Long-term experience with intranasal bevacizumab therapy

Area of Science:

• Otolaryngology
• Vascular Medicine
• Pharmacology

Background:

• Hereditary hemorrhagic telangiectasia (HHT) is a genetic disorder causing abnormal blood vessel formation.
• Epistaxis (nosebleeds) is a common and often severe symptom of HHT, significantly impacting quality of life.
• Intranasal bevacizumab has emerged as a potential treatment for HHT-associated epistaxis.

Purpose of the Study:

• To evaluate the long-term effectiveness and safety of intranasal bevacizumab injections for treating epistaxis in HHT patients.
• To assess treatment durability and identify potential resistance phenomena.

Main Methods:

• A prospective, noncomparative study involving 33 HHT patients treated with intranasal submucosal bevacizumab injections.
• Patients were followed long-term, with effectiveness assessed using the Epistaxis Severity Score (ESS) and the Epistaxis Intensity, Frequency, and Blood Transfusion (IFT) score.
• Hemoglobin levels were also monitored.

Main Results:

• Of 33 patients, 12 (36.3%) showed sustained positive response to intranasal bevacizumab at the study's end.
• Eleven patients (33.3%) had initial improvement, but efficacy waned over time, necessitating treatment discontinuation.
• Four patients (12.1%) showed no improvement; one patient experienced osteonecrosis of the knees.

Conclusions:

• Intranasal bevacizumab is an effective treatment for moderate to severe HHT-related epistaxis.
• Treatment duration and response variability are significant, with common primary and late resistance.
• While generally safe locally, systemic side effects like osteonecrosis warrant consideration.