Real-world evidence on clinical outcomes in immune thrombocytopenia treated with thrombopoietin receptor agonists

J Comp Eff Res. 2020 May;9(7):447-457. doi: 10.2217/cer-2019-0177. Epub 2020 Mar 16.

Abstract

Aim: Eltrombopag and romiplostim are US FDA approved for treatment of immune thrombocytopenia in patients with insufficient response to other treatments. Clinical or real-world data comparing outcomes of the two drugs are limited. Methods: This retrospective cross-sectional study sought information on bleeding-related episodes (BREs), adverse events (AEs) and other outcomes of eltrombopag or romiplostim treatment in immune thrombocytopenia. Results: Patients receiving eltrombopag experienced significantly reduced BREs, severe BREs, rescue medication use and platelet transfusions. Diarrhea and headache were significantly less frequent in patients receiving eltrombopag; other AEs occurred equally in both groups. Conclusion: There may be a potential advantage for the use of eltrombopag versus romiplostim in the practice settings studied, based on rates of BREs and AEs and rescue medication utilization.

Keywords: adverse events; bleeding episodes; eltrombopag; immune thrombocytopenia; romiplostim; treatment outcomes.

Publication types

  • Observational Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aged
  • Benzoates / adverse effects
  • Benzoates / therapeutic use*
  • Cross-Sectional Studies
  • Female
  • Humans
  • Hydrazines / adverse effects
  • Hydrazines / therapeutic use*
  • Male
  • Middle Aged
  • Platelet Count
  • Purpura, Thrombocytopenic, Idiopathic / drug therapy*
  • Pyrazoles / adverse effects
  • Pyrazoles / therapeutic use*
  • Receptors, Fc / therapeutic use*
  • Receptors, Thrombopoietin / agonists*
  • Recombinant Fusion Proteins / adverse effects
  • Recombinant Fusion Proteins / therapeutic use*
  • Retrospective Studies
  • Thrombopoietin / adverse effects
  • Thrombopoietin / therapeutic use*

Substances

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