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

dc.creatorDornelles, Alícia Dorneles
dc.creatorJunges, Ana Paula Pedroso
dc.creatorPereira, Tiago Veiga
dc.creatorKrug, Bárbara Corrêa
dc.creatorGonçalves, Candice Beatriz Treter
dc.creatorLlerena Junior, Juan Clinton
dc.creatorKishnani, Priya Sunil
dc.creatorOliveira Junior, Haliton Alves de
dc.creatorSchwartz, Ida Vanessa Doederlein
dc.date2021-11-23T12:45:33Z
dc.date2021-11-23T12:45:33Z
dc.date2021
dc.date.accessioned2023-09-26T21:10:45Z
dc.date.available2023-09-26T21:10:45Z
dc.identifierDORNELLES, 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.identifier2077-0383
dc.identifierhttps://www.arca.fiocruz.br/handle/icict/49951
dc.identifier10.3390/jcm10214828
dc.identifier2077-0383
dc.identifier.urihttps://repositorioslatinoamericanos.uchile.cl/handle/2250/8870398
dc.description2019-0495/Hospital de Clínicas de Porto Alegre
dc.descriptionPompe 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.
dc.formatapplication/pdf
dc.languageeng
dc.publisherMDPI
dc.rightsopen access
dc.subjectGlycogen storage disease type II
dc.subjectAlpha-glucosidase
dc.subjectPompe disease
dc.subjectEnzyme replacement therapy
dc.titleA systematic review and meta-analysis of enzyme replacement therapy in late-onset Pompe disease
dc.typeArticle


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