Enzyme replacement therapy for Fabry disease : a systematic review and meta-analysis
Autor(a) principal: | |
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Data de Publicação: | 2012 |
Outros Autores: | , , , |
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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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 |
dc.date.issued.fl_str_mv |
2012 |
dc.date.accessioned.fl_str_mv |
2014-02-28T01:50:31Z |
dc.type.driver.fl_str_mv |
info:eu-repo/semantics/article info:eu-repo/semantics/other |
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http://hdl.handle.net/10183/87945 |
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1415-4757 |
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000876252 |
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http://hdl.handle.net/10183/87945 |
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eng |
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eng |
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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info:eu-repo/semantics/openAccess |
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openAccess |
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