Efficacy and Safety of Various Medications in the Treatment of Hereditary Hemorrhagic Telangiectasia Epistaxis

OBJECTIVES: To compare the efficacy and safety of pharmacologic therapies for spontaneous epistaxis in hereditary hemorrhagic telangiectasia (HHT). DATA SOURCES: PubMed, Scopus, Embase, Web of Science, and the Cochrane Library were systematically searched up to August 2025. REVIEW METHODS: Eligible randomized controlled trials evaluating bevacizumab, doxycycline, tranexamic acid, or β-blockers versus placebo or standard care were included. Outcomes included epistaxis frequency and duration, hemoglobin (Hb), epistaxis severity score (ESS), transfusion or emergent-care requirements, quality of life (QOL), and adverse events. RESULTS: Tranexamic acid did not significantly reduce epistaxis duration or frequency or improve Hb, and it did not change transfusion or emergent-care needs, but it increased diarrhea (odds ratio 3.8, 95% confidence interval [CI] [1.7; 8.5]). Bevacizumab produced a modest reduction in epistaxis frequency (standardized mean difference -0.25, 95% CI [-0.47; -0.02]), without significant effects on duration, ESS, Hb, or QOL. Doxycycline showed no significant benefit for duration or frequency. Topical or systemic β-blockers did not significantly affect ESS, QOL, Hb, or overall adverse events. Across agents, between-study heterogeneity was generally low to moderate and CIs were wide, reflecting limited sample sizes. CONCLUSION: No pharmacologic agent demonstrated consistent, clinically robust superiority over placebo across primary endpoints. Bevacizumab may modestly reduce bleeding burden, whereas tranexamic acid increases gastrointestinal toxicity without clear efficacy. Pharmacologic control of HHT-related epistaxis remains suboptimal, underscoring the need for adequately powered trials with standardized outcome measures and optimized dosing and delivery strategies.