Cardiac Intervention Improves Heart Disease and Clinical Outcomes in Patients With Muscular Dystrophy in a Multidisciplinary Care Setting

• Published in: Journal of the American Heart Association 2020-01, Vol.9 (2), p.e014004-e014004
• Main Authors: Nikhanj, Anish, Yogasundaram, Haran, Miskew Nichols, Bailey, Richman-Eisenstat, Janice, Phan, Cecile, Bakal, Jeffrey A, Siddiqi, Zaeem A, Oudit, Gavin Y
• Format: Article
• Language: English
• Subjects: Adolescent; Adult; Ambulatory Care; Arrhythmias, Cardiac - diagnosis; Arrhythmias, Cardiac - etiology; Arrhythmias, Cardiac - physiopathology; Arrhythmias, Cardiac - therapy; Cardiomyopathies - diagnosis; Cardiomyopathies - etiology; Cardiomyopathies - physiopathology; Cardiomyopathies - therapy; Case-Control Studies; Female; heart disease; Heart Rate; Hospitalization; Humans; Male; medical therapy; Middle Aged; multidisciplinary care; Muscular Dystrophies, Limb-Girdle - complications; Muscular Dystrophies, Limb-Girdle - diagnosis; Muscular Dystrophies, Limb-Girdle - physiopathology; Muscular Dystrophies, Limb-Girdle - therapy; muscular dystrophy; Myotonic Dystrophy - complications; Myotonic Dystrophy - diagnosis; Myotonic Dystrophy - physiopathology; Myotonic Dystrophy - therapy; Original Research; outcome data; Patient Care Team; Prospective Studies; Stroke Volume; Time Factors; Treatment Outcome; Ventricular Function, Left; Young Adult
• ISSN: 2047-9980; 2047-9980
• DOI: 10.1161/JAHA.119.014004

Background Patients with muscular dystrophy (MD) represent a vulnerable patient population with no clearly defined care model in modern-day clinical practice to manage a high burden of heart disease and comorbidities. We demonstrate the effectiveness of cardiac interventions, namely the initiation and optimization of medical and device therapies, as part of a multidisciplinary care approach to improve clinical outcomes in patients with MD. Methods and Results We conducted a prospective cohort study at the Neuromuscular Multidisciplinary clinic following patients with dystrophinopathies, limb-girdle MD, type 1 myotonic dystrophy, and facioscapulohumeral MD. A negative control group classified as non-MD myopathies without heart disease, was also tracked. Our cohort of 185 patients (median age: 42 years; 79 [42.7%] women), included 145 patients with MD. Cardiomyopathy was present in 65.6% of the patients with dystrophinopathies (21 of 32) and 27.3% of the patients with limb-girdle MD (9 of 33). Conduction abnormalities were common in type 1 myotonic dystrophy (33.3% [20/60] patients). Cardiac intervention reversed systolic dysfunction, with left ventricular ejection fraction improving from 43% to 50.0% over a 3-year period. A sustained reduction in healthcare utilization was also observed. The number of outpatient clinic visits decreased from 3.0 to 1.5 visits per year, the duration of hospitalizations was reduced from 14.2 to 0.9 days per year, and the number of cardiac-related hospitalizations decreased from 0.4 to 0.1 hospitalizations per year associated with low mortality. Conclusions Our study demonstrates that cardiac intervention as part of a comprehensive multidisciplinary care approach to treating patients with MD leads to a sustained improvement in clinical outcomes.