Why the study?
Cardiomyopathy in Fabry disease is a major determinant of morbidity and mortality, prompting investigation into the effects of enzyme replacement therapy and chaperone therapy on left atrial function using 2DSTE.
Does enzyme replacement therapy or migalastat stabilize left atrial strain in patients with Fabry disease?
Does enzyme replacement therapy or migalastat stabilize left atrial strain in patients with Fabry disease?
In patients with Fabry disease, treatment with enzyme replacement therapy or migalastat is associated with stabilization of left atrial strain and left ventricular mass index over time.
Answers are grounded in this paper and cite their sources.
Stabilization of LA strain with ERT or migalastat suggests disease control in FD; leaves open causal benefits in this observational cohort.
Aims 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). Methods and results 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 ( p > 0.05). LVMi was also stable in treated FD patients during observation ( p > 0.05). Conclusion 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.
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Pogoda et al. (2023) studied this question.
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