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

Copyright © 2022 The Authors. Published by Elsevier Inc. All rights reserved..

BACKGROUND: 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.

OBJECTIVES: 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.

METHODS: 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.

RESULTS: The highest performing, internally validated, parsimonious multivariable model included age, native myocardial T1 dispersion (SD of per voxel myocardial T1 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.

CONCLUSIONS: 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.

Errataetall:

CommentIn: J Am Coll Cardiol. 2022 Sep 6;80(10):995-997. - PMID 36049807

Medienart:

E-Artikel

Erscheinungsjahr:

2022

Erschienen:

2022

Enthalten in:

Zur Gesamtaufnahme - volume:80

Enthalten in:

Journal of the American College of Cardiology - 80(2022), 10 vom: 06. Sept., Seite 982-994

Sprache:

Englisch

Beteiligte Personen:

Orsborne, Christopher [VerfasserIn]
Bradley, Joshua [VerfasserIn]
Bonnett, Laura J [VerfasserIn]
Pleva, Luke A [VerfasserIn]
Naish, Josephine H [VerfasserIn]
Clark, David G [VerfasserIn]
Abidin, Nik [VerfasserIn]
Woolfson, Peter [VerfasserIn]
Nucifora, Gaetano [VerfasserIn]
Schmitt, Matthias [VerfasserIn]
Jovanovic, Ana [VerfasserIn]
Miller, Christopher A [VerfasserIn]
Reid, Anna B [VerfasserIn]

Links:

Volltext

Themen:

Cardiac magnetic resonance imaging
Fabry cardiomyopathy
Fabry disease
Journal Article
Research Support, Non-U.S. Gov't
T(1) mapping

Anmerkungen:

Date Completed 08.09.2022

Date Revised 22.03.2023

published: Print

CommentIn: J Am Coll Cardiol. 2022 Sep 6;80(10):995-997. - PMID 36049807

Citation Status MEDLINE

doi:

10.1016/j.jacc.2022.06.022

funding:

Förderinstitution / Projekttitel:

PPN (Katalog-ID):

NLM34567085X