoietin / therapeutic use*
  • Treatment Outcome


  • Benzoates
  • Hydrazines
  • Pyrazoles
  • Receptors, Fc
  • Recombinant Fusion Proteins
  • Thrombopoietin
  • romiplostim
  • eltrombopag