Long-term effectiveness of agalsidase alfa enzyme replacement in Fabry disease: A Fabry Outcome Survey analysis

Molecular Genetics and Metabolism Reports

Volumn 3, Issue , 2015, Pages 21-27

  Beck, Michael ^a   Hughes, Derralynn ^b   Kampmann, Christoph ^a   Larroque, Sylvain ^c   Mehta, Atul ^b   Pintos Morell, Guillem ^d   Ramaswami, Uma ^b   West, Michael ^e   Wijatyk, Anna ^f   Giugliani, Roberto ^g  

^a JOHANNES GUTENBERG UNIVERSITY   (Germany)

^b UNIVERSITY COLLEGE LONDON   (United Kingdom)

^c SHIRE   (Switzerland)

^d HOSPITAL UNIVERSITARI GERMANS TRIAS I PUJOL   (Spain)

^e DALHOUSIE UNIVERSITY   (Canada)

^f SHIRE   (United States)

^g HOSPITAL DE CLÍNICAS DE PORTO ALEGRE   (Brazil)

Author keywords

Agalsidase alfa; Enzyme replacement therapy; Fabry disease; Long term effectiveness

Indexed keywords

AGALSIDASE ALFA; PLACEBO;

ADULT; ARTICLE; CARDIOMYOPATHY; CONTROLLED STUDY; CROSS-SECTIONAL STUDY; DISEASE COURSE; DOUBLE BLIND PROCEDURE; ENZYME REPLACEMENT; FABRY DISEASE; FEMALE; GLOMERULUS FILTRATION RATE; HEART LEFT VENTRICLE HYPERTROPHY; HEART LEFT VENTRICLE MASS; HUMAN; KIDNEY FUNCTION; LONG TERM CARE; LONGITUDINAL STUDY; MAJOR CLINICAL STUDY; MALE; MORBIDITY; MORTALITY; RANDOMIZED CONTROLLED TRIAL; RETROSPECTIVE STUDY; RISK FACTOR; TREATMENT DURATION;

EID: 84930011520     PISSN: None     EISSN: 22144269     Source Type: Journal    
DOI: 10.1016/j.ymgmr.2015.02.002     Document Type: Article

References (30)

1. S.C. Garman, and D.N. Garboczi The molecular defect leading to Fabry disease: structure of human alpha-galactosidase J. Mol. Biol. 337 2004 319 335
2. A. Mehta, J.T. Clarke, and R. Giugliani Natural course of Fabry disease: changing pattern of causes of death in FOS - Fabry Outcome Survey J. Med. Genet. 46 2009 548 552
3. K.D. MacDermot, A. Holmes, and A.H. Miners Anderson-Fabry disease: clinical manifestations and impact of disease in a cohort of 60 obligate carrier females J. Med. Genet. 38 2001 769 775
4. K.D. MacDermot, A. Holmes, and A.H. Miners Natural history of Fabry disease in affected males and obligate carrier females J. Inherit. Metab. Dis. 24 Suppl. 2 2001 13 14 (discussion 11-12)
5. M. West, K. Nicholls, and A. Mehta Agalsidase alfa and kidney dysfunction in Fabry disease J. Am. Soc. Nephrol. 20 2009 1132 1139
6. K. Sims, J. Politei, M. Banikazemi, and P. Lee Stroke in Fabry disease frequently occurs before diagnosis and in the absence of other clinical events: natural history data from the Fabry Registry Stroke 40 2009 788 794
7. M. Branton, R. Schiffmann, and J.B. Kopp Natural history and treatment of renal involvement in Fabry disease J. Am. Soc. Nephrol. 13 Suppl. 2 2002 S139 S143
8. C. Kampmann, A. Linhart, and F. Baehner Onset and progression of the Anderson-Fabry disease related cardiomyopathy Int. J. Cardiol. 130 2008 367 373
9. R. Schiffmann, J.B. Kopp, and H.A. Austin III Enzyme replacement therapy in Fabry disease: a randomized controlled trial JAMA 285 2001 2743 2749
10. F. Breunig, F. Weidemann, J. Strotmann, A. Knoll, and C. Wanner Clinical benefit of enzyme replacement therapy in Fabry disease Kidney Int. 69 2006 1216 1221
11. A. Mehta, M. Beck, and P. Elliott Enzyme replacement therapy with agalsidase alfa in patients with Fabry's disease: an analysis of registry data Lancet 374 2009 1986 1996
12. R.P. El Dib, P. Nascimento, and G.M. Pastores Enzyme replacement therapy for Anderson-Fabry disease Cochrane Database Syst. Rev. 2 2013 CD006663
13. D.A. Hughes, P.M. Elliott, and J. Shah Effects of enzyme replacement therapy on the cardiomyopathy of Anderson-Fabry disease: a randomised, double-blind, placebo-controlled clinical trial of agalsidase alfa Heart 94 2008 153 158
14. E. Hernberg-Stahl Organization and technical aspects of FOS - the Fabry Outcome Survey A. Mehta, M. Beck, G. Sunder-Plassmann, 2006 Fabry Disease: Perspectives From 5 Years of FOS. Oxford PharmaGenesis Oxford
15. M. Beck, R. Ricci, and U. Widmer Fabry disease: overall effects of agalsidase alfa treatment Eur. J. Clin. Investig. 34 2004 838 844
16. S. Feriozzi, J. Torras, M. Cybulla, K. Nicholls, G. Sunder-Plassmann, and M. West The effectiveness of long-term agalsidase alfa therapy in the treatment of Fabry nephropathy Clin. J. Am. Soc. Nephrol. 7 2012 60 69
17. A. Schwarting, F. Dehout, S. Feriozzi, M. Beck, A. Mehta, and G. Sunder-Plassmann Enzyme replacement therapy and renal function in 201 patients with Fabry disease Clin. Nephrol. 66 2006 77-74
18. A. Linhart, C. Kampmann, and J.L. Zamorano Cardiac manifestations of Anderson-Fabry disease: results from the international Fabry outcome survey Eur. Heart J. 28 2007 1228 1235
19. A. Mehta, R. Ricci, and U. Widmer Fabry disease defined: baseline clinical manifestations of 366 patients in the Fabry Outcome Survey Eur. J. Clin. Investig. 34 2004 236 242
20. M. Banikazemi, J. Bultas, and S. Waldek Agalsidase-beta therapy for advanced Fabry disease: a randomized trial Ann. Intern. Med. 146 2007 77 86
21. R. Schiffmann, D.G. Warnock, and M. Banikazemi Fabry disease: progression of nephropathy, and prevalence of cardiac and cerebrovascular events before enzyme replacement therapy Nephrol. Dial. Transplant. 24 2009 2102 2111
22. K/DOQI clinical practice guidelines for chronic kidney disease: evaluation, classification, and stratification Am. J. Kidney Dis. 39 2002 S1 S266
23. R.B. Devereux, M.J. Koren, G. de Simone, M.J. Roman, and J.H. Laragh Left ventricular mass as a measure of preclinical hypertensive disease Am. J. Hypertens. 5 1992 175S 181S
24. R. Schiffmann, M. Ries, M. Timmons, J.T. Flaherty, and R.O. Brady Long-term therapy with agalsidase alfa for Fabry disease: safety and effects on renal function in a home infusion setting Nephrol. Dial. Transplant. 21 2006 345 354
25. C. Kampmann, A. Linhart, R.B. Devereux, and R. Schiffmann Effect of agalsidase alfa replacement therapy on Fabry disease-related hypertrophic cardiomyopathy: a 12- to 36-month, retrospective, blinded echocardiographic pooled analysis Clin. Ther. 31 2009 1966 1976
26. J. Zamorano, V. Serra, and L. Perez de Isla Usefulness of tissue Doppler on early detection of cardiac disease in Fabry patients and potential role of enzyme replacement therapy (ERT) for avoiding progression of disease Eur. J. Echocardiogr. 12 2011 671 677
27. S.M. Rombach, B.E. Smid, M.G. Bouwman, G.E. Linthorst, M.G. Dijkgraaf, and C.E. Hollak Long term enzyme replacement therapy for Fabry disease: effectiveness on kidney, heart and brain Orphanet J. Rare Dis. 8 2013 47
28. L.J. Anderson, K.M. Wyatt, and W. Henley Long-term effectiveness of enzyme replacement therapy in Fabry disease: results from the NCS-LSD cohort study J. Inherit. Metab. Dis. 37 2014 969 978
29. J. Politei, H. Amartino, and A.B. Schenone Fabry disease: multidisciplinary evaluation after 10 years of treatment with agalsidase beta JIMD Rep. 16 2014 7 14
30. F. Weidemann, M. Niemann, and S. Stork Long-term outcome of enzyme-replacement therapy in advanced Fabry disease: evidence for disease progression towards serious complications J. Intern. Med. 274 2013 331 341