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Abstract Background Aspirations refer to wishes, ways of defining quality of life (QOL), and life goals. Living with chronic illness likely impacts a person’s life aspirations. Duchenne Muscular Dystrophy (DMD) is an inherited disorder characterized by the inevitable and progressive loss of ambulation and independence. The present cross-sectional case–control study investigated how aspirations differed between people with DMD and a stratified comparison group of nationally representative children/adults. Methods A web-based survey was administered October through December 2020. Recruitment was stratified by age group: 8–12, 13–17, and > = 18, reflecting the DMD disability trajectory. Aspirations were measured using qualitative (open-ended) and quantitative (closed-ended) questions. Qualitative prompts asked participants about wishes, how they would define “QOL”; and goals; answers to the prompts were then coded by six trained raters. Quantitative questions included 29 closed-ended goal-delineation items from the QOL Appraisal Profilev2. These data were analyzed using multivariate models adjusting for propensity scores (demographic differences), and testing for the effects of role (patient or comparison), age, and role-by-age interactions. Results The study sample of DMD (n = 285) and comparison (n = 292) participants provided open-text data: 577 wishes statements, 283 QOL-definition statements, and 149 goals statements. Inter-rater reliability (kappa = 0.77) reflected good agreement between different raters’ codes. Results suggested that people with DMD have aspirations that differ from their peers in several important ways. Both open-text and closed-ended data in both unadjusted and adjusted analyses generally showed that people with DMD were more focused on intrinsic aspirations such as health, healthcare, and independence than their peers. Compared to non-DMD persons, DMD individuals were much less focused on financial or housing concerns, community contributions, or spiritual growth. With age, patients’ aspirations focused less on extrinsic aspirations such as careers and work and increasingly emphasized emotion-oriented goals. Patients were markedly less likely to give a direct answer to the open-ended goals question. Conclusion There were important differences in aspirations between people with DMD and their peers. These findings may be helpful for developing psychosocial interventions.

Abstract Background The inevitable and progressive loss of independence in Duchenne Muscular Dystrophy (DMD) patients may have an impact on their siblings’ life aspirations. The present cross-sectional case-control study investigated how aspirations differed between brothers and sisters of people with DMD and a stratified comparison group of nationally representative children/adults. Methods A web-based survey was administered October through December 2020. Aspirations were measured using qualitative (open-ended) and quantitative (closed-ended) questions. Qualitative prompts asked participants about wishes, goals, and how they define quality of life (QOL) and were coded by six trained raters. Quantitative questions included 29 closed-ended goal-delineation items from the QOL Appraisal Profilev2. These data were analyzed using multivariate models adjusting for propensity scores (demographic differences) and testing for the effects of role (sibling or comparison), age, and role-by-age interactions. Results The study sample of DMD sibling (n = 349) and comparison (n = 619) participants provided open-ended data on 968 wishes statements, 390 QOL-definition statements, and 328 goals statements. Inter-rater reliability (kappa = 0.77) reflected good agreement between raters. Results of both open-ended and closed-ended data, and of both unadjusted and adjusted analyses suggested that DMD siblings, with age, were more focused on DMD-related, family/community, intimacy, and work concerns than their peers. They were less focused on improving mood, independence, pragmatics, or subtle fine-tuning of problem-solving in life. In contrast, the comparison group was more focused on goals related to growth, purpose, and reflection. Some group differences were amplified amongst older siblings. Conclusion This is the first study to evaluate DMD siblings’ aspirations in comparison to their peers. While there were many similarities between groups, the differences in aspirations between DMD siblings and their peers encompassed not just DMD, family/community, and intimacy, but also more work concerns. Directions for future quantitative and qualitative research are discussed.