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

Обзор

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.

Вмешательства

• Препарат Bevacizumab
  This is a retrospective observational study of patients who received Bevacizumab as part of their routine clinical care. The study will describe efficacy and tolerance outcomes based on existing data (including echocardiographic, right heart catheterization, respiratory function, biologic and clinical data before and after treatment)

Первичные конечные точки

• Cardiac impairement before and after bevacizumab [Срок оценки: 6 months]

Вторичные конечные точки (8)

• Outcome [Срок оценки: 1 year]
• Adverse effects of bevacizumab [Срок оценки: 6 months]
• Right ventricular function [Срок оценки: 6 months]
• peak tricuspid regurgitant velocity [Срок оценки: 6 months]
• Liver ultrasound before and after bevacizumab [Срок оценки: 6 months]
• Clinical evolution before and after bevacizumab [Срок оценки: 6 months]
• Cardiac output [Срок оценки: 6 months]
• right ventricular function [Срок оценки: 6 months]

Критерии участия

Критерии включения

• 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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Только случаи

Центры проведения

Франция · 1 центр

• Centre hospitalier universitaire de Poitiers — Poitiers

Идентификаторы

NCT: NCT07445347 · BevaHHearT

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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