An Umbrella Review of Meta-Analyses on the Efficacy and Safety of Eltrombopag in Immune Thrombocytopenia.

BACKGROUND AND AIMS: Immune thrombocytopenia (ITP) is an autoimmune disorder with increased bleeding risk. Eltrombopag, an oral thrombopoietin receptor agonist, is widely used after corticosteroids or immunoglobulins. Multiple meta-analyses support its efficacy, yet safety concerns persist. We conducted an umbrella review to synthesize evidence on efficacy and safety across age groups.

METHODS

We searched PubMed, Scopus, and Web of Science through April 2025 for systematic reviews/meta-analyses on eltrombopag in ITP. Methodological quality was appraised using AMSTAR 2. Outcomes included platelet response, bleeding, and adverse events. We mapped primary trials across reviews to address overlap and qualitatively summarized findings; no new pooling was performed.

RESULTS

Nine meta-analyses met inclusion. Across adult/mixed populations, eltrombopag increased platelet response (e.g., RR ≈ 3.4, p < 0.001) and reduced bleeding; pediatric effects were directionally similar but less precise with higher heterogeneity. Reported adverse events included hepatobiliary abnormalities and thrombotic events, underscoring the need for monitoring.

CONCLUSION

Eltrombopag is effective in raising platelet counts and reducing bleeding in ITP. Benefits appear to increase up to ~50 mg daily and plateau thereafter. Age-related variability and safety considerations support individualized dosing and vigilant follow-up.