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
{"title":"BEST研究：贝伐单抗治疗遗传性出血性毛细血管扩张后babh介入研究的一年描述性随访。","authors":"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","doi":"10.1177/20406207241300828","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>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.</p><p><strong>Objective: </strong>To follow and describe evolution and treatments of patients with HHT post-BABH study.</p><p><strong>Design: </strong>This study is a 1-year, multi-centre descriptive study.</p><p><strong>Methods: </strong>We collected clinical (nose and GI bleeding, red blood cell transfusions) and biological (haemoglobin and ferritin levels) data and treatment information.</p><p><strong>Results: </strong>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.</p><p><strong>Conclusion: </strong>Bevacizumab effectively reduces RBC transfusions and is efficient for treating severe bleeding in patients with HHT.</p><p><strong>Trial registration: </strong>This trial was registered with the ClinicalTrials.gov Identifier #NCT06039124.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"16 ","pages":"20406207241300828"},"PeriodicalIF":3.4000,"publicationDate":"2025-04-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12033602/pdf/","citationCount":"0","resultStr":"{\"title\":\"BEST study: one-year descriptive follow-up of bevacizumab treatment in hereditary haemorrhagic telangiectasia post-BABH interventional study.\",\"authors\":\"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\",\"doi\":\"10.1177/20406207241300828\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background: </strong>Hereditary haemorrhagic telangiectasia (HHT) is a genetic vascular disorder characterised by telangiectases, which cause nasal and gastrointestinal (GI) bleeding, and visceral arteriovenous malformations. 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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 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.
期刊介绍：
Therapeutic Advances in Hematology delivers the highest quality peer-reviewed articles, reviews, and scholarly comment on pioneering efforts and innovative studies across all areas of hematology. The journal has a strong clinical and pharmacological focus and is aimed at clinicians and researchers in hematology, providing a forum in print and online for publishing the highest quality articles in this area.
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