Major cardiovascular adverse events in Fabry disease patients receiving agalsidase alfa

FERRARI G; REISIN R; KISINOVSKY I; NEUMANN P; DRAGONETTI L; CHOUA M; ROZENFELD PA; MARCHESONI C; FINN V

MEDICINA (BUENOS AIRES)

MEDICINA (BUENOS AIRES)

Lugar: Buenos Aires; Año: 2021

0025-7680

Introduction: Cardiovascular mortality (CVM) is a major cause of death in Fabry disease (FD). Our primary objectives were to describe causes and potential predictors of mortality in Argentinean FD adult patients and to assess risk of major adverse cardiovascular events (MACE) in the enzyme replacement therapy (ERT) era. Methods: We retrospectively studied 93 consecutive patients with FD treated with agalsidase alpha with median follow up of 9.5 years from start of ERT.Results: Mean age at start of ERT was 35 ± 16.3 years and 56% of our patients were females. Prevalence of cardiomyopathy and renal disease reached 47% and 41%, respectively. Eleven subjects (11.8%, 95% CI: 5-18%) died during follow up, equivalent to 1.24 per 100 patient-years. Mean overall survival from date of birth was 71 years (95% CI: 66-75 years). Seven cases were considered as CVM; main causes of CVM were sudden death and stroke. Risk of MACE was 14% (95% CI: 6.9-21.1%), equivalent to 1.47 events per 100 patient-years from start of ERT. All but 2 subjects had at least one comorbid conventional cardiovascular risk factor; however, 86% of our patients remained free of MACE during the follow-up period. Even though CVM events remained small and our study was underpowered for detection of predictors of mortality, it is worth noting that age at diagnosis and starting of ERT, left ventricular mass index and renal disease have shown at least a positive trend of relationship with CVM, as previously observed. Prevalence of hypertension, diabetes and dyslipidemia were lower in FD patients when compared to data from population level in Argentina. Importance of renal failure in the epidemiology of MACE in this population is highlighted.Conclusion: To our best knowledge, our research represents the first report about epidemiology of MACE and causes of mortality among a large cohort of consecutive FD patients treated with agalsidase alpha in Argentina. CVM was the leading cause of death.