S.P.47 CARE-NMD: Evaluation and implementation of relevant health related QoL instruments in Duchenne muscular dystrophy

CARE-NMD aims to disseminate and implement best-practice standards of care for Duchenne muscular dystrophy (DMD) in Europe. As part of the project a survey of health related quality of life was to be conducted in seven countries. To select relevant methods of measurement a screening of seven existing instruments was carried out. Main criteria for selection were frequency of use, correspondence with domains in the International Classification of Functioning, Disability and Health (ICF), comparability with background population, and scope of cross cultural and geographical assessments. Three pediatric (one disease specific and two generic) and two (generic) instruments were chosen. A comprehensive questionnaire containing the selected instruments was designed and implemented through patient registries in Bulgaria, Czech Republic, Denmark, Germany, Hungary, Poland, and the UK. The overall response rate was 66%. In addition to information on the experience of 1100 European patients and families living with DMD, the data will provide important knowledge about the options and feasibilities of cross national QoL assessments in Duchenne muscular dystrophy.