Title:	Efetividade clínica da terapia de reposição enzimática com Galsulfase no tratamento da Mucopolissacaridose tipo vi : revisão sistemática
Authors:	Gomes, Dalila Fernandes
Orientador(es)::	Silva, Everton Nunes da
Assunto::	Terapia Enzimas Mucopolissacaridoses Galsulfase
Issue Date:	23-Jan-2018
Data de defesa::	25-Sep-2017
Citation:	GOMES, Dalila Fernandes. Efetividade clínica da terapia de reposição enzimática com Galsulfase no tratamento da Mucopolissacaridose tipo vi: revisão sistemática. 2017. 77 f., il. Dissertação (Mestrado em Saúde Coletiva)—Universidade de Brasília, Brasília, 2017.
Abstract:	Introduction: Mucopolysaccharidosis VI is a rare disease characterized by the deficiency of the enzyme arylsulfatase B, responsible for different clinical manifestations. Treatment consists of enzyme replacement therapy with intravenous administration of the galsulfase drug. Objective: To evaluate the efficacy of enzyme replacement therapy with galsulfase in the treatment of mucopolysaccharidosis VI. Method: This is a systematic review of observational studies. The studies were searched in the PubMed, Cochrane Library, Lilacs and Journal of Inherited Metabolic Disease databases. The reference lists of relevant studies for consultation to identify potentially eligible articles. No restrictions were applied regarding publishing data, language, or publishing status. The studies were selected by two independent evaluators who also extracted the data and evaluated the methodological quality of the studies. Results: nineteen studies completed the inclusion criteria, of which nine were case series, five case reports, three cohorts and two cross-sectional. A total of 414 people with mucopolysaccharidosis VI were evaluated and fourteen different outcomes related to the treatment effect identified. Eight outcomes presented positive results, characterized as (1) reduction of urinary glycosaminoglycans, (2) improvement of respiratory function, (3) increased range of joint motion, (4) increased patient growth rate, (5) increased physical resistance, (6) reduction of liver and spleen size, (7) improvement of quality of life, and (8) normalization of cardiac dimensions. Other outcomes remained stable, such as cognitive development, visual acuity, hearing capacity and sleep apnea. About safety, mild adverse reactions of hypersensitivity such as urticaria and pyrexia have been reported. Conclusion: Methodological limitations made it impossible to analyze the effectiveness of the treatment. However, the study findings indicate that enzyme replacement therapy has a positive effect on most disease associated outcomes. Therefore, further studies should be fomented to improve the quality of the evidence.
metadata.dc.description.unidade:	Faculdade de Ciências da Saúde (FS)
Description:	Mestrado (dissertação)—Universidade de Brasília, Faculdade de Ciências da Saúde, Programa de Pós-Graduação em Saúde Coletiva, 2017.
metadata.dc.description.ppg:	Programa de Pós-Graduação em Saúde Coletiva
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Appears in Collections:	Teses, dissertações e produtos pós-doutorado

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