Department of Medicine, Division of Cardiology, University Hospital, Wuerzburg, Germany.
Annu Rev Med. 2011;62:59-67. doi: 10.1146/annurev-med-090910-085119.
Fabry disease is an X-linked lysosomal storage disorder caused by α-galactosidase A deficiency. Intracellular accumulation of globotriaosylceramide starts in utero and progressively develops in various tissues and organs. Cardiac involvement is frequent, and its presentation as concentric nonobstructive left ventricular hypertrophy serves as a model for other hypertrophic cardiomyopathies. This review describes the Fabry cardiomyopathy, its treatment, and multidisciplinary patient care models. These models will help clinicians in diagnosing, assessing, and treating patients with Fabry disease. As the models can be extrapolated to other diseases, they might contribute to more optimal clinical management of patients with other cardiac disorders.
法布瑞氏病是一种 X 连锁溶酶体贮积症,由α-半乳糖苷酶 A 缺乏引起。胞内神经酰胺三己糖苷的积累始于宫内,并在各种组织和器官中逐渐发展。心脏受累很常见,其表现为同心性非阻塞性左心室肥厚,是其他肥厚型心肌病的模型。这篇综述描述了法布瑞氏心肌病及其治疗和多学科患者护理模式。这些模式将帮助临床医生诊断、评估和治疗法布瑞氏病患者。由于这些模式可以外推到其他疾病,它们可能有助于更好地管理其他心脏疾病患者的临床治疗。