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A simple core dataset and disease severity score for hereditary transthyretin (ATTRv) amyloidosis

Damy, T; Conceição, I; García-Pavía, P; Gillmore, J; Jandhyala, R; Sabbat, J; Wixner, J; (2021) A simple core dataset and disease severity score for hereditary transthyretin (ATTRv) amyloidosis. Amyloid 10.1080/13506129.2021.1931099. (In press). Green open access

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Abstract

BACKGROUND: Hereditary transthyretin (ATTRv) amyloidosis is a progressive multisystemic disease of adult-onset that arises from an inherited mutation in the transthyretin gene. Currently available disease severity and progression evaluation tools only cover one single organ or system, impacting data collection uniformity and its use in clinical settings. METHODS: The Jandhyala Method, including a systematic literature review and SMART interviews, was used to observe expert opinion from eight leaders in the treatment of ATTRv across Europe. The aim was to propose a multidisciplinary core dataset (CD) and disease severity scoring (DSS) tools. RESULTS: The multidisciplinary team of experts identified 140 indicators that form part of the standard diagnostic and monitoring practice (SDMP) and should be collected as the ATTRv CD. Thirty-one (22%) of these indicators informed disease severity and comprised the ATTRv DSS, whilst 25 (18%) were deemed to monitor disease progression. CONCLUSIONS: The resulting CD and DSS have different purposes. The ATTRv CD supports the collection of high-quality data for clinical research, whereas the ATTRv DSS can be rapidly conducted in a clinical setting and aid patient management.

Type: Article
Title: A simple core dataset and disease severity score for hereditary transthyretin (ATTRv) amyloidosis
Location: England
Open access status: An open access version is available from UCL Discovery
DOI: 10.1080/13506129.2021.1931099
Publisher version: https://doi.org/10.1080/13506129.2021.1931099
Language: English
Additional information: 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/),
Keywords: ATTRv, core dataset, disease progression, disease severity score, hereditary transthyretin amyloidosis
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine > Inflammation
URI: https://discovery.ucl.ac.uk/id/eprint/10128918
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