Evaluating enzyme replacement therapies for Anderson-Fabry disease : commentary on a recent report

Detalhes bibliográficos
Autor(a) principal: Giugliani, Roberto
Data de Publicação: 2018
Outros Autores: Westwood, Stephanie, Wellhoefer, Hartmann, Schenk, Jörn Magnus, Gurevich, Andrey, Kampmann, Christoph
Tipo de documento: Artigo
Idioma: eng
Título da fonte: Repositório Institucional da UFRGS
Texto Completo: http://hdl.handle.net/10183/194775
Resumo: Anderson-Fabry disease (AFD) is a rare lysosomal storage disorder. Randomized controlled clinical trials (RCTs) are preferred as the highest category of evidence, but limited availability of robust evidence in rare diseases may necessitate the use of less rigorous evidence. An analysis of cohort studies of enzyme replacement therapies for AFD published in 2017 by El Dib and coworkers made treatment recommendations that contradict previously published findings from RCTs and a systematic Cochrane review. Our commentary outlines concerns regarding selection criteria and statistical methods with their analysis.
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spelling Giugliani, RobertoWestwood, StephanieWellhoefer, HartmannSchenk, Jörn MagnusGurevich, AndreyKampmann, Christoph2019-05-30T02:40:37Z20181415-4757http://hdl.handle.net/10183/194775001090254Anderson-Fabry disease (AFD) is a rare lysosomal storage disorder. Randomized controlled clinical trials (RCTs) are preferred as the highest category of evidence, but limited availability of robust evidence in rare diseases may necessitate the use of less rigorous evidence. An analysis of cohort studies of enzyme replacement therapies for AFD published in 2017 by El Dib and coworkers made treatment recommendations that contradict previously published findings from RCTs and a systematic Cochrane review. Our commentary outlines concerns regarding selection criteria and statistical methods with their analysis.application/pdfengGenetics and molecular biology. Ribeirão Preto. vol. 41, no. 4 (Oct./Dec. 2018), p. 790-793.Terapia de reposição de enzimasDoença de FabryAgalsidase alfaAgalsidase betaAnderson-Fabry diseaseEnzyme replacement therapyEvaluating enzyme replacement therapies for Anderson-Fabry disease : commentary on a recent reportinfo: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:UFRGSTEXT001090254.pdf.txt001090254.pdf.txtExtracted Texttext/plain20432http://www.lume.ufrgs.br/bitstream/10183/194775/2/001090254.pdf.txtc3ef25733d322e0b4dcb76397fef2eaeMD52ORIGINAL001090254.pdfTexto completo (inglês)application/pdf450827http://www.lume.ufrgs.br/bitstream/10183/194775/1/001090254.pdfddf034448bbcc0fa787a00dff2c22c81MD5110183/1947752023-06-16 03:32:15.326393oai:www.lume.ufrgs.br:10183/194775Repositório de PublicaçõesPUBhttps://lume.ufrgs.br/oai/requestopendoar:2023-06-16T06:32:15Repositório Institucional da UFRGS - Universidade Federal do Rio Grande do Sul (UFRGS)false
dc.title.pt_BR.fl_str_mv Evaluating enzyme replacement therapies for Anderson-Fabry disease : commentary on a recent report
title Evaluating enzyme replacement therapies for Anderson-Fabry disease : commentary on a recent report
spellingShingle Evaluating enzyme replacement therapies for Anderson-Fabry disease : commentary on a recent report
Giugliani, Roberto
Terapia de reposição de enzimas
Doença de Fabry
Agalsidase alfa
Agalsidase beta
Anderson-Fabry disease
Enzyme replacement therapy
title_short Evaluating enzyme replacement therapies for Anderson-Fabry disease : commentary on a recent report
title_full Evaluating enzyme replacement therapies for Anderson-Fabry disease : commentary on a recent report
title_fullStr Evaluating enzyme replacement therapies for Anderson-Fabry disease : commentary on a recent report
title_full_unstemmed Evaluating enzyme replacement therapies for Anderson-Fabry disease : commentary on a recent report
title_sort Evaluating enzyme replacement therapies for Anderson-Fabry disease : commentary on a recent report
author Giugliani, Roberto
author_facet Giugliani, Roberto
Westwood, Stephanie
Wellhoefer, Hartmann
Schenk, Jörn Magnus
Gurevich, Andrey
Kampmann, Christoph
author_role author
author2 Westwood, Stephanie
Wellhoefer, Hartmann
Schenk, Jörn Magnus
Gurevich, Andrey
Kampmann, Christoph
author2_role author
author
author
author
author
dc.contributor.author.fl_str_mv Giugliani, Roberto
Westwood, Stephanie
Wellhoefer, Hartmann
Schenk, Jörn Magnus
Gurevich, Andrey
Kampmann, Christoph
dc.subject.por.fl_str_mv Terapia de reposição de enzimas
Doença de Fabry
topic Terapia de reposição de enzimas
Doença de Fabry
Agalsidase alfa
Agalsidase beta
Anderson-Fabry disease
Enzyme replacement therapy
dc.subject.eng.fl_str_mv Agalsidase alfa
Agalsidase beta
Anderson-Fabry disease
Enzyme replacement therapy
description Anderson-Fabry disease (AFD) is a rare lysosomal storage disorder. Randomized controlled clinical trials (RCTs) are preferred as the highest category of evidence, but limited availability of robust evidence in rare diseases may necessitate the use of less rigorous evidence. An analysis of cohort studies of enzyme replacement therapies for AFD published in 2017 by El Dib and coworkers made treatment recommendations that contradict previously published findings from RCTs and a systematic Cochrane review. Our commentary outlines concerns regarding selection criteria and statistical methods with their analysis.
publishDate 2018
dc.date.issued.fl_str_mv 2018
dc.date.accessioned.fl_str_mv 2019-05-30T02:40:37Z
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info:eu-repo/semantics/other
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dc.identifier.uri.fl_str_mv http://hdl.handle.net/10183/194775
dc.identifier.issn.pt_BR.fl_str_mv 1415-4757
dc.identifier.nrb.pt_BR.fl_str_mv 001090254
identifier_str_mv 1415-4757
001090254
url http://hdl.handle.net/10183/194775
dc.language.iso.fl_str_mv eng
language eng
dc.relation.ispartof.pt_BR.fl_str_mv Genetics and molecular biology. Ribeirão Preto. vol. 41, no. 4 (Oct./Dec. 2018), p. 790-793.
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