Agalsidase alfa (ReplagalTM) in the treatment of Anderson-Fabry disease

Biologics: Targets and Therapy

Volumn 1, Issue 3, 2007, Pages 291-300

  Pastores, Gregory M ^a  

^a NEW YORK UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

Adjunctive therapy; Agalsidase alfa; Agalsidase beta; Enzyme replacement therapy

Indexed keywords

EID: 83255164188     PISSN: 11775475     EISSN: 11775491     Source Type: Journal    
DOI: None     Document Type: Review

References (62)

1. Altarescu G, Hill S, Wiggs E, et al. 2001. The efficacy of enzyme replacement therapy in patients with chronic neuronopathic Gaucher's disease. J Pediatr, 138:539-47.
2. Baker MP, Jones TD. 2007. Identification and removal of immunogenicity in therapeutic proteins. Curr Opin Drug Discov Devel, 10:219-27.
3. Banikazemi M, Bultas J, Waldek S, et al. 2007. Agalsidase-beta therapy for advanced Fabry disease: a randomized trial. Ann Intern Med, 146:77-86.
4. Barbey F, Brakch N, Linhart A, et al. 2006. Cardiac and vascular hypertrophy in Fabry disease: evidence for a new mechanism independent of blood pressure and glycosphingolipid deposition. Arterioscler Thromb Vasc Biol, 26:839-44.
5. Barbey F, Brakch N, Linhart A, et al. 2006. Increased carotid intima-media thickness in the absence of atherosclerotic plaques in an adult population with Fabry disease. Acta Paediatr Suppl, 95:63-8.
6. Basi S, Lewis JB. 2006. Microalbuminuria as a target to improve cardiovascular and renal outcomes. Am J Kidney Dis, 47:927-46.
7. Berthiaume JT, Davis J, Taira DA, et al. 2007. A managed care organization's use of integrated health management to improve secondary prevention of coronary artery disease. Am J Manag Care, 13:142-7.
8. Bigazzi R, Bianchi S, Nenci R, et al. 1995. Increased thickness of the carotid artery in patients with essential hypertension and microalbuminuria. J Hum Hypertens, 9:827-33.
9. Breunig F, Weidemann F, Strotmann J, et al. 2006. Clinical benefit of enzyme replacement therapy in Fabry disease. Kidney Int, 69:1216-21.
10. Cai SR, Xu G, Becker-Hapak M, et al. 2006. The kinetics and tissue distribution of protein transduction in mice. Eur J Pharm Sci, 27:311-19.
11. Campese VM, Park J. 2007. HMG-CoA reductase inhibitors and the kidney. Kidney Int, 71:1215-22.
12. Clarke JT. 2007. Narrative review: Fabry disease. Ann Intern Med, 146:425-33.
13. Clarke JTR, West ML, Bultas J, et al. 2007. Pharmacokinetics and pharmacodynamics of agalsidase alfa used for enzyme replacement therapy of Fabry disease. Genetics in Medicine, 9:504-9.
14. Close L, Elliott P. 2007. Optimization of concomitant medication in Fabry cardiomyopathy. Acta Paediatr Suppl, 96:81-3.
15. Coppo R, D'Amico G. 2005. Factors predicting progression of IgA nephropathies. J Nephrol, 18:503-12.
16. Elleder M, Bradova V, Smid F, et al. 1990. Cardiocyte storage and hypertrophy as a sole manifestation of Fabry's disease. Report on a case simulating hypertrophic non-obstructive cardiomyopathy. Virchows Arch A Pathol Anat Histopathol, 417:449-55.
17. Elliott PM, Kindler H, Shah JS, et al. 2006. Coronary microvascular dysfunction in male patients with Anderson-Fabry disease and the effect of treatment with alpha galactosidase A. Heart, 92:357-60.
18. Eng CM, Fletcher J, Wilcox WR, et al. 2007. Fabry disease: baseline medical characteristics of a cohort of 1765 males and females in the Fabry Registry. J Inherit Metab Dis, 30:184-92.
19. Eng CM, Guffon N, Wilcox WR, et al. 2001. Safety and efficacy of recombinant human alpha-galactosidase A-replacement therapy in Fabry's disease. N Engl J Med, 345:9-16.
20. Ferrari P. 2007. Prescribing angiotensin-converting enzyme inhibitors and angiotensin receptor blockers in chronic kidney disease. Nephrology (Carlton), 12:81-9.
21. Germain DP, Waldek S, Banikazemi M, et al. 2007. Sustained, long-term renal stabilization after 54 months of agalsidase Beta therapy in patients with fabry disease. J Am Soc Nephrol, 18:1547-57.
22. Gold KF, Pastores GM, Botteman MF, et al. 2002. Quality of life of patients with Fabry disease. Qual Life Res, 11:317-27.
23. Gupta S, Ries M, Kotsopoulos S, et al. 2005. The relationship of vascular glycolipid storage to clinical manifestations of Fabry disease: a crosssectional study of a large cohort of clinically affected heterozygous women. Medicine (Baltimore), 84:261-8.
24. Hughes DA, Elliott PM, Shah J, et al. 2007. Effects of enzyme replacement therapy on the cardiomyopathy of Anderson-Fabry disease: a randomized, double-blind, placebo-controlled clinical trial of agalsidase-alfa. Heart, May 4[Epub ahead of print].
25. Ichinose M, Nakayama M, Ohashi T, et al. 2005. Significance of screening for Fabry disease among male dialysis patients. Clin Exp Nephrol, 9:228-32.
26. Imbriaco M, Spinelli L, Cuocolo A, et al. 2007. MRI characterization of myocardial tissue in patients with Fabry's disease. AJR Am J Roentgenol, 188:850-3.
27. Kikano GE, Brown MT. 2007. Antiplatelet therapy for atherothrombotic disease: an update for the primary care physician. Mayo Clin Proc, 82:583-98.
28. Lidove O, Joly D, Barbey F, et al. 2007. Clinical results of enzyme replacement therapy in Fabry disease: a comprehensive review of literature. Int J Clin Pract, 61:293-302.
29. Linthorst GE, Hollak CE, Donker-Koopman WE, et al. 2004. Enzyme therapy for Fabry disease: neutralizing antibodies toward agalsidase alpha and beta. Kidney Int, 66:1589-95.
30. MacDermot KD, Holmes A, Miners AH. 2001a. Anderson-Fabry disease: clinical manifestations and impact of disease in a cohort of 98 hemizygous males. J Med Genet, 38:750-60.
31. MacDermot KD, Holmes A, Miners AH. 2001b. Anderson-Fabry disease: clinical manifestations and impact of disease in a cohort of 60 obligate carrier females. J Med Genet, 38:769-75.
32. Mehta A, Ricci R, Widmer U, et al. 2004. Fabry disease defined: baseline clinical manifestations of 366 patients in the Fabry Outcome Survey. Eur J Clin Invest, 34:236-42.
33. Moore DF, Gelderman MP, Ferreira PA, et al. 2007. Genomic abnormalities of the murine model of Fabry disease after disease-related perturbation, a systems biology approach. Proc Natl Acad Sci USA, 104:8065-70.
34. Moore DF, Kaneski CR, Askari H, et al. 2007. The cerebral vasculopathy of Fabry disease. J Neurol Sci, 257:258-63.
35. Moore DF, Ries M, Forget EL, et al. 2007. Enzyme replacement therapy in orphan and ultra-orphan diseases: the limitations of standard economic metrics as exemplified by Fabry-Anderson disease. Pharmacoeconomics, 25:201-8.
36. Murray GJ, Anver MR, Kennedy MA, et al. 2007. Cellular and tissue distribution of intravenously administered agalsidase alfa. Mol Genet Metab, 90:307-12.
37. Owens CL, Russell SD, Halushka MK. 2006. Histologic and electron microscopy findings in myocardium of treated Fabry disease. Hum Pathol, 37:764-8.
38. Pastores GM. 2003. Enzyme therapy for the lysosomal storage disorders: principles, patents, practice and prospects. Expert Opin Ther Patents, 13:1157-72.
39. Pastores GM. 2004. Agalsidase alfa (Replagal™): enzyme therapy for Anderson-Fabry disease. Therapy, 1:203-11.
40. Pastores GM, Boyd E, Crandall K, et al. 2007. Safety and pharmacokinetics of agalsidase alfa in patients with Fabry disease and end-stage renal disease. Nephrol Dial Transplant, 22:1920-5.
41. Pastores GM, Thadhani R. 2002. Advances in the management of AndersonFabry disease: enzyme replacement therapy. Expert Opin Biol Ther, 2:325-33.
42. Ramaswami U, Wendt S, Pintos-Morell G, et al. 2007. Enzyme replacement therapy with agalsidase alfa in children with Fabry disease. Acta Paediatr, 96:122-7.
43. Ries M, Clarke JT, Whybra C, et al. 2006. Enzyme-replacement therapy with agalsidase alfa in children with Fabry disease. Pediatrics, 118:924-32.
44. Rolfs A, Bottcher T, Zschiesche M, et al. 2005. Prevalence of Fabry disease in patients with cryptogenic stroke: a prospective study. Lancet, 366:1794-6.
45. Schellekens H. 2005. Factors influencing the immunogenicity of therapeutic proteins. Nephrol Dial Transplant, 20 Suppl 6:63-9.
46. Schiffmann R, Askari H, Timmons M, et al. 2007. 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, 18:1576-83.
47. Schiffmann R, Floeter MK, Dambrosia JM, et al. 2003. Enzyme replacement therapy improves peripheral nerve and sweat function in Fabry disease. Muscle Nerve, 28:703-10.
48. Schiffmann R, Kopp JB, Austin HAI, et al. 2001. Enzyme replacement therapy in Fabry disease: a randomized controlled trial. JAMA, 285:2743-49.
49. Schiffmann R, Murray GJ, Treco D, et al. 2000. Infusion of alpha-galactosidase A reduces tissue globotriaosylceramide storage in patients with Fabry disease. Proc Natl Acad Sci USA, 97:365-70.
50. Schiffmann R, Rapkiewicz A, Abu-Asab M, et al. 2006. Pathological findings in a patient with Fabry disease who died after 2.5 years of enzyme replacement. Virchows Arch, 448:337-43.
51. Schiffmann R, Ries M, Timmons M, et al. 2006. Long-term therapy with agalsidase alfa for Fabry disease: safety and effects on renal function in a home infusion setting. Nephrol Dial Transplant, 21:345-54.
52. Sessa A, Meroni M, Battini G, et al. 2004. Chronic renal failure, dialysis, and renal transplantation in Anderson-Fabry disease. Semin Nephrol, 24:532-6.
53. Shah JS, Hughes DA, Sachdev B, et al. 2005. Prevalence and clinical significance of cardiac arrhythmia in Anderson-Fabry disease. Am J Cardiol, 96:842-6.
54. Shah JS, Hughes DA, Tayebjee MH, et al. 2007. Extracellular matrix turnover and disease severity in Anderson-Fabry disease. J Inherit Metab Dis, 30:88-95.
55. Tanaka M, Ohashi T, Kobayashi M, et al. 2005. Identification of Fabry's disease by the screening of alpha-galactosidase A activity in male and female hemodialysis patients. Clin Nephrol, 64:281-7.
56. Vance DE, Krivit W, Sweeley CC. 1975. Metabolism of neutral glycosphingolipids in plasma of a normal human and a patient with Fabry's disease. J Biol Chem, 250:8119-25.
57. van de Weert M, Jorgensen L, Horn Moeller E, et al. 2005. Factors of importance for a successful delivery system for proteins. Expert Opin Drug Deliv, 2:1029-37.
58. Vedder AC, Linthorst GE, Houge G, et al. 2007. Treatment of Fabry disease: outcome of a comparative trial with agalsidase alfa or beta at a dose of 0.2 mg/kg. PLoS ONE, 2:e598.
59. Vedder AC, Linthorst GE, van Breemen MJ, et al. 2007. The Dutch Fabry cohort: diversity of clinical manifestations and Gb3 levels. J Inherit Metab Dis, 30:68-78.
60. Warnock DG. 2007. Enzyme replacement therapy and Fabry kidney disease: quo vadis?. J Am Soc Nephrol, 18:1368-70.
61. Warnock DG. 2005. Fabry disease: diagnosis and management, with emphasis on the renal manifestations. Curr Opin Nephrol Hypertens, 14:87-95.
62. Whybra C, Schwarting A, Kriegsmann J, et al. 2006. IgA nephropathy in two adolescent sisters heterozygous for Fabry disease. Pediatr Nephrol, 21:1251-6.