Key result
Enzyme replacement therapy over 3 years significantly reduced left ventricular mass (238 to 202 g, P<0.001) and improved exercise capacity only in Fabry patients without baseline myocardial fibrosis.
Why the study?
Does enzyme replacement therapy with recombinant alpha-galactosidase A improve myocardial morphology, function, and exercise capacity in patients with Fabry cardiomyopathy over long-term follow-up?
Population
32 patients with Fabry disease (9 with severe myocardial fibrosis, 11 with mild fibrosis, 12 without fibrosis)
Design
Cohort
Follow-up
3 years
Authors
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May support early enzyme replacement in nonfibrotic Fabry cardiomyopathy; leaves open need for randomized confirmation.
Cohort (n=32)
Does enzyme replacement therapy with recombinant alpha-galactosidase A improve myocardial morphology, function, and exercise capacity in patients with Fabry cardiomyopathy over long-term follow-up?
Enzyme replacement therapy in Fabry cardiomyopathy improves myocardial morphology, function, and exercise capacity only if initiated before the development of myocardial fibrosis.
Weidemann et al. (2009) conducted a cohort in Fabry cardiomyopathy (n=32). Enzyme replacement therapy with recombinant alpha-galactosidase A was evaluated on Disease progression and clinical outcome (left ventricular mass, myocardial function, exercise capacity). Enzyme replacement therapy over 3 years significantly reduced left ventricular mass (238 to 202 g, P<0.001) and improved exercise capacity only in Fabry patients without baseline myocardial fibrosis.
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