Multiparametric Monitoring of Disease Progression in Contemporary Patients with Wild-Type Transthyretin Amyloid Cardiomyopathy Initiating Tafamidis Treatment

Svenja Ney; Roman Johannes Gertz; Lenhard Pennig; Richard J. Nies; Udo Holtick; Linus A. Völker; Gilbert Wunderlich; Katharina Seuthe; Christopher Hohmann; Clemens Metze; Claas Philip Nähle; Jennifer von Stein; Monique Brüwer; Henrik ten Freyhaus; Roman Pfister

doi:10.3390/jcm13010284

Journal of Clinical Medicine (Jan 2024)

Multiparametric Monitoring of Disease Progression in Contemporary Patients with Wild-Type Transthyretin Amyloid Cardiomyopathy Initiating Tafamidis Treatment

Svenja Ney,
Roman Johannes Gertz,
Lenhard Pennig,
Richard J. Nies,
Udo Holtick,
Linus A. Völker,
Gilbert Wunderlich,
Katharina Seuthe,
Christopher Hohmann,
Clemens Metze,
Claas Philip Nähle,
Jennifer von Stein,
Monique Brüwer,
Henrik ten Freyhaus,
Roman Pfister

Affiliations

Svenja Ney: Department III of Internal Medicine, Faculty of Medicine and University Hospital Cologne, University of Cologne, 50937 Cologne, Germany
Roman Johannes Gertz: Department of Radiology, Faculty of Medicine and University Hospital Cologne, University of Cologne, 50937 Cologne, Germany
Lenhard Pennig: Department of Radiology, Faculty of Medicine and University Hospital Cologne, University of Cologne, 50937 Cologne, Germany
Richard J. Nies: Department III of Internal Medicine, Faculty of Medicine and University Hospital Cologne, University of Cologne, 50937 Cologne, Germany
Udo Holtick: Department I of Internal Medicine, Faculty of Medicine and University Hospital Cologne, University of Cologne, 50937 Cologne, Germany
Linus A. Völker: Department II of Internal Medicine and Center for Molecular Medicine Cologne, Faculty of Medicine and University Hospital Cologne, University of Cologne, 50937 Cologne, Germany
Gilbert Wunderlich: Department of Neurology and Center for Rare Diseases, Faculty of Medicine and University Hospital Cologne, University of Cologne, 50937 Cologne, Germany
Katharina Seuthe: Department III of Internal Medicine, Faculty of Medicine and University Hospital Cologne, University of Cologne, 50937 Cologne, Germany
Christopher Hohmann: Department III of Internal Medicine, Faculty of Medicine and University Hospital Cologne, University of Cologne, 50937 Cologne, Germany
Clemens Metze: Department III of Internal Medicine, Faculty of Medicine and University Hospital Cologne, University of Cologne, 50937 Cologne, Germany
Claas Philip Nähle: Department of Radiology, Faculty of Medicine and University Hospital Cologne, University of Cologne, 50937 Cologne, Germany
Jennifer von Stein: Department III of Internal Medicine, Faculty of Medicine and University Hospital Cologne, University of Cologne, 50937 Cologne, Germany
Monique Brüwer: Department III of Internal Medicine, Faculty of Medicine and University Hospital Cologne, University of Cologne, 50937 Cologne, Germany
Henrik ten Freyhaus: Department III of Internal Medicine, Faculty of Medicine and University Hospital Cologne, University of Cologne, 50937 Cologne, Germany
Roman Pfister: Department III of Internal Medicine, Faculty of Medicine and University Hospital Cologne, University of Cologne, 50937 Cologne, Germany

DOI: https://doi.org/10.3390/jcm13010284
Journal volume & issue: Vol. 13, no. 1
p. 284

Abstract

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Background: Recently, a disease modifying therapy has become available for transthyretin amyloid cardiomyopathy (ATTR-CM). A validated monitoring concept of treatment is lacking, but a current expert consensus recommends three clinical domains (clinical, biomarker and ECG/imaging) assessed by several measurable features to define disease progression. Methods: We retrospectively analyzed data of wild-type ATTR-CM patients initiating tafamidis therapy assessed within our local routine protocol at baseline and 6-months follow-up with respect to the frequency of values beyond the proposed thresholds defining disease progression. Additionally, associations of cardiac magnetic resonance (CMR) tomography with clinical domains were examined within a subgroup. Results: Sixty-two ATTR-CM patients were included (88.7% male, mean age 79 years). In total, 16.1% of patients had progress in the clinical and functional domain, 33.9% in the biomarker domain and 43.5% in the imaging/electrocardiography (ECG) domain, with the latter driven by deterioration of the diastolic dysfunction grade and global longitudinal strain. In total, 35.5% of patients showed progress in none, 35.5% in one, 29.0% in two and no patient in three domains, the latter indicating overall disease progression. A subgroup analysis of twenty-two patients with available baseline and follow-up CMR data revealed an increase in CMR-based extracellular volume by more than 5% in 18.2% of patients, with no significant correlation with progress in one of the clinical domains. Conclusions: We provide first frequency estimates of the markers of disease progression according to a recent expert consensus statement, which might help refine the multiparametric monitoring concept in patients with ATTR-CM.

Published in Journal of Clinical Medicine

ISSN: 2077-0383 (Online)
Publisher: MDPI AG
Country of publisher: Switzerland
LCC subjects: Medicine
Website: http://www.mdpi.com/journal/jcm

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