Search

Aim: To investigate whether in-the-moment diary reports of daily experience, taken collectively, are a valid representation of health-related quality of life (HRQL).Methods: A total of 35 boys with Duchenne muscular dystrophy (DMD) were recruited through four neuromuscular care providers across Australia. Participants completed the PedsQL™ Generic Core scales and one week of experience-sampling diary reporting on a personal digital assistant. Rasch analysis was undertaken on the diary data to derive a single valid measure score. The resulting measure score for each participant was correlated with the summary score from the PedsQL™ Generic Core scales to examine whether daily experience was representative of HRQL.Results: The daily diary method showed good metric properties, with adequate goodness of fit for data from items and participants suggesting unidimensionality of the construct: quality of everyday experience. The correlation of the daily diary measure score with overall PedsQL™ summary score showed moderate agreement (r = .60, p = 0.001).Conclusions: The benefits of measuring daily quality of life include detailed descriptions of day-to-day experiences of children without the need for retrospective recall. Diary methods on an electronic platform or software application for personal devices may be a useful tool to understand HRQL as the repeated measures data provide a detailed experience directly from the child and the platform makes data completion highly motivating. [ABSTRACT FROM AUTHOR]

Aim: To investigate parent-reported health status of boys with Duchenne muscular dystrophy (DMD) compared with a large Australian normative population and a cohort of children with Charcot-Marie-Tooth disease type 1A (CMT1A). Methods: The Child Health Questionnaire parent form (CHQ-PF50) was completed by parents of 34 boys with confirmed DMD. Seventeen parents were followed up at 6 months. CHQ-PF50 data were compared with 2620 age-matched norms and 90 children with CMT1A. Results: All domains of the CHQ-PF50 for the DMD cohort were significantly lower than the general paediatric population, particularly for physical functioning ( t=−17.2, P < 0.001) and the child's ability to fulfil school and social roles because of physical limitations ( t=−9.4, P < 0.001). Parents experienced greatest emotional impact of their child's DMD around the time of loss of ambulation. Children with DMD had lower health status compared with children with CMT1A with the exception of the behaviour and pain domains. Physical functioning worsened during 6 months ( P= 0.04); no other changes in health status were observed at follow-up. Conclusions: Parents report the impact of DMD on health status to be considerably worse when compared with CMT1A. Interventions should target minimising the impact of physical limitations on role functioning. [ABSTRACT FROM AUTHOR]