World Heart Federation Consensus on Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM).

Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive and fatal condition that requires early diagnosis, management, and specific treatment. The availability of new disease-modifying therapies has made successful treatment a reality. Transthyretin amyloid cardiomyopathy can be either age-r...

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Autores: Brito, Dulce, Albrecht, Fabiano Castro, de Arenaza, Diego Perez, Bart, Nicole, Better, Nathan, Carvajal-Juarez, Isabel, Conceição, Isabel, Damy, Thibaud, Dorbala, Sharmila, Fidalgo, Jean-Christophe, Garcia-Pavia, Pablo, Ge, Junbo, Gillmore, Julian D, Grzybowski, Jacek, Obici, Laura, Piñero, Daniel, Rapezzi, Claudio, Ueda, Mitsuharu, Pinto, Fausto J
Tipo de recurso: artículo
Fecha de publicación:2023
País:España
Institución:Instituto de Salud Carlos III (ISCIII)
Repositorio:Repisalud
Idioma:inglés
OAI Identifier:oai:repisalud.isciii.es:20.500.12105/19259
Acceso en línea:http://hdl.handle.net/20.500.12105/19259
Access Level:acceso abierto
Palabra clave:Amyloid Neuropathies, Familial
Cardiomyopathies
Humans
Consensus
Prealbumin
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spelling World Heart Federation Consensus on Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM).Brito, DulceAlbrecht, Fabiano Castrode Arenaza, Diego PerezBart, NicoleBetter, NathanCarvajal-Juarez, IsabelConceição, IsabelDamy, ThibaudDorbala, SharmilaFidalgo, Jean-ChristopheGarcia-Pavia, PabloGe, JunboGillmore, Julian DGrzybowski, JacekObici, LauraPiñero, DanielRapezzi, ClaudioUeda, MitsuharuPinto, Fausto JAmyloid Neuropathies, FamilialCardiomyopathiesHumansConsensusPrealbuminTransthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive and fatal condition that requires early diagnosis, management, and specific treatment. The availability of new disease-modifying therapies has made successful treatment a reality. Transthyretin amyloid cardiomyopathy can be either age-related (wild-type form) or caused by mutations in the TTR gene (genetic, hereditary forms). It is a systemic disease, and while the genetic forms may exhibit a variety of symptoms, a predominant cardiac phenotype is often present. This document aims to provide an overview of ATTR-CM amyloidosis focusing on cardiac involvement, which is the most critical factor for prognosis. It will discuss the available tools for early diagnosis and patient management, given that specific treatments are more effective in the early stages of the disease, and will highlight the importance of a multidisciplinary approach and of specialized amyloidosis centres. To accomplish these goals, the World Heart Federation assembled a panel of 18 expert clinicians specialized in TTR amyloidosis from 13 countries, along with a representative from the Amyloidosis Alliance, a patient advocacy group. This document is based on a review of published literature, expert opinions, registries data, patients' perspectives, treatment options, and ongoing developments, as well as the progress made possible via the existence of centres of excellence. From the patients' perspective, increasing disease awareness is crucial to achieving an early and accurate diagnosis. Patients also seek to receive care at specialized amyloidosis centres and be fully informed about their treatment and prognosis.ElsevierPfizerAstraZeneca20242024-05-0720232023-01-0120232023-01-01journal articlehttp://purl.org/coar/resource_type/c_6501VoRhttp://purl.org/coar/version/c_970fb48d4fbd8a85info:eu-repo/semantics/articleapplication/pdfhttp://hdl.handle.net/20.500.12105/19259reponame:Repisaludinstname:Instituto de Salud Carlos III (ISCIII)Inglésengopen accesshttp://purl.org/coar/access_right/c_abf2Attribution-NonCommercial-NoDerivatives 4.0 Internacionalhttp://creativecommons.org/licenses/by-nc-nd/4.0/info:eu-repo/semantics/openAccessoai:repisalud.isciii.es:20.500.12105/192592026-06-12T12:43:37Z
dc.title.none.fl_str_mv World Heart Federation Consensus on Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM).
title World Heart Federation Consensus on Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM).
spellingShingle World Heart Federation Consensus on Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM).
Brito, Dulce
Amyloid Neuropathies, Familial
Cardiomyopathies
Humans
Consensus
Prealbumin
title_short World Heart Federation Consensus on Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM).
title_full World Heart Federation Consensus on Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM).
title_fullStr World Heart Federation Consensus on Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM).
title_full_unstemmed World Heart Federation Consensus on Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM).
title_sort World Heart Federation Consensus on Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM).
dc.creator.none.fl_str_mv Brito, Dulce
Albrecht, Fabiano Castro
de Arenaza, Diego Perez
Bart, Nicole
Better, Nathan
Carvajal-Juarez, Isabel
Conceição, Isabel
Damy, Thibaud
Dorbala, Sharmila
Fidalgo, Jean-Christophe
Garcia-Pavia, Pablo
Ge, Junbo
Gillmore, Julian D
Grzybowski, Jacek
Obici, Laura
Piñero, Daniel
Rapezzi, Claudio
Ueda, Mitsuharu
Pinto, Fausto J
author Brito, Dulce
author_facet Brito, Dulce
Albrecht, Fabiano Castro
de Arenaza, Diego Perez
Bart, Nicole
Better, Nathan
Carvajal-Juarez, Isabel
Conceição, Isabel
Damy, Thibaud
Dorbala, Sharmila
Fidalgo, Jean-Christophe
Garcia-Pavia, Pablo
Ge, Junbo
Gillmore, Julian D
Grzybowski, Jacek
Obici, Laura
Piñero, Daniel
Rapezzi, Claudio
Ueda, Mitsuharu
Pinto, Fausto J
author_role author
author2 Albrecht, Fabiano Castro
de Arenaza, Diego Perez
Bart, Nicole
Better, Nathan
Carvajal-Juarez, Isabel
Conceição, Isabel
Damy, Thibaud
Dorbala, Sharmila
Fidalgo, Jean-Christophe
Garcia-Pavia, Pablo
Ge, Junbo
Gillmore, Julian D
Grzybowski, Jacek
Obici, Laura
Piñero, Daniel
Rapezzi, Claudio
Ueda, Mitsuharu
Pinto, Fausto J
author2_role author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
dc.contributor.none.fl_str_mv Pfizer
AstraZeneca
dc.subject.none.fl_str_mv Amyloid Neuropathies, Familial
Cardiomyopathies
Humans
Consensus
Prealbumin
topic Amyloid Neuropathies, Familial
Cardiomyopathies
Humans
Consensus
Prealbumin
description Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive and fatal condition that requires early diagnosis, management, and specific treatment. The availability of new disease-modifying therapies has made successful treatment a reality. Transthyretin amyloid cardiomyopathy can be either age-related (wild-type form) or caused by mutations in the TTR gene (genetic, hereditary forms). It is a systemic disease, and while the genetic forms may exhibit a variety of symptoms, a predominant cardiac phenotype is often present. This document aims to provide an overview of ATTR-CM amyloidosis focusing on cardiac involvement, which is the most critical factor for prognosis. It will discuss the available tools for early diagnosis and patient management, given that specific treatments are more effective in the early stages of the disease, and will highlight the importance of a multidisciplinary approach and of specialized amyloidosis centres. To accomplish these goals, the World Heart Federation assembled a panel of 18 expert clinicians specialized in TTR amyloidosis from 13 countries, along with a representative from the Amyloidosis Alliance, a patient advocacy group. This document is based on a review of published literature, expert opinions, registries data, patients' perspectives, treatment options, and ongoing developments, as well as the progress made possible via the existence of centres of excellence. From the patients' perspective, increasing disease awareness is crucial to achieving an early and accurate diagnosis. Patients also seek to receive care at specialized amyloidosis centres and be fully informed about their treatment and prognosis.
publishDate 2023
dc.date.none.fl_str_mv 2023
2023-01-01
2023
2023-01-01
2024
2024-05-07
dc.type.none.fl_str_mv journal article
http://purl.org/coar/resource_type/c_6501
VoR
http://purl.org/coar/version/c_970fb48d4fbd8a85
dc.type.openaire.fl_str_mv info:eu-repo/semantics/article
format article
dc.identifier.none.fl_str_mv http://hdl.handle.net/20.500.12105/19259
url http://hdl.handle.net/20.500.12105/19259
dc.language.none.fl_str_mv Inglés
eng
language_invalid_str_mv Inglés
language eng
dc.rights.none.fl_str_mv open access
http://purl.org/coar/access_right/c_abf2
Attribution-NonCommercial-NoDerivatives 4.0 Internacional
http://creativecommons.org/licenses/by-nc-nd/4.0/
dc.rights.openaire.fl_str_mv info:eu-repo/semantics/openAccess
rights_invalid_str_mv open access
http://purl.org/coar/access_right/c_abf2
Attribution-NonCommercial-NoDerivatives 4.0 Internacional
http://creativecommons.org/licenses/by-nc-nd/4.0/
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.publisher.none.fl_str_mv Elsevier
publisher.none.fl_str_mv Elsevier
dc.source.none.fl_str_mv reponame:Repisalud
instname:Instituto de Salud Carlos III (ISCIII)
instname_str Instituto de Salud Carlos III (ISCIII)
reponame_str Repisalud
collection Repisalud
repository.name.fl_str_mv
repository.mail.fl_str_mv
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score 15,811543

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