Dupuis-Girod Sophie, Decullier Evelyne, Rivière Sophie, Lavigne Christian, Grobost Vincent, Leguy-Seguin Vanessa, Maillard Hélène, Chinet Thierry, Fargeton Anne-Emmanuelle, Guilhem Alexandre, Hermann Ruben
Hospices 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.
Laboratory Biology of Cancer and Infection, University Grenoble Alpes, Inserm, CEA, Grenoble, France.
Ther Adv Hematol. 2025 Apr 14;16:20406207241300828. doi: 10.1177/20406207241300828. eCollection 2025.
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.
To follow and describe evolution and treatments of patients with HHT post-BABH study.
This study is a 1-year, multi-centre descriptive study.
We collected clinical (nose and GI bleeding, red blood cell transfusions) and biological (haemoglobin and ferritin levels) data and treatment information.
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.
Bevacizumab effectively reduces RBC transfusions and is efficient for treating severe bleeding in patients with HHT.
This trial was registered with the ClinicalTrials.gov Identifier #NCT06039124.
遗传性出血性毛细血管扩张症（HHT）是一种遗传性血管疾病，其特征为毛细血管扩张，可导致鼻出血和胃肠道（GI）出血，以及内脏动静脉畸形。自2012年以来，贝伐单抗，一种靶向血管内皮生长因子的单克隆抗体，一直是治疗HHT相关出血的一种有前景的药物，并在II期BABH研究中进行了评估。
在BABH研究后随访并描述HHT患者的病情演变及治疗情况。
本研究是一项为期1年的多中心描述性研究。
我们收集了临床数据（鼻出血和胃肠道出血、红细胞输血情况）、生物学数据（血红蛋白和铁蛋白水平）以及治疗信息。
在4个中心纳入的22例患者中，15例接受了贝伐单抗治疗。其中，12例（86%）在治疗后3个月输注红细胞单位数量减少了50%以上。平均血红蛋白水平从83.08 g/L升至105.98 g/L。
贝伐单抗可有效减少红细胞输血，对治疗HHT患者的严重出血有效。
本试验已在ClinicalTrials.gov注册，标识符为#NCT06039124。
