Praxis Medical Insights

Est. 2024 • Clinical Guidelines Distilled

Made possible by volunteer editors from the University of Calgary & University of Alberta

Last Updated: 11/9/2025

Eltrombopag Use in Immune Thrombocytopenia

Patient Selection Criteria

  • Use eltrombopag in patients with ITP lasting ≥3 months who are corticosteroid-dependent or have no response to corticosteroids, as recommended by the American Society of Hematology 1

Positioning in Treatment Algorithm

  • The American Society of Hematology guidelines do not provide preferential recommendation between eltrombopag and romiplostim for patients with ITP ≥3 months who are corticosteroid-dependent or non-responsive 1
  • TPO-RAs like eltrombopag are increasingly preferred over splenectomy due to the increased risk of septicemia, pulmonary embolism, and non-Hodgkin lymphoma associated with splenectomy 3
  • Splenectomy requires lifelong sepsis prevention management and has 20-30% relapse rates, whereas TPO-RAs demonstrate 70-80% response rates in clinical trials 3, 2
  • The choice between eltrombopag (oral) versus romiplostim (subcutaneous) depends primarily on patient preference for route of administration, with both agents being well-tolerated with long-term use and showing similar efficacy profiles 2

Potential for Treatment-Free Remission

  • Approximately 30% of patients achieve long-term (≥6 months) remission that is sustained off treatment with eltrombopag, compared to only 9% spontaneous remission rate in untreated adults, with disease-modifying activity linked to TPO-RA-mediated restoration of impaired regulatory T-cell function and immune tolerance 3
  • For patients with stable responses for ≥6 months, tapering can be considered, though most patients require continuous therapy 3, 2

Common Pitfalls to Avoid

  • Do not delay switching from corticosteroids to second-line therapy, such as eltrombopag, in patients requiring on-demand corticosteroid administration after completing first-line induction, as recommended by the American Society of Hematology 2
  • Do not continue initial corticosteroid treatment beyond 6-8 weeks before considering alternative therapy, such as eltrombopag, to avoid prolonged corticosteroid exposure 2