Long‐term follow‐up of intranasal bevacizumab therapy in hereditary hemorrhagic telangiectasia (HHT).

Prospective, noncomparative study.

Patients treated for HHT‐associated epistaxis by intranasal submucosal bevacizumab injections between June 2011 and August 2013 were included and followed prospectively. The effectiveness of the treatment was evaluated by the epistaxis severity score (ESS); the epistaxis intensity, frequency, and the need of blood transfusion (IFT) score; and hemoglobin levels.

Thirty‐three patients were included. The total number of treatments with intranasal bevacizumab injection was 210. The mean number of treatments per patient was 6.2 ± 4.6 (range, 1–16), and the mean treatment and observation period was 38.8 ± 21.8 months (range, 2–66 months). Four patients showed no improvement after treatment. Eleven patients (33.3%) showed initial improvement in both ESS and IFT, but the treatment was discontinued before the end of the study because the effect became gradually shorter lasting despite repeated injections. Twelve patients (36.3%) continued to have a positive response to the treatment at the end of the study. No local adverse effects were observed, but one patient developed osteonecrosis in both knees during the treatment period.

Intranasal bevacizumab injection is an effective treatment for most of the moderate and severe grades of HHT‐associated epistaxis. The duration of the effect of the treatment was variable. Primary and late resistance phenomena to the treatment were quite common.

4. Laryngoscope, 128:2237–2244, 2018