The effectiveness of long-term agalsidase alfa therapy in the treatment of fabry nephropathy

Clinical Journal of the American Society of Nephrology

Volumn 7, Issue 1, 2012, Pages 60-69

  Feriozzi, Sandro ^a   Torras, Joan ^b   Cybulla, Markus ^c   Nicholls, Kathy ^d   Sunder Plassmann, Gere ^e   West, Michael ^f  

^a BELCOLLE HOSPITAL   (Italy)

^b UNIVERSITY OF BARCELONA   (Spain)

^c UNIVERSITY OF FREIBURG   (Germany)

^d ROYAL MELBOURNE HOSPITAL   (Australia)

^e MEDICAL UNIVERSITY OF VIENNA   (Austria)

^f DALHOUSIE UNIVERSITY   (Canada)

Author keywords

[No Author keywords available]

Indexed keywords

AGALSIDASE ALFA; CREATININE; PROTEIN;

ADULT; ARTICLE; BLOOD PRESSURE MEASUREMENT; CREATININE BLOOD LEVEL; DISEASE REGISTRY; ENZYME REPLACEMENT; FABRY DISEASE; FEMALE; FOLLOW UP; GLOMERULUS FILTRATION RATE; HUMAN; KIDNEY FUNCTION; MAJOR CLINICAL STUDY; MALE; OBSERVATIONAL STUDY; PATIENT ASSESSMENT; PROTEIN URINE LEVEL; TREATMENT OUTCOME; URINARY EXCRETION;

ADULT; ALPHA-GALACTOSIDASE; ANGIOTENSIN RECEPTOR ANTAGONISTS; ANGIOTENSIN-CONVERTING ENZYME INHIBITORS; ENZYME REPLACEMENT THERAPY; FABRY DISEASE; FEMALE; GLOMERULAR FILTRATION RATE; HUMANS; HYPERTENSION; ISOENZYMES; KIDNEY DISEASES; MALE; MIDDLE AGED; PROTEINURIA;

EID: 84857082168     PISSN: 15559041     EISSN: 1555905X     Source Type: Journal    
DOI: 10.2215/CJN.03130411     Document Type: Article

References (40)

1. Ortiz A, Oliveira JP, Waldek S, Warnock DG, Cianciaruso B, Wanner C Fabry Registry: Nephropathy in males and females with Fabry disease: Cross-sectional description of patients before treatment with enzyme replacement therapy. Nephrol Dial Transplant 23: 1600-1607, 2008
2. Hughes DA, Elliott PM, Shah J, Zuckerman J, Coghlan G, Brookes J, Mehta AB: Effects of enzyme replacement therapy on the car-diomyopathy of Anderson-Fabry disease: A randomised, double-blind, placebo-controlled clinical trial of agalsidase alfa. Heart 94: 153-158, 2008
3. Imbriaco M, Pisani A, Spinelli L, Cuocolo A, Messalli G, Capuano E, Marmo M, Liuzzi R, Visciano B, Cianciaruso B, Salvatore M: Effects of enzyme-replacement therapy in patients with Anderson-Fabry disease: A prospective long-term cardiac magnetic resonance imaging study. Heart 95: 1103-1107, 2009
4. Mehta A, Clarke JT, Giugliani R, Elliott P, Linhart A, Beck M, Sunder-Plassmann G FOS Investigators: Natural course of Fabry disease: Changing pattern of causes of death in FOS - Fabry Outcome Survey. J Med Genet 46: 548-552, 2009
5. Germain DP, Waldek S, Banikazemi M, Bushinsky DA, Charrow J, Desnick RJ, Lee P, Loew T, Vedder AC, Abichandani R, Wilcox WR, Guffon N: Sustained, long-term renal stabilization after 54 months of agalsidase beta therapy in patients with Fabry disease. J Am Soc Nephrol 18: 1547-1557, 2007
6. Schiffmann R, Ries M, Timmons M, Flaherty JT, Brady RO: Long-term therapy with agalsidase alfa for Fabry disease: Safety and effects on renal function in a home infusion setting. Nephrol Dial Transplant 21: 345-354, 2006
7. West M, Nicholls K, Mehta A, Clarke JT, Steiner R, Beck M, Barshop BA, Rhead W, Mensah R, Ries M, Schiffmann R: Agalsidase alfa and kidney dysfunction in Fabry disease. J Am Soc Nephrol 20: 1132-1139, 2009
8. Ortiz A, Oliveira JP, Wanner C, Brenner BM, Waldek S, Warnock DG: Recommendations and guidelines for the diagnosis and treatment of Fabry nephropathy in adults. Nat Clin Pract Nephrol 4: 327-336, 2008
9. Levey AS, Stevens LA, Schmid CH, Zhang YL, Castro AF, Feldman HI, Kusek JW, Eggers P, Van Lente F, Greene T, Coresh J CKD-EPI (Chronic Kidney Disease Epidemiology Collaboration): A new equation to estimate glomerular filtration rate. Ann Intern Med 150: 604-612, 2009
10. Levey AS, Bosch JP, Lewis JB, Greene T, Rogers N, Roth D Modification of Diet in Renal Disease Study Group: A more accurate method to estimate glomerular filtration rate from serum creatinine: A new prediction equation. Ann Intern Med 130: 461-470, 1999
11. Matsushita K, Selvin E, Bash LD, Astor BC, Coresh J: Risk implications of the new CKD Epidemiology Collaboration (CKD-EPI) equation compared with the MDRD Study equation for estimated GFR: The Atherosclerosis Risk in Communities (ARIC) Study. Am J Kidney Dis 55: 648-659, 2010
12. 2. Am J Kidney Dis 56: 486-495, 2010
13. Peake M, Whiting M: Measurement of serum creatinine- Current status and future goals. Clin Biochem Rev 27: 173-184, 2006
14. Vickery S, Stevens PE, Dalton RN, van Lente F, Lamb EJ: Does the ID-MS traceable MDRD equation work and is it suitable for use with compensated Jaffe and enzymatic creatinine assays? Nephrol Dial Transplant 21: 2439-2445, 2006
15. National Kidney Foundation: K/DOQI clinical practice guidelines for chronic kidney disease: Evaluation, classification, and stratification. Am J Kidney Dis 39[Suppl 1]: S1-S266, 2002
16. Schiffmann R, Kopp JB, Austin HA, Sabnis S, Moore DF, Weibel T, Balow JE, Brady RO: Enzyme replacement therapy in Fabry disease: A randomized controlled trial. JAMA 285: 2743-2749, 2001
17. Stevens LA, Coresh J, Feldman HI, Greene T, Lash JP, Nelson RG, Rahman M, Deysher AE, Zhang YL, Schmid CH, Levey AS: Evaluation of the Modification of Diet in Renal Disease study equation in a large diverse population. J Am Soc Nephrol 18: 2749-2757, 2007
18. Rombach SM, Baas MC, ten Berge IJ, Krediet RT, Bemelman FJ, Hollak CE: The value of estimated GFR in comparison to measured GFR for the assessment of renal function in adult patients with Fabry disease. Nephrol Dial Transplant 25: 2549-2556, 2010
19. Levey AS, Stevens LA: Estimating GFR using the CKD Epidemiology Collaboration (CKD-EPI) creatinine equation: More accurate GFR estimates, lower CKD prevalence estimates, and better risk predictions. Am J Kidney Dis 55: 622-627, 2010
20. Stevens LA, Schmid CH, Zhang YL, Coresh J, Manzi J, Landis R, Bakoush O, Contreras G, Genuth S, Klintmalm GB, Poggio E, Rossing P, Rule AD, Weir MR, Kusek J, Greene T, Levey AS: Development and validation of GFR-estimating equations using diabetes, transplant and weight. Nephrol Dial Transplant 25: 449-457, 2010
21. Stevens LA, Coresh J, Greene T, Levey AS: Assessing kidney function-Measured and estimated glomerular filtration rate. N Engl J Med 354: 2473-2483, 2006
22. Mehta A, Beck M, Elliott P, Giugliani R, Linhart A, Sunder-Plassmann G, Schiffmann R, Barbey F, Ries M, Clarke JT Fabry Outcome Survey investigators: Enzyme replacement therapy with agalsidase alfa in patients with Fabry's disease: An analysis of registry data. Lancet 374: 1986-1996, 2009
23. Mehta A, Beck M, Elliott P, Giugliani R, Linhart A, Sunder-Plassmann G, Schiffmann R, Barbey F, Ries M, Clarke JT: Department of error. Lancet 375: 200, 2010
24. Schiffmann R, Warnock DG, Banikazemi M, Bultas J, Linthorst GE, Packman S, Sorensen SA, Wilcox WR, Desnick RJ: Fabry disease: Progression of nephropathy, and prevalence of cardiac and cerebrovascular events before enzyme replacement therapy. Nephrol Dial Transplant 24: 2102-2111, 2009
25. Barbey F, Lidove O, Schwarting A: Fabry nephropathy: 5 years of enzyme replacement-A short review. NDT Plus 1: 11-19, 2008
26. Zoja C, Benigni A, Remuzzi G: Cellular responses to protein overload: Key event in renal disease progression. Curr Opin Nephrol Hypertens 13: 31-37, 2004
27. Banikazemi M, Bultas J, Waldek S, Wilcox WR, Whitley CB, McDonald M, Finkel R, Packman S, Bichet DG, Warnock DG, Desnick RJ Fabry Disease Clinical Trial Study Group: Agalsidase-beta therapy foradvanced Fabry disease: A randomized trial. Ann Intern Med 146: 77-86, 2007
28. Wanner C, Oliveira JP, Ortiz A, Mauer M, Germain DP, Linthorst GE, Serra AL, Maródi L, Mignani R, Cianciaruso B, Vujkovac B, Lemay R, Beitner-Johnson D, Waldek S, Warnock DG: Prognostic indicators of renal disease progression in adults with Fabry disease: Natural history data from the Fabry Registry. Clin J Am Soc Nephrol 5: 2220-2228, 2010
29. Schiffmann R, Askari H, Timmons M, Robinson C, Benko W, Brady RO, Ries M: Weekly enzyme replacement therapy may slow decline of renal function in patients with Fabry disease who are on long-term biweekly dosing. J Am Soc Nephrol 18: 1576-1583, 2007
30. Breunig F, Weidemann F, Strotmann J, Knoll A, Wanner C: Clinical benefit of enzyme replacement therapy in Fabry disease. Kidney Int 69: 1216-1221, 2006
31. Thofehrn S, Netto C, Cecchin C, Burin M, Matte U, Brustolin S, Nunes AC, Coelho J, Tsao M, Jardim L, Giugliani R, Barros EJ: Kidney function and 24-hour proteinuria in patients with Fabry disease during 36 months of agalsidase alfa enzyme replacement therapy: A Brazilian experience. Ren Fail 31: 773-778, 2009
32. Tahir H, Jackson LL, Warnock DG: Antiproteinuric therapy and Fabry nephropathy: Sustained reduction of proteinuria in patients receiving enzyme replacement therapy with agalsidase-beta. J Am Soc Nephrol 18: 2609-2617, 2007
33. Jain G, Warnock DG: Blood pressure, proteinuria and nephrop-athy in Fabry disease. Nephron Clin Pract 118: c43-c48, 2011
34. Warnock D, Ortiz A, Mauer M, Linthorst GE, Oliveira JP, Serra AL, Marodi L, Mignani R, Vujkovac B, Beitner-Johnson D, Lemay R, Cole JA, Svarstad E, Waldek S, Germain DP, Wanner C; On behalf of the Fabry Registry: Renal outcome of agalsidase beta treatment for Fabry disease: Role of proteinuria and timing of treatment initiation [published online ahead of print July 29, 2011]. Nephrol Dial Transplant doi:10.1093/ndt/gfr420
35. Feriozzi S, Schwarting A, Sunder-Plassmann G, West M, Cybulla M International Fabry Outcome Survey Investigators: Agalsidase alfa slows the decline in renal function in patients with Fabry disease. Am J Nephrol 29: 353-361, 2009
36. Taal MW, Brenner BM: Predicting initiation and progression of chronic kidney disease: Developing renal risk scores. Kidney Int 70: 1694-1705, 2006
37. Remuzzi G, Ruggenenti P, Perico N: Chronic renal diseases: Renoprotective benefits of renin-angiotensin system inhibition. Ann Intern Med 136: 604-615, 2002
38. Wilmer WA, Rovin BH, Hebert CJ, Rao SV, Kumor K, Hebert LA: Management of glomerular proteinuria: A commentary. J Am Soc Nephrol 14: 3217-3232, 2003
39. Chiurchiu C, Remuzzi G, Ruggenenti P: Angiotensin-converting enzyme inhibition and renal protection in nondiabetic patients: The data of the meta-analyses. J Am Soc Nephrol 16[Suppl 1]: S58-S63, 2005
40. Batista EC, Carvalho LR, Casarini DE, Carmona AK, dos Santos EL, da Silva ED, dos Santos RA, Nakaie CR, Rojas MV, de Oliveira SM, Bader M, D'Almeida V, Martins AM, de Picoly Souza K, Pesquero JB: ACE activity is modulated by the enzyme a-galactosidase A. J Mol Med (Berl) 89: 65-74, 2011