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Expert consensus on the monitoring of transthyretin amyloid cardiomyopathy

Garcia-Pavia, P; Bengel, F; Brito, D; Damy, T; Duca, F; Dorbala, S; Nativi-Nicolau, J; ... Elliott, PM; + view all (2021) Expert consensus on the monitoring of transthyretin amyloid cardiomyopathy. European Journal of Heart Failure , 23 (6) pp. 895-905. 10.1002/ejhf.2198. Green open access

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Abstract

Transthyretin amyloid cardiomyopathy (ATTR-CM) is a life-threatening condition with a heterogeneous clinical presentation. The recent availability of treatment for ATTR-CM has stimulated increased awareness of the disease and patient identification. Stratification of patients with ATTR-CM is critical for optimal management and treatment; however, monitoring disease progression is challenging and currently lacks best-practice guidance. In this report, experts with experience in treating amyloidosis and ATTR-CM developed consensus recommendations for monitoring the course of patients with ATTR-CM and proposed meaningful thresholds and frequency for specific parameters. A set of 11 measurable features across three separate domains were evaluated: (i) clinical and functional endpoints, (ii) biomarkers and laboratory markers, and (iii) imaging and electrocardiographic parameters. Experts recommended that one marker from each of the three domains provides the minimum requirements for assessing disease progression. Assessment of cardiac disease status should be part of a multiparametric evaluation in which progression, stability or improvement of other involved systems in transthyretin amyloidosis should also be considered. Additional data from placebo arms of clinical trials and future studies assessing ATTR-CM will help to elucidate, refine and define these and other measurements.

Type: Article
Title: Expert consensus on the monitoring of transthyretin amyloid cardiomyopathy
Open access status: An open access version is available from UCL Discovery
DOI: 10.1002/ejhf.2198
Publisher version: https://doi.org/10.1002/ejhf.2198
Language: English
Additional information: This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions.
Keywords: Science & Technology, Life Sciences & Biomedicine, Cardiac & Cardiovascular Systems, Cardiovascular System & Cardiology, Transthyretin amyloid cardiomyopathy, Monitoring tools, Heart failure, Amyloidosis, Laboratory markers, Cardiac imaging, CARDIOVASCULAR MAGNETIC-RESONANCE, CARDIAC TROPONIN-T, HEART-FAILURE, PROGNOSTIC VALUE, FUNCTIONAL CLASS, NATURAL-HISTORY, RENAL-FUNCTION, MANAGEMENT, DIAGNOSIS
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 Population Health Sciences > Institute of Cardiovascular Science
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > Institute of Cardiovascular Science > Clinical Science
URI: https://discovery.ucl.ac.uk/id/eprint/10140394
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