Validated Model for Prediction of Adverse Cardiac Outcome in Patients With Fabry Disease

Orsborne, Christopher, Bradley, Joshua, Bonnett, Laura J ORCID: 0000-0002-6981-9212, Pleva, Luke A, Naish, Josephine H, Clark, David G, Abidin, Nik, Woolfson, Peter, Nucifora, Gaetano, Schmitt, Matthias
et al (show 3 more authors) (2022) Validated Model for Prediction of Adverse Cardiac Outcome in Patients With Fabry Disease. Journal of the American College of Cardiology, 80 (10). pp. 982-994.

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<h4>Background</h4>The cardiac manifestations of Fabry disease are the leading cause of death, but risk stratification remains inadequate. Identifying patients who are at risk of adverse cardiac outcome may facilitate more evidence-based treatment guidance. Contemporary cardiovascular cardiac magnetic resonance biomarkers have become widely adopted, but their prognostic value remains unclear.<h4>Objectives</h4>The objective of this study was to develop, internally validate, and evaluate the performance of, a prognostic model, including contemporary deep phenotyping, which can be used to generate individual risk estimates for adverse cardiac outcome in patients with Fabry disease.<h4>Methods</h4>This longitudinal prospective cohort study consisted of 200 consecutive patients with Fabry disease undergoing clinical cardiac magnetic resonance. Median follow-up was 4.5 years (IQR: 2.7-6.3 years). Prognostic models were developed using Cox proportional hazards modeling. Outcome was a composite of adverse cardiac events. Model performance was evaluated. A risk calculator, which provides 5-year estimated risk of adverse cardiac outcome for individual patients, including men and women, was generated.<h4>Results</h4>The highest performing, internally validated, parsimonious multivariable model included age, native myocardial T<sub>1</sub> dispersion (SD of per voxel myocardial T<sub>1</sub> relaxation times), and indexed left ventricular mass. Median optimism-adjusted c-statistic across 5 imputed model development data sets was 0.77 (95% CI: 0.70-0.84). Model calibration was excellent across the full risk profile.<h4>Conclusions</h4>This study developed and internally validated a risk prediction model that accurately predicts 5-year risk of adverse cardiac outcome for individual patients with Fabry disease, including men and women, which could easily be integrated into clinical care. External validation is warranted.

Item Type: Article
Uncontrolled Keywords: cardiac magnetic resonance imaging, Fabry cardiomyopathy, Fabry disease, T-1 mapping
Divisions: Faculty of Health and Life Sciences
Faculty of Health and Life Sciences > Institute of Population Health
Depositing User: Symplectic Admin
Date Deposited: 19 Oct 2022 09:27
Last Modified: 22 Feb 2023 09:19
DOI: 10.1016/j.jacc.2022.06.022
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