A systematic review and meta-analysis of enzyme replacement therapy in late-onset Pompe disease

Detalhes bibliográficos
Autor(a) principal: Dornelles, Alícia Dorneles
Data de Publicação: 2021
Outros Autores: Junges, Ana Paula Pedroso, Pereira, Tiago Veiga, Krug, Bárbara Corrêa, Gonçalves, Candice Beatriz Treter, Llerena Junior, Juan Clinton, Kishnani, Priya Sunil, Oliveira Junior, Haliton Alves de, Schwartz, Ida Vanessa Doederlein
Tipo de documento: Artigo
Idioma: eng
Título da fonte: Repositório Institucional da FIOCRUZ (ARCA)
Texto Completo: https://www.arca.fiocruz.br/handle/icict/49951
Resumo: 2019-0495/Hospital de Clínicas de Porto Alegre
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spelling Dornelles, Alícia DornelesJunges, Ana Paula PedrosoPereira, Tiago VeigaKrug, Bárbara CorrêaGonçalves, Candice Beatriz TreterLlerena Junior, Juan ClintonKishnani, Priya SunilOliveira Junior, Haliton Alves deSchwartz, Ida Vanessa Doederlein2021-11-23T12:45:33Z2021-11-23T12:45:33Z2021DORNELLES, Alícia Dorneles et al. A systematic review and meta-analysis of enzyme replacement therapy in late-onset Pompe disease. Journal of Clinical Medicine, v. 10, n. 21, p. 1-19, 21 Oct. 2021.2077-0383https://www.arca.fiocruz.br/handle/icict/4995110.3390/jcm102148282077-03832019-0495/Hospital de Clínicas de Porto AlegreUniversidade Federal do Rio Grande do Sul. Faculdade de Medicina. Programa de Pós-Graduação em Ciências Médicas. Porto Alegre, RS, Brasil / Hospital de Clínicas de Porto Alegre. Serviço de Genética Médica. Porto Alegre, RS, Brasil.Hospital de Clínicas de Porto Alegre. Serviço de Genética Médica. Porto Alegre, RS, Brasil / Universidade Federal do Rio Grande do Sul. Faculdade de Medicina. Porto Alegre, RS, Brasil.Applied Health Research Centre. Li Ka Shing Knowledge Institute. St Michael's Hospital. Toronto, ON, Canada / University of Leicester. College of Medicine. Department of Health Sciences. Leicester, UK.Hospital de Clínicas de Porto Alegre. Centro de Pesquisa Clínica. Núcleo de Investigação Clínica em Medicamentos. Porto Alegre, RS, Brasil.Hospital de Clínicas de Porto Alegre. Centro de Pesquisa Clínica. Núcleo de Investigação Clínica em Medicamentos. Porto Alegre, RS, Brasil.Fundação Oswaldo Cruz. Instituto Nacional de Saúde da Mulher, da Criança e do Adolescente Fernandes Figueira. Rio de Janeiro, RJ, Brasil.Duke University Medical Center. Department of Pediatrics. Durham, NC, USA.Hospital Alemão Oswaldo Cruz. Avaliação em Tecnologias da Saúde. São Paulo, SP, Brasil.Universidade Federal do Rio Grande do Sul. Faculdade de Medicina. Programa de Pós-Graduação em Ciências Médicas. Porto Alegre, RS, Brasil / Hospital de Clínicas de Porto Alegre. Serviço de Genética Médica. Porto Alegre, RS, Brasil / Universidade Federal do Rio Grande do Sul. Faculdade de Medicina. Porto Alegre, RS, Brasil / Hospital de Clínicas de Porto Alegre. Centro de Pesquisa Clínica. Núcleo de Investigação Clínica em Medicamentos. Porto Alegre, RS, Brasil / Universidade Federal do Rio Grande do Sul. Faculdade de Medicina. Departamento de Genética. Porto Alegre, RS, Brasil.Pompe disease (PD) is a glycogen storage disorder caused by deficient activity of acid alpha-glucosidase (GAA). We sought to review the latest available evidence on the safety and efficacy of recombinant human GAA enzyme replacement therapy (ERT) for late-onset PD (LOPD). Methods: We systematically searched the MEDLINE (via PubMed), Embase, and Cochrane databases for prospective clinical studies evaluating ERT for LOPD on pre-specified outcomes. A meta-analysis was also performed. Results: Of 1601 articles identified, 22 were included. Studies were heterogeneous and with very low certainty of evidence for most outcomes. The following outcomes showed improvements associated with GAA ERT, over a mean follow-up of 32.5 months: distance walked in the 6-min walking test (6MWT) (mean change 35.7 m (95% confidence interval [CI] 7.78, 63.75)), physical domain of the SF-36 quality of life (QOL) questionnaire (mean change 1.96 (95% CI 0.33, 3.59)), and time on ventilation (TOV) (mean change −2.64 h (95% CI −5.28, 0.00)). There were no differences between the pre- and post-ERT period for functional vital capacity (FVC), Walton and Gardner-Medwin Scale score, upper-limb strength, or total SF-36 QOL score. Adverse events (AEs) after ERT were mild in most cases. Conclusion: Considering the limitations imposed by the rarity of PD, our data suggest that GAA ERT improves 6MWT, physical QOL, and TOV in LOPD patients. ERT was safe in the studied population. PROSPERO register: 135102.engMDPIA systematic review and meta-analysis of enzyme replacement therapy in late-onset Pompe diseaseinfo:eu-repo/semantics/publishedVersioninfo:eu-repo/semantics/articleGlycogen storage disease type IIAlpha-glucosidasePompe diseaseEnzyme replacement therapyinfo:eu-repo/semantics/openAccessreponame:Repositório Institucional da FIOCRUZ (ARCA)instname:Fundação Oswaldo Cruz (FIOCRUZ)instacron:FIOCRUZLICENSElicense.txtlicense.txttext/plain; charset=utf-83132https://www.arca.fiocruz.br/bitstream/icict/49951/1/license.txt33967fcc8ef681b8f5c2c34178ebc114MD51ORIGINALve_Juan_Llerena Junior_etal_IFF_2021.pdfve_Juan_Llerena Junior_etal_IFF_2021.pdfapplication/pdf3136501https://www.arca.fiocruz.br/bitstream/icict/49951/2/ve_Juan_Llerena%20Junior_etal_IFF_2021.pdf239baf70fbf468a2e8f104acd3e0238fMD52icict/499512021-11-25 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dc.title.en.fl_str_mv A systematic review and meta-analysis of enzyme replacement therapy in late-onset Pompe disease
title A systematic review and meta-analysis of enzyme replacement therapy in late-onset Pompe disease
spellingShingle A systematic review and meta-analysis of enzyme replacement therapy in late-onset Pompe disease
Dornelles, Alícia Dorneles
Glycogen storage disease type II
Alpha-glucosidase
Pompe disease
Enzyme replacement therapy
title_short A systematic review and meta-analysis of enzyme replacement therapy in late-onset Pompe disease
title_full A systematic review and meta-analysis of enzyme replacement therapy in late-onset Pompe disease
title_fullStr A systematic review and meta-analysis of enzyme replacement therapy in late-onset Pompe disease
title_full_unstemmed A systematic review and meta-analysis of enzyme replacement therapy in late-onset Pompe disease
title_sort A systematic review and meta-analysis of enzyme replacement therapy in late-onset Pompe disease
author Dornelles, Alícia Dorneles
author_facet Dornelles, Alícia Dorneles
Junges, Ana Paula Pedroso
Pereira, Tiago Veiga
Krug, Bárbara Corrêa
Gonçalves, Candice Beatriz Treter
Llerena Junior, Juan Clinton
Kishnani, Priya Sunil
Oliveira Junior, Haliton Alves de
Schwartz, Ida Vanessa Doederlein
author_role author
author2 Junges, Ana Paula Pedroso
Pereira, Tiago Veiga
Krug, Bárbara Corrêa
Gonçalves, Candice Beatriz Treter
Llerena Junior, Juan Clinton
Kishnani, Priya Sunil
Oliveira Junior, Haliton Alves de
Schwartz, Ida Vanessa Doederlein
author2_role author
author
author
author
author
author
author
author
dc.contributor.author.fl_str_mv Dornelles, Alícia Dorneles
Junges, Ana Paula Pedroso
Pereira, Tiago Veiga
Krug, Bárbara Corrêa
Gonçalves, Candice Beatriz Treter
Llerena Junior, Juan Clinton
Kishnani, Priya Sunil
Oliveira Junior, Haliton Alves de
Schwartz, Ida Vanessa Doederlein
dc.subject.en.en.fl_str_mv Glycogen storage disease type II
Alpha-glucosidase
Pompe disease
Enzyme replacement therapy
topic Glycogen storage disease type II
Alpha-glucosidase
Pompe disease
Enzyme replacement therapy
description 2019-0495/Hospital de Clínicas de Porto Alegre
publishDate 2021
dc.date.accessioned.fl_str_mv 2021-11-23T12:45:33Z
dc.date.available.fl_str_mv 2021-11-23T12:45:33Z
dc.date.issued.fl_str_mv 2021
dc.type.status.fl_str_mv info:eu-repo/semantics/publishedVersion
dc.type.driver.fl_str_mv info:eu-repo/semantics/article
format article
status_str publishedVersion
dc.identifier.citation.fl_str_mv DORNELLES, Alícia Dorneles et al. A systematic review and meta-analysis of enzyme replacement therapy in late-onset Pompe disease. Journal of Clinical Medicine, v. 10, n. 21, p. 1-19, 21 Oct. 2021.
dc.identifier.uri.fl_str_mv https://www.arca.fiocruz.br/handle/icict/49951
dc.identifier.issn.pt_BR.fl_str_mv 2077-0383
dc.identifier.doi.none.fl_str_mv 10.3390/jcm10214828
dc.identifier.eissn.pt_BR.fl_str_mv 2077-0383
identifier_str_mv DORNELLES, Alícia Dorneles et al. A systematic review and meta-analysis of enzyme replacement therapy in late-onset Pompe disease. Journal of Clinical Medicine, v. 10, n. 21, p. 1-19, 21 Oct. 2021.
2077-0383
10.3390/jcm10214828
url https://www.arca.fiocruz.br/handle/icict/49951
dc.language.iso.fl_str_mv eng
language eng
dc.rights.driver.fl_str_mv info:eu-repo/semantics/openAccess
eu_rights_str_mv openAccess
dc.publisher.none.fl_str_mv MDPI
publisher.none.fl_str_mv MDPI
dc.source.none.fl_str_mv reponame:Repositório Institucional da FIOCRUZ (ARCA)
instname:Fundação Oswaldo Cruz (FIOCRUZ)
instacron:FIOCRUZ
instname_str Fundação Oswaldo Cruz (FIOCRUZ)
instacron_str FIOCRUZ
institution FIOCRUZ
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collection Repositório Institucional da FIOCRUZ (ARCA)
bitstream.url.fl_str_mv https://www.arca.fiocruz.br/bitstream/icict/49951/1/license.txt
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