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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| Format: | Article |
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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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| author | Sophie Dupuis-Girod Evelyne Decullier Sophie Rivière Christian Lavigne Vincent Grobost Vanessa Leguy-Seguin Hélène Maillard Thierry Chinet Anne-Emmanuelle Fargeton Alexandre Guilhem Ruben Hermann |
| author_facet | Sophie Dupuis-Girod Evelyne Decullier Sophie Rivière Christian Lavigne Vincent Grobost Vanessa Leguy-Seguin Hélène Maillard Thierry Chinet Anne-Emmanuelle Fargeton Alexandre Guilhem Ruben Hermann |
| author_sort | Sophie Dupuis-Girod |
| collection | DOAJ |
| description | 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. |
| format | Article |
| id | doaj-art-0672b0282f404707aa997e6aba924019 |
| institution | OA Journals |
| issn | 2040-6215 |
| language | English |
| publishDate | 2025-04-01 |
| publisher | SAGE Publishing |
| record_format | Article |
| series | Therapeutic Advances in Hematology |
| spelling | doaj-art-0672b0282f404707aa997e6aba9240192025-08-20T02:20:25ZengSAGE PublishingTherapeutic Advances in Hematology2040-62152025-04-011610.1177/20406207241300828BEST study: one-year descriptive follow-up of bevacizumab treatment in hereditary haemorrhagic telangiectasia post-BABH interventional studySophie Dupuis-GirodEvelyne DecullierSophie RivièreChristian LavigneVincent GrobostVanessa Leguy-SeguinHélène MaillardThierry ChinetAnne-Emmanuelle FargetonAlexandre GuilhemRuben HermannBackground: 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.https://doi.org/10.1177/20406207241300828 |
| spellingShingle | Sophie Dupuis-Girod Evelyne Decullier Sophie Rivière Christian Lavigne Vincent Grobost Vanessa Leguy-Seguin Hélène Maillard Thierry Chinet Anne-Emmanuelle Fargeton Alexandre Guilhem Ruben Hermann BEST study: one-year descriptive follow-up of bevacizumab treatment in hereditary haemorrhagic telangiectasia post-BABH interventional study Therapeutic Advances in Hematology |
| title | BEST study: one-year descriptive follow-up of bevacizumab treatment in hereditary haemorrhagic telangiectasia post-BABH interventional study |
| title_full | BEST study: one-year descriptive follow-up of bevacizumab treatment in hereditary haemorrhagic telangiectasia post-BABH interventional study |
| title_fullStr | BEST study: one-year descriptive follow-up of bevacizumab treatment in hereditary haemorrhagic telangiectasia post-BABH interventional study |
| title_full_unstemmed | BEST study: one-year descriptive follow-up of bevacizumab treatment in hereditary haemorrhagic telangiectasia post-BABH interventional study |
| title_short | BEST study: one-year descriptive follow-up of bevacizumab treatment in hereditary haemorrhagic telangiectasia post-BABH interventional study |
| title_sort | best study one year descriptive follow up of bevacizumab treatment in hereditary haemorrhagic telangiectasia post babh interventional study |
| url | https://doi.org/10.1177/20406207241300828 |
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