Intranasal submucosal bevacizumab for epistaxis in hereditary hemorrhagic telangiectasia: a double-blind, randomized, placebo-controlled trial.

BACKGROUND: The purpose of this study was to investigate the efficacy of bevacizumab ("Avastin") for the treatment of epistaxis in hereditary hemorrhagic telangiectasia (HHT). METHODS: In this double blind, placebo controlled trial, 15 adult patients with HHT with a minimum of 2 epistaxis episodes per week were randomized. A history of thromboembolic events or recent or planned surgery led to exclusion. Patients received a single intranasal submucosal injection (10 mL) of 100 mg bevacizumab or placebo. The primary outcome was the relative reduction of average daily epistaxis visual analog score (VAS). RESULTS: Average daily posttreatment VAS scores decreased by 27% in the bevacizumab group and by 3% in the placebo group (p = .57). The reduction in HHT epistaxis severity scores was -0.61 greater in the bevacizumab group compared to the placebo group (p = .34). CONCLUSION: Results show a trend toward reduced epistaxis with bevacizumab. This further supports the use of bevacizumab in HHT. Clincial Trials. gov number: NCT01314274