Randomized, controlled, double-blinded clinical trial of effect of bevacizumab injection in management of epistaxis in hereditary hemorrhagic telangiectasia patients undergoing surgical cauterization.

BACKGROUND

Given its role in the disease pathophysiology, inhibition of vascular endothelial growth factor (VEGF)-mediated angiogenesis has received attention as a potential strategy to reduce epistaxis associated with hereditary hemorrhagic telangiectasia (HHT). In this study we evaluated 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 the 12-item Short Form (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 a reduced ESS vs baseline at 1 (-1.2; p = 0.01) and 4 (-1.2; p = 0.05) months postprocedure. The bevacizumab group demonstrated a reduced ESS score vs 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 postprocedure. The additive benefit of bevacizumab over saline exceeded the MCID at 1, 2, and 4 months, but the difference was not statistically significant.

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 with electrocautery alone.