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

作者:Germain Dominique P*; Weidemann Frank; Abiose Ademola; Patel Manesh R; Cizmarik Marta; Cole J Alexander; Beitner Johnson Dana; Benistan Karelle; Cabrera Gustavo; Charrow Joel; Kantola Ilkka; Linhart Ales; Nicholls Kathy; Niemann Markus; Scott C Ronald; Sims Katherine; Waldek Stephen; Warnock David G; Strotmann Joerg
来源:Genetics in Medicine, 2013, 15(12): 958-965.
DOI:10.1038/gim.2013.53

摘要

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-beta (recombinant human alpha-galactosidase A) on left ventricular hypertrophy. %26lt;br%26gt;Methods: Longitudinal Fabry Registry data were analyzed from 115 men treated with agalsidase-beta (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 %26gt;= 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. %26lt;br%26gt;Results: For men in whom treatment was initiated at the age of 18 to %26lt;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 %26lt; 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 %26gt;= 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. %26lt;br%26gt;Conclusion: Agalsidase-beta treatment for %26gt;= 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.

  • 出版日期2013-12