Objective:
To understand caregiver perspectives on Duchenne Muscular Dystrophy (DMD) care, including barriers to recommended care and areas for improvement.
Background:
DMD is a progressive neuromuscular disorder requiring multidisciplinary management, yet adherence to U.S. care guidelines remains inconsistent.
Design/Methods:
Caregivers (≥18 years) of individuals with DMD were recruited via Albany Medical Center and national patient advocacy organizations. Participants completed an online survey assessing compliance with 2018 care considerations. Descriptive and bivariate analyses were performed for closed-ended data, and thematic analyses for open-ended data. For individuals aged ≥ 6 years, compliance scores were calculated based on the number of care recommendations met.
Results:
A total of 146 caregivers completed the survey. The mean age of individuals with DMD was 16.6 ± 8.3 years (range 2–50), with 59% non-ambulatory (n=86). The mean compliance score was 9.8 ± 2.7 out of 14 care recommendations (69.6% ± 19.5). Compliance scores were higher in children (p<0.001), clinical trial participants (p=0.018), individuals diagnosed before age 1 (p=0.003), and individuals from households with another disability (p=0.025) compared to counterparts. Over half of unmet specialist visits resulted from insufficient communication from providers about their importance (56.0%, n=229). Caregivers identified neuromuscular, cardiology, and psychosocial care as the top three specialties needing improvement. Thematic analyses revealed common barriers to care: cost and insurance (20.5%, n=30), limited provider availability (15.1%, n=22), and difficulty obtaining equipment or treatments (10.3%, n=15). Regarding helpful resources, caregivers expressed a need for checklists (74.8%, n=107) and brochures (57.3%, n=82), as well as stronger emotional and social support (35.6%, n=52), up-to-date information on disease progression (28.1%, n=41), and earlier connections to advocacy organizations (12.3%, n=18). Conclusions: Despite compliance to many aspects of care, notable gaps persist. These findings underscore the importance of caregiver-informed strategies to strengthen adherence to guidelines and improve multidisciplinary DMD management.