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

Cardiomyopathy in Fabry disease (FD) is a major determinant of morbidity and mortality. This study investigates the effects of FD-specific treatment using 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. The 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 at baseline, whereas untreated control patients showed normal baseline values. FD-specific treated (including migalastat and ERT) patients showed stabilization of LAS over time ( In patients with FD, treated with either ERT or chaperone therapy, LAS values measured by echocardiographic speckle tracking were stable over time, pointing toward disease stabilization.

Informations de copyright

© 2023 Pogoda, Brand, Duning, Schmidt-Pogoda, Sindermann, Lenders and Brand.

Déclaration de conflit d'intérêts

CP received speaker honoraria and travel funding from Amicus Therapeutics, Sanofi Genzyme, Chiesi, and Shire/Takeda. ML received speaker honoraria, travel funding and research grants from Amicus Therapeutics, Sanofi Genzyme, Chiesi, and Shire/Takeda. EB received research grants and speaker honoraria from Sanofi Genzyme, Shire/Takeda, Chiesi, Greenovation, and Amicus Therapeutics. SB received research grants from Shire/Takeda. AS received speaker honoraria from Sanofi Genzyme, TD received honorariums and travel expenses from Genzyme, Shire, Sanofi Aventis, and Amicus Therapeutics for serving as a speaker and consultant. TD received research support from Genzyme, Shire, Amicus Therapeutics. The remaining author declares that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.