Cardiac Intervention Improves Heart Disease and Clinical Outcomes in Patients With Muscular Dystrophy in a Multidisciplinary Care Setting.
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.
['Nikhanj|Anish|A|', 'Yogasundaram|Haran|H|', 'Miskew Nichols|Bailey|B|', 'Richman-Eisenstat|Janice|J|', 'Phan|Cecile|C|', 'Bakal|Jeffrey A|JA|', 'Siddiqi|Zaeem A|ZA|', 'Oudit|Gavin Y|GY|']
D000293:Adolescent D000328:Adult D000553:Ambulatory Care D001145:Arrhythmias, Cardiac D009202:Cardiomyopathies D016022:Case-Control Studies D005260:Female D006339:Heart Rate D006760:Hospitalization D006801:Humans D008297:Male D008875:Middle Aged D049288:Muscular Dystrophies, Limb-Girdle D009223:Myotonic Dystrophy D010348:Patient Care Team D011446:Prospective Studies D013318:Stroke Volume D013997:Time Factors D016896:Treatment Outcome D016277:Ventricular Function, Left D055815:Young Adult
heart disease muscular dystrophy multidisciplinary care medical therapy outcome data