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

Abstract

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.