Characteristics of Healthcare Users in Pediatric Patients with Muscular Dystrophy in the United States


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

Poster Number: 117


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


1. Children's Hospital of Philadelphia, Division of Neurology, 2. Children's Hospital of Philadelphia, Division of Cardiology, 3. Children's Hospital of Philadelphia, Division of Cardiology, 4. Children's Hospital of Philadelphia, 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: Regular access to health services is critical in optimizing the health of patients with Duchenne and Becker muscular dystrophy (MD), and access to new therapies most often occurs through neurology visits.

Objectives: To characterize factors associated with healthcare use in pediatric MD.

Approach: 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 and costs were summarized using descriptive statistics and reported per patient per year (PPPY); comparisons are also presented for patients with ≥1 neurology visit PPPY compared with those with <1 visit PPPY.

Results: 1,341 patients were included with a median follow up of 2.8y (IQR 1.8, 4.3). 22% were age 1-6y, 37% 7-12y, and 41% 13-18y at index date. 49.5% had commercial insurance and 50.5% had Medicaid. Median healthcare costs PPPY were lowest in the 1-6y group [$7,746 (IQR $2,620, $15,689) compared to the 7-12y [$9,933 (IQR $3,646, $23,263)] (p = 0.0149)] and 13-18y groups [$12,447 (IQR $3,319, $28,577)] (p=<0.0001). 20% had ≥1 neurology visit PPPY (median 1.7, IQR 1.3, 2.2). Those with <1 neurology visit PPPY were more likely to have Medicaid (58.5% vs 18.5%, p <0.0001). There was no difference between those with ≥1 vs <1 neurology visit PPPY in the proportion with heart failure (3.7% vs 5.8%, p=0.1789), respiratory failure (8.9% vs 10.0%, p=0.6008), ≥1 complex chronic condition (CCC) (34.6% vs 29.9%, p=0.1418), or mortality (1.1% vs 1.0%, p=0.9999).

Conclusions: Only 20% of patients with DMD/BMD visit outpatient neurology annually suggesting limited access to vital specialty care. Insurance status was associated with healthcare use while heart failure, respiratory failure and CCC’s were not, indicating that factors other than illness severity affect care access. Further understanding barriers to care is critical to improving outcomes.