title: Analysis of left ventricular mass in untreated men and in men treated with agalsidase-β: data from the Fabry Registry.
abstract: OBJECTIVE
  The aim of this study was to evaluate the progression of left ventricular hypertrophy in untreated men with Fabry disease and to assess the effects of agalsidase-β (recombinant human α-galactosidase A) on left ventricular hypertrophy.
  
  
  METHODS
  Longitudinal Fabry Registry data were analyzed from 115 men treated with agalsidase-β (1 mg/kg/2 weeks) and 48 untreated men. Measurements included baseline left-ventricular mass and at least one additional left-ventricular mass assessment over ≥ 2 years. Patients were grouped into quartiles, based on left-ventricular mass slopes. Multivariate logistic regression analyses identified factors associated with left ventricular hypertrophy progression.
  
  
  RESULTS
  For men in whom treatment was initiated at the age of 18 to <30 years, mean left ventricular mass slope was -3.6 g/year (n = 31) compared with +9.5 g/year in untreated men of that age (n = 15) (P < 0.0001). Untreated men had a 3.4-fold higher risk of having faster increases in left-ventricular mass compared with treated men (odds ratio: 3.43; 95% confidence interval: 1.05-11.22; P = 0.0415). A baseline age of ≥ 40 years was also associated with left--ventricular hypertrophy progression (odds ratio: 5.03; 95% confidence interval: 1.03-24.49; P = 0.0457) compared with men younger than 30 years.
  
  
  CONCLUSIONS
  Agalsidase-β treatment for ≥2 years may improve or stabilize left-ventricular mass in men with Fabry disease. Further investigations may determine whether early intervention and stabilization of LVM are correlated with clinical outcomes.
authors.affiliation: University of Versailles, Hôpital Raymond Poincaré (AP-HP), Garches, France.
authors.forename: Dominique P
authors.initials: DP
authors.lastname: Germain
authors.forename: Frank
authors.initials: F
authors.lastname: Weidemann
authors.forename: Ademola
authors.initials: A
authors.lastname: Abiose
authors.forename: Manesh R
authors.initials: MR
authors.lastname: Patel
authors.forename: Marta
authors.initials: M
authors.lastname: Cizmarik
authors.forename: J Alexander
authors.initials: JA
authors.lastname: Cole
authors.forename: Dana
authors.initials: D
authors.lastname: Beitner-Johnson
authors.forename: Karelle
authors.initials: K
authors.lastname: Benistan
authors.forename: Gustavo
authors.initials: G
authors.lastname: Cabrera
authors.forename: Joel
authors.initials: J
authors.lastname: Charrow
authors.forename: Ilkka
authors.initials: I
authors.lastname: Kantola
authors.forename: Ales
authors.initials: A
authors.lastname: Linhart
authors.forename: Kathy
authors.initials: K
authors.lastname: Nicholls
authors.forename: Markus
authors.initials: M
authors.lastname: Niemann
authors.forename: C Ronald
authors.initials: CR
authors.lastname: Scott
authors.forename: Katherine
authors.initials: K
authors.lastname: Sims
authors.forename: Stephen
authors.initials: S
authors.lastname: Waldek
authors.forename: David G
authors.initials: DG
authors.lastname: Warnock
authors.forename: Jörg
authors.initials: J
authors.lastname: Strotmann
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D018450:Disease Progression; D000795:Fabry Disease / Q000150:complications* / Q000188:drug therapy* / Q000503:physiopathology; D006801:Humans; D017379:Hypertrophy, Left Ventricular / Q000188:drug therapy*; D007527:Isoenzymes / Q000627:therapeutic use*; D008137:Longitudinal Studies; D008297:Male; D008875:Middle Aged; D015999:Multivariate Analysis; D012042:Registries; D016896:Treatment Outcome; D055815:Young Adult; D000519:alpha-Galactosidase / Q000627:therapeutic use*
chemical_list: D007527:Isoenzymes; D000519:alpha-Galactosidase; C459420:agalsidase beta