Analysis of left ventricular mass in untreated men and in men treated with agalsidase-β: data from the Fabry Registry.

Journal Article (Journal Article)

PURPOSE: 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. CONCLUSION: 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.

Full Text

Duke Authors

Cited Authors

  • Germain, DP; Weidemann, F; Abiose, A; Patel, MR; Cizmarik, M; Cole, JA; Beitner-Johnson, D; Benistan, K; Cabrera, G; Charrow, J; Kantola, I; Linhart, A; Nicholls, K; Niemann, M; Scott, CR; Sims, K; Waldek, S; Warnock, DG; Strotmann, J; Fabry Registry,

Published Date

  • December 2013

Published In

Volume / Issue

  • 15 / 12

Start / End Page

  • 958 - 965

PubMed ID

  • 23703683

Electronic International Standard Serial Number (EISSN)

  • 1530-0366

Digital Object Identifier (DOI)

  • 10.1038/gim.2013.53


  • eng

Conference Location

  • United States