MedicalResearch.com Interview with: Dr. Kevin J. Whitehead MD Adjunct Associate Professor, Pediatrics Associate Professor, Internal Medicine University of Utah MedicalResearch.com: What is the background for this study? Response: Hereditary hemorrhagic telangiectasia (or Osler-Weber-Rendu Syndrome) is a genetic vascular malformation syndrome that results in arteriovenous malformations. This syndrome is found in 1:5000 individuals, and is inherited in autosomal dominant fashion. It is often under diagnosed by the medical community (some estimate that 90% of patients do not know that they are affected). In the skin and mucous membranes small AVMs - termed telangiectasias - form, and they have the potential to rupture and bleed. This happens most commonly in the nose, and 95% of patients with HHT have recurrent nose bleeding (epistaxis). While other dangerous internal organ AVMs also form, epistaxis is the most important determinant of quality of life for these patients. This epistaxis can lead to anemia, heart failure and rarely death. Patients often seek therapy for epistaxis, but cautery or laser treatment of the telangiectasias tends to provide only temporary benefit. Definitive therapy to eliminate epistaxis requires surgical closure of the nasal passages, and is too drastic for most patients. We sought to test medical therapy for HHT-related epistaxis with our best candidate drugs in nose spray form. The drugs with the most anecdotal support include bevacizumab (an anti-angiogenic drug), estriol (an estrogen), and tranexamic acid (an inhibitor of fibrinolysis). Our patients received a nose spray with one of these drugs or a saline placebo and used two sprays in each nostril daily for 12 weeks and recorded their nose bleed frequency and duration in a daily diary. (more…)