Abstract
Introduction Children with Duchenne muscular dystrophy (DMD) are at risk of experiencing fatigue
that negatively impacts their health-related quality of life (HRQoL). This study aimed
to assess the association between fatigue and HRQoL, by examining fatigue trajectories
over 48 weeks, and assessing factors associated with these fatigue trajectories.
Methods The study sample consisted of 173 DMD subjects enrolled in a 48-week-long phase 2
clinical trial (NCT00592553) for a novel therapeutic who were between the ages of
5 and 16 years.
Results The results of regression modeling show baseline fatigue and baseline HRQoL (R
2 = 0. 54 for child self-report and 0.51 for parent proxy report) and change in fatigue
and HRQoL over 48 weeks (R
2 = 0.47 for child self-report and 0.36 for parent proxy report) were significantly
associated with one another. Three unique fatigue trajectories using Latent Class
Growth Models were identified for child and parent proxy reported fatigue. The risk
of being in the high fatigue group as compared to the low fatigue group increased
by 24% with each year increase in age and also with decreasing walking distance, as
reported by children and parent proxy, respectively.
Conclusion This study identified fatigue trajectories and risk factors associated with greater
fatigue, helping clinicians and researchers identify the profile of fatigue in DMD
children.
Keywords
Duchenne muscular dystrophy - fatigue - health-related quality of life
