Enzyme replacement therapy with idursulfase for mucopolysaccharidosis type ii (hunter syndrome)

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dc.contributor.author da Silva, Edina M. K. [UNIFESP]
dc.contributor.author Louzada Strufaldi, Maria Wany [UNIFESP]
dc.contributor.author Andriolo, Regis B.
dc.contributor.author Silva, Laercio A. [UNIFESP]
dc.date.accessioned 2019-01-21T10:30:03Z
dc.date.available 2019-01-21T10:30:03Z
dc.date.issued 2016
dc.identifier http://dx.doi.org/10.1002/14651858.CD008185.pub4
dc.identifier.citation Cochrane Database Of Systematic Reviews. Hoboken, n. 2, p. CD008185, 2016.
dc.identifier.issn 1469-493X
dc.identifier.uri http://repositorio.unifesp.br/handle/11600/49548
dc.description.abstract Background Mucopolysaccharidosis II, also known as Hunter syndrome, is a rare, X-linked disease caused by a deficiency of the lysosomal enzyme iduronate-2-sulfatase, which catalyses a step in the catabolism of glycosaminoglycans. The glycosaminoglycans accumulate within tissues affecting multiple organs and physiologic systems. The clinical manifestations include neurologic involvement, severe airways obstruction, skeletal deformities and cardiomyopathy. The disease has a variable age of onset and variable rate of progression. In those with severe disease, death usually occurs in the second decade of life, whereas those individuals with less severe disease may survive into adulthood. Enzyme replacement therapy with intravenous infusions of idursulfase has emerged as a new treatment for mucopolysaccharidosis type II. This is an update of a previously published version of this review. Objectives To evaluate the effectiveness and safety of enzyme replacement therapy with idursulfase compared to other interventions, placebo or no intervention, for treating mucopolysaccharidosis type II. Search methods We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group's Trials Register (date of last search 23 November 2015). We also searched Embase, PubMed and the Literature Latino-Americana e do Caribe em Ciencias da Saude (LILACS) (date of last search 28 November 2015). Selection criteria Randomised and quasi-randomised controlled trials of enzyme replacement therapy with idursulfase compared to no intervention, placebo or other options (e.g. behavioral strategies, transplantation). Data collection and analysis Two authors independently screened the trials identified, appraised quality of papers and extracted data. Main results One study (96 male participants) met the inclusion criteria, although the primary outcome of this review -z score for height and weight, was not assessed in the study. This trial was considered to be of overall good quality. Following 53 weeks of treatment, participants in the weekly idursulfase 0.5 mg/kg group demonstrated a significant improvement rate compared with placebo for the primary outcome: distance walked in sixminutes on the basis of the sum of ranks of change from baseline, mean difference 37.00 (95% confidence interval 6.52 to 67.48). The every-other-week idursulfase 0.5 mg/kg group also showed an improvement, which was not significant compared with placebo, mean difference 23.00 (95% confidence interval -4.49 to 50.49). After 53 weeks, there was no statistical significance difference in per cent predicted forced vital capacity between the three groups and absolute forced vital capacity was significantly increased from baseline in the weekly dosing group compared to placebo, mean difference 0.16 (95% confidence interval CI 0.05 to 0.27). No difference was observed between the every-other-week idursulfase 0.5 mg/kg group and placebo. In addition, liver and spleen volumes and urine glycosaminoglycan excretion were significantly reduced from baseline by both idursulfase dosing regimens. Idursulfase was generally well tolerated, but infusion reactions did occur. Idursulfase antibodies were detected in 31.7% of participants at the end of the study and they were related to a smaller reduction in urine glycosaminoglycan levels. Authors' conclusions The current evidence is limited. While the randomised clinical trial identified was considered to be of good quality, it failed to describe important outcomes. It has been demonstrated that enzyme replacement therapy with idursulfase is effective in relation to functional capacity (distance walked in six minutes and forced vital capacity), liver and spleen volumes and urine glycosaminoglycan excretion in people with mucopolysaccharidosis type II compared with placebo. There is no available evidence in the included study and in the literature on outcomes such as improvement in growth, sleep apnoea, cardiac function, quality of life and mortality. More studies are needed to obtain more information on the long-term effectiveness and safety of enzyme replacement therapy. en
dc.description.sponsorship National Institute for Health Research, UK
dc.description.sponsorship National Institute for Health Research
dc.format.extent CD008185
dc.language.iso eng
dc.publisher Wiley
dc.relation.ispartof Cochrane Database Of Systematic Reviews
dc.rights Acesso aberto
dc.subject Drug Administration Schedule en
dc.subject Enzyme Replacement Therapy [methods] en
dc.subject Iduronate Sulfatase [administration & Dosage] Mucopolysaccharidosis Ii [drug Therapy] en
dc.subject Randomized Controlled Trials As Topic en
dc.subject Rare Diseases [drug Therapy en
dc.subject Enzymology] en
dc.subject HumansI/Ii Clinical-Trial en
dc.subject Outcome Survey Hos en
dc.subject Phase-I/Ii en
dc.subject Mps-Ii en
dc.subject Storage Disorders en
dc.subject Metaanalyses en
dc.subject Experience en
dc.subject Diagnosis en
dc.subject Children en
dc.subject Younger en
dc.title Enzyme replacement therapy with idursulfase for mucopolysaccharidosis type ii (hunter syndrome) en
dc.type Revisão
dc.description.affiliation Emergency Medicine and Evidence Based Medicine, Universidade Federal de São Paulo, São Paulo, Brazil
dc.description.affiliation Universidade Federal de São Paulo, São Paulo, Brazil
dc.description.affiliation Department of Public Health, Universidade do Estado do Pará, Belém, Brazil
dc.description.affiliation Department of Urology,Universidade Federal de São Paulo, São Paulo, Brazil
dc.description.affiliationUnifesp Emergency Medicine and Evidence Based Medicine, Universidade Federal de São Paulo, Rua Borges Lagoa 564 Cj 64, BR-04038000 Sao Paulo, SP, Brazil
dc.description.affiliationUnifesp Universidade Federal de São Paulo, São Paulo, Brazil
dc.description.affiliationUnifesp Department of Urology,Universidade Federal de São Paulo, São Paulo, Brazil
dc.identifier.file WOS000373285000036.pdf
dc.identifier.doi 10.1002/14651858.CD008185.pub4
dc.description.source Web of Science
dc.identifier.wos WOS:000373285000036



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