OBJECTIVES : Duchenne muscular dystrophy (DMD) is a rare pediatric-onset neuromuscular disorder. One therapy is approved for all patients, regardless of genotype: the corticosteroid Emflaza, which slows loss of function. There are limited patient experience data even though corticosteroids have been standard of care for over 10 years. This study, led by Parent Project Muscular Dystrophy, uses a community-engaged approach to understand corticosteroid decision making and anticipated or experienced benefits and side effects.

METHODS : Teenagers and adults with DMD and parents participated in semi-structured interviews via Zoom led by experienced interviewers. Data were systematically analyzed by corticosteroid-use and cohort using a rapid analysis approach.

RESULTS : 25 respondents were parents and 16 living with DMD. 26 of DMD index cases were current corticosteroid users; 3 prior users; and 10 parents were deciding whether to initiate use in young children. Most respondents characterized corticosteroids as the only available treatment and better than no treatment. Users reported a range of benefits, with the majority indicating maintained strength, prolonged independent daily activities, and improved mobility. Users reported a range of bothersome side effects, the most common of which were weight gain and mood changes. Most parents of users reported difficult decisions to initiate corticosteroids in the pediatric period. Most users with DMD endorsed their parents’ choice and chose to continue use. Among non-users, individuals who discontinued did so due to side effects. Parents of children still deciding on use had serious concerns about negative effects and almost all reported they were unlikely to choose corticosteroids for treatment.

CONCLUSIONS : Though corticosteroids were described as an imperfect and only moderately-effective therapy, users appreciated benefits and reported side effects as manageable. Decision making to initiate was challenging to many. Non-users had strong concerns about side effects. Parents of younger children especially were optimistic about future alternatives to corticosteroids.