Efficacy and Tolerance of Treatment With Bevacizumab for Severe Liver Involvement With High Cardiac Output in Hereditary Hemorrhagic Telangiectasia Within the French Hereditary Hemorrhagic Telangiectasia Network
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 入组人数
- 111
- 试验地点
- 1
- 主要终点
- Cardiac impairement before and after bevacizumab
研究概览
简要总结
High cardiac output secondary to hepatic arteriovenous malformations may be isolated or associated with left heart failure with post-capillary pulmonary hypertension. More rarely, precapillary pulmonary hypertension develops, linked to obstructive pulmonary arterial remodeling, referred to as pulmonary arterial hypertension (PAH), which affects younger patients and is not necessarily associated with hepatic arteriovenous malformation.
BEVACIZUMAB is an anti-VEGF treatment indicated under compassionate use guidelines for hereditary hemorrhagic telangiectasia in cases of symptomatic hepatic arteriovenous malformations, when complicated by isolated high cardiac output or post-capillary pulmonary hypertension, and in cases of refractory chronic bleeding. However, the efficacy of this treatment on pulmonary hypertension related to high cardiac output, isolated or associated with left heart failure, is poorly understood. In addition, this treatment is classified as a "possible association" for the development of PAH, according to the 7th World Congress Symposium on Pulmonary Hypertension. Indeed, Hlavaty et al. found, based on pharmacovigilance data and by searching for disproportionate effects using the Bayesian network method, a possible link between the use of BEVACIZUMAB and the development of PAH. This treatment is therefore not recommended in cases of PAH associated with hereditary hemorrhagic telangiectasia.
The objective of this study is to investigate the efficacy and tolerability of Bevacizumab treatment in hereditary hemorrhagic telangiectasia with cardiac involvement (isolated symptomatic high cardiac output or associated with post-capillary PAH) secondary to severe liver damage, based on the experience of the French hereditary hemorrhagic telangiectasia network since the CIROCO registry was opened in 2009.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •hereditary hemorrhagic telangiectasia
- •treated with BEVACIZUMAB for severe liver involvement with high cardiac output (symptomatic isolated high cardiac output or associated with post-capillary pulmonary hypertension or combined pulmonary hypertension or any pulmonary hypertension)
- •hemodynamic data (cardiac ultrasound or right heart catheterization) within 12 months prior to the start of Bevacizumab treatment
- •in the French hereditary hemorrhagic telangiectasia network and registered in the CIROCO national hereditary hemorrhagic telangiectasia
排除标准
- 未提供
研究组 & 干预措施
hereditary hemorrhagic telangiectasia patients
Adult patients with hereditary hemorrhagic telangiectasia who have received treatment with BEVACIZUMAB for severe liver involvement with high cardiac output (symptomatic isolated high cardiac output or associated with post-capillary pulmonary hypertension or combined pulmonary hypertension or any pulmonary hypertension) in hereditary hemorrhagic telangiectasia within the French hereditary hemorrhagic telangiectasia network and registered in the CIROCO national hereditary hemorrhagic telangiectasia registry and having hemodynamic data (cardiac ultrasound or right heart catheterization) within 12 months prior to the start of Bevacizumab treatment
干预措施: Bevacizumab (Drug)
结局指标
主要结局
Cardiac impairement before and after bevacizumab
时间窗: 6 months
mean pulmonary arterial pressure (right heart catheterization) or systolic pulmonary arterial pressure (cardiac ultrasound) in mmHg before (M0) and after (M6) treatment (bevacizumab)
次要结局
- Cardiac output(6 months)
- right ventricular function(6 months)
- Clinical evolution before and after bevacizumab(6 months)
- Outcome(1 year)
- Adverse effects of bevacizumab(6 months)
- Right ventricular function(6 months)
- peak tricuspid regurgitant velocity(6 months)
- Liver ultrasound before and after bevacizumab(6 months)