The uptake of recombinant glucocerebrosidases by blood monocytes from type 1 Gaucher disease patients is variable

British Journal of Haematology

Volumn 157, Issue 2, 2012, Pages 274-277

  Berger, Juliette ^a   Stirnemann, Jérôme ^b   Bourgne, Céline ^a   Pereira, Bruno ^c   Pigeon, Pascale ^a   Heraoui, Djazia ^d   Froissart, Roseline ^e   Rapatel, Chantal ^a   Rose, Christian ^f   Belmatoug, Nadia ^d   Berger, Marc G ^a  

^a UNIVERSITY HOSPITAL OF CLERMONT FERRAND   (France)

^b HÔPITAL JEAN VERDIER   (France)

^c SERVICE DE RHUMATOLOGIE   (France)

^d BEAUJON HOSPITAL   (France)

^e Centre de Biologie Est   (France)

^f HÔPITAL SAINT VINCENT DE PAUL   (France)

Author keywords

Enzyme replacement therapy; Gaucher disease; Monocyte

Indexed keywords

GLUCOSYLCERAMIDASE; IMIGLUCERASE; MANNOSE RECEPTOR; PROTALIX; TALIGLUCERASE ALFA; UNCLASSIFIED DRUG; VELAGLUCERASE ALFA;

ADULT; ASTHENIA; BONE DISEASE; CLINICAL ARTICLE; CONCENTRATION RESPONSE; CONTROLLED STUDY; DRUG DOSE COMPARISON; DRUG EXPOSURE; DRUG MECHANISM; DRUG UPTAKE; ENZYME ACTIVITY; ENZYME DEFICIENCY; ENZYME REPLACEMENT; ENZYME SYNTHESIS; FEMALE; FLOW CYTOMETRY; GAUCHER DISEASE; GAUCHER DISEASE TYPE 1; HUMAN; HUMAN CELL; IN VITRO STUDY; IN VIVO STUDY; INCUBATION TIME; LETTER; MALE; MONOCYTE; MONONUCLEAR CELL; POLYMORPHONUCLEAR CELL; PRIORITY JOURNAL; PROTEIN EXPRESSION; THROMBOCYTOPENIA;

ENZYME REPLACEMENT THERAPY; FEMALE; GAUCHER DISEASE; GLUCOSYLCERAMIDASE; HUMANS; MALE; MONOCYTES;

EID: 84859214491     PISSN: 00071048     EISSN: 13652141     Source Type: Journal    
DOI: 10.1111/j.1365-2141.2011.08989.x     Document Type: Letter

References (10)

1. Berger, J., Lecourt, S., Vanneaux, V., Rapatel, C., Boisgard, S., Caillaud, C., Boiret-Dupre, N., Chomienne, C., Marolleau, J.P., Larghero, J. & Berger, M.G. (2010) Glucocerebrosidase deficiency dramatically impairs human bone marrow haematopoiesis in an in vitro model of Gaucher disease. British Journal of Haematology, 150, 93-101.
2. Boot, R.G., Verhoek, M., de Fost, M., Hollak, C.E., Maas, M., Bleijlevens, B., van Breemen, M.J., van Meurs, M., Boven, L.A., Laman, J.D., Moran, M.T., Cox, T.M. & Aerts, J.M. (2004) Marked elevation of the chemokine CCL18/PARC in Gaucher disease: a novel surrogate marker for assessing therapeutic intervention. Blood, 103, 33-39.
3. Brumshtein, B., Salinas, P., Peterson, B., Chan, V., Silman, I., Sussman, J.L., Savickas, P.J., Robinson, G.S. & Futerman, A.H. (2010) Characterization of gene-activated human acid-beta-glucosidase: crystal structure, glycan composition, and internalization into macrophages. Glycobiology, 20, 24-32.
4. Edmunds, T. (1998) Cerezyme: a case study. Developments in Biological Standardization, 96, 131-140.
5. Friedman, B., Vaddi, K., Preston, C., Mahon, E., Cataldo, J.R. & McPherson, J.M. (1999) A comparison of the pharmacological properties of carbohydrate remodeled recombinant and placental-derived beta-glucocerebrosidase: implications for clinical efficacy in treatment of Gaucher disease. Blood, 93, 2807-2816.
6. Shaaltiel, Y., Bartfeld, D., Hashmueli, S., Baum, G., Brill-Almon, E., Galili, G., Dym, O., Boldin-Adamsky, S.A., Silman, I., Sussman, J.L., Futerman, A.H. & Aviezer, D. (2007) Production of glucocerebrosidase with terminal mannose glycans for enzyme replacement therapy of Gaucher's disease using a plant cell system. Plant Biotechnology Journal, 5, 579-590.
7. Stirnemann, J., Boutten, A., Vincent, C., Mekinian, A., Heraoui, D., Fantin, B., Fain, O., Mentre, F. & Belmatoug, N. (2011) Impact of imiglucerase on the serum glycosylated-ferritin level in Gaucher disease. Blood Cells, Molecules, and Diseases, 46, 34-38.
8. Van Patten, S.M., Hughes, H., Huff, M.R., Piepenhagen, P.A., Waire, J., Qiu, H., Ganesa, C., Reczek, D., Ward, P.V., Kutzko, J.P. & Edmunds, T. (2007) Effect of mannose chain length on targeting of glucocerebrosidase for enzyme replacement therapy of Gaucher disease. Glycobiology, 17, 467-478.
9. Weinreb, N.J., Charrow, J., Andersson, H.C., Kaplan, P., Kolodny, E.H., Mistry, P., Pastores, G., Rosenbloom, B.E., Scott, C.R., Wappner, R.S. & Zimran, A. (2002) Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2-5years of treatment: a report from the Gaucher Registry. American Journal of Medicine, 113, 112-119.
10. Zimran, A., Loveday, K., Fratazzi, C. & Elstein, D. (2007) A pharmacokinetic analysis of a novel enzyme replacement therapy with Gene-Activated human glucocerebrosidase (GA-GCB) in patients with type 1 Gaucher disease. Blood Cells, Molecules, and Diseases, 39, 115-118.