This is not the most recent version of the article. View current version (5 FEB 2016)

Intervention Review

You have free access to this content

Enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II (Hunter syndrome)

  1. Edina MK da Silva1,*,
  2. Maria Wany Louzada Strufaldi2,
  3. Régis B Andriolo3,
  4. Laercio A Silva4

Editorial Group: Cochrane Cystic Fibrosis and Genetic Disorders Group

Published Online: 8 JAN 2014

Assessed as up-to-date: 6 NOV 2013

DOI: 10.1002/14651858.CD008185.pub3


How to Cite

da Silva EMK, Strufaldi MWL, Andriolo RB, Silva LA. Enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II (Hunter syndrome). Cochrane Database of Systematic Reviews 2014, Issue 1. Art. No.: CD008185. DOI: 10.1002/14651858.CD008185.pub3.

Author Information

  1. 1

    Universidade Federal de São Paulo, Emergency Medicine and Evidence Based Medicine, São Paulo, São Paulo, Brazil

  2. 2

    Universidade Federal de São Paulo, São Paulo, São Paulo, Brazil

  3. 3

    Universidade do Estado do Pará, Department of Public Health, Belém, Pará, Brazil

  4. 4

    Universidade Federal de São Paulo, Department of Urology, São Paulo, São Paulo, Brazil

*Edina MK da Silva, Emergency Medicine and Evidence Based Medicine, Universidade Federal de São Paulo, Rua Borges Lagoa 564 cj 64, Vl. Clementino, São Paulo, São Paulo, 04038-000, Brazil. edinaksilva@terra.com.br.

Publication History

  1. Publication Status: New search for studies and content updated (no change to conclusions)
  2. Published Online: 8 JAN 2014

SEARCH

This is not the most recent version of the article. View current version (05 FEB 2016)

References

References to studies included in this review

  1. References to studies included in this review
  2. References to studies excluded from this review
  3. Additional references
  4. References to other published versions of this review
Muenzer 2006 {published data only}
  • Beck M, Wraith E, Muenzer J, Giugliani R, Harmatz P, Eng CM, et al.  Long-term weekly dosing of idursulfase in the treatment of mucopolysaccharidosis II (MPS II, Hunter syndrome) [abstract]. Journal of Inherited Metabolic Disease 2007;30(Suppl 1):116.
  • Muenzer J, Beck M, Eng CM, Giugliani R, Harmatz P, Martin R, et al. Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome. Genetics in Medicine 2011;13(2):95-101.
  • Muenzer J, Wraith E, Beck M, Giugliani R, Harmatz P, Eng CM, et al. Clinical benefit of enzyme replacement therapy (ERT) in mucopolysaccharidosis II (MPS II, Hunter syndrome) [abstract]. Journal of Inherited Metabolic Disease 2006;29(Suppl 1):28.
  • 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). Genetics in Medicine 2006;8(8):465-73.

References to studies excluded from this review

  1. References to studies included in this review
  2. References to studies excluded from this review
  3. Additional references
  4. References to other published versions of this review
Gutiérrez-Solana 2007 {published data only}
  • Gutiérrez-Solana LG. Clinical study of enzyme replacement therapy with idursulfase. Revista de Neurologia 2007;44(Suppl 1):S7-S11.
Muenzer 2007 {published data only}
  • Muenzer J, Calikoglu MG, Conway AM, Kimura A. The long-term experience of enzyme replacement therapy for Hunter syndrome (MPS II) [abstract]. Journal of Inherited Metabolic Disorder 2005;28(Suppl 1):188.
  • Muenzer J, Gucsavas-Calikoglu M, McCandless SE, Schuetz TJ, Kimura A. A phase I/II clinical trial of enzyme replacement therapy in mucopolysaccharidosis II (Hunter syndrome). Molecular Genetics and Metabolism. 2007;90(3):329-37.
  • Muenzer J, Towle D, Calikoglu M, McCandless S. The 12-month experience of enzyme replacement for mucopolysaccharidosis II (Hunter syndrome) [abstract]. Journal of Inherited Metabolic Disease 2003;26(Suppl 2):144.
Sohn 2013 {published data only}
Tylki-Szymanska 2008 {published data only}
  • Tylki-Szymanska A, Giugliani R, Hwu WL. A clinical trial of idursulfase in Hunter syndrome patients 5 years old and younger [abstract]. Journal of Inherited Metabolic Disease 2008;31(Suppl 1):130.

Additional references

  1. References to studies included in this review
  2. References to studies excluded from this review
  3. Additional references
  4. References to other published versions of this review
Alcade-Martín 2010
  • Alcalde-Martín C, Muro-Tudelilla JM, Cancho-Candela R, Gutiérrez-Solana LG, Pintos-Morell G, Martí-Herrero M, et al. First experience of enzyme replacement therapy with idursulfase in Spanish patients with Hunter syndrome under 5 years of age: Case observations from the Hunter Outcome Survey (HOS). European Journal of Medical Genetics 2010;53(6):371-7.
ATS 2002
de Jong 1992
  • de Jong JG, Wevers RA, Liebrand-van Sambeek R. Measuring urinary glycosaminoglycans in the presence of protein: an improved screening procedure for mucopolysaccharidoses based on dimethylmethylene blue. Clinical Chemistry 1992;38(6):803-7.
Elbourne 2002
  • Elbourne DR, Altman DG, Higgins JP, Curtin F, Worthington HV, Vail A. Meta-analyses involving cross-over trials: methodological issues. International Journal of Epidemiology 2002;31(1):140-9. [PUBMED: 11914310]
Higgins 2003
Higgins 2011a
  • Higgins JPT, Altman DG. Chapter 8: Assessing risk of bias in included studies. In: Higgins JPT, Green S (editors). Cochrane Handbook for Systematic Reviews of Interventions Version 5.1 [updated March 2011]. The Cochrane Collaboration, 2011. Available from www.cochrane-handbook.org.
Higgins 2011b
  • Higgins JPT, Deeks JJ, Altman DG. Chpater 16: Special topics in statistics. Intention to treat issues. In: Higgins JPT, Green S (editors). Cochrane Handbook for Systematic Reviews of Interventions Version 5.1 [updated March 2011]. The Cochrane Collaboration, 2011. Available from www.cochrane-handbook.org.
Jones 2008
  • Jones SA, Almássy Z, Beck M, Burt K, Clarke JT, Giugliani R, et al. Mortality and cause of death in mucopolysaccharidosis type II-a historical review based on data from the Hunter Outcome Survey (HOS). Journal of Inherited Metabolic Disease 2008;32(4):534-43.
Jones 2013
  • Jones SA, Parini R, Harmatz P, Giugliani R, Fang J, Mendelsohn NJ, et al. The effect of idursulfase on growth in patients with Hunter syndrome: data from the Hunter Outcome Survey (HOS). Molecular Genetics and Metabolism 2013;109(1):41-8.
Kresse 1982
Martin 2008
Meikle 1999
Muenzer 2011
  • Muenzer J, Beck M, Eng CM, Giugliani R, Harmatz P, Martin R, et al. Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome. Genetics in Medicine 2011;13(2):95-101.
National Horizon Scanning Centre 2005
  • National Horizon Scanning Centre. New and Emerging Technology Briefing: Iduronate-2-sulfatase for Hunter Syndrome. The University of Birmingham 2005.
Neufeld 2001
  • Neufeld EF, Muenzer J. The mucopolysaccharidoses. The Metabolic and Molecular Bases of Inherited Disease. New York, NY: McGraw-Hill, 2001:3421-52.
Schulze-Frenking 2011
  • Schulze-Frenking G, Jones SA, Roberts J, Beck M, Wraith JE. Effects of enzyme replacement therapy on growth in patients with mucopolysaccharidosis type II. Journal of Inherited Metabolic Disease 2011;34(1):203-8.
Tuschl 2005
Vellodi 2007
  • Vellodi A, Wraith JE, Cleary M, Ramaswami U, Lavery C, Jessop E. Guidelines for the investigation and management of patients with mucopolysaccharidosis type II. www.specialisedservices.nhs.uk/library/23/Guidelines_for_Mucopolysaccharidosis_Type_II.pdf (accessed September 2009).
Wraith 2008a
  • Wraith JE, Scarpa M, Beck M, Bodamer OA, De Meirleir L, Guffon N, et al. Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy. European Journal of Pediatrics 2008;167(3):267-77.
Wraith 2008b
Young 1983

References to other published versions of this review

  1. References to studies included in this review
  2. References to studies excluded from this review
  3. Additional references
  4. References to other published versions of this review
da Silva 2011