Andrea Bever

Introduction Studies have reported health-related quality-of-life impacts of Duchenne muscular dystrophy (DMD); however, further research is needed to understand how those with DMD experience their condition and how psychosocial impacts evolve over time in response to disease progression. This qualitative study explores the social and emotional implications of key transitions, challenges and adaptations throughout the disease course from the perspective of patients and family caregivers. Methods Semi-structured interviews were conducted with men and boys with DMD, and/or their caregivers, in the USA. Thematic analysis was used to examine patterns in data collected across the interviews. Results Nineteen participants were included. Three major themes were identified: (1) barriers to participation are multifaceted; (2) an emotional journey shaped by ‘inevitable progression;' (3) family provides critical tangible and emotional support. This study illustrates that psychosocial impacts of DMD are shaped by knowledge of the condition’s natural history alongside other factors including the extent of social barriers, personal growth and adaptation, and family support. Conclusions Findings provide insight into the strength and resilience with which individuals and their families respond to daily challenges and major clinical milestones and highlight the relative importance of loss of upper limb function as a transition in DMD affecting health-related quality-of-life.

Background: Lung cancer is the most common cancer and leading cause of cancer death worldwide. In lung cancer clinical trials, overall survival (OS) is a widely used endpoint; however, DFS – the time to cancer recurrence or death from any cause – may be a better indicator of transformative patient outcomes. While the use of DFS is growing, patient perceptions of the validity of this endpoint have not been established. Objective: To understand the importance of DFS as a treatment endpoint, from the perspective of Canadian lung cancer survivors. Methods: This qualitative study included Canadian survivors of stage Ib to IIIa lung cancer, who participated in web-based semi-structured interviews in early 2021. Participants described their experience of cancer diagnosis and treatment and provided their perspectives on DFS and OS, including how well each endpoint aligned with their own treatment priorities. Thematic analysis was used to explore patterns in responses. Results: Of 14 participants, the mean age was 64 years, 14% were male, 86% had surgery and 58% were treated with chemotherapy. Most participants viewed DFS as intrinsically relevant to their treatment priorities. One individual’s interest in DFS was limited to its ability to serve as a surrogate for OS. All participants emphasized that new treatments should be quickly approved based on DFS when OS data are not yet available; an issue that was often viewed in the context of promoting patient agency in treatment decision-making. Conclusions: These findings validate DFS as a meaningful endpoint from the perspective of lung cancer survivors and highlight patients’ desire for rapid approval of treatments that have been demonstrated to improve DFS.