BEST study: one-year descriptive follow-up of bevacizumab treatment in hereditary haemorrhagic telangiectasia post-BABH interventional study
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...
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| Main Authors: | , , , , , , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
SAGE Publishing
2025-04-01
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| Series: | Therapeutic Advances in Hematology |
| Online Access: | https://doi.org/10.1177/20406207241300828 |
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| Summary: | 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. |
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| ISSN: | 2040-6215 |