Journal article

Agalsidase Alfa and Kidney Dysfunction in Fabry Disease

Michael West, Kathy Nicholls, Atul Mehta, Joe TR Clarke, Robert Steiner, Michael Beck, Bruce A Barshop, William Rhead, Robert Mensah, Markus Ries, Raphael Schiffmann

JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY | AMER SOC NEPHROLOGY | Published : 2009

Abstract

In male patients with Fabry disease, an X-linked disorder of glycosphingolipid metabolism caused by deficient activity of the lysosomal enzyme alpha-galactosidase A, kidney dysfunction becomes apparent by the third decade of life and invariably progresses to ESRD without treatment. Here, we summarize the effects of agalsidase alfa on kidney function from three prospective, randomized, placebo-controlled trials and their open-label extension studies involving 108 adult male patients. The mean baseline GFR among 54 nonhyperfiltrating patients (measured GFR <135 ml/min per 1.73 m(2)) treated with placebo was 85.4 +/- 29.6 ml/min per 1.73 m(2); during 6 mo of placebo, the mean annualized rate of..

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Grants

Funding Acknowledgements

Michael West has received research funding, consultancy fees, and/or speaker's fees from Shire Human Genetic Therapies (formerly TKT), Genzyme, and Amicus Therapeutics.Kathy Nicholls has received research funding, travel support, consultancy, and/or speaker honoraria from Shire Human Genetic Therapies, Genzyrne, and Amicus Therapeutics, and is a member of the Fabry Outcome Study I nternational Advisorv Board.Atul Mehta has received research funding, consultancy fees, and/or speaker's fees from Shire Human Genetic Therapies, Genzyme, and Amicus Therapeutics.Joe T.R. Clarke has received research funding, consultancy fees, and/or speaker's fees from Shire Human Genetic Therapies, Genzyme, and Actelion.Robert Steiner has received research funding, consultancy fees, honoraria, and/or speaker's fees from Shire Human Genetic Therapies, Genzyme, Actelion, Biomarin, and Amicus Therapeutics.Michael Beck has received travel support, honoraria, and unrestricted grants from Genzvme, Shire Human Genetic Therapies, and Biomarin.Bruce Barshop has received research funding, consultation fees, and/or speaker's fees from Genzyme and Biomarin.William Rhead has received research funding, consultancy fees, and/or speaker's fees from Shire Human Genetic Therapies, Genzyme, Hyperion, Ucyclyd, and Actelion.Robert Mensah is an employee of Shire Human Genetic Therapies. Markus Ries reports no conflict during the conduct of these studies and is currently an employee of Shire Human Genetic Therapies.Raphael Schiffmarm has received research funding, consultancy fees, and/or speaker's fees from Shire Human Genetic Therapies, Genzyme, and Amicus Therapeutics.