Abstract 435 Impact of enzyme replacement therapy and migalastat on left atrial strain and cardiomyopathy in patients with Fabry disease

Aims

Cardiomyopathy in Fabry disease (FD) is major course of 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.

Methods & Results

In this prospective observational single-center study, 20 Fabry patients (10 [50%] females) under migalastat, 48 Fabry patients (24 [50%] females) under ERT (agalsidase-alfa and agalsidase-beta) and 30 untreated Fabry patients (all females) as controls were analyzed. Mean follow-up time ranged from 26 to 81 months. 2-dimensional strain echocardiography was performed using speckle-tracking to measure LV strain, RV strain and LA strain.
FD-specific treated patients present with increased LVMi and higher frequency of LVH at baseline, whereas untreated control patients show normal baseline values. FD-specific treated (including migalastat and ERT with agalsidase-alfa or agalsidase-beta) patients showed stabilization of 2D speckle-tracking derived LA phasic strain (LAS) over time (p>0.05). Left ventricular mass index (LVMi) was also stable in FD-specific treated patients (p>0.05) during observation.

Conclusion

In patients with FD treated with either ERT or chaperone therapy, LAS phases measured by echocardiographic speckle tracking are stable over time. This highlights a disease stabilization and an improvement of prognosis.

Clin Res Cardiol (2023). https://doi.org/10.1007/s00392-023-02180-w
Impact of enzyme replacement therapy and migalastat on left atrial strain and cardiomyopathy in patients with Fabry disease
C. Pogoda¹, S.-M. Brand², T. Duning³, A. Schmidt-Pogoda⁴, J. Sindermann¹, M. Lenders⁵, E. Brand⁵
¹Klinik für Kardiologie I: Koronare Herzkrankheit, Herzinsuffizienz und Angiologie, Universitätsklinikum Münster, Münster; ²Institut für Sportmedizin, Universitätsklinikum Münster, Münster; ³Klinik für Neurologie mit Institut für klinische Neurophysiologie und Neurologische Frührehabilitation, Gesundheit Nord GmbH, Klinikum Bremen Ost, Bremen; ⁴Klinik für Neurologie mit Institut für Translationale Neurologie, Universitätsklinikum Münster, Münster; ⁵Medizinische Klinik D, Allgemeine Innere Medizin sowie Nieren- und Hochdruckkrankheiten und Rheumatologie, Universitätsklinikum Münster, Münster;
Aims Cardiomyopathy in Fabry disease (FD) is major course of 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. Methods & Results In this prospective observational single-center study, 20 Fabry patients (10 [50%] females) under migalastat, 48 Fabry patients (24 [50%] females) under ERT (agalsidase-alfa and agalsidase-beta) and 30 untreated Fabry patients (all females) as controls were analyzed. Mean follow-up time ranged from 26 to 81 months. 2-dimensional strain echocardiography was performed using speckle-tracking to measure LV strain, RV strain and LA strain. FD-specific treated patients present with increased LVMi and higher frequency of LVH at baseline, whereas untreated control patients show normal baseline values. FD-specific treated (including migalastat and ERT with agalsidase-alfa or agalsidase-beta) patients showed stabilization of 2D speckle-tracking derived LA phasic strain (LAS) over time (p>0.05). Left ventricular mass index (LVMi) was also stable in FD-specific treated patients (p>0.05) during observation. Conclusion In patients with FD treated with either ERT or chaperone therapy, LAS phases measured by echocardiographic speckle tracking are stable over time. This highlights a disease stabilization and an improvement of prognosis.
https://dgk.org/kongress_programme/jt2023/aP2184.html