Different dose-dependent correction of MIP-1β and chitotriosidase during initial enzyme replacement therapy

Journal of Inherited Metabolic Disease

Volumn 32, Issue 2, 2009, Pages 274-279

  Breemen, M J ^a   Fost, M ^b   Maas, M ^b   Wiersma, M G ^b   Hollak, C E M ^b   Poll, L W ^c   Dahl, S ^d   Boot, R G ^a   Aerts, J M F G ^a  

^a UNIVERSITY OF AMSTERDAM   (Netherlands)

^b ACADEMIC MEDICAL CENTER   (Netherlands)

^c UNIVERSITY OF DUISBURG ESSEN   (Germany)

^d HEINRICH HEINE UNIVERSITY   (Germany)

Author keywords

[No Author keywords available]

Indexed keywords

CHITOTRIOSIDASE; GLUCOSYLCERAMIDASE; MACROPHAGE INFLAMMATORY PROTEIN 1BETA; MANNOSE RECEPTOR;

ADULT; AGED; ARTICLE; CLINICAL ARTICLE; CONTROLLED STUDY; DOSE TIME EFFECT RELATION; DRUG DOSE COMPARISON; DRUG DOSE REDUCTION; DRUG EFFICACY; ENZYME REPLACEMENT; FEMALE; GAUCHER DISEASE; HUMAN; MALE; PHAGOCYTE; PROTEIN BLOOD LEVEL; PROTEIN EXPRESSION; TREATMENT DURATION;

ADOLESCENT; AGED; CHEMOKINE CCL4; DOSE-RESPONSE RELATIONSHIP, DRUG; FEMALE; GAUCHER DISEASE; GLUCOSYLCERAMIDASE; HEXOSAMINIDASES; HUMANS; MALE; MIDDLE AGED; SPLENECTOMY;

EID: 64449083217     PISSN: 01418955     EISSN: 15732665     Source Type: Journal    
DOI: 10.1007/s10545-009-1064-5     Document Type: Article

References (18)

1. Aerts JM, Hollak CE (1997) Plasma and metabolic abnormalities in Gaucher's disease. Baillières Clin Haematol 10(4): 691-709. doi: 10.1016/S0950-3536(97)80034-0.
2. Aerts JM, van Breemen MJ, Bussink AP, et al (2008) Biomarkers for lysosomal storage disorders: Identification and application as exemplified by chitotriosidase in Gaucher disease. Acta Paediatr Supplement 97(s457): 7-14. doi: 10.1111/j.1651-2227.2007.00641.x.
3. Barton NW, Brady RO, Dambrosia JM, et al (1991) Replacement therapy for inherited enzyme deficiency - macrophage-targeted glucocerebrosidase for Gaucher's disease. N Engl J Med 324(21): 1464-1470.
4. Beutler E, Grabowski GA (1995) Gaucher disease. In: Scriver CR, Beaudet AL, Sly WS, Valle D, eds; Childs B, Kinzler KW, Vogelstein B, assoc. eds. The Metabolic and Molecular Bases of Inherited Disease, 8th edn. New York: McGraw-Hill, 2641-2670.
5. Boot RG, Hollak CE, Verhoek M, et al (1997) Glucocerebrosidase genotype of Gaucher patients in the Netherlands: Limitations in prognostic value. Hum Mutat 10(5): 348-358. doi: 10.1002/ (SICI)1098-1004(1997)10:5<348::AID-HUMU3>3.0.CO;2-B.
6. Boot RG, Renkema GH, Verhoek M, et al (1998) The human chitotriosidase gene: Nature of inherited enzyme deficiency. J Biol Chem 273(40): 25680-25685. doi: 10.1074/jbc.273.40.25680.
7. Boot RG, Verhoek M, de Fost M, et al (2004) Marked elevation of the chemokine CCL18/PARC in Gaucher disease: A novel surrogate marker for assessing therapeutic intervention. Blood 103(1): 33-39. doi: 10.1182/ blood-2003-05-1612.
8. Boven LA, van Meurs M, Boot RG, et al (2004) Gaucher cells demonstrate a distinct macrophage phenotype and resemble alternatively activated macrophages. Am J Clin Pathol 122(3): 359-369. doi: 10.1309/ BG5VA8JRDQH1M7HN.
9. de Fost M, Hollak CE, Groener JE, et al (2006) 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 108(3): 830-835. doi: 10.1182/blood-2005-12-5072.
10. Grabowski GA, Barton NW, Pastores G, et al (1995) Enzyme therapy in type I Gaucher disease: Comparative efficacy of mannose-terminated glucocerebrosidase from natural and recombinant sources. Ann Intern Med 122(1): 33-39.
11. Hollak CE, van Weely S, van Oers MH, Aerts JM (1994) Marked elevation of plasma chitotriosidase activity: A novel hallmark of Gaucher disease. J Clin Invest 93(3): 1288-1292. doi: 10.1172/JCI117084.
12. Hollak CE, Aerts JM, Goudsmit R, et al (1995) Individualised low-dose alglucerase therapy for type 1 Gaucher's disease. Lancet 345(8963): 1474-1478. doi: 10.1016/S0140-6736(95)91037-9.
13. Hollak CE, Evers L, Aerts JM, van Oers MH (1997) Elevated levels of M-CSF, sCD14 and IL8 in type 1 Gaucher disease. Blood Cells Mol Dis 23(2): 201-212. doi: 10.1006/bcmd.1997.0137.
14. Michelakakis H, Spanou C, Kondyli A, et al (1996) Plasma tumor necrosis factor-a (TNF-a) levels in Gaucher disease. Biochim Biophys Acta 1317(3): 219-222.
15. Mistry PK, Wraight EP, Cox TM (1996) Therapeutic delivery of proteins to macrophages: Implications for treatment of Gaucher's disease. Lancet 348(9041): 1555-1559. doi: 10.1016/S0140-6736(96)04451-0.
16. Schoonhoven A, Rudensky B, Elstein D, et al (2007) Monitoring of Gaucher patients with a novel chitotriosidase assay. Clin Chim Acta 381(2): 136-139. doi: 10.1016/j.cca.2007.02.042.
17. van Breemen MJ, de Fost M, Voerman JS, et al (2007) Increased plasma macrophage inflammatory protein (MIP)-1α and MIP-1β levels in type 1 Gaucher disease. Biochim Biophys Acta 1772(7): 788-796.
18. van Weely S, van Leeuwen MB, Jansen ID, et al (1991) Clinical phenotype of Gaucher disease in relation to properties of mutant glucocerebrosidase in cultured fibroblasts. Biochim Biophys Acta 1096(4): 301-311.