dc.creator | Gomes, Dalila Fernandes | |
dc.creator | Gallo, Luciana Guerra | |
dc.creator | Leite, Betânia Ferreira | |
dc.creator | Silva, Roberta Borges | |
dc.creator | Silva, Everton Nunes da | |
dc.date | 2020-06-23T14:02:48Z | |
dc.date | 2020-06-23T14:02:48Z | |
dc.date | 2019 | |
dc.date.accessioned | 2023-09-26T20:20:26Z | |
dc.date.available | 2023-09-26T20:20:26Z | |
dc.identifier | GOMES, 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.identifier | 0141-8955 | |
dc.identifier | https://www.arca.fiocruz.br/handle/icict/41827 | |
dc.identifier | 10.1002/jimd.12028 | |
dc.identifier.uri | https://repositorioslatinoamericanos.uchile.cl/handle/2250/8854636 | |
dc.description | Luciana 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.description | Introduction: 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.description | 2022-12-31 | |
dc.format | application/pdf | |
dc.format | application/pdf | |
dc.format | application/pdf | |
dc.format | application/pdf | |
dc.language | eng | |
dc.publisher | Wiley | |
dc.rights | restricted access | |
dc.subject | Systematic review | |
dc.subject | Cross-Sectional Studies | |
dc.subject | Enzyme Replacement Therapy | |
dc.subject | Mucopolysaccharidosis VI | |
dc.subject | N-Acetylgalactosamine-4-Sulfatase | |
dc.subject | Observational Studies as Topic | |
dc.subject | Recombinant Proteins | |
dc.subject | Enzyme replacement therapy | |
dc.subject | Mucopolysaccharidosis VI | |
dc.subject | Systematic review | |
dc.subject | Terapia de Reposição de Enzimas | |
dc.subject | Mucopolissacaridose IV | |
dc.subject | Revisão Sistemática | |
dc.subject | Estudos Transversais | |
dc.subject | Estudos Observacionais como Assunto | |
dc.title | Clinical effectiveness of enzyme replacement therapy with galsulfase in mucopolysaccharidosis type VI treatment: systematic review | |
dc.type | Article | |