Effect of 6 years of enzyme replacement therapy on plasma and urine glycosaminoglycans in attenuated MPS i patients

Glycobiology

Volumn 20, Issue 10, 2010, Pages 1259-1273

  Coppa, Giovanni V ^a   Buzzega, Dania ^b   Zampini, Lucia ^a   MacCari, Francesca ^b   Galeazzi, Tiziana ^a   Pederzoli, Francesca ^b   Gabrielli, Orazio ^a   Volpi, Nicola ^b  

^a UNIVERSITÀ POLITECNICA DELLE MARCHE   (Italy)

^b UNIVERSITY OF MODENA AND REGGIO EMILIA   (Italy)

Author keywords

dermatan sulfate; enzyme replacement therapy; glycosaminoglycans; heparan sulfate; mucopolysaccharidoses

Indexed keywords

CHONDROITIN SULFATE; DERMATAN SULFATE; DISACCHARIDE; GLYCOSAMINOGLYCAN; HEPARAN SULFATE; IDURONIC ACID; LEVO IDURONIDASE;

ARTICLE; BLOOD SAMPLING; CASE REPORT; ENZYME REPLACEMENT; HUMAN; HURLER SYNDROME; MOLECULAR WEIGHT; PRIORITY JOURNAL; PROTEIN BLOOD LEVEL; PROTEIN URINE LEVEL; SCHEIE SYNDROME; URINALYSIS;

BIOLOGICAL MARKERS; CASE-CONTROL STUDIES; DISACCHARIDES; ENZYME REPLACEMENT THERAPY; GLYCOSAMINOGLYCANS; HUMANS; MUCOPOLYSACCHARIDOSIS I;

EID: 77956502514     PISSN: 09596658     EISSN: 14602423     Source Type: Journal    
DOI: 10.1093/glycob/cwq088     Document Type: Article

References (35)

1. Andrade F, Prieto JA, Elorz J, Martín S, Sanjurjo P, Aldámiz-Echevarría L. 2008. Stability of urinary glycosaminoglycans in patients with mucopoly-saccharidoses. Clin Chim Acta. 388:73-77.
2. Burlingame RW, Thomas GH, Stevens RL, Schmid K, Moser HW. 1981. Direct quantitation of glycosaminoglycans in 2 mL of urine from patients with mucopolysaccharidoses. Clin Chem. 27:124-128.
3. Buzzega D, Pederzoli F, Maccari F, Aslan D, Türk M, Volpi N. 2010. Comparison of CPC and CETAB extractive procedures for quantification and characterization of human urinary glycosaminoglycans. Clin Chem Lab Med. [Epub Ahead of Print] DOI: 10.1515/CCLM.2010.217.
4. Chi L, Wolff JJ, Laremore TN, Restaino OF, Xie J, Schiraldi C, Toida T, Amster IJ, Linhardt RJ. 2008. Structural analysis of bikunin glycosamino-glycan. J Am Chem Soc. 130:2617-2625.
5. Conrad HE. 2001. Nitrous acid degradation of glycosaminoglycans. Curr Protoc Mol Biol. Chapter 17:Unit 17.22A.
6. Coppa GV, Catassi C, Gabrielli O, Giorgi PL, Dall'Amico R, Naia S, Panin G, Chiandetti L. 1987. Clinical application of a new simple method for the identification of mucopolysaccharidoses. Helv Paediatr Acta. 42:419-423.
7. de Lima CR, Baccarin RY, Michelacci YM. 2007. Reliability of 1, 9-di-methylmethylene blue tests in comparison to agarose gel electrophoresis for quantification of urinary glycosaminoglycans. Clin Chim Acta. 378:206-215.
8. Dietrich CP, Martins JR, Sampaio LO, Nader HB. 1993. Anomalous structure of urinary chondroitin sulfate from cancer patients A potential new marker for diagnosis of neoplasias. Lab Invest. 68:439-445.
9. Edens RE, al-Hakim A, Weiler JM, Rethwisch DG, Fareed J, Linhardt RJ. 1982. Gradient polyacrylamide gel electrophoresis for determination of molecular weights of heparin preparations and low-molecular-weight heparin derivatives. JPharm Sci. 81:823-827.
10. Fuller M, Rozaklis T, Ramsay SL, Hopwood JJ, Meikle PJ. 2004. Disease-specific markers for the mucopolysaccharidoses. Pediatr Res. 56:733-738.
11. Fuller M, Meikle PJ, Hopwood JJ. 2004. Glycosaminoglycan degradation fragments in mucopolysaccharidosis I. Glycobiology. 14:443-450.
12. Gabrielli O, Clarke LA, Bruni S, Coppa GV. 2010. Enzyme-replacement therapy in a 5-month-old boy with attenuated presymptomatic MPS I: 5-year follow-up. Pediatrics. 125:183-187.
13. Guimarães MA, Mourão PA. 1997. Urinary excretion of sulfated polysaccharides administered to Wistar rats suggests a renal permselectivity to these polymers based on molecular size. Biochim Biophys Acta. 1335:161-172.
14. Juvani M, Friman C, Ranta H, Wegelius O.1975. Isolation and characterization of undersulphated chondroitin-4-sulphate from normal human plasma. Biochim Biophys Acta. 411:1-10.
15. Maccari F, Gheduzzi D, Volpi N. 2003. Anomalous structure of urinary glycosaminoglycans in patients with pseudoxanthoma elasticum. Clin Chem. 49:380-388.
16. Maroclo MV, Pereira SD, Sampaio FJ, Cardoso LE. 2005. Urinary glycosaminoglycan excretion during the menstrual cycle in normal young women. J Urol. 173:1789-1792.
17. Martins AM, Dualibi AP, Norato D, Takata ET, Santos ES, Valadares ER, Porta G, de Luca G, Moreira G, Pimentel H, et al. 2009. Guidelines for the management of mucopolysaccharidosis type I. J Pediatr. 155(4 Suppl): S32-46.
18. Mason KE, Meikle PJ, Hopwood JJ, Fuller M. 2006. Characterization of sulfated oligosaccharides in mucopolysaccharidosis type IIIA by electro-spray ionization mass spectrometry. Anal Chem. 78:4534-4542.
19. Masuda H, Takakura Y, Hashida M. 1999. Pharmacokinetics and disposition characteristics of recombinant decorin after intravenous injection into mice. Biochim Biophys Acta. 1426:420-428.
20. Meikle PJ, Hopwood JJ, Clague AE, Carey WF. 1999. Prevalence of lysosomal storage disorders. JAMA. 281:249-254.
21. Oguma T, Tomatsu S, Montano AM, Okazaki O. 2007. Analytical method for the determination of disaccharides derived from keratan, heparan, and der-matan sulfates in human serum and plasma by high-performance liquid chromatography/turbo ionspray ionization tandem mass spectrometry. Anal Biochem. 368:79-86.
22. Pecly IM, Melo-Filho NM, Mourão PA. 2006. Effects of molecular size and chemical structure on renal and hepatic removal of exogenously administered chondroitin sulfate in rats. Biochim Biophys Acta. 1760:865-876.
23. Randall DR, Colobong KE, Hemmelgarn H, Sinclair GB, Hetty E, Thomas A, Bodamer OA, Volkmar B, Fernhoff PM, Casey R, et al. 2008. Heparin co-factor II-thrombin complex: A biomarker of MPS disease. Mol Genet Metab. 94:456-461.
24. Tomatsu S, Montaño AM, Oguma T, Dung VC, Oikawa H, de Carvalho TG, Gutiérrez ML, Yamaguchi S, Suzuki Y, Fukushi M, et al. 2010. Dermatan sulfate and heparan sulfate as a biomarker for mucopolysaccharidosis I. J Inherit Metab Dis. 33:141-150.
25. Tomatsu S, Montaño AM, Oguma T, Dung VC, Oikawa H, Gutiérrez ML, Yamaguchi S, Suzuki Y, Fukushi M, Barrera LA, et al. 2010. Validation of disaccharide compositions derived from dermatan sulfate and heparan sulfate in mucopolysaccharidoses and mucolipidoses II and III by tandem mass spectrometry. Mol Genet Metab. 99:124-131.
26. Volpi N. 1993. "Fast moving" and "slow moving" heparins, dermatan sulfate, and chondroitin sulfate: Qualitative and quantitative analysis by agarose-gel electrophoresis. Carbohydr Res. 247:263-278.
27. Volpi N. 2007. Analytical aspects of pharmaceutical grade chondroitin sulfates. J Pharm Sci. 96:3168-3180.
28. Volpi N. 2009. High-performance liquid chromatography and on-line mass spectrometry detection for the analysis of chondroitin sulfates/hyaluronan disaccharides derivatized with 2-aminoacridone. Anal Biochem. 397:12-23.
29. Volpi N, Bolognani L. 1993. Glycosaminoglycans and proteins: Different behaviours in high-performance size-exclusion chromatography. J Chromatogr. 630:390-396.
30. Volpi N, Maccari F. 2002. Detection of submicrogram quantities of glycosaminoglycans on agarose-gels by sequential staining with toluidine blue and Stains-All. Electrophoresis. 23:4060-4066.
31. Volpi N, Maccari F. 2005. Microdetermination of chondroitin sulfate in normal human plasma by fluorophore-assisted carbohydrate electrophoresis (FACE). Clin Chim Acta. 356:125-133.
32. Volpi N, Maccari F. 2006. Electrophoretic approaches to the analysis of complex polysaccharides. J Chromatogr B Analyt Technol Biomed Life Sci. 834:1-13.
33. Volpi N, Maccari F. 2009. Structural characterization and antithrombin activity of dermatan sulfate purified from marine clam Scapharca inaequivalvis. Glycobiology. 19:356-367.
34. Wang RY, Cambray-Forker EJ, Ohanian K, Karlin DS, Covault KK, Schwartz PH, Abdenur JE. 2009. Treatment reduces or stabilizes brain imaging abnormalities in patients with MPS I and II. Mol Genet Metab. 98:406-411.
35. Whitley CB, Ridnour MD, Draper KA, Dutton CM, Neglia JP. 1989. Diagnostic test for mucopolysaccharidosis I. Direct method for quantifying excessive urinary glycosaminoglycan excretion. Clin Chem. 35:374-379.