Enzyme replacement therapy and fabry nephropathy

Clinical Journal of the American Society of Nephrology

Volumn 5, Issue 2, 2010, Pages 371-378

  Warnock, David G ^a   Daina, Erica ^b,c   Remuzzi, Giuseppe ^b,c   West, Michael ^d  

^a University of Alabama at Birmingham   (United States)

^b MARIO NEGRI INST PHARMACOL RES   (Italy)

^c OSPEDALI RIUNITI   (Italy)

^d DALHOUSIE UNIVERSITY   (Canada)

Author keywords

[No Author keywords available]

Indexed keywords

AGALSIDASE ALFA; AGALSIDASE BETA; ALBUMIN; ANGIOTENSIN RECEPTOR ANTAGONIST; CREATININE; DIPEPTIDYL CARBOXYPEPTIDASE INHIBITOR; IRBESARTAN; LOSARTAN;

ANEMIA; BLOOD PRESSURE; BLOOD PRESSURE REGULATION; CHRONIC KIDNEY DISEASE; CLINICAL TRIAL; CONFERENCE PAPER; COUGHING; CREATININE BLOOD LEVEL; DISEASE COURSE; ENZYME REPLACEMENT; FABRY DISEASE; GLOMERULUS FILTRATION RATE; HUMAN; HYPERKALEMIA; KIDNEY FUNCTION; OUTCOME ASSESSMENT; PROTEIN URINE LEVEL; PROTEINURIA; TREATMENT DURATION; URINARY EXCRETION;

ADULT; ALPHA-GALACTOSIDASE; ANGIOTENSIN II TYPE 1 RECEPTOR BLOCKERS; ANGIOTENSIN-CONVERTING ENZYME INHIBITORS; BIOLOGICAL MARKERS; CREATININE; DISEASE PROGRESSION; ENZYME REPLACEMENT THERAPY; FABRY DISEASE; FEMALE; GLOMERULAR FILTRATION RATE; HUMANS; KIDNEY DISEASES; MALE; MIDDLE AGED; PROTEINURIA; SEVERITY OF ILLNESS INDEX; TREATMENT OUTCOME;

EID: 77749320320     PISSN: 15559041     EISSN: 1555905X     Source Type: Journal    
DOI: 10.2215/CJN.06900909     Document Type: Conference Paper

References (50)

1. Gubler MC, Lenoir G, Grunfeld JP, Ulmann A, Droz D, Habib R: Early renal changes in hemizygous and heterozygous patients with Fabry's disease. Kidney Int 13: 223-235, 1978
2. Sessa A, Meroni M, Battini G, Maglio A, Brambilla PL, Bertella M, Nebuloni M, Pallotti F, Giordano F, Bertagnolio B, Tosoni A: Renal pathological changes in Fabry disease. J Inherit Metab Dis 24[Suppl 2]: 66-70, discussion 65, 2001
3. Thurberg BL, Rennke H, Colvin RB, Dikman S, Gordon RE, Collins AB, Desnick RJ, O'Callaghan M: Globotriaosylceramide accumulation in the Fabry kidney is cleared from multiple cell types after enzyme replacement therapy. Kidney Int 62: 1933-1946, 2002
4. Tondel C, Bostad L, Hirth A, Svarstad E: Renal biopsy findings in children and adolescents with Fabry disease and minimal albuminuria. Am J Kidney Dis 51: 767-776, 2008
5. Valbuena C, Carvalho E, Bustorff M, Ganhão M, Relvas S, Nogueira R, Carneiro F, Oliveira JP: Kidney biopsy findings in heterozygous Fabry disease females with early nephropathy. Virchows Arch 453: 329-338, 2008
6. Branton MH, Schiffmann R, Sabnis SG, Murray GJ, Quirk JM, Altarescu G, Goldfarb L, Brady RO, Balow JE, Austin HA 3rd, Kopp JB: Natural history of Fabry renal disease: Influence of alpha-galactosidase A activity and genetic mutations on clinical course. Medicine (Baltimore) 81: 122-138, 2002
7. Alroy J, Sabnis S, Kopp JB: Renal pathology in Fabry disease. J Am Soc Nephrol 13[Suppl 2]: S134-S138, 2002
8. Fogo A, Bostad L, Svarstad E, Cook WJ, Moll S, Barbey F, Geldenhuys L, West M, Ferluga D, Vujkovac B, Howie A, Burns A, Reeve R, Waldek S, Noel LH, Grunfeld JP, Valbuena C, Oliveira JP, Muller J, Breunig F, Zhang X, Warnock DG: Scoring system for renal pathology in Fabry disease: Report of the International Study Group of Fabry Nephropathy (ISGFN). Nephrol Dial Transplant October 15, 2009 [epub ahead of print]
9. MacDermot KD, Holmes A, Miners AH: Anderson-Fabry disease: Clinical manifestations and impact of disease in a cohort of 98 hemizygous males. J Med Genet 38: 750-760, 2001
10. 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
11. Ortiz A, Oliveira JP, Waldek S, Warnock DG, Cianciaruso B, Wanner C: 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
12. Schiffmann R, Kopp JB, Austin HA 3rd, 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
13. Eng CM, Banikazemi M, Gordon RE, Goldman M, Phelps R, Kim L, Gass A, Winston J, Dikman S, Fallon JT, Brodie S, Stacy CB, Mehta D, Parsons R, Norton K, O'Callaghan M, Desnick RJ: A phase 1/2 clinical trial of enzyme replacement in Fabry disease: Pharmacokinetic, substrate clearance, and safety studies. Am J Hum Genet 68: 711-722, 2001
14. Banikazemi M, Bultas J, Waldek S, Wilcox WR, Whitley CB, McDonald M, Finkel R, Packman S, Bichet DG, Warnock DG, Desnick RJ: Agalsidase-beta therapy for ad-vanced Fabry disease: A randomized trial. Ann Intern Med 146: 77-86, 2007
15. Schiffmann R, Askari H, Timmons M, Robinson C, Benko W, Brady R, Ries M: Weekly enzyme replacement therapy may slow decline of renal function in Fabry patients who are on long-term biweekly dosing. J Am Soc Nephrol 18: 1576-1583, 2007
16. Warnock DG: Fabry disease: Diagnosis and management, with emphasis on the renal manifestations. Curr Opin Nephrol Hypertens 14: 87-95, 2005
17. Fervenza FC, Torra R, Warnock DG: Safety and efficacy of enzyme replacement therapy in the nephropathy of Fabry disease. Biologics 2: 1-22, 2008
18. Feriozzi S, Schwarting A, Sunder-Plassmann G, West M, Cybulla M: Agalsidase alfa slows the decline in renal function in patients with Fabry disease. Am J Nephrol 29: 353-361, 2009
19. 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
20. Levey AS: Clinical practice: Nondiabetic kidney disease. N Engl J Med 347: 1505-1511, 2002
21. Germain D, Waldek S, Banikazemi M, Bushinsky D, Charrow J, 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
22. 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
23. Schwarting A, Dehout F, Feriozzi S, Beck M, Mehta A, Sunder-Plassmann G: Enzyme replacement therapy and renal function in 201 patients with Fabry disease. Clin Nephrol 66: 77-84, 2006
24. 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
25. Ruggenenti P, Schieppati A, Remuzzi G: Progression, remission, regression of chronic renal diseases. Lancet 357: 1601-1608, 2001
26. Lea J, Greene T, Hebert L, Lipkowitz M, Massry S, Middleton J, Rostand SG, Miller E, Smith W, Bakris GL: The relationship between magnitude of proteinuria reduction and risk of end-stage renal disease: Results of the African American Study of Kidney Disease and Hypertension. Arch Intern Med 165: 947-953, 2005
27. Ruggenenti P, Perna A, Remuzzi G: Retarding progression of chronic renal disease: The neglected issue of residual proteinuria. Kidney Int 63: 2254-2261, 2003
28. Ruggenenti P, Perticucci E, Cravedi P, Gambara V, Costantini M, Sharma SK, Perna A, Remuzzi G: Role of remission clinics in the longitudinal treatment of CKD. J Am Soc Nephrol 19: 1213-1224, 2008
29. Tahir H, Jackson LL, Warnock DG: Antiproteinuric therapy and Fabry nephropathy: Sustained reduction in proteinuria in patients receiving enzyme replacement therapy with agalsidase-beta. J Am Soc Nephrol 18: 2609 -2617, 2007
30. 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
31. Warnock DG. The Fabrazyme® and Arbs and ACE Inhibitor Treatment (FAACET) Study (NCT00446862). Available at: http://www.clinicaltrials.gov/ct/ search;jsessionid=87169CD1104ADB522E4B53168DEA01DD?term=NCT00446862&submit= Search. Accessed September 9, 2007
32. Schiffmann R, Waldek S, Benigni A, Auray-Blais C: Biomarkers of Fabry disease nephropathy. Clin J Am Soc Nephrol 5: 360-364, 2010
33. Canadian Fabry Research Consortium: Canadian Fabry Disease Initiative (CFDI) Enzyme Replacement Therapy (ERT) Study (NCT00455104) [database on the Internet]. Available at: http://clinicaltrials.gov/ct2/show/NCT00455104?term= west+fabry&rank=1. Accessed September 16, 2009
34. Brenner BM, Cooper ME, de Zeeuw D, Keane WF, Mitch WE, Parving HH, Remuzzi G, Snapinn SM, Zhang Z, Shahinfar S, RENAAL Study Investigators: Effects of losartan on renal and cardiovascular outcomes in patients with type 2 diabetes and nephropathy. N Engl J Med 345: 861-869, 2001
35. Lewis EJ, Hunsicker LG, Clarke WR, Berl T, Pohl MA, Lewis JB, Ritz E, Atkins RC, Rohde R, Raz I, Collaborative Study Group: Renoprotective effect of the angiotensin-receptor antagonist irbesartan in patients with neuropathy due to type 2 diabetes. N Engl J Med 345: 851-860, 2001
36. Hofstetter TH: Prevention of end-stage renal disease due to type 2 diabetes. N Engl J Med 345: 910-912, 2001
37. The ESCAPE Trial Group: Strict blood-pressure control and progression of renal failure in children. N Engl J Med 361: 1639-1650, 2009
38. Remuzzi G, Benigni A, Remuzzi A: Mechanisms of progression and regression of renal lesions of chronic nephropathies and diabetes. J Clin Invest 116: 288-296, 2006
39. 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
40. Eng CM, Germain DP, Banikazemi M, Warnock DG, Wanner C, Hopkin RJ, Bultas J, Lee P, Sims K, Brodie SE, Pastores GM, Strotmann JM, Wilcox WR: Fabry disease: Guidelines for the evaluation and management of multiorgan system involvement. Genet Med 8: 539-548, 2006
41. Levey AS, Stevens LA, Schmid CH, Zhang Y, Castro AF 3rd, Feldman HI, Kusek JW, Eggers P, Van Lente F, Greene T, Coresh J, CKD-EPI: A new equation to estimate glomerular filtration rate. Ann Intern Med 150: 604-612, 2009
42. Jain G, Campbell RC, Warnock DG: Mineralocorticoid receptor blockers and chronic kidney disease. Clin J Am Soc Nephrol 4: 1685-1691, 2009
43. Kent DM, Jafar TH, Hayward RA, Tighiouart H, Landa M, de Jong P, de Zeeuw D, Remuzzi G, Kamper AL, Levey AS: Progression risk, urinary protein excretion, and treatment effects of angiotensin-converting enzyme inhibitors in nondiabetic kidney disease. J Am Soc Nephrol 18: 1959-1965, 2007
44. Chobanian AV, Bakris GL, Black HR, Cushman WC, Green LA, Izzo JL Jr, Jones DW, Materson BJ, Oparil S, Wright JT Jr, Roccella EJ: The Seventh Report of the Joint National Committee on Prevention, Detection, Evaluation, and Treatment of High Blood Pressure: The JNC 7 report. JAMA 289: 2560-2572, 2003
45. K/DOQI clinical practice guidelines on hypertension and antihypertensive agents in chronic kidney disease. Am J Kidney Dis 43: S1-S290, 2004
46. Kleinert J, Dehout F, Schwarting A, de Lorenzo AG, Ricci R, Kampmann C, Beck M, Ramaswami U, Linhart A, Gal A, Houge G, Widmer U, Mehta A, Sunder-Plassmann G: Prevalence of uncontrolled hypertension in patients with Fabry disease. Am J Hypertens 19: 782-787, 2006
47. Bakris GL, Weir MR: Angiotensin-converting enzyme inhibitor-associated elevations in serum creatinine: Is this a cause for concern? Arch Intern Med 160: 685-693, 2000
48. Vedder AC, Breunig F, Donker-Koopman WE, Mills K, Young E, Winchester B, Ten Berge IJ, Groener JE, Aerts JM, Wanner C, Hollak CE: Treatment of Fabry disease with different dosing regimens of agalsidase: Effects on antibody formation and GL-3. Mol Genet Metab 94: 319-325, 2008
49. Wang J, Lozier J, Johnson G, Kirshner S, Verthelyi D, Pariser A, Shores E, Rosenberg A: Neutralizing antibodies to therapeutic enzymes: Considerations for testing, prevention and treatment. Nat Biotechnol 26: 901-908, 2008
50. Young E, Mills K, Morris P, Vellodi A, Lee P, Waldek S, Winchester B: Is globotriaosylceramide a useful biomarker in Fabry disease? Acta Paediatr Suppl 94: 51-54, discussion 37-58, 2005