Cystatin C as a marker of early changes of renal function in Fabry nephropathy.
BACKGROUND
A sensitive, feasible and reproducible marker for renal function is necessary to evaluate the clinical efficacy of enzyme replacement therapy (ERT) in Fabry nephropathy. Serum creatinine has some limitations and cystatin C has been proposed, in other nephropathies, as a useful marker of renal function. The use of cystatin C as a marker of glomerular filtration rate (GFR) was investigated in Fabry patients receiving ERT.


METHODS
Renal function was evaluated with serum creatinine, serum cystatin C and estimated GFR (through Modification of Diet in Renal Disease [MDRD], Cockcroft-Gault [C&G] and Hoek formulae) in 21 Fabry patients receiving ERT with agalsidase alfa for 3 years and in 13 Fabry patients receiving agalsidase alfa for 4 years.


RESULTS
During years of ERT while serum creatinine remained stable, cystatin C values showed a significant, increasing trend right from the first year of ERT.


CONCLUSIONS
In Fabry disease, cystatin C is a sensitive and reliable marker of renal function, and it should be taken into account when evaluating GFR trends during ERT.
Feriozzi
Sandro
S
Nephrology and Dialysis Unit, Ospedale Belcolle, Viterbo, Italy. sandro.feriozzi@tiscali.it
Germain
Dominique P
DP
Di Vito
Raffaele
R
Legrand
Alain
A
Ricci
Roberta
R
Barbey
Frédéric
F
D000328:Adult; D000368:Aged; D015415:Biomarkers / Q000097:blood; D003404:Creatinine / Q000097:blood; D055316:Cystatin C; D015891:Cystatins / Q000097:blood*; D000795:Fabry Disease / Q000150:complications / Q000188:drug therapy*; D005260:Female; D005919:Glomerular Filtration Rate; D006801:Humans; D007674:Kidney Diseases / Q000175:diagnosis* / Q000209:etiology / Q000503:physiopathology; D008297:Male; D008875:Middle Aged; D000519:alpha-Galactosidase / Q000627:therapeutic use*
D015415:Biomarkers; C525030:CST3 protein, human; D055316:Cystatin C; D015891:Cystatins; D003404:Creatinine; D000519:alpha-Galactosidase