OBJECTIVES: Duchenne muscular dystrophy (DMD) is a childhood-onset genetic disorder, characterized by muscle degeneration, leading to limb weakness, loss of ambulation and progressive cardiac and pulmonary dysfunction. Previous research has described the economic impact of DMD with age and disease progression, however few have utilized real-world data. This study describes healthcare resource utilization (HCRU) for DMD patients aged 18 and under.

METHODS: Data were drawn from the Adelphi DMD Disease Specific Program™, a cross sectional survey of neurologists including retrospective data collection. Data was collected in France, Germany, Italy, Japan, Spain, the United Kingdom, and the United States from October 2022, an interim cut was taken in May 2023. Neurologists reported patient demographics, consultation history, DMD-related hospitalizations, and use of mobility aids or home adaptations.

RESULTS: 124 neurologists reported data on 425 male DMD patients, aged 0-18 years. Patients were characterized as child (≤12, n=264) or adolescent (≥13, n=161). Proportion of patients classified as non-ambulatory was higher in adolescents (13% vs. 56%). In the 12 months prior to survey, adolescents had a greater mean number of consultations (16.9 vs. 28.5), different physician types involved in their care (3.7 vs. 4.5), and number of tests/assessments monitoring their condition (9.7 vs. 13.7). Adolescents had been hospitalized more often due to DMD (13% vs. 18%), with a greater proportion of those hospitalized requiring intensive care (9% vs. 21%) and having surgery (6% vs. 31%). Adolescents more frequently required mobility aids/supports (60% vs. 85%) and adaptations to their home (52% vs. 83%).

CONCLUSIONS: Greater HCRU was reported for adolescents with DMD compared to children, in both inpatient and outpatient care. This data suggests that younger patients and thus patients with lower disease progression represent less economic burden on the healthcare system. Data collection is ongoing, with a complete dataset expected later in 2023.