BEST研究：遗传性出血性毛细血管扩张症BABH介入研究后贝伐单抗治疗的一年期描述性随访
BEST study: one-year descriptive follow-up of bevacizumab treatment in hereditary haemorrhagic telangiectasia post-BABH interventional study.
作者信息
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
机构信息
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.
出版信息
Ther Adv Hematol. 2025 Apr 14;16:20406207241300828. doi: 10.1177/20406207241300828. eCollection 2025.
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.
背景
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。
目的
在BABH研究后随访并描述HHT患者的病情演变及治疗情况。
设计
本研究是一项为期1年的多中心描述性研究。
方法
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。
结果
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。
结论
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。
试验注册
本试验已在ClinicalTrials.gov注册，标识符为#NCT06039124。
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