Key result
End-stage renal disease in men with Fabry disease on enzyme replacement was associated with a 100% rate of cardiovascular events over 10 years compared to 26% in those without (p<0.0001).
Why the study?
Does end-stage renal disease (CKD5) worsen cardiovascular outcomes and disease progression in male patients with Fabry disease on enzyme replacement therapy?
Cohort (n=25)
Does end-stage renal disease (CKD5) worsen cardiovascular outcomes and disease progression in male patients with Fabry disease on enzyme replacement therapy?
Absolute Event Rate: 100% vs 26%
p-value: p=<0.0001
In male patients with Fabry disease on enzyme replacement therapy, the presence of end-stage renal disease is strongly associated with progressive left ventricular hypertrophy and a significantly higher rate of cardiovascular events over 10 years.
CKD5 linked to higher CV events and LV mass in Fabry on ERT; hypothesis-generating, prospective trials needed before practice change.
OBJECTIVES: Assess the impact of end-stage renal disease (chronic kidney disease stage 5 (CKD5)) on cardiovascular outcomes in patients with Fabry disease on enzyme replacement therapy. BACKGROUND: Fabry disease, an X-linked lysosomal storage disease, causes hypertrophic cardiomyopathy and cardiovascular dysfunction. METHODS: Cardiac and renal function of 25 male patients with Fabry disease were analysed at 0, 1, 2, 5, 7 and 10 years after initiation of treatment. Patients were grouped at baseline into those with CKD5 (n=10) and those without (n=15). ECG and echocardiography were performed 6 and 12 monthly, respectively, while renal function was measured yearly. RESULTS: After 10 years of treatment, cardiac and renal function in non-CKD5 patients remained unchanged. In contrast, CKD5 was associated with worse baseline cardiac parameters and progressive LV hypertrophy. LV mass index grew by 35.4±31.8 g/m(2.7) in CKD5 versus 5.7±7.9 g/m(2.7), p=0.044 in non-CKD5, predominantly due to increased interventricular septal wall thickness (7.7±5.5 mm vs 1.3±1.7 mm, p=0.003). Cardiovascular events, including sudden death, arrhythmia and pacing device insertion, occurred in 100% patients with CKD5 (21 events) and 26% non-CKD5 patients (7 events), p<0.0001. Additionally, estimated LV filling pressure (E/Ea) was significantly higher in patients having cardiovascular events (21.1±7.7 vs 12.5±4.5, p=0.008) irrespective of renal function. CONCLUSIONS: End-stage renal disease was the strongest indicator of cardiovascular disease progression in Fabry disease. Enzyme replacement initiated prior to CKD5 was associated with stability in cardiac and renal disease while patients with CKD5 showed ongoing deterioration. Additionally, E/Ea ≥15 may predict risk of cardiac events.
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Talbot et al. (2014) conducted a cohort in Fabry disease (n=25). End-stage renal disease (CKD5) vs. No CKD5 was evaluated on Cardiovascular events (including sudden death, arrhythmia and pacing device insertion) (p=<0.0001). End-stage renal disease in men with Fabry disease on enzyme replacement was associated with a 100% rate of cardiovascular events over 10 years compared to 26% in those without (p<0.0001).
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