Intranasal submucosal bevacizumab for epistaxis in hereditary hemorrhagic telangiectasia: A double‐blind, randomized, placebo‐controlled trial

Abstract

Background The purpose of this study was to investigate the efficacy of bevacizumab (“Avastin”) for the treatment of epistaxis in hereditary hemorrhagic telangiectasia (HHT). Methods In this double blind, placebo controlled trial, 15 adult patients with HHT with a minimum of 2 epistaxis episodes per week were randomized. A history of thromboembolic events or recent or planned surgery led to exclusion. Patients received a single intranasal submucosal injection (10 mL) of 100 mg bevacizumab or placebo. The primary outcome was the relative reduction of average daily epistaxis visual analog score (VAS). Results Average daily posttreatment VAS scores decreased by 27% in the bevacizumab group and by 3% in the placebo group (p = .57). The reduction in HHT epistaxis severity scores was ‐0.61 greater in the bevacizumab group compared to the placebo group (p = .34). Conclusion Results show a trend toward reduced epistaxis with bevacizumab. This further supports the use of bevacizumab in HHT. Clincial Trials. gov number: NCT01314274 © 2014 Wiley Periodicals, Inc. Head Neck 37: 783–787, 2015

Treatment of epistaxis in hereditary hemorrhagic telangiectasia with tranexamic acid - a double-blind placebo-controlled cross-over phase IIIB study

“…drugs related to female sex hormones are increased thrombotic events and feminization of men. A fourth double blind placebo controlled study on HHT showed that intranasal submucosal injection of bevacizumab led to an improvement in epistaxis; however, this was not statistically significant [15]. Potential side effects of this antibody against vascular endothelial growth factor (VEGF) include thrombembolic events.…”

Hereditary hemorrhagic telangiectasia: diagnosis and management from the hematologist’s perspective

“…6,7 However, data on intranasal bevacizumab have been conflicting, and studies investigating the use of intravenous bevacizumab are limited to case reports and retrospective series. 8 Treatment of HHT involves a multidisciplinary approach of specialists in cardiology, pulmonology, hepatology, interventional radiology, ear, nose and throat (ENT), genetics, and hematology.…”

Genetic and Molecular Basis for Hereditary Hemorrhagic Telangiectasia

“…In addition, serum VEGF is reportedly increased in HHT patients, although levels do not correlate with disease severity [100][101][102]. Administration of Bevacizumab (Avastin Ò ), a VEGF antibody, can ameliorate liver AVMs [103][104][105] and reduce epistaxis [106][107][108][109][110][111][112], although a recent Phase I clinical trial (nasal spray administration) disappointingly showed no efficacy in the treatment of epistaxis [113]. Thalidomide is a second HHT therapeutic that targets angiogenesis.…”