Abstract
To explore impact of long-term high dose steroid use side effects with perceived high burden on Duchenne Muscular Dystrophy (DMD) patient and parent health-related quality of life (HRQoL) impact, personal carer burden, and health service resource use. This study is being conducted as part of Project HERCULES. A targeted literature review (TLR) was performed to identify side effects of steroid use in DMD, focussing on quantifying the HRQoL and resource use impacts. An online survey was also circulated via patient organizations in the UK to ascertain steroid side-effects of concern to parents and young people with DMD (University of Glasgow). The next phase of the research consists of the use of a Focus Group of patient-parent pairings, with a pre-Focus Group questionnaire, to qualitatively determine key themes in steroid side effect burden. The TLR identified 211 studies related to steroid side effects and burden in DMD. The majority covered steroid treatment response (154 records) or side effects (149 records). Only 11 studies covered quality of life impact, and 4 studies covered resource use and costs associated with steroid side effects, hence there is limited published evidence on burden. The online survey identified fractures (vertebral and long bone), obesity, and delayed puberty as side effects of greatest concern to parents, whereas short stature, obesity, and cushingoid features were of greatest concern to patients. These results will be used to guide discussions in the Focus Group. Steroid side effects that concern parents and the young person with DMD have not been previously explored in detail. The current research will ultimately enable recommendations for further research to quantify the HRQoL and resource use burden of steroid use in DMD, for Health Technology Assessment and development of care in areas that concern the patient community.
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