Intranasal Therapy for Epistaxis in Hereditary Hemorrhagic Telangiectasia

Epistaxis is a frequent and debilitating feature of hereditary hemorrhagic telangiectasia (HHT). Nasal packing, laser ablation, and septodermoplasty are often required to control persistent bleeding, and severe iron-deficiency anemia secondary to chronic blood loss is common.

To determine whether topical application of agents with various mechanisms of action decreases epistaxis frequency, investigators conducted a multicenter, double-blind, placebo-controlled, randomized phase II trial of bevacizumab, estriol, tranexamic acid, or saline placebo spray (twice daily for 12 weeks) in 121 HHT patients having weekly episodes of epistaxis lasting at least 1 minute.

Median epistaxis frequency during weeks 5 through 12 (the primary outcome) and the duration of epistaxis were not significantly different among patients using any of the agents or placebo. Epistaxis severity scores declined in all groups by week 12, then rose by week 24 but remained statistically lower than at baseline (P<0.001). The agents had no effect on the levels of hemoglobin, ferritin, or need for transfusion. Tranexamic acid was associated with gastrointestinal symptoms; the other agents were well-tolerated.

In a separate multicenter, double-blind, placebo-controlled, randomized phase II trial, French investigators examined whether bevacizumab spray shortened the duration of epistaxis in 80 HHT patients with mean epistaxis duration of more than 20 minutes per month during the previous 3 months. Patients were randomly assigned to receive bevacizumab (25, 50, or 75 mg) or saline placebo spray intranasally (3 times at 14-day intervals).

During the 3 months following treatment, no effect was observed with any dose on the duration or number of episodes of epistaxis, transfusion requirements, or levels of hemoglobin and ferritin. No adverse effects were recorded.