Iorio Annamaria, Lucà Fabiana, Pozzi Andrea, Rao Carmelo Massimiliano, Chimenti Cristina, Di Fusco Stefania Angela, Rossini Roberta, Caretta Giorgio, Cornara Stefano, Giubilato Simona, Di Matteo Irene, Di Nora Concetta, Pilleri Anna, Gelsomino Sandro, Ceravolo Roberto, Riccio Carmine, Grimaldi Massimo, Colivicchi Furio, Oliva Fabrizio, Gulizia Michele Massimo
Cardiology Department, Papa Giovanni XXIII Hospital, 24127 Bergamo, Italy.
Cardiology Department, Grande Ospedale Metropolitano, GOM, AO Bianchi Melacrino Morelli, 89129 Reggio Calabria, Italy.
Diagnostics (Basel). 2024 Jan 18;14(2):208. doi: 10.3390/diagnostics14020208.
Anderson-Fabry disease (AFD) is a lysosome storage disorder resulting from an X-linked inheritance of a mutation in the galactosidase A (GLA) gene encoding for the enzyme alpha-galactosidase A (α-GAL A). This mutation results in a deficiency or absence of α-GAL A activity, with a progressive intracellular deposition of glycosphingolipids leading to organ dysfunction and failure. Cardiac damage starts early in life, often occurring sub-clinically before overt cardiac symptoms. Left ventricular hypertrophy represents a common cardiac manifestation, albeit conduction system impairment, arrhythmias, and valvular abnormalities may also characterize AFD. Even in consideration of pleiotropic manifestation, diagnosis is often challenging. Thus, knowledge of cardiac and extracardiac diagnostic "red flags" is needed to guide a timely diagnosis. Indeed, considering its systemic involvement, a multidisciplinary approach may be helpful in discerning AFD-related cardiac disease. Beyond clinical pearls, a practical approach to assist clinicians in diagnosing AFD includes optimal management of biochemical tests, genetic tests, and cardiac biopsy. We extensively reviewed the current literature on AFD cardiomyopathy, focusing on cardiac "red flags" that may represent key diagnostic tools to establish a timely diagnosis. Furthermore, clinical findings to identify patients at higher risk of sudden death are also highlighted.
安德森-法布里病(AFD)是一种溶酶体贮积症,由编码α-半乳糖苷酶A(α-GAL A)的半乳糖苷酶A(GLA)基因的X连锁遗传突变引起。这种突变导致α-GAL A活性缺乏或缺失,糖鞘脂在细胞内逐渐沉积,导致器官功能障碍和衰竭。心脏损害在生命早期就开始,通常在明显的心脏症状出现之前以亚临床形式发生。左心室肥厚是常见的心脏表现,尽管传导系统损害、心律失常和瓣膜异常也可能是AFD的特征。即使考虑到多效性表现,诊断往往也具有挑战性。因此,需要了解心脏和心脏外的诊断“红旗”来指导及时诊断。事实上,考虑到其全身受累,多学科方法可能有助于识别AFD相关的心脏病。除了临床要点外,协助临床医生诊断AFD的实用方法包括对生化检查、基因检测和心脏活检的优化管理。我们广泛回顾了目前关于AFD心肌病的文献,重点关注可能是及时诊断的关键诊断工具的心脏“红旗”。此外,还强调了识别猝死风险较高患者的临床发现。