Hereditary hemorrhagic telangiectasia (HHT) is a disease characterized by mucocutaneous
telangiectasias and visceral
arteriovenous malformations. The genetic mutations that cause this disease result in elevated levels of
vascular endothelial growth factor, which is inhibited by
bevacizumab. Previous studies have shown
bevacizumab treatment to be effective in reducing symptoms, but study protocols have all used oncological dosing parameters, which carry several well-described serious side effects. This study investigates whether drastically lower dosages of
bevacizumab than normally used in oncological treatment could control
epistaxis in patients with HHT and medically refractory
epistaxis. A prospective, open-label, noncomparative study enrolled six patients receiving 0.125-mg/kg infusions of
bevacizumab once every 4 weeks for a total of six infusions. Severity of
epistaxis was assessed with the
epistaxis severity score, and quality-of-life measures were followed with the 20-item Sino-Nasal Outcome Test and 36-item Short Form surveys. A statistically significant improvement was seen in the control of
epistaxis severity and frequency, with minimal negative side effects and high patient satisfaction. Very low dose
bevacizumab treatment is an effective method of controlling medically refractory
epistaxis in patients with HHT and additional investigation to optimize dosing guidelines is warranted.