Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease.

ABSTRACT:

Background

Fabry disease results from deficient α-galactosidase A activity and globotriaosylceramide accumulation causing renal insufficiency, strokes, hypertrophic cardiomyopathy and early demise. We assessed the 10-year outcome of recombinant α-galactosidase A therapy.

Methods

The outcomes (severe clinical events, renal function, cardiac structure) of 52/58 patients with classic Fabry disease from the phase 3 clinical trial and extension study, and the Fabry Registry were evaluated. Disease progression rates for patients with low renal involvement (LRI, n=32) or high renal involvement (HRI, n=20) at baseline were assessed.

Results

81% of patients (42/52) did not experience any severe clinical event during the treatment interval and 94% (49/52) were alive at the end

SUBMITTER: Germain DP 

PROVIDER: S-EPMC4413801 | biostudies-literature | 2015 May

REPOSITORIES: biostudies-literature