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Updated: Jul 29, 2025

Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
09:18

Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy

Published on: January 12, 2019

10.4K

Duchenne Muscular Dystrophy Fatigue Trajectories

Yi Sally Wei1, Mona Hnaini2, Basmah ElAloul2

  • 1Children's Hospital London Health Sciences Centre, London, Canada.

Neuropediatrics
|May 26, 2023
PubMed

Insights

Children with Duchenne muscular dystrophy (DMD) experience fatigue impacting quality of life. This study identified fatigue trajectories and risk factors like age and walking distance, aiding clinical identification of fatigue profiles in DMD children.

Area of Science:

  • Neurology
  • Pediatrics
  • Quality of Life Research

Background:

  • Duchenne muscular dystrophy (DMD) significantly impacts children's health-related quality of life (HRQoL).
  • Fatigue is a common and detrimental symptom in children with DMD, affecting their daily functioning and overall well-being.

Purpose of the Study:

  • To assess the association between fatigue and HRQoL in children with DMD.
  • To examine fatigue trajectories over a 48-week period.
  • To identify factors associated with distinct fatigue trajectories in this population.

Main Methods:

  • A cohort of 173 children aged 5-16 with DMD participated in a 48-week phase 2 clinical trial.
  • Latent Class Growth Models were used to identify unique fatigue trajectories.
  • Regression modeling analyzed associations between baseline and changes in fatigue and HRQoL, and identified risk factors.

Main Results:

  • Baseline fatigue and HRQoL were significantly associated (R²=0.54 child, 0.51 parent proxy).
  • Changes in fatigue and HRQoL over 48 weeks also showed significant association (R²=0.47 child, 0.36 parent proxy).
  • Higher risk of high fatigue was linked to increased age and decreased walking distance.

Conclusions:

  • Distinct fatigue trajectories were identified in children with DMD.
  • Age and reduced walking distance are significant risk factors for higher fatigue levels.
  • Understanding these fatigue profiles aids clinicians in managing DMD symptoms and improving patient outcomes.
Abstract

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