Prognostic Indicators of Renal Disease Progression in Adults with Fabry Disease: Natural History Data from the Fabry Registry

• Type: Article in International Scientific Journal
• Date: 2010

Authors

Wanner, C (Author) Other; João Paulo Oliveira (Author) FMUP; Ortiz, A (Author) Other; Mauer, M (Author) Other; Germain, DP (Author) Other; Linthorst, GE (Author) Other; Serra, AL (Author) Other; Marodi, L (Author) Other; Mignani, R (Author) Other; Cianciaruso, B (Author) Other; Vujkovac, B (Author) Other; Lemay, R (Author) Other; Beitner Johnson, D (Author) Other; Waldek, S (Author) Other; Warnock, DG (Author) Other

Journal

Title: Clinical journal of the American Society of Nephrology : CJASN

Vol. 5 No. 2

Pages: 2220-2228

ISSN: 1555-9041

Publisher: American Society of Nephrology

Indexing

ISI Web of Knowledge - 98 Citations

Scopus - 117 Citations

Scientific classification

FOS: Medical and Health sciences > Other medical sciences

Other information

Authenticus ID: P-003-0CW

DOI: 10.2215/cjn.04340510

Abstract (EN): Background and objectives: These analyses were designed to characterize renal disease progression in untreated adults with Fabry disease. Design, setting, participants, & measurements: Data from the Fabry Registry for 462 untreated adults (121 men and 341 women) who had at least two estimated GFR (eGFR) values over a span >= 12 months before starting enzyme replacement therapy were included. Results: Most men (86 of 121, 71%) had more rapid loss of kidney function than the normal adult population (loss of eGFR > -1 ml/min per 1.73 m(2) per year), whereas fewer women (133 of 341, 39%) had rapid loss of kidney function. Patients with rapid progression had significantly higher mean averaged urinary protein to urinary creatinine ratios (UP/Cr) than patients with slower progression (1.5 versus 0.2 for men; 1.4 versus 0.5 for women; P < 0.0001). Patients were grouped into quartiles based on averaged UP/Cr; renal function in men declined more rapidly with higher UP/Cr, with the steepest declines observed in men with UP/Cr > 1.5 (mean eGFR slope, -5.6 ml/min per 1.73 m(2) per year; n = 30). eGFR slope declined more slowly in women, with the steepest declines observed in women with UP/Cr > 1.2 (mean eGFR slope, -1.3 ml/min per 1.73 m(2) per year; n = 85). Regression models of eGFR slope indicated that UP/Cr is the most important indicator of renal disease progression in adult Fabry patients. In women, lower baseline eGFR and age were also associated with renal disease progression. Women who had clinical events had more rapid loss of kidney function. Conclusions: Urinary protein excretion is strongly associated with renal disease progression in men and women with Fabry disease. Clin J Am Soc Nephrol 5: 2220-2228, 2010. doi: 10.2215/CJN.04340510

Language: English

Type (Professor's evaluation): Scientific

No. of pages: 9