Key result
Multimodal risk model predicts 1-year disease progression in treated ATTR-CM with AUC ~0.75.
Why the study?
Although a validated approach for detecting disease progression in ATTR-CM exists, predicting progression under disease-modifying therapy remains an unmet clinical need. This study aimed to identify discriminative parameters and develop a risk stratification model for disease progression in ATTR-CM patients receiving disease-modifying therapy.
Does a multimodal assessment integrating baseline clinical, laboratory, and echocardiographic parameters predict disease progression at 1 year in patients with ATTR-CM receiving disease-modifying therapy?
Cohort (n=171)
No
Does a multimodal assessment integrating baseline clinical, laboratory, and echocardiographic parameters predict disease progression at 1 year in patients with ATTR-CM receiving disease-modifying therapy?
A multimodal risk model combining nonspecific intraventricular conduction delay, high-sensitivity troponin T, and peak left atrial longitudinal strain moderately predicts 1-year disease progression in ATTR-CM patients on disease-modifying therapy, potentially identifying high-risk patients needing intensified monitoring.
Background Validated approach for the detection of disease progression (DiP) in transthyretin amyloid cardiomyopathy (ATTR-CM) exists, yet predicting DiP under disease-modifying therapy remains an unmet clinical need. Therefore, aims of this study were to identify parameters with discriminative ability for predicting DiP and to develop a risk stratification model for DiP in ATTR-CM patients receiving disease-modifying therapy. Methods In this prospective registry study, we included all ATTR-CM patients who received disease-modifying therapy and completed one-year follow-up at our hospital. DiP was defined according to validated criteria involving sufficient NT-proBNP rise or any loop diuretic dose increase at one-year follow-up (Ioannou, A, Cappelli, F, Emdin, M. et al. Stratifying Disease Progression in Patients With Cardiac ATTR Amyloidosis. JACC. 2024 Apr, 83 (14) 1276–1291.). Except for DiP and follow-up-related parameters, all data were obtained exclusively from examinations performed as part of the initial diagnostic assessment. Left atrial strain measurements were performed in line with current guidelines, using 2D speckle tracking echocardiography, measured according to the zero strain reference at end-diastole. Results Among 171 included patients, 62 (36%) met criteria for DiP. General characteristics were largely comparable between groups; however, progressors exhibited greater dyspnea burden and nearly 2.5-fold higher prevalence of nonspecific intraventricular conduction delay (NICD). Median high-sensitivity troponin T levels were 70% higher among progressors (34 vs. 58 ng/L, p < 0.001). Moderate tricuspid regurgitation was the only conventional echocardiographic parameter significantly associated with DiP, occurring approximately twice as frequently among progressors. Left atrial function analysis identified peak left atrial longitudinal strain (PALS) < 7.90% as the most discriminative among all evaluated parameters (Youden’s index 37.2%). A multivariable risk model integrating NICD, high-sensitivity troponin T, and PALS achieved an AUC of 0.75 (95%CI: 0.68-0.83) for predicting DiP. Conclusion Multimodal evaluation provides prognostic information regarding DiP risk in ATTR-CM patients undergoing disease-modifying therapy. Findings of this study may support clinical decision-making and improve identification of high-risk patients, who may benefit from intensified monitoring.
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Piwowarski et al. (2026) conducted a cohort in Transthyretin amyloid cardiomyopathy (ATTR-CM) (n=171). Multimodal risk model (nonspecific intraventricular conduction delay, high-sensitivity troponin T, and peak left atrial longitudinal strain) was evaluated on Disease progression (sufficient NT-proBNP rise or any loop diuretic dose increase at one-year follow-up) (AUC 0.75, 95% CI 0.68-0.83). A multivariable risk model integrating NICD, hs-troponin T, and PALS predicted 1-year disease progression in treated ATTR-CM patients (AUC 0.75; 95% CI 0.68-0.83).
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