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

Published

Journal Article

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.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.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.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

Pubmed Central ID

  • 23703683

Electronic International Standard Serial Number (EISSN)

  • 1530-0366

International Standard Serial Number (ISSN)

  • 1098-3600

Digital Object Identifier (DOI)

  • 10.1038/gim.2013.53

Language

  • eng