Longitudinal observations of serum heparin cofactor II-thrombin complex in treated Mucopolysaccharidosis i and II patients

Journal of Inherited Metabolic Disease

Volumn 35, Issue 2, 2012, Pages 355-362

  Clarke, Lorne Andrew ^a   Hemmelgarn, Harmony ^a   Colobong, Karen ^a   Thomas, Anita ^a   Stockler, Sylvia ^b   Casey, Robin ^c   Chan, Alicia ^d   Fernoff, Paul ^e   Mitchell, John ^f  

^a UNIVERSITY OF BRITISH COLUMBIA   (Canada)

^b UNIVERSITY OF BRITISH COLUMBIA   (Canada)

^c UNIVERSITY OF CALGARY   (Canada)

^d UNIVERSITY OF ALBERTA   (Canada)

^e EMORY UNIVERSITY SCHOOL OF MEDICINE   (United States)

^f MCGILL UNIVERSITY   (Canada)

Author keywords

[No Author keywords available]

Indexed keywords

ENZYME ANTIBODY; GLYCOSAMINOGLYCAN; HEPARIN COFACTOR II; IDURONATE 2 SULFATASE; LARONIDASE;

ADOLESCENT; ANTIBODY DETECTION; ARTICLE; CHILD; CLINICAL ARTICLE; CONTROLLED STUDY; DISEASE SEVERITY; ENZYME BLOOD LEVEL; ENZYME LINKED IMMUNOSORBENT ASSAY; ENZYME REPLACEMENT; FEMALE; GLYCOSAMINOGLYCAN URINE LEVEL; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; HUNTER SYNDROME; HURLER SYNDROME; INFANT; LONGITUDINAL STUDY; MALE; PRESCHOOL CHILD; RADIOIMMUNOPRECIPITATION; SCHOOL CHILD; TREATMENT DURATION; TREATMENT OUTCOME; URINE LEVEL;

ADOLESCENT; ADULT; BIOLOGICAL MARKERS; CHILD; CHILD, PRESCHOOL; GLYCOSAMINOGLYCANS; HEPARIN COFACTOR II; HUMANS; IDURONATE SULFATASE; INFANT; LONGITUDINAL STUDIES; MUCOPOLYSACCHARIDOSIS I; MUCOPOLYSACCHARIDOSIS II; THROMBIN;

EID: 84863493157     PISSN: 01418955     EISSN: 15732665     Source Type: Journal    
DOI: 10.1007/s10545-011-9369-6     Document Type: Article

References (18)

1. Andersson HC, Charrow J, Kaplan P et al. (2005) Individualization of long-term enzyme replacement therapy for Gaucher disease. Genet Med 7:105-110
2. Clarke LA (2008) The mucopolysaccharidoses: a success ofmolecular medicine. Expert Rev Mol Med 10:e1
3. Clarke LA, Wraith JE, Beck M et al. (2009) Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I. Pediatrics 123:229-240
4. de Jong JG, Heijs WM, Wevers RA et al. (1994) Mucopolysacchar- idoses screening: dimethylmethylene blue versus alcian blue. Ann Clin Biochem 31:267-271
5. Giugliani R, Rojas VM, Martins AM et al. (2008) A dose-optimization trial of laronidase (Aldurazyme) in patients with mucopolysaccharidosis I. Mol Genet Metab 96:13-19
6. Huntington JA (2003) Mechanisms of glycosaminoglycan activation of the serpins in hemostasis. J Thromb Haemost 1:1535-1549
7. Langford-Smith K, Arasaradnam M, Wraith JE, Wynn R, Bigger BW (2010) Evaluation of heparin cofactor II-thrombin complex as a biomarker on blood spots from mucopolysaccharidosis I, IIIA and IIIB mice. Mol Genet Metab 99:269-274
8. Langford-Smith KJ, Mercer J, Petty J et al. (2011) Heparin cofactor II-thrombin complex and dermatan sulphate:chondroitin sul-phate ratio are biomarkers of short- and long-term treatment effects in mucopolysaccharide diseases. J Inherit Metab Dis 34:499-508
9. Mistry P, Germain DP (2006) Therapeutic goals in Gaucher disease. Rev Med Interne 27(Suppl 1):S30-S38
10. Mistry PK, Cappellini MD, Lukina E et al. (2011) A reappraisal of Gaucher disease-diagnosis and disease management algorithms. Am J Hematol 86:110-115
11. Muenzer J, Wraith JE, Beck M et al. (2006) A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysacchar-idosis II (Hunter syndrome). Genet Med 8:465-473
12. Muenzer J, Gucsavas-Calikoglu M, McCandless SE, Schuetz TJ, Kimura A (2007) A phase I/II clinical trial of enzyme replacement therapy in mucopolysaccharidosis II (Hunter syndrome). Mol Genet Metab 90:329-337
13. Muenzer J, Beck M, Eng CM et al. (2011) Long-term, open-labeled extension study ofidursulfase in the treatment ofHunter syndrome. GenetMed 13:95-101
14. Pike RN, Buckle AM, Le Bonniec BF, Church FC et al. (2005) Control of the coagulation system by serpins. Getting by with a little help from glycosaminoglycans. FEBS J 272:4842-4851
15. Prasad VK, Kurtzberg J (2010) Transplant outcomes in mucopoly- saccharidoses. Semin Hematol 47:59-69
16. Randall DR, Sinclair GB, Colobong KE, Hetty E, Clarke LA (2006) Heparin cofactor II-thrombin complex in MPS I: a biomarker of MPS disease. Mol Genet Metab 88:235-243
17. Randall DR, Colobong KE, Hemmelgarn H et al (2008) Heparin cofactor II-thrombin complex: a biomarker of MPS disease. Mol Genet Metab 94:456-461
18. Wraith JE, Clarke LA, Beck M et al. (2004) Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase). J Pediatr 144:581-588