Cardiac Medication Use in ACTION for Duchenne Muscular Dystrophy Cardiomyopathy

Area of Science:

• Cardiology
• Neuromuscular Disorders
• Pharmacology

Background:

• Duchenne Muscular Dystrophy (DMD) frequently involves cardiac complications, leading to increased mortality.
• Consensus-directed medical therapy (CDMT) is recommended for DMD-associated cardiomyopathy.
• Understanding current cardiac medication practices in DMD is crucial for optimizing patient care.

Purpose of the Study:

• To analyze cardiac medication use and adherence to CDMT in a prospective cohort of males with DMD.
• To assess the prevalence of left ventricular (LV) systolic dysfunction and its management.
• To identify potential barriers to achieving target dosing of cardiac medications.

Main Methods:

• Prospective analysis of patients enrolled in the ACTION Dystrophinopathy Registry since 2021.
• Evaluation of cardiac medication use at enrollment and follow-up.
• Definition of CDMT as concurrent use of ACEi/ARB/ARNI, BB, and MRA.

Main Results:

• Over half (57.7%) of 265 DMD patients had decreased LV systolic function; 25.3% had moderate/severe dysfunction.
• Among those with moderate/severe dysfunction, 73.1% received CDMT at follow-up, similar to enrollment (70.6%).
• Target CDMT doses were achieved by only 23-28% of patients on individual medication classes.

Conclusions:

• A significant proportion of DMD patients with moderate/severe LV dysfunction are not on optimal CDMT.
• Barriers to achieving target medication dosing persist, necessitating further investigation.
• Improved understanding of optimal cardiac medication strategies is vital given cardiac mortality in DMD.