Heart failure guideline-directed medical therapy in young adults with dystrophin-related cardiomyopathy: the HOPE-MD registry

AIMS: Duchenne (DMD) and Becker (BMD) muscular dystrophies often progress to dilated cardiomyopathy and heart failure with reduced ejection fraction (HFrEF), a major cause of death in affected patients. Although guideline-directed medical therapy (GDMT) improves HFrEF outcomes, its real-world use in dystrophinopathies is poorly defined. We assessed GDMT utilization and optimization in the study population. METHODS AND RESULTS: HOPE-MD (Heart failure treatment OPtimization in patiEnts with Muscular Dystrophy) is a European, multicentre, registry including adults with genetically confirmed DMD or BMD and left ventricular ejection fraction (LVEF) ≤ 40%. GDMT was defined as concurrent use of a renin-angiotensin system inhibitor (RASi), beta-blocker, mineralocorticoid receptor antagonist (MRA), and sodium-glucose cotransporter-2 inhibitor (SGLT2i). Doses were categorized as target (≥100%), intermediate (50-99%), or low (