心血管多模态成像在评估安德森-法布里病中的作用:从早期诊断到治疗监测》ESC 心肌和心包疾病工作组和欧洲心血管成像协会的临床共识声明。

IF 6.7 1区 医学 Q1 CARDIAC & CARDIOVASCULAR SYSTEMS
Matteo Cameli, Maurizio Pieroni, Maria Concetta Pastore, Antonio Brucato, Silvia Castelletti, Lia Crotti, Marc Dweck, Andrea Frustaci, Alessia Gimelli, Karin Klingel, Petr Kuchynka, Johanna Kuusisto, George Lazaros, Giulia Elena Mandoli, Marco Merlo, James Moon, Denisa Muraru, Antonis Pantazis, Angelos G Rigopoulos, Arsen Ristic, Leyla Elif Sade, Mary N Sheppard, Carsten Tschöpe, Steffen E Petersen, Massimo Imazio, Yohann Bohbot, Maja Cikes, Pankaj Garg, Niall Keenan, Aniela Petrescu, Ivan Stankovic, Liliana Szabo, Valtteri Uusitalo
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引用次数: 0

摘要

安德森-法布里病(AFD)是一种罕见的x连锁遗传疾病,其特征是α -半乳糖苷酶a (α -gal)缺陷,其损害鞘糖脂代谢并导致球三烷基神经酰胺(Gb3)在溶酶体内过度储存。AFD累及肾脏、心脏、血管和神经系统,主要见于儿童期发病的男性患者,成人也常出现心脏表现。AFD心肌病是由心肌细胞内Gb3的积累引起的,首先表现为左心室肥厚和舒张功能障碍,导致限制性心肌病和收缩期心力衰竭并累及双心室。AFD心肌病的诊断在最初阶段可能是隐匿的,需要与其他肥厚型心肌病准确鉴别诊断。然而,及时启动已经显示出有希望的结果的特定疗法,特别是早期治疗,是至关重要的。诊断累及心脏的AFD需要仔细整合临床评估、基因检测和心脏成像。基础和高级超声心动图、心脏磁共振和核成像可以为早期诊断提供关键信息(中央插图),这些患者的管理通常仅限于在该领域具有高专业知识的中心。这份临床共识声明由欧洲心脏病学会(ESC)心肌和心包疾病工作组和ESC欧洲心血管成像协会的专家制定,旨在为所有临床医生提供实用的建议,以使用多模态成像来简化AFD的诊断评估、预后分层和心脏受累的管理。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
The role of cardiovascular multimodality imaging in the evaluation of Anderson-Fabry disease: from early diagnosis to therapy monitoring.

Anderson-Fabry disease (AFD) is a rare genetic disease with X-linked transmission characterized by a defect in the enzyme alpha-galactosidase A, which impairs glycosphingolipid metabolism and leads to an excessive storage of globotriaosylceramide (Gb3) within lysosomes. AFD involves renal, cardiac, vascular, and nervous systems and is mainly observed in male patients with onset in childhood, although cardiac manifestation is often shown in adults. AFD cardiomyopathy is caused by the accumulation of Gb3 within myocytes first showed by left ventricular hypertrophy and diastolic dysfunction, leading to restrictive cardiomyopathy and systolic heart failure with biventricular involvement. The diagnosis of AFD cardiomyopathy may be insidious in the first stages and requires accurate differential diagnosis with other cardiomyopathies with hypertrophic phenotype. However, it is fundamental to promptly initiate specific therapies that have shown promising results, particularly for early treatment. A careful integration between clinical evaluation, genetic tests, and cardiac imaging is required to diagnose AFD with cardiac involvement. Basic and advanced echocardiography, cardiac magnetic resonance, and nuclear imaging may offer pivotal information for early diagnosis (Graphical Abstract), and the management of these patients is often limited to centres with high expertise in the field. This clinical consensus statement, developed by experts from the European Society of Cardiology (ESC) Working Group on Myocardial and Pericardial Diseases and the European Association of Cardiovascular Imaging of the ESC, aims to provide practical advice for all clinicians regarding the use of multimodality imaging to simplify the diagnostic evaluation, prognostic stratification, and management of cardiac involvement in AFD.

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来源期刊
European Heart Journal - Cardiovascular Imaging
European Heart Journal - Cardiovascular Imaging CARDIAC & CARDIOVASCULAR SYSTEMS-
CiteScore
11.60
自引率
9.70%
发文量
708
审稿时长
4-8 weeks
期刊介绍: European Heart Journal – Cardiovascular Imaging is a monthly international peer reviewed journal dealing with Cardiovascular Imaging. It is an official publication of the European Association of Cardiovascular Imaging, a branch of the European Society of Cardiology. The journal aims to publish the highest quality material, both scientific and clinical from all areas of cardiovascular imaging including echocardiography, magnetic resonance, computed tomography, nuclear and invasive imaging. A range of article types will be considered, including original research, reviews, editorials, image focus, letters and recommendation papers from relevant groups of the European Society of Cardiology. In addition it provides a forum for the exchange of information on all aspects of cardiovascular imaging.
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