Survival in transthyretin familial amyloid polyneuropathy : a review

Detalhes bibliográficos
Autor(a) principal: Inês, Mónica
Data de Publicação: 2019
Outros Autores: Costa, João
Tipo de documento: Artigo
Idioma: eng
Título da fonte: Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
Texto Completo: http://hdl.handle.net/10451/40155
Resumo: © 2019 Costa J. This article is distributed under the terms of the Creative Commons Attribution 4.0 International License
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spelling Survival in transthyretin familial amyloid polyneuropathy : a reviewAmyloidosisTransthyretin familial amyloid polyneuropathyLiver transplantationTafamidisSurvival© 2019 Costa J. This article is distributed under the terms of the Creative Commons Attribution 4.0 International LicenseTransthyretin-associated familial amyloid polyneuropathy (TTR-FAP) is a neurological disease that affects severely patients and their families and caregivers over generations. It is a rare, progressive, and if untreated fatal autosomal dominant hereditary disorder. The disease may affect multiple organ systems and if untreated progress rapidly to death. TTR-FAP affects nearly 10,000 people worldwide, with known endemic regions in Portugal, Sweden, and Japan. Until recently only liver transplantation and tafamidis were treatment options across several world regions. Despite the worldwide use of these disease-modifying treatments to delay disease progression, challenges in clinical assessment and management remain because of disease heterogeneity, phenotypic diversity, small patient populations, incomplete natural history and uncertainty of treatment effect in survival. The two new treatment options (inotersen and patisiran) appear to provide important benefits for patients, based on clinical trials short-term evidence. In this review, we discuss the disease natural survival course and currently available treatments impact on survival. We also discuss the importance of treatment choice (and or sequence of treatments) to maximize survival, while preserving the patient’s health-related quality of life.Mónica Inês is PhD candidate and Outcomes & Evidence full-time employee of Pfizer in Portugal and hold stock and/or stock options.Sciaccess Inc.Repositório da Universidade de LisboaInês, MónicaCosta, João2019-11-18T14:22:40Z20192019-01-01T00:00:00Zinfo:eu-repo/semantics/publishedVersioninfo:eu-repo/semantics/articleapplication/pdfhttp://hdl.handle.net/10451/40155engJ Neurol Neuromed (2019) 4(1): 22-252572-942Xinfo:eu-repo/semantics/openAccessreponame:Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)instname:Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informaçãoinstacron:RCAAP2023-11-08T16:39:17Zoai:repositorio.ul.pt:10451/40155Portal AgregadorONGhttps://www.rcaap.pt/oai/openaireopendoar:71602024-03-19T21:53:52.459804Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) - Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informaçãofalse
dc.title.none.fl_str_mv Survival in transthyretin familial amyloid polyneuropathy : a review
title Survival in transthyretin familial amyloid polyneuropathy : a review
spellingShingle Survival in transthyretin familial amyloid polyneuropathy : a review
Inês, Mónica
Amyloidosis
Transthyretin familial amyloid polyneuropathy
Liver transplantation
Tafamidis
Survival
title_short Survival in transthyretin familial amyloid polyneuropathy : a review
title_full Survival in transthyretin familial amyloid polyneuropathy : a review
title_fullStr Survival in transthyretin familial amyloid polyneuropathy : a review
title_full_unstemmed Survival in transthyretin familial amyloid polyneuropathy : a review
title_sort Survival in transthyretin familial amyloid polyneuropathy : a review
author Inês, Mónica
author_facet Inês, Mónica
Costa, João
author_role author
author2 Costa, João
author2_role author
dc.contributor.none.fl_str_mv Repositório da Universidade de Lisboa
dc.contributor.author.fl_str_mv Inês, Mónica
Costa, João
dc.subject.por.fl_str_mv Amyloidosis
Transthyretin familial amyloid polyneuropathy
Liver transplantation
Tafamidis
Survival
topic Amyloidosis
Transthyretin familial amyloid polyneuropathy
Liver transplantation
Tafamidis
Survival
description © 2019 Costa J. This article is distributed under the terms of the Creative Commons Attribution 4.0 International License
publishDate 2019
dc.date.none.fl_str_mv 2019-11-18T14:22:40Z
2019
2019-01-01T00:00:00Z
dc.type.status.fl_str_mv info:eu-repo/semantics/publishedVersion
dc.type.driver.fl_str_mv info:eu-repo/semantics/article
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status_str publishedVersion
dc.identifier.uri.fl_str_mv http://hdl.handle.net/10451/40155
url http://hdl.handle.net/10451/40155
dc.language.iso.fl_str_mv eng
language eng
dc.relation.none.fl_str_mv J Neurol Neuromed (2019) 4(1): 22-25
2572-942X
dc.rights.driver.fl_str_mv info:eu-repo/semantics/openAccess
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.publisher.none.fl_str_mv Sciaccess Inc.
publisher.none.fl_str_mv Sciaccess Inc.
dc.source.none.fl_str_mv reponame:Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
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