OBJECTIVES: To gather qualitative evidence from patients and their parents on the impact of long-term, high-dose steroid use side effects in Duchenne Muscular Dystrophy (DMD), including patient and parent health-related quality of life (HRQoL) impact, personal carer burden, and health service utilisation. This study was conducted as part of Project HERCULES.

METHODS: Following a targeted literature review and two patient/parent surveys, dyads and small group interviews on steroid burden in DMD were performed with patients and their parents, followed by a focus group which also included DMD health-care professionals. Key themes in steroid side effect burden were identified and discussed to determine what mattered most to the patients and their families.

RESULTS: Explorative, thematic analysis of two dyads (n=2 for each) and one small group interview (n=4) attended in September 2021, identified the following themes for discussion at the focus group: emotional outbursts and behaviour, anxiety/mood, broken bones, sleep/daytime tiredness, physical appearance, delayed puberty, gastrointestinal issues, caregiver burden and adrenal crisis, and additional resource use. The opinions on each topic were attained from attendees at the focus group, resulting in a narrative on the burden each theme has on the daily lives of people with DMD and their family, with illustrative quotes derived from patient, parent, and health-care professional perspectives.

CONCLUSIONS: The current research has, for the first time through qualitative research, explored which steroid side effects concern patients with DMD and their families the most, and enabled open discussions between the families and health-care professionals about how this burden effects their daily lives. The evidence gathered can be utilised as an input to describe steroid side effect burden in Heath Technology Assessment (e.g. NICE) submissions, as a base for further quantitative data collection, or as the foundation for vignette development for health economic utility studies in DMD.