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

Abstract

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.

• Authors: Dominique P Germain, Frank Weidemann, Ademola Abiose, Manesh R Patel, Marta Cizmarik, J Alexander Cole, Dana Beitner-Johnson, Karelle Benistan, Gustavo Cabrera, Joel Charrow, Ilkka Kantola, Ales Linhart, Kathy Nicholls, Markus Niemann, C Ronald Scott, Katherine Sims, Stephen Waldek, David G Warnock, Jörg Strotmann, Fabry Registry
• Journal: Genetics in medicine : official journal of the American College of Medical Genetics (Genet Med) Vol. 15 Issue 12 Pg. 958-65 (Dec 2013) ISSN: 1530-0366 [Electronic] United States
• PMID: 23703683 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't)

Chemical References

• Isoenzymes
• alpha-Galactosidase
• agalsidase beta

Topics

• Adolescent
• Adult
• Aged
• Disease Progression
• Fabry Disease (complications, drug therapy, physiopathology)
• Humans
• Hypertrophy, Left Ventricular (drug therapy)
• Isoenzymes (therapeutic use)
• Longitudinal Studies
• Male
• Middle Aged
• Multivariate Analysis
• Registries
• Treatment Outcome
• Young Adult
• alpha-Galactosidase (therapeutic use)