Prognostic Implications of the Extent of Cardiac Damage in Patients With Fabry Disease.
Fabry disease
cardiac damage
echocardiography
hypertrophic cardiomyopathy
Journal
Journal of the American College of Cardiology
ISSN: 1558-3597
Titre abrégé: J Am Coll Cardiol
Pays: United States
ID NLM: 8301365
Informations de publication
Date de publication:
10 10 2023
10 10 2023
Historique:
received:
14
06
2023
revised:
24
07
2023
accepted:
26
07
2023
medline:
1
11
2023
pubmed:
5
10
2023
entrez:
4
10
2023
Statut:
ppublish
Résumé
There is limited evidence on the risk stratification of cardiovascular outcomes in patients with Fabry disease (FD). This study sought to classify FD patients into disease stages, based on the extent of the cardiac damage evaluated by echocardiography, and to assess their prognostic impact in a multicenter cohort. Patients with FD from 5 Italian referral centers were categorized into 4 stages: stage 0, no cardiac involvement; stage 1, left ventricular (LV) hypertrophy (LV maximal wall thickness >12 mm); stage 2, left atrium (LA) enlargement (LA volume index >34 mL/m A total of 314 patients were included. Among them, 174 (56%) were classified as stage 0, 41 (13%) as stage 1, 57 (18%) as stage 2 and 42 (13%) as stage 3. A progressive increase in the composite event rate at 8 years was observed with worsening stages of cardiac damage (log-rank P < 0.001). On multivariable Cox regression analysis, the staging was independently associated with the risk of cardiovascular events (HR: 2.086 per 1-stage increase; 95% CI: 1.487-2.927; P < 0.001). Notably, cardiac staging demonstrated a stronger and additive prognostic value, as compared with the degree of LV hypertrophy. In FD patients, a novel staging classification of cardiac damage, evaluated by echocardiography, is strongly associated with cardiovascular outcomes and may be helpful to refine risk stratification.
Sections du résumé
BACKGROUND
There is limited evidence on the risk stratification of cardiovascular outcomes in patients with Fabry disease (FD).
OBJECTIVES
This study sought to classify FD patients into disease stages, based on the extent of the cardiac damage evaluated by echocardiography, and to assess their prognostic impact in a multicenter cohort.
METHODS
Patients with FD from 5 Italian referral centers were categorized into 4 stages: stage 0, no cardiac involvement; stage 1, left ventricular (LV) hypertrophy (LV maximal wall thickness >12 mm); stage 2, left atrium (LA) enlargement (LA volume index >34 mL/m
RESULTS
A total of 314 patients were included. Among them, 174 (56%) were classified as stage 0, 41 (13%) as stage 1, 57 (18%) as stage 2 and 42 (13%) as stage 3. A progressive increase in the composite event rate at 8 years was observed with worsening stages of cardiac damage (log-rank P < 0.001). On multivariable Cox regression analysis, the staging was independently associated with the risk of cardiovascular events (HR: 2.086 per 1-stage increase; 95% CI: 1.487-2.927; P < 0.001). Notably, cardiac staging demonstrated a stronger and additive prognostic value, as compared with the degree of LV hypertrophy.
CONCLUSIONS
In FD patients, a novel staging classification of cardiac damage, evaluated by echocardiography, is strongly associated with cardiovascular outcomes and may be helpful to refine risk stratification.
Identifiants
pubmed: 37793750
pii: S0735-1097(23)06393-3
doi: 10.1016/j.jacc.2023.07.026
pii:
doi:
Types de publication
Multicenter Study
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
1524-1534Commentaires et corrections
Type : CommentIn
Informations de copyright
Copyright © 2023 American College of Cardiology Foundation. Published by Elsevier Inc. All rights reserved.
Déclaration de conflit d'intérêts
Funding Support and Author Disclosures Dr Lillo has received advisory board fees from Amicus Therapeutics, Sanofi Genzyme, Takeda, and Shire. Dr Biagini has received speaker fees from Amicus Therapeutics, Sanofi Genzyme, Takeda, and Bristol Myers Squibb. Dr Pieroni has received speaker and/or advisory board fees from Sanofi Genzyme, Pfizer, and Bristol Myers Squibb. Dr Crea has received personal fees from Amgen, AstraZeneca, Abbott, Menarini, Chiesi, and Daiichi-Sankyo. Dr Limongelli has received advisory board fees from Amicus Therapeutics; and has received an unrestricted grant from Sanofi. Dr Olivotto has received research grants from Bristol Myers Squibb, Cytokinetics, Sanofi Genzyme, Shire, Bayer, Amicus, Chiesi, and Menarini International; and has received speaker and/or advisory board fees from Bristol Myers Squibb, Cytokinetics, Sanofi Genzyme, Amicus, Shire, Tenaya, and Rocket Pharma. Dr Graziani has received research grants from Takeda and Pfizer; and has received advisory board fees from Amicus Therapeutics, Sanofi Genzyme, and Shire. All other authors have reported that they have no relationships relevant to the contents of this paper to disclose.