Cardioprotection and Heart Failure Management in Duchenne and Becker Muscular Dystrophies: A State-of-the-Art Review Across Pediatric and Transitional Age
5 affiliations
- 1Department of Pediatrics, San Bassiano Hospital, AULSS 7 Pedemontana, 36061 Bassano del Grappa, Italy
- 2Department of Pediatric Cardiology, IRCCS Istituto Giannina Gaslini, 16147 Genoa, Italy
- 3Centre of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, 16147 Genoa, Italy
- 4Pediatric Neurology and Muscular Diseases Unit, IRCCS Istituto Giannina Gaslini, 16147 Genoa, Italy
- 5Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health—DINOGMI, University of Genoa, 16147 Genoa, Italy
Abstract
Advances in respiratory care have significantly extended the life expectancy of patients with Duchenne and Becker muscular dystrophies (DMD/BMD), making progressive dilated cardiomyopathy the leading cause of mortality in this population. Historically, a conservative “watch and wait” approach was often maintained until overt systolic dysfunction became evident. However, the advent of advanced echocardiographic technologies, particularly Global Longitudinal Strain (GLS) imaging, has demonstrated that myocardial impairment begins in early childhood, long before a decline in ejection fraction occurs. This critical evidence strongly advocates for a paradigm shift toward early, proactive cardioprotective strategies. As patients progress into the transitional age and adulthood, the pediatric prophylactic approach (traditionally based on ACE inhibitors and beta-blockers) must integrate with evolving adult heart failure guidelines. This comprehensive review critically evaluates the pathophysiological continuum of dystrophic cardiomyopathy. We highlight the prognostic value of early subclinical detection (GLS) and specifically discuss the transition to modern guideline-directed medical therapies (GDMTs), exploring the emerging role of Angiotensin Receptor-Neprilysin Inhibitors (ARNI, e.g., sacubitril/valsartan) and SGLT2 inhibitors (e.g., dapagliflozin) in this specific cohort. Finally, we address the role of advanced device therapies (ICD, CRT, and left ventricular assist devices) in end-stage disease. By bridging pediatric neurology and adult cardiology, this review aims to equip clinicians with an updated, multidisciplinary framework to optimize cardiovascular outcomes in the growing population of transitional-age DMD/BMD patients.
Related
- Duchenne muscular dystrophy-associated cardiomyopathy: advances in diagnosis and management
- Non-invasive adjunctive diagnostic models incorporating high-frequency ultrasound and shear wave elastography for pediatric Duchenne muscular dystrophy
- Snail-Inspired Metabolic Rewiring Offers a New Avenue to Cardioprotection.
- Pediatric Headache: A Practical Approach to Clinical Diagnosis and Management
- Familial Hypercholesterolemia Screening: From Pediatric Detection to Life-long Management
BibTeX
@article{pasquinucci2026cardioprotection,
title = {{Cardioprotection and Heart Failure Management in Duchenne and Becker Muscular Dystrophies: A State-of-the-Art Review Across Pediatric and Transitional Age}},
author = {Mattia Pasquinucci and Maria Elena Derchi and Alessandra Siboldi and Sara Bondanza and Chiara Panicucci and Federica Trucco and Claudio Bruno and Davide Meneghesso},
journal = {Journal of Clinical Medicine},
year = {2026},
month = nov,
doi = {10.3390/jcm15218554},
url = {https://www.mdpi.com/2077-0383/15/21/8554},
note = {CC BY 4.0},
}
RIS
TY - JOUR
TI - Cardioprotection and Heart Failure Management in Duchenne and Becker Muscular Dystrophies: A State-of-the-Art Review Across Pediatric and Transitional Age
AU - Mattia Pasquinucci
AU - Maria Elena Derchi
AU - Alessandra Siboldi
AU - Sara Bondanza
AU - Chiara Panicucci
AU - Federica Trucco
AU - Claudio Bruno
AU - Davide Meneghesso
PY - 2026
DA - 2026/11/03
JO - Journal of Clinical Medicine
DO - 10.3390/jcm15218554
UR - https://www.mdpi.com/2077-0383/15/21/8554
AB - Advances in respiratory care have significantly extended the life expectancy of patients with Duchenne and Becker muscular dystrophies (DMD/BMD), making progressive dilated cardiomyopathy the leading cause of mortality in this population. Historically, a conservative “watch and wait” approach was often maintained until overt systolic dysfunction became evident. However, the advent of advanced echocardiographic technologies, particularly Global Longitudinal Strain (GLS) imaging, has demonstrated that myocardial impairment begins in early childhood, long before a decline in ejection fraction occurs. This critical evidence strongly advocates for a paradigm shift toward early, proactive cardioprotective strategies. As patients progress into the transitional age and adulthood, the pediatric prophylactic approach (traditionally based on ACE inhibitors and beta-blockers) must integrate with evolving adult heart failure guidelines. This comprehensive review critically evaluates the pathophysiological continuum of dystrophic cardiomyopathy. We highlight the prognostic value of early subclinical detection (GLS) and specifically discuss the transition to modern guideline-directed medical therapies (GDMTs), exploring the emerging role of Angiotensin Receptor-Neprilysin Inhibitors (ARNI, e.g., sacubitril/valsartan) and SGLT2 inhibitors (e.g., dapagliflozin) in this specific cohort. Finally, we address the role of advanced device therapies (ICD, CRT, and left ventricular assist devices) in end-stage disease. By bridging pediatric neurology and adult cardiology, this review aims to equip clinicians with an updated, multidisciplinary framework to optimize cardiovascular outcomes in the growing population of transitional-age DMD/BMD patients.
SN - 2077-0383
ER -