Assistant Professor University of Colorado Aurora, Colorado
Introduction: Given its role in the disease pathophysiology, inhibition of VEGF-mediated angiogenesis has received attention as a potential strategy to reduce epistaxis associated with hereditary hemorrhagic telangiectasia (HHT). This study evaluates the efficacy of a submucosal injection of bevacizumab, a VEGF-inhibitor, in reducing the severity of epistaxis and improving quality of life when given at the time of operative electrocautery.
Methods: This randomized, double-blinded placebo-controlled trial was conducted at a single institution from 2014 to 2019. Patients scheduled to undergo operative bipolar electrocautery of nasal telangiectasias were randomized to receive a submucosal injection of saline or bevacizumab at time of surgery. Surveys to assess epistaxis severity and quality-of-life (QOL), including the Epistaxis Severity Score (ESS) and Short Form 12 (SF-12), were administered preoperatively and at 1, 2, 4, and 6 months postoperatively. The minimal clinically important difference (MCID) of the ESS instrument is reported to be 0.71.
Results: Of 39 patients enrolled, 37 (94.9%) completed the study. The saline group demonstrated reduced ESS versus baseline at 1 (-1.2, p=0.01) and 4 (-1.2, p=0.05) months post-procedure. The bevacizumab group demonstrated reduced ESS versus baseline at 1 (-2.3, p<0.001), 2 (-2.3, p<0.001), 4 (-2.0, p=0.003), and 6 (-1.3, p=0.05) months post-procedure. The additive benefit of bevacizumab over saline was not statistically significant but exceeded the MCID at 1, 2, and 4 months.
Conclusion: The addition of a single treatment of submucosal bevacizumab may be associated with additional clinically meaningful benefit for up to 4 months when compared to electrocautery alone.