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, Gurevich,Andrey, Kampmann,Christoph
Tipo de documento: Artigo
Idioma: eng
Título da fonte: Genetics and Molecular Biology
Texto Completo: http://old.scielo.br/scielo.php?script=sci_arttext&pid=S1415-47572018000500790
Resumo: Abstract 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 Evaluating enzyme replacement therapies for Anderson-Fabry disease: commentary on a recent reportAgalsidase alfaagalsidase betaAnderson-Fabry diseaseenzyme replacement therapyAbstract 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.Sociedade Brasileira de Genética2018-12-01info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersiontext/htmlhttp://old.scielo.br/scielo.php?script=sci_arttext&pid=S1415-47572018000500790Genetics and Molecular Biology v.41 n.4 2018reponame:Genetics and Molecular Biologyinstname:Sociedade Brasileira de Genética (SBG)instacron:SBG10.1590/1678-4685-gmb-2017-0345info:eu-repo/semantics/openAccessGiugliani,RobertoWestwood,StephanieWellhoefer,HartmannSchenk,JörnGurevich,AndreyKampmann,Christopheng2019-01-14T00:00:00Zoai:scielo:S1415-47572018000500790Revistahttp://www.gmb.org.br/ONGhttps://old.scielo.br/oai/scielo-oai.php||editor@gmb.org.br1678-46851415-4757opendoar:2019-01-14T00:00Genetics and Molecular Biology - Sociedade Brasileira de Genética (SBG)false
dc.title.none.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
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
Gurevich,Andrey
Kampmann,Christoph
author_role author
author2 Westwood,Stephanie
Wellhoefer,Hartmann
Schenk,Jörn
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
Gurevich,Andrey
Kampmann,Christoph
dc.subject.por.fl_str_mv Agalsidase alfa
agalsidase beta
Anderson-Fabry disease
enzyme replacement therapy
topic Agalsidase alfa
agalsidase beta
Anderson-Fabry disease
enzyme replacement therapy
description Abstract 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.none.fl_str_mv 2018-12-01
dc.type.driver.fl_str_mv info:eu-repo/semantics/article
dc.type.status.fl_str_mv info:eu-repo/semantics/publishedVersion
format article
status_str publishedVersion
dc.identifier.uri.fl_str_mv http://old.scielo.br/scielo.php?script=sci_arttext&pid=S1415-47572018000500790
url http://old.scielo.br/scielo.php?script=sci_arttext&pid=S1415-47572018000500790
dc.language.iso.fl_str_mv eng
language eng
dc.relation.none.fl_str_mv 10.1590/1678-4685-gmb-2017-0345
dc.rights.driver.fl_str_mv info:eu-repo/semantics/openAccess
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv text/html
dc.publisher.none.fl_str_mv Sociedade Brasileira de Genética
publisher.none.fl_str_mv Sociedade Brasileira de Genética
dc.source.none.fl_str_mv Genetics and Molecular Biology v.41 n.4 2018
reponame:Genetics and Molecular Biology
instname:Sociedade Brasileira de Genética (SBG)
instacron:SBG
instname_str Sociedade Brasileira de Genética (SBG)
instacron_str SBG
institution SBG
reponame_str Genetics and Molecular Biology
collection Genetics and Molecular Biology
repository.name.fl_str_mv Genetics and Molecular Biology - Sociedade Brasileira de Genética (SBG)
repository.mail.fl_str_mv ||editor@gmb.org.br
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