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dc.contributor.authorAlegra, Tacianept_BR
dc.contributor.authorVairo, Filippo Pinto ept_BR
dc.contributor.authorSouza, Monica Vinhas dept_BR
dc.contributor.authorKrug, Bárbara Côrreapt_BR
dc.contributor.authorSchwartz, Ida Vanessa Doederleinpt_BR
dc.date.accessioned2014-02-28T01:50:31Zpt_BR
dc.date.issued2012pt_BR
dc.identifier.issn1415-4757pt_BR
dc.identifier.urihttp://hdl.handle.net/10183/87945pt_BR
dc.description.abstractThe specific treatment available for Fabry disease (FD) is enzyme replacement therapy (ERT) with agalsidase alfa or beta. A systematic review and meta-analysis was conducted to assess the efficacy and safety of ERT for FD. Only double-blind, randomized clinical trials (RCTs) comparing agalsidase alfa or beta and placebo were included. ERT with either agalsidase alfa or beta was considered similar for the purposes of analysis. Ten RCTs were identified, which showed improvements in neuropathic pain, in heart abnormalities and in globotriaosylceramide (GL-3) levels. A meta-analysis showed increased odds for fever, rigors, development of IgG antibodies to agalsidase, and no significant association with development of hypertension or reduction in the QRS complex duration on electrocardiogram. The RCTs included in this comparison enrolled few patients, were highly heterogeneous, and were focused mainly on surrogate endpoints, limiting any conclusions as to the real effect of ERT for FD. The available evidence suggests that response to ERT is variable across patient subgroups and that agalsidase may slow progression of FD, with slight improvement of existing changes. Nevertheless, many uncertainties remain, and further studies are necessary.en
dc.format.mimetypeapplication/pdfpt_BR
dc.language.isoengpt_BR
dc.relation.ispartofGenetics and molecular biology. Ribeirão Preto. Vol. 35, n. 4 supl (Dec. 2012), p. 947-954pt_BR
dc.rightsOpen Accessen
dc.subjectDoença de Fabrypt_BR
dc.subjectFabry diseaseen
dc.subjectFabry disease/therapyen
dc.subjectTerapia de reposição de enzimaspt_BR
dc.subjectenzyme replacement therapyen
dc.subjectalpha-Galactosidaseen
dc.titleEnzyme replacement therapy for Fabry disease : a systematic review and meta-analysispt_BR
dc.typeArtigo de periódicopt_BR
dc.identifier.nrb000876252pt_BR
dc.type.originNacionalpt_BR


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