Publicação
Assessment of patients with hereditary transthyretin amyloidosis : understanding the impact of management and disease progression
| Resumo: | Timely diagnosis of hereditary variant transthyretin (ATTRv) amyloidosis is critical for appropriate treatment and optimal outcomes. Significant differences are seen between patients receiving treatment and those who are not, though disease progression may continue despite treatment in some patients. Healthcare professionals caring for patients with ATTRv amyloidosis therefore need reliable ongoing assessments to understand the continuing course of disease and make appropriate treatment choices on an individual basis. Various signs and symptoms experienced by patients may be evaluated as indicators of disease progression, though there is currently no validated score that can be used for such ongoing assessment. Recognizing this situation, a group of clinicians highly experienced in ATTR amyloidosis developed an approach to understand and define disease progression in diagnosed and treated patients with ATTRv amyloidosis. The suggested approach is based on the recognition of distinct phenotypes which may usefully inform the particular tools, tests and investigations that are most likely to be appropriate for individual patients. It is aimed at implementing appropriate and ongoing assessment of patients being treated for ATTRv amyloidosis, such that the effectiveness of management can be usefully assessed throughout the course of disease and management can be tailored according to the patient’s requirements. |
|---|---|
| Autores principais: | Conceição, isabel |
| Outros Autores: | Coelho, Teresa; Rapezzi, Claudio; Parman, Yeşim; Obici, Laura; Galán, Lucía; Rousseau, Antoine |
| Assunto: | ATTR Amyloidosis Follow-up Hereditary Monitoring Progression Transthyretin |
| Ano: | 2019 |
| País: | Portugal |
| Tipo de documento: | artigo |
| Tipo de acesso: | acesso aberto |
| Instituição associada: | Universidade de Lisboa |
| Idioma: | inglês |
| Origem: | Repositório da Universidade de Lisboa |
| _version_ | 1866810984934408192 |
|---|---|
| author | Conceição, isabel |
| author2 | Coelho, Teresa Rapezzi, Claudio Parman, Yeşim Obici, Laura Galán, Lucía Rousseau, Antoine |
| author2_role | author author author author author author |
| author_facet | Conceição, isabel Coelho, Teresa Rapezzi, Claudio Parman, Yeşim Obici, Laura Galán, Lucía Rousseau, Antoine |
| author_role | author |
| contributor_name_str_mv | Repositório Científico de Acesso Aberto da ULisboa |
| country_str | PT |
| creators_json_txt | [{\"Person.name\":\"Conceição, isabel\",\"Person.identifier.orcid\":\"0000-0003-0934-9631\"},{\"Person.name\":\"Coelho, Teresa\"},{\"Person.name\":\"Rapezzi, Claudio\"},{\"Person.name\":\"Parman, Yeşim\"},{\"Person.name\":\"Obici, Laura\"},{\"Person.name\":\"Galán, Lucía\"},{\"Person.name\":\"Rousseau, Antoine\"}] |
| datacite.contributors.contributor.contributorName.fl_str_mv | Repositório Científico de Acesso Aberto da ULisboa |
| datacite.creators.creator.creatorName.fl_str_mv | Conceição, isabel Coelho, Teresa Rapezzi, Claudio Parman, Yeşim Obici, Laura Galán, Lucía Rousseau, Antoine |
| datacite.date.Accepted.fl_str_mv | 2019-01-01T00:00:00Z |
| datacite.date.available.fl_str_mv | 2021-02-19T14:32:38Z |
| datacite.date.embargoed.fl_str_mv | 2021-02-19T14:32:38Z |
| datacite.rights.fl_str_mv | http://purl.org/coar/access_right/c_abf2 |
| datacite.subjects.subject.fl_str_mv | ATTR Amyloidosis Follow-up Hereditary Monitoring Progression Transthyretin |
| datacite.titles.title.fl_str_mv | Assessment of patients with hereditary transthyretin amyloidosis : understanding the impact of management and disease progression |
| dc.contributor.none.fl_str_mv | Repositório Científico de Acesso Aberto da ULisboa |
| dc.creator.none.fl_str_mv | Conceição, isabel Coelho, Teresa Rapezzi, Claudio Parman, Yeşim Obici, Laura Galán, Lucía Rousseau, Antoine |
| dc.date.Accepted.fl_str_mv | 2019-01-01T00:00:00Z |
| dc.date.available.fl_str_mv | 2021-02-19T14:32:38Z |
| dc.date.embargoed.fl_str_mv | 2021-02-19T14:32:38Z |
| dc.format.none.fl_str_mv | application/pdf |
| dc.identifier.none.fl_str_mv | http://hdl.handle.net/10451/46443 |
| dc.language.none.fl_str_mv | eng |
| dc.publisher.none.fl_str_mv | Taylor & Francis Group |
| dc.rights.cclincense.fl_str_mv | http://creativecommons.org/licenses/by-nc-nd/4.0/ |
| dc.rights.none.fl_str_mv | http://purl.org/coar/access_right/c_abf2 |
| dc.subject.none.fl_str_mv | ATTR Amyloidosis Follow-up Hereditary Monitoring Progression Transthyretin |
| dc.title.fl_str_mv | Assessment of patients with hereditary transthyretin amyloidosis : understanding the impact of management and disease progression |
| dc.type.none.fl_str_mv | http://purl.org/coar/resource_type/c_6501 |
| description | Timely diagnosis of hereditary variant transthyretin (ATTRv) amyloidosis is critical for appropriate treatment and optimal outcomes. Significant differences are seen between patients receiving treatment and those who are not, though disease progression may continue despite treatment in some patients. Healthcare professionals caring for patients with ATTRv amyloidosis therefore need reliable ongoing assessments to understand the continuing course of disease and make appropriate treatment choices on an individual basis. Various signs and symptoms experienced by patients may be evaluated as indicators of disease progression, though there is currently no validated score that can be used for such ongoing assessment. Recognizing this situation, a group of clinicians highly experienced in ATTR amyloidosis developed an approach to understand and define disease progression in diagnosed and treated patients with ATTRv amyloidosis. The suggested approach is based on the recognition of distinct phenotypes which may usefully inform the particular tools, tests and investigations that are most likely to be appropriate for individual patients. It is aimed at implementing appropriate and ongoing assessment of patients being treated for ATTRv amyloidosis, such that the effectiveness of management can be usefully assessed throughout the course of disease and management can be tailored according to the patient’s requirements. |
| dirty | 0 |
| eu_rights_str_mv | openAccess |
| format | article |
| fulltext.url.fl_str_mv | https://repositorio.ulisboa.pt/bitstreams/1c87f9d9-3421-4b7e-8acf-42f63962c1f3/download |
| id | ul_706d330f2aebbce5e6aaa2bb89e9357a |
| identifier.url.fl_str_mv | http://hdl.handle.net/10451/46443 |
| instacron_str | ul |
| institution | Universidade de Lisboa |
| instname_str | Universidade de Lisboa |
| language | eng |
| network_acronym_str | ul |
| network_name_str | Repositório da Universidade de Lisboa |
| oai_identifier_str | oai:repositorio.ulisboa.pt:10451/46443 |
| organization_str_mv | urn:organizationAcronym:ul |
| person_str_mv | Conceição, isabel Conceição, isabel http://orcid.org/0000-0003-0934-9631 0000-0003-0934-9631 Coelho, Teresa Rapezzi, Claudio Parman, Yeşim Obici, Laura Galán, Lucía Rousseau, Antoine |
| publishDate | 2019 |
| publisher.none.fl_str_mv | Taylor & Francis Group |
| reponame_str | Repositório da Universidade de Lisboa |
| repository_id_str | urn:repositoryAcronym:ul |
| service_str_mv | urn:repositoryAcronym:ul |
| spelling | engTaylor & Francis Grouppt_PTTimely diagnosis of hereditary variant transthyretin (ATTRv) amyloidosis is critical for appropriate treatment and optimal outcomes. Significant differences are seen between patients receiving treatment and those who are not, though disease progression may continue despite treatment in some patients. Healthcare professionals caring for patients with ATTRv amyloidosis therefore need reliable ongoing assessments to understand the continuing course of disease and make appropriate treatment choices on an individual basis. Various signs and symptoms experienced by patients may be evaluated as indicators of disease progression, though there is currently no validated score that can be used for such ongoing assessment. Recognizing this situation, a group of clinicians highly experienced in ATTR amyloidosis developed an approach to understand and define disease progression in diagnosed and treated patients with ATTRv amyloidosis. The suggested approach is based on the recognition of distinct phenotypes which may usefully inform the particular tools, tests and investigations that are most likely to be appropriate for individual patients. It is aimed at implementing appropriate and ongoing assessment of patients being treated for ATTRv amyloidosis, such that the effectiveness of management can be usefully assessed throughout the course of disease and management can be tailored according to the patient’s requirements.application/pdfpt_PTAssessment of patients with hereditary transthyretin amyloidosis : understanding the impact of management and disease progressionPersonalConceição, isabelDSpacehttp://dspace.org/items/52ba30d8-6cbf-424d-b334-4d06a2ecc6e5DSpacehttp://dspace.org/items/52ba30d8-6cbf-424d-b334-4d06a2ecc6e5CONCEICAOISABELORCIDhttp://orcid.org0000-0003-0934-9631Scopus Author IDhttps://www.scopus.com6701694419Coelho, TeresaRapezzi, ClaudioParman, YeşimObici, LauraGalán, LucíaRousseau, AntoineHostingInstitutionOrganizationalRepositório Científico de Acesso Aberto da ULisboae-mailmailto:repositorio@reitoria.ulisboa.ptrepositorio@reitoria.ulisboa.ptISSNIsPartOf1350-6129ISSNIsPartOf1744-2818DOIIsPartOf10.1080/13506129.2019.16273122021-02-19T14:32:38Z20192019-01-01T00:00:00ZHandlehttp://hdl.handle.net/10451/46443http://purl.org/coar/access_right/c_abf2open accessATTRAmyloidosisFollow-upHereditaryMonitoringProgressionTransthyretin1210423 bytesliteraturehttp://purl.org/coar/resource_type/c_6501journal article2019http://creativecommons.org/licenses/by-nc-nd/4.0/http://purl.org/coar/access_right/c_abf2application/pdffulltexthttps://repositorio.ulisboa.pt/bitstreams/1c87f9d9-3421-4b7e-8acf-42f63962c1f3/downloadAmyloid263103111 |
| spellingShingle | Assessment of patients with hereditary transthyretin amyloidosis : understanding the impact of management and disease progression Conceição, isabel ATTR Amyloidosis Follow-up Hereditary Monitoring Progression Transthyretin |
| status | SINGLETON |
| subject.fl_str_mv | ATTR Amyloidosis Follow-up Hereditary Monitoring Progression Transthyretin |
| title | Assessment of patients with hereditary transthyretin amyloidosis : understanding the impact of management and disease progression |
| title_full | Assessment of patients with hereditary transthyretin amyloidosis : understanding the impact of management and disease progression |
| title_fullStr | Assessment of patients with hereditary transthyretin amyloidosis : understanding the impact of management and disease progression |
| title_full_unstemmed | Assessment of patients with hereditary transthyretin amyloidosis : understanding the impact of management and disease progression |
| title_short | Assessment of patients with hereditary transthyretin amyloidosis : understanding the impact of management and disease progression |
| title_sort | Assessment of patients with hereditary transthyretin amyloidosis : understanding the impact of management and disease progression |
| topic | ATTR Amyloidosis Follow-up Hereditary Monitoring Progression Transthyretin |
| topic_facet | ATTR Amyloidosis Follow-up Hereditary Monitoring Progression Transthyretin |
| url | http://hdl.handle.net/10451/46443 |
| visible | 1 |