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Thrombopoietin-receptor agonists for children with immune thrombocytopenia: a systematic review

Jiaxing Zhang1,2, Yi Liang3, Yuan Ai4

  • 1a Chinese Evidence-based Medicine Center , Sichuan University , Chengdu , China.

Expert Opinion on Pharmacotherapy
|August 29, 2017
PubMed

Insights

Thrombopoietin-receptor agonists (TPOras) show promise in improving platelet response for pediatric immune thrombocytopenia (ITP). These treatments may increase durable and overall platelet counts compared to placebo.

Area of Science:

  • Hematology
  • Pediatric Medicine
  • Pharmacology

Background:

  • Immune thrombocytopenia (ITP) is an autoimmune disorder characterized by low platelet counts.
  • Pediatric ITP presents unique management challenges, necessitating safe and effective treatment options.

Purpose of the Study:

  • To systematically review the efficacy and safety of Thrombopoietin-receptor agonists (TPOras) in treating pediatric immune thrombocytopenia (ITP).

Main Methods:

  • A systematic review of randomized controlled trials (RCTs) was conducted, searching PubMed, Embase, and Cochrane Library up to January 2017.
  • Primary outcomes included durable platelet response and clinically significant bleeding.
  • Secondary outcomes assessed overall response, bleeding events, rescue medication use, and adverse events.

Main Results:

  • Five RCTs involving 261 participants were analyzed.
  • TPOras (Eltrombopag, Romiplostim) significantly increased durable and overall platelet response compared to placebo.
  • Eltrombopag demonstrated a significant reduction in both clinically significant and total bleeding events.

Conclusions:

  • Thrombopoietin-receptor agonists (TPOras) appear to be effective in enhancing platelet response in pediatric ITP.
  • Further research may be warranted to fully elucidate the safety profile and comparative effectiveness of different TPOras in this population.
Abstract

Keywords:
Pediatric immune thrombocytopeniaeltrombopagromiplostimsystematic reviewthrombopoietin-receptor agonists

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