Enzyme replacement therapy for Fabry disease : a systematic review and meta-analysis

Detalhes bibliográficos
Autor(a) principal: Alegra, Taciane
Data de Publicação: 2012
Outros Autores: Vairo, Filippo Pinto e, Souza, Monica Vinhas de, Krug, Bárbara Côrrea, Schwartz, Ida Vanessa Doederlein
Tipo de documento: Artigo
Idioma: eng
Título da fonte: Repositório Institucional da UFRGS
Texto Completo: http://hdl.handle.net/10183/87945
Resumo: The 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.
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spelling Alegra, TacianeVairo, Filippo Pinto eSouza, Monica Vinhas deKrug, Bárbara CôrreaSchwartz, Ida Vanessa Doederlein2014-02-28T01:50:31Z20121415-4757http://hdl.handle.net/10183/87945000876252The 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.application/pdfengGenetics and molecular biology. Ribeirão Preto. Vol. 35, n. 4 supl (Dec. 2012), p. 947-954Doença de FabryTerapia de reposição de enzimasFabry diseaseFabry disease/therapyenzyme replacement therapyalpha-GalactosidaseEnzyme replacement therapy for Fabry disease : a systematic review and meta-analysisinfo:eu-repo/semantics/articleinfo:eu-repo/semantics/otherinfo:eu-repo/semantics/publishedVersioninfo:eu-repo/semantics/openAccessreponame:Repositório Institucional da UFRGSinstname:Universidade Federal do Rio Grande do Sul (UFRGS)instacron:UFRGSORIGINAL000876252.pdf000876252.pdfTexto completo (inglês)application/pdf611633http://www.lume.ufrgs.br/bitstream/10183/87945/1/000876252.pdf864c876cd01373a3bba76d4c79181524MD51TEXT000876252.pdf.txt000876252.pdf.txtExtracted Texttext/plain38963http://www.lume.ufrgs.br/bitstream/10183/87945/2/000876252.pdf.txt85d5330eefedada5de7014152f6c7d82MD52THUMBNAIL000876252.pdf.jpg000876252.pdf.jpgGenerated Thumbnailimage/jpeg1811http://www.lume.ufrgs.br/bitstream/10183/87945/3/000876252.pdf.jpgb22ae19d262c8f4d4826d788d753f138MD5310183/879452023-09-21 03:40:19.718351oai:www.lume.ufrgs.br:10183/87945Repositório de PublicaçõesPUBhttps://lume.ufrgs.br/oai/requestopendoar:2023-09-21T06:40:19Repositório Institucional da UFRGS - Universidade Federal do Rio Grande do Sul (UFRGS)false
dc.title.pt_BR.fl_str_mv Enzyme replacement therapy for Fabry disease : a systematic review and meta-analysis
title Enzyme replacement therapy for Fabry disease : a systematic review and meta-analysis
spellingShingle Enzyme replacement therapy for Fabry disease : a systematic review and meta-analysis
Alegra, Taciane
Doença de Fabry
Terapia de reposição de enzimas
Fabry disease
Fabry disease/therapy
enzyme replacement therapy
alpha-Galactosidase
title_short Enzyme replacement therapy for Fabry disease : a systematic review and meta-analysis
title_full Enzyme replacement therapy for Fabry disease : a systematic review and meta-analysis
title_fullStr Enzyme replacement therapy for Fabry disease : a systematic review and meta-analysis
title_full_unstemmed Enzyme replacement therapy for Fabry disease : a systematic review and meta-analysis
title_sort Enzyme replacement therapy for Fabry disease : a systematic review and meta-analysis
author Alegra, Taciane
author_facet Alegra, Taciane
Vairo, Filippo Pinto e
Souza, Monica Vinhas de
Krug, Bárbara Côrrea
Schwartz, Ida Vanessa Doederlein
author_role author
author2 Vairo, Filippo Pinto e
Souza, Monica Vinhas de
Krug, Bárbara Côrrea
Schwartz, Ida Vanessa Doederlein
author2_role author
author
author
author
dc.contributor.author.fl_str_mv Alegra, Taciane
Vairo, Filippo Pinto e
Souza, Monica Vinhas de
Krug, Bárbara Côrrea
Schwartz, Ida Vanessa Doederlein
dc.subject.por.fl_str_mv Doença de Fabry
Terapia de reposição de enzimas
topic Doença de Fabry
Terapia de reposição de enzimas
Fabry disease
Fabry disease/therapy
enzyme replacement therapy
alpha-Galactosidase
dc.subject.eng.fl_str_mv Fabry disease
Fabry disease/therapy
enzyme replacement therapy
alpha-Galactosidase
description The 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.
publishDate 2012
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dc.relation.ispartof.pt_BR.fl_str_mv Genetics and molecular biology. Ribeirão Preto. Vol. 35, n. 4 supl (Dec. 2012), p. 947-954
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