Safety and Efficacy of Carvedilol Therapy for Patients with Dilated Cardiomyopathy Secondary to Muscular Dystrophy

Background By the age of 20 years, almost all patients with Duchenne’s or Becker’s muscular dystrophy have experienced dilated cardiomyopathy (DCM), a condition that contributes significantly to their morbidity and mortality. Although studies have shown carvedilol to be an effective therapy for patients with other forms of DCM, few data exist concerning its safety and efficacy for patients with muscular dystrophy. This study aimed to evaluate the safety and efficacy of carvedilol for patients with DCM. Methods A clinical trial at an outpatient clinic investigated 22 muscular dystrophy patients, ages 14 to 46 years, with DCM and left ventricular ejection fraction (LVEF) less than 50%. Carvedilol up-titrated over 8 weeks then was administered at the maximum or highest tolerated dose for 6 months. Baseline and posttreatment cardiac magnetic resonance imaging (CMR), echocardiography, and Holter monitoring were recorded. Results Carvedilol therapy was associated with a modest but statistically significant improvement in CMR-derived ejection fraction (41% ± 8.3% to 43% ± 8%; p