title: Prognostic indicators of renal disease progression in adults with Fabry disease: natural history data from the Fabry Registry.
abstract: OBJECTIVE
  These analyses were designed to characterize renal disease progression in untreated adults with Fabry disease.
  
  
  METHODS
  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 of ≥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.
authors.affiliation: Department of Medicine, Division of Nephrology, University of Würzburg, Würzburg, Germany. Wanner_C@medizin.uni-wuerzburg.de
authors.forename: Christoph
authors.initials: C
authors.lastname: Wanner
authors.forename: João P
authors.initials: JP
authors.lastname: Oliveira
authors.forename: Alberto
authors.initials: A
authors.lastname: Ortiz
authors.forename: Michael
authors.initials: M
authors.lastname: Mauer
authors.forename: Dominique P
authors.initials: DP
authors.lastname: Germain
authors.forename: Gabor E
authors.initials: GE
authors.lastname: Linthorst
authors.forename: Andreas L
authors.initials: AL
authors.lastname: Serra
authors.forename: László
authors.initials: L
authors.lastname: Maródi
authors.forename: Renzo
authors.initials: R
authors.lastname: Mignani
authors.forename: Bruno
authors.initials: B
authors.lastname: Cianciaruso
authors.forename: Bojan
authors.initials: B
authors.lastname: Vujkovac
authors.forename: Roberta
authors.initials: R
authors.lastname: Lemay
authors.forename: Dana
authors.initials: D
authors.lastname: Beitner-Johnson
authors.forename: Stephen
authors.initials: S
authors.lastname: Waldek
authors.forename: David G
authors.initials: DG
authors.lastname: Warnock
mesh_terms: D000328:Adult; D003404:Creatinine / Q000652:urine; D018450:Disease Progression; D056947:Enzyme Replacement Therapy; D000795:Fabry Disease / Q000150:complications* / Q000628:therapy; D005260:Female; D005919:Glomerular Filtration Rate; D006801:Humans; D007674:Kidney Diseases / Q000209:etiology*; D007676:Kidney Failure, Chronic / Q000209:etiology; D008297:Male; D008875:Middle Aged; D011379:Prognosis; D011507:Proteinuria / Q000150:complications; D012042:Registries
chemical_list: D003404:Creatinine
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