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

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

doi:10.1038/gim.2013.53

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

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

^*Corresponding author for this work

Pediatrics

Research output: Contribution to journal › Article › peer-review

79 Scopus citations

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.

Original language	English (US)
Pages (from-to)	958-965
Number of pages	8
Journal	Genetics in Medicine
Volume	15
Issue number	12
DOIs	https://doi.org/10.1038/gim.2013.53
State	Published - Dec 2013

Keywords

Fabry disease
cardiomyopathy
enzyme replacement therapy
lysosomal storage diseases
recombinant a-galactosidase A

ASJC Scopus subject areas

Genetics(clinical)

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

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10.1038/gim.2013.53

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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. (2013). Analysis of left ventricular mass in untreated men and in men treated with agalsidase-β: Data from the Fabry Registry. Genetics in Medicine, 15(12), 958-965. https://doi.org/10.1038/gim.2013.53

@article{2ed7c158cff84d6aa24dc2d777103602,

title = "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.",

keywords = "Fabry disease, cardiomyopathy, enzyme replacement therapy, lysosomal storage diseases, recombinant a-galactosidase A",

author = "Germain, {Dominique P.} and Frank Weidemann and Ademola Abiose and Patel, {Manesh R.} and Marta Cizmarik and Cole, {J. Alexander} and Dana Beitner-Johnson and Karelle Benistan and Gustavo Cabrera and Joel Charrow and Ilkka Kantola and Ales Linhart and Kathy Nicholls and Markus Niemann and Scott, {C. Ronald} and Katherine Sims and Stephen Waldek and Warnock, {David G.} and J{\"o}rg Strotmann",

year = "2013",

month = dec,

doi = "10.1038/gim.2013.53",

language = "English (US)",

volume = "15",

pages = "958--965",

journal = "Genetics in Medicine",

issn = "1098-3600",

publisher = "Lippincott Williams and Wilkins",

number = "12",

}

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 2013, 'Analysis of left ventricular mass in untreated men and in men treated with agalsidase-β: Data from the Fabry Registry', Genetics in Medicine, vol. 15, no. 12, pp. 958-965. https://doi.org/10.1038/gim.2013.53

TY - JOUR

T1 - Analysis of left ventricular mass in untreated men and in men treated with agalsidase-β

T2 - Data from the Fabry Registry

AU - Germain, Dominique P.

AU - Weidemann, Frank

AU - Abiose, Ademola

AU - Patel, Manesh R.

AU - Cizmarik, Marta

AU - Cole, J. Alexander

AU - Beitner-Johnson, Dana

AU - Benistan, Karelle

AU - Cabrera, Gustavo

AU - Charrow, Joel

AU - Kantola, Ilkka

AU - Linhart, Ales

AU - Nicholls, Kathy

AU - Niemann, Markus

AU - Scott, C. Ronald

AU - Sims, Katherine

AU - Waldek, Stephen

AU - Warnock, David G.

AU - Strotmann, Jörg

PY - 2013/12

Y1 - 2013/12

N2 - 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.

AB - 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.

KW - Fabry disease

KW - cardiomyopathy

KW - enzyme replacement therapy

KW - lysosomal storage diseases

KW - recombinant a-galactosidase A

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

Abstract

Keywords

ASJC Scopus subject areas

UN SDGs

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