AUTHOR=Pogoda Christian , Brand Stefan-Martin , Duning Thomas , Schmidt-Pogoda Antje , Sindermann Jürgen , Lenders Malte , Brand Eva 

TITLE=Impact of enzyme replacement therapy and migalastat on left atrial strain and cardiomyopathy in patients with Fabry disease

JOURNAL=Frontiers in Cardiovascular Medicine

VOLUME=Volume 10 - 2023

YEAR=2023

URL=https://www.frontiersin.org/journals/cardiovascular-medicine/articles/10.3389/fcvm.2023.1223635

DOI=10.3389/fcvm.2023.1223635

ISSN=2297-055X

ABSTRACT=Cardiomyopathy in Fabry disease (FD) is a major determinant for morbidity and mortality. This study investigates the effects of FD-specific treatment by enzyme replacement therapy (ERT) and chaperone therapy on left atrial (LA) function using two-dimensional speckle-tracking echocardiography (2DSTE).In this prospective observational single-center study, 20 FD patients (10 [50%] females) treated with migalastat, 48 FD patients (24 [50%] females) treated with ERT (agalsidase-alfa and agalsidase-beta) and 30 untreated FD patients (all females) as controls were analyzed. Mean follow-up time ranged from 26 to 81 months. 2DSTE was performed for left ventricle strain, right ventricle strain and LA strain (LAS). FD-specific treated patients presented with increased left ventricular mass index (LVMi) and higher frequency of left ventricular hypertrophy (LVH) at baseline, whereas untreated control patients showed normal baseline values. FD-specific treated (including migalastat and ERT) patients showed stabilization of LAS over time (p>0.05). LVMi was also stable in treated FD patients during observation (p>0.05).In patients with FD, treated with either ERT or chaperone therapy, LAS values measured by echocardiographic speckle tracking were stable over time, pointing towards a disease stabilization.