Clinical effectiveness of enzyme replacement therapy with galsulfase in mucopolysaccharidosis type VI treatment: systematic review

dc.creatorGomes, Dalila Fernandes
dc.creatorGallo, Luciana Guerra
dc.creatorLeite, Betânia Ferreira
dc.creatorSilva, Roberta Borges
dc.creatorSilva, Everton Nunes da
dc.date2020-06-23T14:02:48Z
dc.date2020-06-23T14:02:48Z
dc.date2019
dc.date.accessioned2023-09-26T20:20:26Z
dc.date.available2023-09-26T20:20:26Z
dc.identifierGOMES, Dalila Fernandes et al. Clinical effectiveness of enzyme replacement therapy with galsulfase in mucopolysaccharidosis type VI treatment: systematic review. Journal Of Inherited Metabolic Disease, [Lancaster], v. 42, n. 1, p. 66-76, 2019.
dc.identifier0141-8955
dc.identifierhttps://www.arca.fiocruz.br/handle/icict/41827
dc.identifier10.1002/jimd.12028
dc.identifier.urihttps://repositorioslatinoamericanos.uchile.cl/handle/2250/8854636
dc.descriptionLuciana Guerra Gallo - Fundação Oswaldo Cruz. Fiocruz Brasília. Brasília, DF, Brasil. Documento produzido em parceria ou por autora vinculada à Fiocruz, mas não consta à informação no documento.
dc.descriptionIntroduction: Mucopolysaccharidosis VI is a rare disease characterized by the arylsulfatase B enzyme deficiency, which is responsible for different clinical manifestations. The treatment consists of enzyme replacement therapy with intravenous administration of galsulfase. Objective: Evaluate the effectiveness of the enzyme replacement therapy with galsulfase for the mucopolysaccharidosis VI treatment. Method: Systematic review of observational studies. The databases of PubMed, Cochrane Library, Lilacs, and Journal of Inherited Metabolic Disease were reviewed. The selection of studies, data mining, and methodological quality assessment were independently conducted by two authors. Results: Eighteen studies fulfilled the inclusion criteria. Two studies were cohorts, one was longitudinal study, one was cross-sectional, one was a case-control, eight were case series, and five were case reports. A total of 362 participants with mucopolysaccharidosis type VI were evaluated, and 14 different outcomes related to the treatment effect were identified. Seven outcomes showed positive results, characterized by the patient survival, quality of life, respiratory function, joint mobility, physical resistance, reduction of urinary glycosaminoglycans, and growth. The hearing function and the cognitive development were stable after the treatment. Other outcomes related to the cardiac function, visual acuity, sleep apnea, and the size of the liver and spleen presented inconclusive outcomes. Concerning safety, light adverse reactions of hypersensitivity were reported. Conclusion: This review provided a broader panoramic view of the outcomes related to mucopolysaccharidosis type VI. Regardless of the inherent limitations of observational studies, the outcomes indicate that the enzyme replacement therapy has a positive effect on most of the outcomes associated to the disease.
dc.description2022-12-31
dc.formatapplication/pdf
dc.formatapplication/pdf
dc.formatapplication/pdf
dc.formatapplication/pdf
dc.languageeng
dc.publisherWiley
dc.rightsrestricted access
dc.subjectSystematic review
dc.subjectCross-Sectional Studies
dc.subjectEnzyme Replacement Therapy
dc.subjectMucopolysaccharidosis VI
dc.subjectN-Acetylgalactosamine-4-Sulfatase
dc.subjectObservational Studies as Topic
dc.subjectRecombinant Proteins
dc.subjectEnzyme replacement therapy
dc.subjectMucopolysaccharidosis VI
dc.subjectSystematic review
dc.subjectTerapia de Reposição de Enzimas
dc.subjectMucopolissacaridose IV
dc.subjectRevisão Sistemática
dc.subjectEstudos Transversais
dc.subjectEstudos Observacionais como Assunto
dc.titleClinical effectiveness of enzyme replacement therapy with galsulfase in mucopolysaccharidosis type VI treatment: systematic review
dc.typeArticle


Este ítem pertenece a la siguiente institución