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

Abstract

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