Journal of the American Society of Nephrology
2008 JASN IMPACT FACTOR 7.505 HOME   AUTHOR INFO   EDITORIAL BOARD   SUBSCRIBE   FEEDBACK   ALERTS   HELP 
    advanced
CURRENT ISSUE ARCHIVES JASN Express ONLINE SUBMISSION


Published ahead of print on April 8, 2009
J Am Soc Nephrol 20: 1132-1139, 2009
© 2009 American Society of Nephrology
doi: 10.1681/ASN.2008080870
This Article
Right arrow Full Text
Right arrow Full Text (PDF)
Right arrow Press Release
Right arrow All Versions of this Article:
ASN.2008080870v1
20/5/1132    most recent
Right arrow Alert me when this article is cited
Right arrow Alert me if a correction is posted
Right arrow Citation Map
Services
Right arrow Email this article to a friend
Right arrow Similar articles in this journal
Right arrow Similar articles in PubMed
Right arrow Alert me to new issues of the journal
Right arrow Download to citation manager
Right arrow reprints & permissions
Citing Articles
Right arrow Citing Articles via HighWire
Right arrow Citing Articles via Google Scholar
Google Scholar
Right arrow Articles by West, M.
Right arrow Articles by Schiffmann, R.
Right arrow Search for Related Content
PubMed
Right arrow PubMed Citation
Right arrow Articles by West, M.
Right arrow Articles by Schiffmann, R.

CLINICAL RESEARCH

Agalsidase Alfa and Kidney Dysfunction in Fabry Disease

Michael West*, Kathy Nicholls{dagger}, Atul Mehta{ddagger}, Joe T.R. Clarke§,||, Robert Steiner, Michael Beck**, Bruce A. Barshop{dagger}{dagger}, William Rhead{ddagger}{ddagger}, Robert Mensah§§, Markus Ries**,|||| and Raphael Schiffmann||||

* Department of Medicine, Dalhousie University, Halifax, Nova Scotia, Canada; {dagger} Royal Melbourne Hospital, Parkville, Victoria, Australia; {ddagger} Royal Free Hospital and University College Medical School, London, United Kingdom; § Division of Clinical and Metabolic Genetics, Hospital for Sick Children and University of Toronto, Toronto, Canada; || Service de génétique médicale, Centre hospitalier universitaire de Sherbrooke, Sherbrooke, Canada; Oregon Health and Science University, Portland, Oregon; ** Center for Lysosomal Storage Diseases, University of Mainz, Mainz, Germany; {dagger}{dagger} University of California San Diego School of Medicine, La Jolla, California; {ddagger}{ddagger} Medical College of Wisconsin, Milwaukee, Wisconsin; §§ Biostatistics, Shire HGT, Cambridge, Massachusetts; and |||| Developmental and Metabolic Neurology Branch, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, Maryland

Correspondence: Dr. Michael L. West, Division of Nephrology, Department of Medicine, Dalhousie University, 5090 ACC QE II Health Sciences Centre, 5820 University Avenue, Halifax, NS, Canada B3H 1V8. Phone: 902-473-4023; Fax: 902-473-2675; E-mail: mlwest{at}dal.ca

Received for publication August 19, 2008. Accepted for publication January 26, 2009.

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 m2) treated with placebo was 85.4 ± 29.6 ml/min per 1.73 m2; during 6 mo of placebo, the mean annualized rate of change in GFR was –7.0 ± 32.9 ml/min per 1.73 m2. Among 85 nonhyperfiltrating patients treated with agalsidase alfa, the annualized rate of change was –2.9 ± 8.7 ml/min per 1.73 m2. Treatment with agalsidase alfa did not affect proteinuria. Multivariate analysis revealed that GFR and proteinuria category (<1 or ≥1 g/d) at baseline significantly predicted the rate of decline of GFR during treatment. This summary represents the largest group of male patients who had Fabry disease and for whom the effects of enzyme replacement therapy on kidney function have been studied. These data suggest that agalsidase alfa may stabilize kidney function in these patients.




This article has been cited by other articles:


Home page
 Nephrol Dial TransplantHome page
C. Tondel, U. Ramaswami, K. M. Aakre, F. Wijburg, M. Bouwman, and E. Svarstad
Monitoring renal function in children with Fabry disease: comparisons of measured and creatinine-based estimated glomerular filtration rate
Nephrol. Dial. Transplant., December 27, 2009; (2009) gfp658v1.
[Abstract] [Full Text] [PDF]

Home page
 Nephrol Dial TransplantHome page
A. Ortiz, B. Cianciaruso, M. Cizmarik, D. P. Germain, R. Mignani, J. P. Oliveira, J. Villalobos, B. Vujkovac, S. Waldek, C. Wanner, et al.
End-stage renal disease in patients with Fabry disease: natural history data from the Fabry Registry
Nephrol. Dial. Transplant., October 21, 2009; (2009) gfp554v1.
[Abstract] [Full Text] [PDF]

Home page
 J. Med. Genet.Home page
A Mehta, J T R Clarke, R Giugliani, P Elliott, A Linhart, M Beck, G Sunder-Plassmann, and on behalf of the FOS Investigators
Natural course of Fabry disease: changing pattern of causes of death in FOS - Fabry Outcome Survey
J. Med. Genet., August 1, 2009; 46(8): 548 - 552.
[Abstract] [Full Text] [PDF]


HOME CURRENT ISSUE ARCHIVES JASN Express ONLINE SUBMISSION AUTHOR INFO
EDITORIAL BOARD SUBSCRIBE FEEDBACK ALERTS HELP

Copyright © 2009 by the American Society of Nephrology. Online ISSN: 1533-3450 Print ISSN: 1046-6673