PMS24 Decision-Making and Benefit-Risk Tradeoffs for Duchenne Muscular Dystrophy Treatment

Duchenne muscular dystrophy (DMD) is a rare pediatric-onset neuromuscular disorder. Emflaza, corticosteroid, the only therapy approved for all patients, regardless of genotype. Corticosteroids are standard care and modestly delay disease course. This study, led by Parent Project Muscular Dystrophy, uses community-engaged approach to understand corticosteroid decision making experiences. Teenagers adults with DMD parents participated in semi-structured interviews via Zoom experienced interviewers. Three vignettes were used glean information about values decision-making willingness try new that, compared corticosteroids, had (1) improved efficacy, but worse side effects, (2) reduced no (3) on long-term risks or benefits. Data systematically analyzed corticosteroid-use cohort using rapid analysis approach. 25 respondents 16 living DMD. 26 index cases current users; 3 prior 10 deciding whether initiate use young children. Parents start corticosteroids critical options more effects despite increased Current users willing an option efficacy citing ability manage effects. They preferred that one uncertain outcomes. corticosteroid-users treatment even limited outcome data. while non-users unwilling accept added expressed tolerance uncertainty than patients. Most research this patient population finds novel therapies, expected benefits far exceeded potential anticipated choices. Comparisons versus therapies provide important data motivations.