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

Germain, D. P.; Weidemann, F.; Abiose, A.; Patel, M. R.; Cizmarik, M.; Cole, J. A.; Beitner-Johnson, D.; Benistan, K.; Cabrera, G.; Charrow, J.; Kantola, I.; Linhart, A.; Nicholls, K.; Niemann, M.; Scott, C. R.; Sims, K.; Waldek, S.; Warnock, D. G.; Strotmann, J.

Genet Med. 2013 May 25; 15(12):958-65


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.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 >/=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-beta 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.Genet Med 15 12, 958-965.Genetics in Medicine (2013); 15 12, 958-965. doi:10.1038/gim.2013.53.

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