Healthcare Utilization of Cardiac Specialty Care in Pediatric Patients with Muscular Dystrophy in the United States


Real World Data - Disease registries, natural history, post marketing surveillance

Poster Number: 170


Erika Mejia, MD, Jonathan Edelson, MD, Katherine Iacobellis, Susan Matesanz, MD, John Brandsema, MD, Heather Griffis, PhD, Oluwatimilehin Okunowo, Kimberly Lin, MD


1. Children's Hospital of Philadelphia, 2. Children's Hospital of Philadelphia, Division of Cardiology, 3. Children's Hospital of Philadelphia, Division of Cardiology, 4. Children's Hospital of Philadelphia, Division of Neurology, 5. Children's Hospital of Pennsylvania, 6. Children's Hospital of Philadelphia, 7. Children's Hospital of Philadelphia, 8. Children's Hospital of Philadelphia, Division of Cardiology

Background: Cardiac disease is a major contributor to morbidity and mortality in patients with Duchenne and Becker muscular dystrophy (MD).

Objectives: Characterize the burden of cardiac disease and management in pediatric MD.

Methods: Males <18y at time of first claim for MD (index date) with continuous enrollment for ≥12 months post-index date were identified from the MarketScan Database (2013-2018). Healthcare resource use were summarized using descriptive statistics and reported per patient per year (PPPY).

Results: 1341 patients were included with a median follow-up of 2.8y (IQR 1.8, 4.3). 295 (22%) were 1-6y, 494 (37%) 7-12y, and 552 (41%) 13-18y at index date. 72 (5.4%) had heart failure with a higher prevalence in the 13-18y group (7%, n=38) compared to the 1-6y (1.7%, n=5) (p=0.001), but not the 7-12y group (6%, n=29) (p=0.50). 568 (42%) of patients had ≥1 outpatient cardiology visits PPPY, including only 244 (44%) of patients in the 13-18y group. The median number of cardiology visits amongst patients with >1 outpatient cardiology visit PPPY was higher in the 13-18y group (median 1.1, IQR [0.7-1.7]) compared to the 7-12y (0.9, IQR [0.5-1.3]) (p=0.0002) and 1-6y group (0.7, IQR [0.4-1.3]) (p=0.0001). 379 (28%) had ≥ 1 echocardiograms (echo) PPPY with the highest rate in the 13-18y group (35%, n=191) compared to the 7-12y (28%, n=136) (p=0.026) and 1-6y group (18%, n=52) (p<0.001). 31% (n=420) did not have an echo. Ambulatory rhythm monitoring was infrequent, observed in 19% of the cohort.

Conclusions: Visits and echos increased with age among patients with ≥1 visit PPPY, which parallels an increased heart failure prevalence with age. However, over half of patients in the 13-18y group did not have yearly cardiac follow up or echocardiograms, and 81% of the study group did not undergo ambulatory rhythm monitoring. Significant changes to management are needed in order to observe updated Duchenne MD Care Considerations and optimize cardiac outcomes.