Myocardial Amyloid Burden in Transthyretin Amyloidosis

作者：Awais A. Sheikh, Anouk Achten, Alberto Aimo, Yousuf Razvi, Josephine Mansell, Muhammad Umaid Rauf, Aldostefano Porcari, Rishi K Patel, Lucia Venneri, Ana Martinez–Naharro, Carol Jane Whelan, Cristina C Quarta, Ruta Virsinskaite, D. Barak, Ashutosh D. Wechalekar, Helen Jane Lachmann, Daniel S. Knight, Tushar Kotecha, Peter Kellman, Charlotte Manisty, James Moon, Michele Emdin, Scott David Solomon, Philip Nigel Hawkins, Julian David Gillmore, Marianna Fontana · 发表于：Journal of the American College of Cardiology · 年份：2025 · DOI：10.1016/j.jacc.2025.10.054 · 被引用次数：15 · 研究领域：Amyloidosis: Diagnosis, Treatment, Outcomes、Parathyroid Disorders and Treatments、Pericarditis and Cardiac Tamponade

BACKGROUND: Stabilizers/silencers limit new transthyretin amyloid formation, whereas emerging agents aim to clear existing deposits. Cardiovascular magnetic resonance (CMR) extracellular volume (ECV) reflects myocardial amyloid and may provide a quantitative framework for therapeutic planning OBJECTIVES: The aim was to define calibrated ECV thresholds, evaluate their diagnostic and prognostic value, and explore how CMR-ECV could provide a quantitative framework for disease staging and therapeutic planning. METHODS: We studied 1,541 subjects undergoing CMR for transthyretin amyloidosis (ATTR) classified as TTR-variant carriers (n = 123), extracardiac ATTR (n = 41), early-stage ATTR-CM (n = 70), or overt ATTR-CM (n = 1,308). The endpoint was all-cause mortality. RESULTS: ECV was similar in carriers and extracardiac ATTR but rose from early-stage to ATTR-cardiomyopathy (CM). Associations with biomarkers, National Amyloidosis Centre (NAC) stage, Perugini grade, and echocardiographic measures were modest, with wide overlap. Diagnostic performance was excellent: ECV <30% excluded and ≥40% confirmed cardiac involvement, whereas 30% to 39% indicated early infiltration. Over a median follow-up of 2.8 years (IQR: 1.4-4.3 years), 612 patients (40%) died. Prognostically, ECV independently predicted mortality (HR: 1.22 per 10% increase; 95% CI: 1.10-1.34 per 10% increase; P < 0.001) after multivariable analysist. Stratifying patients by ECV categories (degree of infiltration: none <30%; m...