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Breakthrough advances enhancing care in ATTR amyloid cardiomyopathy

Porcari, Aldostefano; Sinagra, Gianfranco; Gillmore, Julian D; Fontana, Marianna; Hawkins, Philip N; (2024) Breakthrough advances enhancing care in ATTR amyloid cardiomyopathy. European Journal of Internal Medicine 10.1016/j.ejim.2024.01.001. (In press). Green open access

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Abstract

Transthyretin amyloid cardiomyopathy (ATTR-CM) has been traditionally considered a rare and inexorably fatal condition. ATTR-CM now is an increasingly recognized cause of heart failure (HF) and mortality worldwide with effective pharmacological treatments. Advances in non-invasive diagnosis, coupled with the development of effective treatments, have transformed the diagnosis of ATTR-CM, which is now possible without recourse to endomyocardial biopsy in ≈70 % of cases. Many patients are now diagnosed at an earlier stage. Echocardiography and cardiac magnetic resonance have enabled identification of patients with possible ATTR-CM and more accurate prognostic stratification. Although radionuclide scintigraphy with 'bone' tracers has an established diagnostic value, the diagnostic performance of the bone tracers validated for non-invasive confirmation of ATTR-CM may not be equal. Characterising the wider clinical phenotype of patients with ATTR-CM has enabled identification of features with potential for earlier diagnosis such as carpal tunnel syndrome. Therapies able to slow or halt ATTR-CM progression and increase survival are now available and there is also evidence that patients may benefit from specific conventional HF medications. Cutting-edge research in the field of antibody-mediated removal of ATTR deposits compellingly suggest that ATTR-CM is a truly reversible disorder, bringing hope for patients even with advanced disease. A wide horizon of possibilities is unfolding and awaits discovery.

Type: Article
Title: Breakthrough advances enhancing care in ATTR amyloid cardiomyopathy
Location: Netherlands
Open access status: An open access version is available from UCL Discovery
DOI: 10.1016/j.ejim.2024.01.001
Publisher version: http://dx.doi.org/10.1016/j.ejim.2024.01.001
Language: English
Additional information: © 2024 The Author(s). Published by Elsevier B.V. on behalf of European Federation of Internal Medicine under a Creative Commons license (http://creativecommons.org/licenses/by-nc-nd/4.0/).
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/10185332
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