The Duchenne muscular dystrophy quality of life (DMD-QOL) is a validated, 14-item questionnaire that assesses physical functioning, psychological impact, and social participation in boys with DMD aged 7+ years by proxy report and boys aged 10+ years by self-report. There is an evidence gap for use of the proxy-completed DMD-QOL among younger boys with DMD.
We conducted qualitative concept elicitation/cognitive interviews with caregivers of children under 7 years of age with DMD in the UK and US. The interviews explored the participant’s experience with DMD and assessed the relevance, comprehensiveness, ease of use, and understandability of the proxy-completed DMD-QOL.
Interviews were completed with 9 caregivers (all parents) of children aged 5–7 years with DMD in the UK (n=3) and US (n=6); the mean age at diagnosis was 5.7 and 6.9 years in the UK and US. Most caregivers (n=8/9) reported that the DMD-QOL instructions were understandable, and all (n=9/9) could explain the instructions’ meaning and demonstrated understanding of the response options. Concepts frequently endorsed as relevant for this age group included feeling tired (n=9/9), difficulty getting around (n=8/9), feeling angry (n=7/9), and the ability to take part in activities with friends (n=7/9). Fewer caregivers endorsed the relevance of psychological impact concepts (eg, feeling embarrassed [n=4/9], which was not reported spontaneously by any of the participants), or they reported that such concepts may not be easily attributable to DMD; however, several caregivers suspected the concepts would be relevant for their child in the future.
Overall caregiver feedback indicated that the proxy-completed DMD-QOL is easy to use, understandable, and face valid for use by caregivers of children aged 5–7 years with DMD. A second phase of interviews should be conducted to further evaluate the measure’s generalizability and accessibility in this age group, including among children aged 4 years with DMD.