Better measuring quality of life in people with DMD (v1)
Research type
Research Study
Full title
Producing a preference-based quality of life (QoL) measure for people with Duchenne Muscular Dystrophy (DMD)
IRAS ID
242647
Contact name
Jill Carlton
Contact email
Sponsor organisation
University of Sheffield
Duration of Study in the UK
1 years, 2 months, 31 days
Research summary
Duchenne Muscular Dystrophy (DMD) is an inherited neuromuscular disorder affecting boys and men, with an estimated incidence of 1:3800 to 1:6200 in live births. The disease causes progressive muscle weakness, functional dependence, and cardiovascular and respiratory problems. Symptoms are evident from two years old and the average life expectancy of people with DMD is 23 to 28 years. To date, there is no known cure, and so treatment is focused on alleviative treatments and interventions to improve the health-related quality of life (QoL) of people with DMD. \n\nA number of existing questionnaires have been used to assess QoL in people with DMD, with conflicting results. There are growing concerns, however, that existing generic ’preference-based’ QoL questionnaires may be insufficient to adequately assess aspects of QoL that may be uniquely important to people with DMD, such as social participation, independence, and dignity. These ‘preference-based’ questionnaires are used in the economic evaluation of treatments and interventions, and so it is important that they are suitable for assessing QoL accurately in the health conditions in which they are used to evaluate treatments. \n\nThe purpose of this mixed-methods study, funded by Duchenne UK, is to develop a robust and valid preference-based questionnaire suitable for measuring QoL in people with DMD. The research involves an initial systematic review of the available evidence, followed by interviews with patients with DMD, recruited through five sites. Themes emerging from these interviews will be further refined using a survey with a larger group of people with DMD to produce a preference-based questionnaire of QoL in DMD. Health states identified from this questionnaire will be valued by a general population sample, in order to produce ’utility values’ to accompany the questionnaire, so it can be used in the accurate economic evaluation of alleviative treatments and interventions for DMD.
REC name
South West - Central Bristol Research Ethics Committee
REC reference
18/SW/0055
Date of REC Opinion
21 Feb 2018
REC opinion
Further Information Favourable Opinion