• Thibaud Damy, Isabel Conceição, Pablo García-Pavía, Julian Gillmore, Ravi Jandhyala, Jan Sabbat, Jonas Wixner, and Teresa Coelho.
• Department of Cardiology, Referral Center for Cardiac Amyloidosis, GRC Amyloid Research Institute, DHU A-TVB, APHP CHU Henri Mondor and Université Paris Est Créteil, Créteil, France.
• Amyloid. 2021 Sep 1; 28 (3): 189-198.

BackgroundHereditary 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.MethodsThe 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.ResultsThe 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.ConclusionsThe 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.

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