@article{a7ba4cb62f01457d88c64f136e98541e,
title = "Expert consensus on the monitoring of transthyretin amyloid cardiomyopathy",
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.",
keywords = "Amyloidosis, Cardiac imaging, Heart failure, Laboratory markers, Monitoring tools, Transthyretin amyloid cardiomyopathy",
author = "Pablo Garcia-Pavia and Frank Bengel and Dulce Brito and Thibaud Damy and Franz Duca and Sharmila Dorbala and Jose Nativi-Nicolau and Laura Obici and Claudio Rapezzi and Yoshiki Sekijima and Elliott, {Perry M.}",
note = "Funding Information: Medical writing and editorial assistance were provided by Aisling Koning and Kyle Lambe of Synergy Medical Communications, London, UK, and was supported by Pfizer. Funding Information: Editorial support for the development of this manuscript was funded by Pfizer. Authors were not paid for their work developing the manuscript and the views and opinions expressed are solely those of the authors. Funding Information: : P.G.‐P. has received speaking fees from Pfizer, Eidos, Alnylam and Akcea; consulting fees from Pfizer, Eidos, Neuroinmmune, Alnylam, AstraZeneca, Prothena and Akcea; research/educational support to his institution from Pfizer, Eidos and Alnylam; and grant support by the Instituto de Salud Carlos III (PI20/01379). F.B. has received personal fees from Pfizer. D.B. has received consultancy fees from Pfizer, Amgen, Boehringer Ingelheim and Novartis; speaker fees from Pfizer; and institutional funding for clinical trials from Amgen, Boehringer Ingelheim and Novartis. T.D. has received a grant and personal fees from Pfizer. F.D. received speaker as well as consulting fees and congress support from Bayer, Novartis, AOP, Alnylam, and Pfizer; and institutional research funding from Pfizer. S.D. has received research grants from Pfizer and GE Healthcare; and consultant fees from Pfizer and GE Healthcare. J.N.‐N. has received institutional funding for clinical trials from Pfizer, Akcea and Eidos; educational grants from Pfizer; and consultancy fees from Pfizer, Eidos, Akcea, and Alnylam. L.O. has received personal consultancy and speaker honoraria from Pfizer, Alnylam and Akcea. C.R. discloses no conflict of interest. Y.S. has received grants, consultancy and speaker honoraria from Pfizer and Alnylam. P.M.E. has received consultancy fees from Pfizer, Alnylam, MyoKardia and Sanofi Genzyme and an unrestricted educational grant from Pfizer. Conflict of interest Publisher Copyright: {\textcopyright} 2021 European Society of Cardiology.",
year = "2021",
doi = "10.1002/ejhf.2198",
language = "English",
journal = "European Journal of Heart Failure",
issn = "1388-9842",
publisher = "John Wiley & Sons, Ltd",
}