Availability, use, efficacy and safety of bevacizumab in European hereditary haemorrhagic telangiectasia centres

Introduction Bevacizumab, a vascular endothelial growth factor inhibitor, is used off‐label for treatment of severe anaemia related to epistaxis, gastrointestinal bleeding and/or severe hepatic arteriovenous malformations (HAVM) and right‐sided cardiac failure in patients with hereditary haemorrhagic telangiectasia (HHT). Aim To report the experience of treatment with bevacizumab within European Reference Network for Rare Multisystemic Vascular Diseases (VASCERN) centres. Method A retrospective analysis of the usage, availability, efficacy and safety in HHT patients treated with bevacizumab in European VASCERN centres. Results A total of 151 patients received treatment with bevacizumab in European VASCERN centres. Most patients were treated in Denmark, France and the Netherlands. There was an improvement in haemoglobin with a mean increase of 2.9 g/dL (95% confidence interval 2.4, 3.6) and significant reduction in the number of red blood cell transfusions. Cardiac output was measured and improved in 85% of patients treated for high output cardiac failure in relation to severe HAVM. No severe adverse events were recorded, but 70 patients experienced at least one adverse event. Conclusion Although this is a retrospective study, we demonstrated convincing efficacy of bevacizumab in the treatment of patients with HHT, with a good risk‐benefit balance.