Agalsidase alfa slows the decline in renal function in patients with Fabry disease.
The aim of this study was to determine the effects of enzyme replacement therapy with agalsidase alpha on renal function in patients with Fabry nephropathy. Serum creatinine data were collected from 165 adult patients during 3 years of treatment. Serum creatinine increased in all men whereas it was stable in women, except in stage II renal disease (Kidney Disease Outcomes Quality Initiative). The estimated glomerular filtration rate (eGFR) declined in males with stage I and II (from 115.0 +/- 22.2 to 98.3 +/- 27.3 and from 76.5 +/- 8.1 to 66.3 +/-21.6 ml/min/1.73 m(2), respectively; both p < 0.01), whereas eGFR was stable in stage III. In females, eGFR was stable in stages I and III, and decreased in stage II (from 72.5 +/- 8.3 to 67.3 +/- 13.6 ml/min/1.73 m(2); p = 0.01). The 24-hour proteinuria was <1 g in all patients, and most patients (96%) were treated with angiotensin receptor blockers (ARBs) or angiotensin-converting enzyme (ACE) inhibitors. Agalsidase alpha in combination with ACE inhibitors/ARB may be effective in slowing the deterioration in renal function in Fabry nephropathy.
Feriozzi
Sandro
S
Nephrology and Dialysis, Belcolle Hospital, Strada Sammartinese snc, Viterbo, Italy. sandro.feriozzi@tiscali.it
Schwarting
Andreas
A
Sunder-Plassmann
Gere
G
West
Michael
M
Cybulla
Markus
M
D000293:Adolescent; D000328:Adult; D000368:Aged; D047228:Angiotensin II Type 1 Receptor Blockers / Q000627:therapeutic use; D000806:Angiotensin-Converting Enzyme Inhibitors / Q000627:therapeutic use; D015331:Cohort Studies; D003404:Creatinine / Q000097:blood; D000795:Fabry Disease / Q000150:complications / Q000188:drug therapy*; D005260:Female; D005919:Glomerular Filtration Rate; D006801:Humans; D006973:Hypertension; D007527:Isoenzymes / Q000627:therapeutic use; D007677:Kidney Function Tests; D008297:Male; D008875:Middle Aged; D011507:Proteinuria / Q000188:drug therapy; D011994:Recombinant Proteins; D051437:Renal Insufficiency / Q000188:drug therapy* / Q000209:etiology; D055815:Young Adult; D000519:alpha-Galactosidase / Q000627:therapeutic use*
D047228:Angiotensin II Type 1 Receptor Blockers; D000806:Angiotensin-Converting Enzyme Inhibitors; D007527:Isoenzymes; D011994:Recombinant Proteins; C000627036:agalsidase alfa; D003404:Creatinine; D000519:alpha-Galactosidase