IDENTIFYING AND PRIORITIZING PARENTAL CONCERNS ASSOCIATED WITH DUCHENNE MUSCULAR DYSTROPHY USING BEST-WORST SCALING

Purpose: Duchenne muscular dystrophy (DMD) is a rare, progressive disorder. Affected individuals typically lose ambulation at 8-12 years and die in their third decade. While the condition is associated with significant emotional and care-related burden on parents, little is about the priorities of the parents and guardians who care for individuals with DMD. We sought to identify and prioritize parental concerns associated with the care of individuals with DMD.

Methods: Through a rigorous engagement of parents, guardians and other stakeholders, a comprehensive set of parental concerns was identified. To prioritize these concerns, we designed and implement a survey of parents and guardians who care for individuals with DMD and utilized best-worst scaling (BWS) case 1 to prioritize their concerns. A national sample of parents and guardians of individuals with DMD in the US were recruited from advocacy organizations, the DuchenneConnect Registry, and via snowball recruiting. Each of 16 BWS choice tasks incorporated a subset of six concerns that where chosen based on a Youden balanced-incomplete-block experimental design, where respondents were asked to choose the item that was most and least concerning to them over the past 7 days. Analysis used an average best-minus-worst (B-W) approach, where items selected as most concerning were scored as +1, those least concern as -1, and zero otherwise. Hypothesis testing utilized a two-tailed t-test based on mean scores.

Results: Of the 119 parents/guardians who completed the survey, the average age was 43.7 (SD 7.7) and were predominately Caucasian (92%), married (90%), biological mothers (67%). A total of 16 concerns were identified, spanning issues associated with the child’s well-being, disease management, family impact and parents’ wellbeing. Overall, weakness was identified as most concerning (B-W score=0.637; p<0.001). Respondents were also concerned that they were “getting the right care for my child over time” (0.254; p<0.001) and that their “child missing out on new treatments” (0.245; p<0.001).

Conclusion: This paper presents a unique and theoretically robust approach to the identification and prioritization of parental concerns. Overall, respondents ranked the child’s well-being and disease management as a greater concern compared to the impact of DMD on the family impact and parents’ wellbeing. Future research will focus on advanced estimation techniques for BWS, exploratory factor analysis and study of preferences heterogeneity and latent-classes.