메뉴 건너뛰기




Volumn 88, Issue 3, 2013, Pages 166-171

Enzyme replacement therapy with velaglucerase alfa in Gaucher disease: Results from a randomized, double-blind, multinational, Phase 3 study

Author keywords

[No Author keywords available]

Indexed keywords

CHITOTRIOSIDASE; DRUG ANTIBODY; GLUCOSYLCERAMIDASE; HEMOGLOBIN; IMMUNOGLOBULIN G; VELAGLUCERASE ALFA;

EID: 84874302849     PISSN: 03618609     EISSN: 10968652     Source Type: Journal    
DOI: 10.1002/ajh.23381     Document Type: Article
Times cited : (64)

References (22)
  • 1
    • 0000216808 scopus 로고    scopus 로고
    • Gaucher disease
    • In: Scriver CR, Beudet AL, Sly WS, Valle D, editors. New York: McGraw-Hill; . pp -.
    • Beutler E, Grabowski GA. Gaucher disease. In: Scriver CR, Beudet AL, Sly WS, Valle D, editors. The Metabolic and Molecular Bases of Inherited Disease. New York: McGraw-Hill; 2001. pp 3635-3668.
    • (2001) The Metabolic and Molecular Bases of Inherited Disease , pp. 3635-3668
    • Beutler, E.1    Grabowski, G.A.2
  • 2
    • 0031436478 scopus 로고    scopus 로고
    • Gaucher's disease: Clinical features and natural history
    • Cox TM, Schofield JP. Gaucher's disease: Clinical features and natural history. Baillieres Clin Haematol 1997;10:657-689.
    • (1997) Baillieres Clin Haematol , vol.10 , pp. 657-689
    • Cox, T.M.1    Schofield, J.P.2
  • 3
    • 0028883136 scopus 로고
    • Enzyme therapy in type 1 Gaucher disease: Comparative efficacy of mannose-terminated glucocerebrosidase from natural and recombinant sources
    • Grabowski GA, Barton NW, Pastores G, et al. Enzyme therapy in type 1 Gaucher disease: Comparative efficacy of mannose-terminated glucocerebrosidase from natural and recombinant sources. Ann Intern Med 1995;122:33-39.
    • (1995) Ann Intern Med , vol.122 , pp. 33-39
    • Grabowski, G.A.1    Barton, N.W.2    Pastores, G.3
  • 4
    • 0029029221 scopus 로고
    • Replacement therapy with imiglucerase for type 1 Gaucher's disease
    • Zimran A, Elstein D, Levy-Lahad E, et al. Replacement therapy with imiglucerase for type 1 Gaucher's disease. Lancet 1995;345:1479-1480.
    • (1995) Lancet , vol.345 , pp. 1479-1480
    • Zimran, A.1    Elstein, D.2    Levy-Lahad, E.3
  • 5
    • 70849126444 scopus 로고    scopus 로고
    • Characterization of gene-activated human acid-β-glucosidase: Crystal structure, glycan composition, and internalization into macrophages
    • Brumshtein B, Salinas P, Peterson B, et al. Characterization of gene-activated human acid-β-glucosidase: Crystal structure, glycan composition, and internalization into macrophages. Glycobiology 2010;20:24-32.
    • (2010) Glycobiology , vol.20 , pp. 24-32
    • Brumshtein, B.1    Salinas, P.2    Peterson, B.3
  • 6
    • 77954693904 scopus 로고    scopus 로고
    • Phase 1/2 and extension study of velaglucerase alfa replacement therapy in adults with type 1 Gaucher disease: 48-month experience
    • Zimran A, Altarescu G, Philips M, et al. Phase 1/2 and extension study of velaglucerase alfa replacement therapy in adults with type 1 Gaucher disease: 48-month experience. Blood 2010;115:4651-4656.
    • (2010) Blood , vol.115 , pp. 4651-4656
    • Zimran, A.1    Altarescu, G.2    Philips, M.3
  • 7
    • 17044455396 scopus 로고    scopus 로고
    • Transglycosidase activity of chitotriosidase: Improved enzymatic assay for the human macrophage chitinase
    • Aguilera B, Ghauharali-van der Vlugt K, Helmond MT, et al. Transglycosidase activity of chitotriosidase: Improved enzymatic assay for the human macrophage chitinase. J Biol Chem 2003;278:40911-40916.
    • (2003) J Biol Chem , vol.278 , pp. 40911-40916
    • Aguilera, B.1    Ghauharali-van der Vlugt, K.2    Helmond, M.T.3
  • 8
    • 34248153885 scopus 로고    scopus 로고
    • Monitoring of Gaucher patients with a novel chitotriosidase assay
    • Schoonhoven A, Rudensky B, Elstein D, et al. Monitoring of Gaucher patients with a novel chitotriosidase assay. Clin Chim Acta 2007;381:136-139.
    • (2007) Clin Chim Acta , vol.381 , pp. 136-139
    • Schoonhoven, A.1    Rudensky, B.2    Elstein, D.3
  • 9
    • 9144222696 scopus 로고    scopus 로고
    • Marked elevation of the chemokine CCL18/PARC in Gaucher disease: A novel surrogate marker for assessing therapeutic intervention
    • Boot RG, Verhoek M, de Fost M, et al. Marked elevation of the chemokine CCL18/PARC in Gaucher disease: A novel surrogate marker for assessing therapeutic intervention. Blood 2004;103:33-39.
    • (2004) Blood , vol.103 , pp. 33-39
    • Boot, R.G.1    Verhoek, M.2    de Fost, M.3
  • 10
    • 80054834248 scopus 로고    scopus 로고
    • Development of a panel of highly sensitive, equivalent assays for detection of antibody responses to velaglucerase alfa or imiglucerase enzyme replacement therapy in patients with Gaucher disease
    • Séllos-Moura M, Barzegar S, Pan L, et al. Development of a panel of highly sensitive, equivalent assays for detection of antibody responses to velaglucerase alfa or imiglucerase enzyme replacement therapy in patients with Gaucher disease. J Immunol Methods 2011;373:45-53.
    • (2011) J Immunol Methods , vol.373 , pp. 45-53
    • Séllos-Moura, M.1    Barzegar, S.2    Pan, L.3
  • 11
    • 0016799829 scopus 로고
    • Sequential treatment assignment with balancing for prognostic factors in the controlled clinical trial
    • Pocock SJ, Simon R. Sequential treatment assignment with balancing for prognostic factors in the controlled clinical trial. Biometrics 1975;31:103-115.
    • (1975) Biometrics , vol.31 , pp. 103-115
    • Pocock, S.J.1    Simon, R.2
  • 12
    • 0002294347 scopus 로고
    • A simple sequentially rejective multiple test procedure
    • Holm S. A simple sequentially rejective multiple test procedure. Scand J Statist 1979;6:65-70.
    • (1979) Scand J Statist , vol.6 , pp. 65-70
    • Holm, S.1
  • 13
    • 33748541288 scopus 로고    scopus 로고
    • The biology of the Gaucher cell: The cradle of human chitinases
    • Bussink AP, van Eijk M, Renkema GH, et al. The biology of the Gaucher cell: The cradle of human chitinases. Int Rev Cytol 2006;252:71-128.
    • (2006) Int Rev Cytol , vol.252 , pp. 71-128
    • Bussink, A.P.1    van Eijk, M.2    Renkema, G.H.3
  • 14
    • 33745722913 scopus 로고    scopus 로고
    • Superior effects of high-dose enzyme replacement therapy in type 1 Gaucher disease on bone marrow involvement and chitotriosidase levels: A 2-center retrospective analysis
    • de Fost M, Hollak CE, Groener JE, et al. Superior effects of high-dose enzyme replacement therapy in type 1 Gaucher disease on bone marrow involvement and chitotriosidase levels: A 2-center retrospective analysis. Blood 2006;108:830-835.
    • (2006) Blood , vol.108 , pp. 830-835
    • de Fost, M.1    Hollak, C.E.2    Groener, J.E.3
  • 16
    • 34248504877 scopus 로고    scopus 로고
    • ® human glucocerebrosidase (GA-GCB) in patients with type 1 Gaucher disease
    • ® human glucocerebrosidase (GA-GCB) in patients with type 1 Gaucher disease. Blood Cells Mol Dis 2007;39:115-118.
    • (2007) Blood Cells Mol Dis , vol.39 , pp. 115-118
    • Zimran, A.1    Loveday, K.2    Fratazzi, C.3
  • 17
    • 33846197993 scopus 로고    scopus 로고
    • The long-term international safety experience of imiglucerase therapy for Gaucher disease
    • Starzyk K, Richards S, Yee J, et al. The long-term international safety experience of imiglucerase therapy for Gaucher disease. Mol Genet Metab 2007;90:157-163.
    • (2007) Mol Genet Metab , vol.90 , pp. 157-163
    • Starzyk, K.1    Richards, S.2    Yee, J.3
  • 18
    • 0036155174 scopus 로고    scopus 로고
    • Gaucher and Niemann-Pick diseases-enzymatic diagnosis in dried blood spots on filter paper: Retrospective diagnoses in newborn-screening cards
    • Chamoles NA, Blanco M, Gaggioli D, et al. Gaucher and Niemann-Pick diseases-enzymatic diagnosis in dried blood spots on filter paper: Retrospective diagnoses in newborn-screening cards. Clin Chim Acta 2002;317:191-197.
    • (2002) Clin Chim Acta , vol.317 , pp. 191-197
    • Chamoles, N.A.1    Blanco, M.2    Gaggioli, D.3
  • 19
    • 33645458448 scopus 로고    scopus 로고
    • Detection of mucopolysaccharidosis type II by measurement of iduronate-2-sulfatase in dried blood spots and plasma samples
    • Dean CJ, Bockmann MR, Hopwood JJ, et al. Detection of mucopolysaccharidosis type II by measurement of iduronate-2-sulfatase in dried blood spots and plasma samples. Clin Chem 2006;52:643-649.
    • (2006) Clin Chem , vol.52 , pp. 643-649
    • Dean, C.J.1    Bockmann, M.R.2    Hopwood, J.J.3
  • 20
    • 77649338367 scopus 로고    scopus 로고
    • Genetic heterozygosity and pseudodeficiency in the Pompe disease newborn screening pilot program
    • Labrousse P, Chien YH, Pomponio RJ, et al. Genetic heterozygosity and pseudodeficiency in the Pompe disease newborn screening pilot program. Mol Genet Metab 2010;99:379-383.
    • (2010) Mol Genet Metab , vol.99 , pp. 379-383
    • Labrousse, P.1    Chien, Y.H.2    Pomponio, R.J.3
  • 21
    • 56749106016 scopus 로고    scopus 로고
    • Two-tier approach for the detection of alpha-galactosidase A deficiency in kidney transplant recipients
    • De Schoenmakere G, Poppe B, Wuyts B, et al. Two-tier approach for the detection of alpha-galactosidase A deficiency in kidney transplant recipients. Nephrol Dial Transplant 2008;23:4044-4048.
    • (2008) Nephrol Dial Transplant , vol.23 , pp. 4044-4048
    • De Schoenmakere, G.1    Poppe, B.2    Wuyts, B.3
  • 22
    • 78049511066 scopus 로고    scopus 로고
    • Early achievement and maintenance of the therapeutic goals using velaglucerase alfa in type 1 Gaucher disease
    • Elstein D, Cohn GM, Wang N, et al. Early achievement and maintenance of the therapeutic goals using velaglucerase alfa in type 1 Gaucher disease. Blood Cells Mol Dis 2011;46:119-123.
    • (2011) Blood Cells Mol Dis , vol.46 , pp. 119-123
    • Elstein, D.1    Cohn, G.M.2    Wang, N.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.