Enzyme replacement therapy for the management of the mucopolysaccharidoses

International Journal of Clinical Pharmacology and Therapeutics

Volumn 47, Issue SUPPL. 1, 2009, Pages

  Wraith, J E ^a  

^a ROYAL MANCHESTER CHILDREN S HOSPITAL   (United Kingdom)

Author keywords

Enzyme replacement therapy; Hematopoietic stem cell therapy; Mucopolysaccharidosis disorders; Palliative care; Surgery

Indexed keywords

GALSULFASE; IDURONATE 2 SULFATASE; LEVO IDURONIDASE; N ACETYLGALACTOSAMINE 4 SULFATASE; RECOMBINANT PROTEIN;

ENZYME REPLACEMENT; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; HUNTER SYNDROME; HURLER SYNDROME; MAROTEAUX LAMY SYNDROME; METHODOLOGY; MULTIMODALITY CANCER THERAPY; REVIEW;

COMBINED MODALITY THERAPY; ENZYME REPLACEMENT THERAPY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; IDURONATE SULFATASE; IDURONIDASE; MUCOPOLYSACCHARIDOSIS I; MUCOPOLYSACCHARIDOSIS II; MUCOPOLYSACCHARIDOSIS VI; N-ACETYLGALACTOSAMINE-4-SULFATASE; RECOMBINANT PROTEINS;

EID: 77649212017     PISSN: 09461965     EISSN: None     Source Type: Journal    
DOI: 10.5414/cpp47063     Document Type: Conference Paper

References (23)

1. Boelens JJ. Trends in hematopoietic cell transplantation for inborn errors of metabolism. J Inherit Metab Dis. 2006; 29: 413-420. (Pubitemid 43880644)
2. Boelens JJ, Wynn RF, O'Meara A, Veys P, Bertrand Y, Souillet G et al. Outcomes of hematopoietic stem cell transplantation for Hurler's syndrome in Europe: a risk factor analysis for graft failure. Bone Marrow Transplant. 2007; 40: 225-233. (Pubitemid 47108465)
3. Clarke LA, Wraith JE, Beck M, Kolodny EH, Pastores GM, Muenzer J et al. Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I. Pediatrics. 2009; 123: 229-240.
4. Cox-Brinkman J, Boelens JJ, Wraith JE, O'Meara A, Veys P, Wijburg FA et al. Haematopoietic cell transplantation (HCT) in combination with enzyme replacement therapy (ERT) in patients with Hurler syndrome. Bone Marrow Transplant. 2006; 38: 17-21. (Pubitemid 43923084)
5. Giugliani R, Harmatz P, Wraith JE. Management guidelines for mucopolysaccharidosis VI. Pediatrics. 2007; 120: 405-418. (Pubitemid 47219512)
6. Grewal SS, Wynn R, Abdenur JE, Burton BK, Gharib M, Haase C et al. Safety and efficacy of enzyme replacement therapy in combination with hematopoietic stem cell transplantation in Hurler syndrome. Genet Med. 2005; 7: 143-146. (Pubitemid 40261841)
7. Harmatz P, Whitley CB, Waber L, Pais R, Steiner R, Plecko B et al. Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome). J Pediatr. 2004; 144: 574-580. (Pubitemid 38594756)
8. Harmatz P, Ketteridge D, Giugliani R, Guffon N, Leão Teles E, Miranda MC et al. Direct comparison of measures of endurance, mobility, and joint function during enzyme-replacement therapy of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): results after 48 weeks in a Phase 2 open-label clinical study of recombinant human N-acetylgalactosamine 4-sulfatase. Pediatrics. 2005; 115: e681-e689. (Pubitemid 43843141)
9. Harmatz P, Giugliani R, Schwartz I, Guffon N, Leão Teles E, Miranda MC et al. Enzyme replacement therapy for mucopolysaccharidosis VI: a Phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension study. J Pediatr. 2006; 148: e533-e539.
10. Hirth A, Berg A, Greve G. Successful treatment of severe heart failure in an infant with Hurler syndrome. J Inherit Metab Dis. 2007; 30: 820.
11. Kakkis ED, Muenzer J, Tiller GE, Waber L, Belmont J, Passage M et al. Enzyme-replacement therapy inmucopolysaccharidosis I.NEngl JMed. 2001; 344: 182-188.
12. Moore D, Connock MJ, Wraith E, Lavery C. The prevalence of and survival in mucopolysaccharidosis I: Hurler, Hurler-Scheie and Scheie syndromes in the UK. Orphanet J Rare Dis. 2008; 3: 24.
13. Muenzer J, Wraith JE, Beck M, Giugliani R, Harmatz P, Eng CM et al. A Phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome). Genet Med. 2006; 8: 465-473.
14. Muenzer J, Gucsavas-Calikoglu M, McCandless SE, Schuetz TJ, Kimura A. APhase I/II clinical trial of enzyme replacement therapy in mucopolysaccharidosis II (Hunter syndrome). Mol Genet Metab. 2007; 90: 329-337. (Pubitemid 46241902)
15. Neufeld EF, Muenzer J. The mucopolysaccharidoses. In: Scriver CR, Beaudet AL, Sly WS, Valle D (eds). The metabolic and molecular bases of inherited disease. New York: McGraw-Hill; 2001. p. 3421-3452.
16. Peters C, Steward CG. Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines. Bone Marrow Transplant. 2003; 31: 229-239. (Pubitemid 36336225)
17. Soni S, Hente M, Breslin N, Hersh J, Whitley C, Cheerva A et al. Pre-stem cell transplantation enzyme replacement therapy in Hurler syndrome does not lead to significant antibody formation or delayed recovery of the endogenous enzyme post-transplant: a case report. Pediatr Transplant. 2007; 11: 563-567. (Pubitemid 47057339)
18. Tolar J, Grewal SS, Bjoraker KJ, Whitley CB, Shapiro EG, Charnas L et al. Combination of enzyme replacement and hematopoietic stem cell transplantation as therapy for Hurler syndrome. Bone Marrow Transplant. 2008; 41: 531-535. (Pubitemid 351472144)
19. Tomatsu S, Montano AM, Ohashi A, Gutierrez MA, Oikawa H, Oguma T et al. Enzyme replacement therapy in a murine model of Morquio Asyndrome. Hum Mol Genet. 2008; 17: 815-824. (Pubitemid 351359698)
20. Wraith JE. Enzyme replacement therapy with idursulfase in patients with mucopolysaccharidosis type II. Acta Paediatr. 2008; 97 (Suppl 457): 76-78. (Pubitemid 351421301)
21. Wraith JE, Clarke LA, Beck M, Kolodny EH, Pastores GM, Muenzer J et al. Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, doubleblinded, placebo-controlled, multinational study of recombinant human α-L-iduronidase (laronidase). J Pediatr. 2004; 144: 581-588. (Pubitemid 38594757)
22. Wraith JE, Beck M, Lane R, van der Ploeg A, Shapiro E, Xue Y et al. Enzyme replacement therapy in patients who have mucopolysaccharidosis I and are younger than 5 years: results of a multinational study of recombinant human α-L-iduronidase (laronidase). Pediatrics. 2007; 120: e37-e46. (Pubitemid 47036214)
23. Wynn RF, Mercer J, Page J, Carr TF, Jones S, Wraith JE. Use of enzyme replacement therapy (laronidase) before hematopoietic stem cell transplantation for mucopolysaccharidosis I: experience in 18 patients. J Pediatr. 2009; 154: 135-139.