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Volumn 41, Issue 7, 2011, Pages 710-718

Modelling the resource implications of managing adults with Fabry disease in Italy

Author keywords

Agalsidase alfa; Agalsidase beta; Fabrazyme; Fabry disease; Italy; Replagal; Servizio Sanitario Nazionale

Indexed keywords

AGALSIDASE ALFA; AGALSIDASE BETA;

EID: 79958722345     PISSN: 00142972     EISSN: 13652362     Source Type: Journal    
DOI: 10.1111/j.1365-2362.2010.02458.x     Document Type: Article
Times cited : (11)

References (33)
  • 1
    • 0035852488 scopus 로고    scopus 로고
    • Anderson-Fabry's disease: α-galactosidase deficiency
    • Peters FPJ, Vermeulen A, Kho TL. Anderson-Fabry's disease: α-galactosidase deficiency. Lancet 2001;357:138-40.
    • (2001) Lancet , vol.357 , pp. 138-140
    • Peters, F.P.J.1    Vermeulen, A.2    Kho, T.L.3
  • 2
    • 12144287518 scopus 로고    scopus 로고
    • Fabry disease defined: baseline clinical manifestations of 366 patients in FOS - the Fabry Outcome Survey
    • Mehta A, Ricci R, Widmer U, Dehout F, Garcia de Lorenzo A, Kampmann C et al. Fabry disease defined: baseline clinical manifestations of 366 patients in FOS - the Fabry Outcome Survey. Eur J Clin Invest, 2004;34:236-42.
    • (2004) Eur J Clin Invest , vol.34 , pp. 236-242
    • Mehta, A.1    Ricci, R.2    Widmer, U.3    Dehout, F.4    Garcia de Lorenzo, A.5    Kampmann, C.6
  • 3
  • 4
    • 68049129847 scopus 로고    scopus 로고
    • Natural course of Fabry disease: changing pattern of causes of death in FOS - Fabry Outcome Survey
    • Mehta A, Clarke JT, Giugliani R, Elliott P, Linhart A, Beck M et al. Natural course of Fabry disease: changing pattern of causes of death in FOS - Fabry Outcome Survey. J Med Genet 2009;46:548-52.
    • (2009) J Med Genet , vol.46 , pp. 548-552
    • Mehta, A.1    Clarke, J.T.2    Giugliani, R.3    Elliott, P.4    Linhart, A.5    Beck, M.6
  • 5
    • 0000889058 scopus 로고    scopus 로고
    • α-Galactosidase A deficiency: fabry disease
    • In: Scriver CR, Beaudet AL, Sly WS, Valle D, editors., 8th edn. New York: McGraw-Hill
    • Desnick RJ, Ioannou YA, Eng CM. α-Galactosidase A deficiency: fabry disease. In: Scriver CR, Beaudet AL, Sly WS, Valle D, editors. The Metabolic and Molecular Bases of Inherited Disease, 8th edn. New York: McGraw-Hill, 2001:pp. 37-74.
    • (2001) The Metabolic and Molecular Bases of Inherited Disease , pp. 37-74
    • Desnick, R.J.1    Ioannou, Y.A.2    Eng, C.M.3
  • 7
    • 0036234750 scopus 로고    scopus 로고
    • Advances in the management of Anderson-Fabry disease: enzyme replacement therapy
    • Pastores GM, Thadhani R. Advances in the management of Anderson-Fabry disease: enzyme replacement therapy. Expert Opin Biol Ther 2002;2:1-9.
    • (2002) Expert Opin Biol Ther , vol.2 , pp. 1-9
    • Pastores, G.M.1    Thadhani, R.2
  • 8
    • 68049134975 scopus 로고    scopus 로고
    • A 4-year study of the efficacy and tolerability of enzyme replacement therapy with agalsidase alfa in 36 women with Fabry disease
    • Whybra C, Miebach E, Mengel E, Gal A, Baron K, Beck M et al. A 4-year study of the efficacy and tolerability of enzyme replacement therapy with agalsidase alfa in 36 women with Fabry disease. Genet Med. 2009;11:441-9.
    • (2009) Genet Med. , vol.11 , pp. 441-449
    • Whybra, C.1    Miebach, E.2    Mengel, E.3    Gal, A.4    Baron, K.5    Beck, M.6
  • 10
    • 54949106603 scopus 로고    scopus 로고
    • Agalsidase alfa slows the decline in renal function in patients with Fabry disease
    • International Fabry Outcome Survey Investigators
    • 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 2009;29:353-61.
    • (2009) Am J Nephrol , vol.29 , pp. 353-361
    • Feriozzi, S.1    Schwarting, A.2    Sunder-Plassmann, G.3    West, M.4    Cybulla, M.5
  • 11
    • 34248190164 scopus 로고    scopus 로고
    • Sustained, long-term renal stabilization after 54months of agalsidase beta therapy in patients with Fabry disease
    • Germain DP, Waldek S, Banikazemi M, Bushinsky DA, Charrow J, Desnick RJ et al. Sustained, long-term renal stabilization after 54months of agalsidase beta therapy in patients with Fabry disease. Am Soc Nephrol 2007;18:1547-57.
    • (2007) Am Soc Nephrol , vol.18 , pp. 1547-1557
    • Germain, D.P.1    Waldek, S.2    Banikazemi, M.3    Bushinsky, D.A.4    Charrow, J.5    Desnick, R.J.6
  • 13
    • 0035811624 scopus 로고    scopus 로고
    • Safety and efficacy of recombinant human α-galactosidase A replacement in Fabry disease
    • Eng CM, Guffon N, Wilcox WR, Germain DP, Lee P, Waldek S et al. Safety and efficacy of recombinant human α-galactosidase A replacement in Fabry disease. N Engl J Med 2001;345:9-16.
    • (2001) N Engl J Med , vol.345 , pp. 9-16
    • Eng, C.M.1    Guffon, N.2    Wilcox, W.R.3    Germain, D.P.4    Lee, P.5    Waldek, S.6
  • 14
    • 31544456336 scopus 로고    scopus 로고
    • Long-term therapy with agalsidase alfa for Fabry disease: safety and effects on renal function in a home infusion setting
    • 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 2005;21:345-54.
    • (2005) Nephrol Dial Transplant , vol.21 , pp. 345-354
    • Schiffmann, R.1    Ries, M.2    Timmons, M.3    Flaherty, J.T.4    Brady, R.O.5
  • 15
    • 31544463530 scopus 로고    scopus 로고
    • Home treatment for Fabry disease: practice guidelines based on 3years experience in The Netherlands
    • Linthorst GE, Vedder AC, Ormel EE, Aerts JM, Hollak CE. Home treatment for Fabry disease: practice guidelines based on 3years experience in The Netherlands. Nephrol Dial Transplant 2006;21:355-60.
    • (2006) Nephrol Dial Transplant , vol.21 , pp. 355-360
    • Linthorst, G.E.1    Vedder, A.C.2    Ormel, E.E.3    Aerts, J.M.4    Hollak, C.E.5
  • 16
    • 33646845939 scopus 로고    scopus 로고
    • Intravenous enzyme replacement therapy: better in home or hospital?
    • Milligan A, Hughes D, Goodwin S, Richfield L, Mehta A. Intravenous enzyme replacement therapy: better in home or hospital? Br J Nurs 2006;15:330-3.
    • (2006) Br J Nurs , vol.15 , pp. 330-333
    • Milligan, A.1    Hughes, D.2    Goodwin, S.3    Richfield, L.4    Mehta, A.5
  • 17
    • 79958726561 scopus 로고    scopus 로고
    • European Medicines Agency reviews treatment recommendations for Fabrazyme. European Medicines Agency. 22 October; EMA/CHMP/654389/2010. Available at:. Accessed on 11 November 2010.
    • European Medicines Agency reviews treatment recommendations for Fabrazyme. European Medicines Agency. 22 October 2010; EMA/CHMP/654389/2010. Available at:. Accessed on 11 November 2010.
    • (2010)
  • 18
    • 79958753994 scopus 로고    scopus 로고
    • ®). Available at:. Accessed on 11 November 2010.
    • ®). Available at:. Accessed on 11 November 2010.
  • 20
    • 79958725051 scopus 로고    scopus 로고
    • OECD Health Data. Available online at
    • OECD Health Data 2008. Available online at.
    • (2008)
  • 21
    • 0013192938 scopus 로고    scopus 로고
    • A biochemical and pharmacological comparison of enzyme replacement therapies for the glycolipid storage disorder Fabry disease
    • Lee K, Jin X, Zhang K, Copertino L, Andrews L, Baker-Malcolm J et al. A biochemical and pharmacological comparison of enzyme replacement therapies for the glycolipid storage disorder Fabry disease. Glycobiology 2003;13:305-13.
    • (2003) Glycobiology , vol.13 , pp. 305-313
    • Lee, K.1    Jin, X.2    Zhang, K.3    Copertino, L.4    Andrews, L.5    Baker-Malcolm, J.6
  • 22
    • 33645218156 scopus 로고    scopus 로고
    • Comparison of the effects of agalsidase alfa and agalsidase beta on cultured human Fabry fibroblasts and Fabry mice
    • Sakuraba H, Murata-Ohsawa M, Kawashima I, Tajima Y, Kotani M, Ohshima T et al. Comparison of the effects of agalsidase alfa and agalsidase beta on cultured human Fabry fibroblasts and Fabry mice. J Hum Genet 2005;51:180-8.
    • (2005) J Hum Genet , vol.51 , pp. 180-188
    • Sakuraba, H.1    Murata-Ohsawa, M.2    Kawashima, I.3    Tajima, Y.4    Kotani, M.5    Ohshima, T.6
  • 23
    • 0035949059 scopus 로고    scopus 로고
    • Enzyme-replacement therapy for Anderson-Fabry disease
    • Pastores GM, Thadhani R. Enzyme-replacement therapy for Anderson-Fabry disease. Lancet 2001;358:601-3.
    • (2001) Lancet , vol.358 , pp. 601-603
    • Pastores, G.M.1    Thadhani, R.2
  • 25
    • 0042305028 scopus 로고    scopus 로고
    • Comparative evaluation of alphagalactosidase A infusions for treatment of Fabry disease
    • Hopkin RJ, Bissler J, Grabowski GA. Comparative evaluation of alphagalactosidase A infusions for treatment of Fabry disease. Genet Med 2003;5:144-53.
    • (2003) Genet Med , vol.5 , pp. 144-153
    • Hopkin, R.J.1    Bissler, J.2    Grabowski, G.A.3
  • 26
    • 3142737171 scopus 로고    scopus 로고
    • Enzyme replacement therapy for Fabry disease: lessons from two alpha-galactosidase A orphan products and one FDA approval
    • Desnick RJ. Enzyme replacement therapy for Fabry disease: lessons from two alpha-galactosidase A orphan products and one FDA approval. Expert Opin Biol Ther 2004;4:1167-76.
    • (2004) Expert Opin Biol Ther , vol.4 , pp. 1167-1176
    • Desnick, R.J.1
  • 27
    • 4243100675 scopus 로고    scopus 로고
    • Enzyme replacement therapy in Fabry's disease: recent advances and clinical applications
    • Mignani R, Cagnoli L. Enzyme replacement therapy in Fabry's disease: recent advances and clinical applications. J Nephrol 2004;17:354-63.
    • (2004) J Nephrol , vol.17 , pp. 354-363
    • Mignani, R.1    Cagnoli, L.2
  • 28
    • 33847050214 scopus 로고    scopus 로고
    • Clinical results of enzyme replacement therapy in Fabry disease: a comprehensive review of literature
    • Lidove O, Joly D, Barbey F, Bekri S, Alexandra JF, Peigne V et al. Clinical results of enzyme replacement therapy in Fabry disease: a comprehensive review of literature. Int J Clin Pract 2007;61:293-302.
    • (2007) Int J Clin Pract , vol.61 , pp. 293-302
    • Lidove, O.1    Joly, D.2    Barbey, F.3    Bekri, S.4    Alexandra, J.F.5    Peigne, V.6
  • 29
    • 78649634506 scopus 로고    scopus 로고
    • Effects of enzyme replacement therapy in Fabry disease - A comprehensive review of the medical literature
    • Lidove O, West M, Pintos-Morell G, Reisin R, Nicholls K, Figuera LE et al. Effects of enzyme replacement therapy in Fabry disease - A comprehensive review of the medical literature. Genetics in Medicine 2010;12:668-79.
    • (2010) Genetics in Medicine , vol.12 , pp. 668-679
    • Lidove, O.1    West, M.2    Pintos-Morell, G.3    Reisin, R.4    Nicholls, K.5    Figuera, L.E.6
  • 30
    • 66349107417 scopus 로고    scopus 로고
    • Evidence and values: requirements for public reimbursement of drugs for rare diseases - a case study in oncology
    • discussion e282-4. Epub 2009.
    • Drummond M, Evans B, LeLorier J, Karakiewicz P, Martin D, Tugwell P et al. Evidence and values: requirements for public reimbursement of drugs for rare diseases - a case study in oncology. Can J Clin Pharmacol 2009;16: e273-81; discussion e282-4. Epub 2009.
    • (2009) Can J Clin Pharmacol , vol.16
    • Drummond, M.1    Evans, B.2    LeLorier, J.3    Karakiewicz, P.4    Martin, D.5    Tugwell, P.6
  • 31
    • 0035816007 scopus 로고    scopus 로고
    • Enzyme replacement therapy in Fabry disease: a randomized controlled trial
    • Schiffmann R, Kopp JB, Austin HA, Sabnis S, Moore DF, Weibel T et al. Enzyme replacement therapy in Fabry disease: a randomized controlled trial. JAMA 2001;285:2743-9.
    • (2001) JAMA , vol.285 , pp. 2743-2749
    • Schiffmann, R.1    Kopp, J.B.2    Austin, H.A.3    Sabnis, S.4    Moore, D.F.5    Weibel, T.6
  • 32
    • 44449108760 scopus 로고    scopus 로고
    • Successful reinstitution of agalsidase beta therapy in Fabry disease patients with previous IgE-antibody or skin-test reactivity to the recombinant enzyme
    • Bodensteiner D, Scott RC, Sims KB, Shepherd GM, Cintron RD, Germain DP. Successful reinstitution of agalsidase beta therapy in Fabry disease patients with previous IgE-antibody or skin-test reactivity to the recombinant enzyme. Genet Med 2008;10:353-8.
    • (2008) Genet Med , vol.10 , pp. 353-358
    • Bodensteiner, D.1    Scott, R.C.2    Sims, K.B.3    Shepherd, G.M.4    Cintron, R.D.5    Germain, D.P.6
  • 33
    • 78649426034 scopus 로고    scopus 로고
    • Modelling the resource implications of managing adults with Fabry disease in Norway favours home infusion
    • Guest JF, Jenssen T, Houge G, Aaseboe W, Tøndel C, Svarstad E. Modelling the resource implications of managing adults with Fabry disease in Norway favours home infusion. Eur J Clin Invest 2010;40:1104-12.
    • (2010) Eur J Clin Invest , vol.40 , pp. 1104-1112
    • Guest, J.F.1    Jenssen, T.2    Houge, G.3    Aaseboe, W.4    Tøndel, C.5    Svarstad, E.6


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