Antiproteinuric therapy and Fabry nephropathy: factors associated with preserved kidney function during agalsidase-beta therapy.

ABSTRACT:

Background

Nephropathy is an important feature of classical Fabry disease, which results in alpha-galactosidase A deficiency and cellular globotriaosylceramide accumulation. We report the safety and efficacy of antiproteinuric therapy with ACE inhibitors or angiotensin II receptor blockers (ARBs) in a study of classical Fabry patients receiving recombinant agalsidase-beta therapy.

Methods and design

The goal was maintenance of urine protein to creatinine ratio (UPCR) <0.5 g/g or a 50% reduction in baseline UPCR for 24 patients at eight study sites. The change in estimated glomerular filtration rate (eGFR) was assessed over 21 months of treatment.

Results

18 out of 24 patients achieved the UPCR goal with eGFR slopes that were significantly better than six patients who

SUBMITTER: Warnock DG 

PROVIDER: S-EPMC4717450 | biostudies-literature | 2015 Dec

REPOSITORIES: biostudies-literature