TY - JOUR
T1 - Availability, use, efficacy and safety of bevacizumab in European hereditary haemorrhagic telangiectasia centres
AU - Haahr, Pernille D
AU - Kjeldsen, Anette D
AU - Fialla, Annette D
AU - Fargeton, Anne-Emmanuelle
AU - Guilhem, Alexandre
AU - Buscarini, Elisabetta
AU - Droege, Freya
AU - Manfredi, Guido
AU - Mager, Hans-Jurgen
AU - Saurin, Jean-Christophe
AU - Kjeldsen, Jens
AU - Hessels, Josefien
AU - Post, Marco C
AU - Mandic, Robert
AU - Boerman, Sanne
AU - Kasper-Virchow, Stefan
AU - Möller, Sören
AU - Geisthoff, Urban
AU - Dupuis-Girod, Sophie
N1 - Publisher Copyright:
© 2025 The Author(s). British Journal of Clinical Pharmacology published by John Wiley & Sons Ltd on behalf of British Pharmacological Society.
PY - 2026/2
Y1 - 2026/2
N2 - 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.
AB - 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.
KW - VASCERN
KW - bevacizumab
KW - hereditary haemorrhagic telangiectasia
UR - https://www.scopus.com/pages/publications/105017049000
U2 - 10.1002/bcp.70273
DO - 10.1002/bcp.70273
M3 - Article
C2 - 40985322
SN - 0306-5251
VL - 92
SP - 535
EP - 544
JO - British Journal of Clinical Pharmacology
JF - British Journal of Clinical Pharmacology
IS - 2
ER -