source: Neuromuscular Disorders
S. Geuens, J. Willen, C. Antonis, N. Goemans, L. De Waele
Despite some medical breakthroughs in Duchenne muscular dystrophy, no cure is available at this moment. New therapies are studied in clinical trials (CT) in boys with DMD. The investments of those boys and their families participating in CT may not be underestimated: time investment, impact on family activities, absence at work and school, side effects of the medication, systematic medical examinations. There is a certain burden that comes with de participation to those trials, and studies investigating the impact of that burden are very scarce.
The aim of this study is to develop an instrument to evaluate this burden and the impact of participation in a CT on the psychosocial wellbeing and quality of life of DMD boys and their parents. In a cross-sectional research design two groups of patients were asked to fulfill questionnaires. The first group were boys with DMD who participated in a CT. The control group consisted of age- and gender-matched boys with DMD. Parents of both groups were asked to answer a child Behavior Checklist (CBCL) reporting the psychosocial wellbeing of their child and an Adult Self Report (ASR) reflecting their own wellbeing.
University Hospitals Leuven, Belgium
10.1016/j.nmd.2018.06.161 read more