Real-DMD: Caregiver Baseline Characteristics From an Electronic Survey of Long-Term Real-World Experiences of Patients With Duchenne Muscular Dystrophy (DMD)

OBJECTIVES: DMD is a rare, progressive neuromuscular disorder primarily affecting young males, leading to muscle weakness and functional decline. Caring for patients with DMD can impact caregivers’ physical and psychological well-being and ability to work. REAL-DMD is a real-world, observational, prospective, longitudinal cohort study surveying caregivers of DMD patients on caregiving experience and patient function. Caregiver characteristics and experiences at baseline are reported.

METHODS: Adult caregivers of ambulatory DMD patients recruited from US patient advocacy groups (Parent Project Muscular Dystrophy; The Akari Foundation) completed a web-based survey reporting their socio-demographics, employment status, ability to work, and daily hours spent caregiving. Patient-Reported Outcome Measurement Information (PROMIS®) Global Health was also collected.

RESULTS: In total, 123 caregivers in REAL-DMD completed the baseline survey (mean±SD age: 40.6±7.3 years). Most participants were female (91.9%) and White (86.2%) and cared for 1 child with DMD (1 child: 82.1%; 2+ children: 17.9%). Mean age of care recipient was 9.6±4.7 years. 57.7% of caregivers reported having anxiety or depression, with 35.8% undergoing treatment. Most caregivers were employed full- (43.1%) or part-time (20.3%). 65.0% reported experiencing impact on their ability to work due to caregiving responsibilities, including lower productivity (22.8%), switching jobs (20.3%), or reducing hours (19.5%). 28.5% temporarily stopped working, went on leave, or permanently left the workforce. Caregivers reported spending a mean±SD of 8.8±7.0 hours daily caring for the patient. Physical health of caregivers (PROMIS T-score: 49.1±7.9) was comparable to the US general population (50±10), while mental health was one-half SD lower (44.7±8.3).

CONCLUSIONS: REAL-DMD baseline caregiver findings provide insights into the characteristics of caregivers of ambulatory DMD patients in a real-world setting. Findings indicate impact on mental health and ability to work, along with substantial time commitments. Longitudinal data from REAL-DMD will characterize changes in caregiver impact over time as the care recipients’ disease progresses.