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BEST study: one-year descriptive follow-up of bevacizumab treatment in hereditary haemorrhagic telangiectasia
Sophie Dupuis-Girod1,2, Evelyne Decullier3,4, Sophie Rivière5
1Hospices Civils de Lyon, Hôpital Femme-Mère-Enfants, Service de Génétique et centre de Référence de la maladie de Rendu-Osler, 59 Bd Pinel, Bron 69677, France.
Background:
Hereditary haemorrhagic telangiectasia (HHT) is a genetic vascular disorder characterised by telangiectases, which cause nasal and gastrointestinal (GI) bleeding, and visceral arteriovenous malformations. Since 2012 bevacizumab, a monoclonal antibody targeting vascular endothelial growth factor, has been a promising treatment for HHT-related bleeding and was evaluated in the phase II BABH study.
Objective:
To follow and describe evolution and treatments of patients with HHT post-BABH study.
Design:
This study is a 1-year, multi-centre descriptive study.
Methods:
We collected clinical (nose and GI bleeding, red blood cell transfusions) and biological (haemoglobin and ferritin levels) data and treatment information.
Results:
Of 22 patients included across 4 centers, 15 received bevacizumab. Among them, 12 (86%) had a >50% decrease in the number of RBC units transfused 3 months post-treatment. Mean haemoglobin levels increased from 83.08 to 105.98 g/L.
Conclusion:
Bevacizumab effectively reduces RBC transfusions and is efficient for treating severe bleeding in patients with HHT.
Trial Registration:
This trial was registered with the ClinicalTrials.gov Identifier #NCT06039124.
