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

Author:

Ney Svenja1,Gertz Roman Johannes2,Pennig Lenhard2,Nies Richard J.1,Holtick Udo3,Völker Linus A.45ORCID,Wunderlich Gilbert6,Seuthe Katharina1,Hohmann Christopher1,Metze Clemens1,Nähle Claas Philip27,von Stein Jennifer1ORCID,Brüwer Monique1ORCID,ten Freyhaus Henrik1,Pfister Roman1ORCID

Affiliation:

1. Department III of Internal Medicine, Faculty of Medicine and University Hospital Cologne, University of Cologne, 50937 Cologne, Germany

2. Department of Radiology, Faculty of Medicine and University Hospital Cologne, University of Cologne, 50937 Cologne, Germany

3. Department I of Internal Medicine, Faculty of Medicine and University Hospital Cologne, University of Cologne, 50937 Cologne, Germany

4. Department II of Internal Medicine and Center for Molecular Medicine Cologne, Faculty of Medicine and University Hospital Cologne, University of Cologne, 50937 Cologne, Germany

5. Cologne Cluster of Excellence on Cellular Stress Responses in Ageing-Associated Diseases, 50923 Cologne, Germany

6. Department of Neurology and Center for Rare Diseases, Faculty of Medicine and University Hospital Cologne, University of Cologne, 50937 Cologne, Germany

7. Radiologische Allianz Hamburg, 20357 Hamburg, Germany

Abstract

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.

Funder

Pfizer. Linus A. Völker received research funding and consulting fees from Alexion, AstraZeneca, Bayer

Sanofi-Genzyme

Cologne Clinician Scientist Program (CCSP)/Faculty of Medicine/University of Cologne

Deutsche Forschungsgemeinschaft

Publisher

MDPI AG

Reference28 articles.

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