AbstractTimely diagnosis of hereditary variant transthyretin (ATTRv) amyloidosis is critical for appropriate treatment and optimal outcomes. Significant differences are seen between patients receiv..

Antoine Rousseau

Claudio Rapezzi

Isabel Conceição

Laura Obici

Lucía Galán

Teresa Coelho

Yeşim Parman

Amyloid

English

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

Rousseau, Antoine

Rapezzi, Claudio

Obici, Laura

Galan, Lucia

Parman, Yesim

Conceicao, Isabel

Coelho, Teresa

İstanbul Üniversitesi Açık Erişim Sistemi

Assessment of patients with hereditary transthyretin amyloidosis - understanding the impact of management and disease progression

Open Access Repository

Assessment of patients with hereditary transthyretin amyloidosis – understanding the impact of management and disease progression

© 2019 The Author(s). Published by Informa UK Limited, trading as Taylor & Francis Group.This is an Open Access article distributed under the terms of the Creative Commons Attribution-NonCommercial-NoDerivatives License (http://creativecommons.org/licenses/by-nc-nd/4.0/),which permits non-commercial re-use, distribution, and reproduction in any medium, provided the original work is properly cited, and is not altered, transformed, or built upon inany way.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.info:eu-repo/semantics/publishedVersio

Conceição, isabel

Parman, Yeşim

Galán, Lucía

Universidade de Lisboa: Repositório.UL

Assessment of patients with hereditary transthyretin amyloidosis : understanding the impact of management and disease progression

Conceicao I.

Coelho T.

Rapezzi C.

Parman Y.

Obici L.

Galan L.

Rousseau A.

Archivio istituzionale della ricerca - Università di Ferrara

Assessment of patients with hereditary transthyretin amyloidosis–understanding the impact of management and disease progression

https://iris.unife.it/bitstream/11392/2416407/1/Assessment%20of%20patients%20with%20hereditary%20transthyretin%20amyloidosis%20understanding%20the%20impact%20of%20management%20and%20disease%20progression.pdf

Assessment of patients with hereditary transthyretin amyloidosis – understanding the impact of management and disease progression

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İstanbul Üniversitesi Açık Erişim Sistemi

Open Access Repository

Universidade de Lisboa: Repositório.UL

Archivio istituzionale della ricerca - Università di Ferrara