Participation and quality of life in children with Duchenne muscular dystrophy using the international classification of functioning, disability, and health
Background:
Duchenne muscular dystrophy (DMD) is characterized by muscle damage and progressive loss of muscle function in male children. DMD is one of the most devastating genetically linked neuromuscular diseases for which there is currently no cure. Most clinical studies for DMD utilize a standard protocol for measurement exploring pathophysiology, muscle strength and timed tasks. However, we propose that beyond traditional measurements, examining broader components of health as emphasized by the International Classification of Functioning, Disability and Health-Children and Youth Version (
ICF-
CY) may be of great value to children and their families, and important outcomes for future clinical trials.
Methods:
Forty boys with DMD and 10 healthy age-matched boys completed two self-report measures: the Children's Assessment of Participation and Enjoyment and the Pediatric Quality of Life Inventory 4.0. We investigated differences between the two groups with regard to participation in real life activities and perceived quality of life (QoL). Additionally, we compared participation in activities and QoL in younger and older boys with DMD.
Results:
Participation in physical activities was significantly lower in boys with DMD than healthy boys. Perceived QoL was markedly diminished in children with DMD relative to healthy controls, except in the emotional domain. The amount of time boys engage in an activity (frequency), as well as participation in social activities, declined for our older cohort of boys with DMD, but QoL in boys with DMD remained constant over time.
Conclusions:
The
ICF-
CY provides a conceptual framework and specific terminology that facilitates investigation of the consequences of impairment in children and youth. Our study is one of the first to explore self-report of participation in a cohort of boys with DMD. It was not surprising that activities of choice for boys with DMD were less physical in nature than healthy boys their age, but the consequences of less social engagement as the boys age is of great concern. Results from our study underscore the need to further evaluate activities that children elect to participate in, with special emphasis on facilitators and barriers to participation and how participation changes throughout the course of a disease.
