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  • Expert consensus on the mon...
    Garcia‐Pavia, Pablo; Bengel, Frank; Brito, Dulce; Damy, Thibaud; Duca, Franz; Dorbala, Sharmila; Nativi‐Nicolau, Jose; Obici, Laura; Rapezzi, Claudio; Sekijima, Yoshiki; Elliott, Perry M.

    European journal of heart failure, June 2021, Letnik: 23, Številka: 6
    Journal Article

    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. This consensus document from an international expert panel recommends a set of clinically feasible tools for the long‐term monitoring of patients with transthyretin amyloid cardiomyopathy (ATTR‐CM), including meaningful thresholds for defining disease progression and the frequency of measurements. 6MWT, 6‐min walk test; ECG, electrocardiogram; EQ‐5D, EuroQol five dimensions; GLS, global longitudinal strain; HF, heart failure; KCCQ, Kansas City Cardiomyopathy Questionnaire; LV, left ventricular; LVEF, LV, left ventricular ejection fraction; NAC, UK National Amyloidosis Centre; NT‐proBNP, N‐terminal pro‐B‐type natriuretic peptide; NYHA, New York Heart Association; QoL, quality of life.