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Neuropediatrics 2024; 55(01): 042-048
DOI: 10.1055/a-2101-7860
Original Article

Duchenne Muscular Dystrophy Fatigue Trajectories

Yi Sally Wei
1   Children's Hospital London Health Sciences Centre, London, Canada
,
Mona Hnaini
2   Schulich School of Medicine & Dentistry, Western University, London, Ontario, Canada
,
Basmah ElAloul
2   Schulich School of Medicine & Dentistry, Western University, London, Ontario, Canada
,
Eugenio Zapata
2   Schulich School of Medicine & Dentistry, Western University, London, Ontario, Canada
,
Craig Campbell
1   Children's Hospital London Health Sciences Centre, London, Canada
2   Schulich School of Medicine & Dentistry, Western University, London, Ontario, Canada
3   Department of Paediatrics, Clinical Neurological Sciences and Epidemiology, Western University, London, Ontario, Canada
› Author Affiliations
Funding This study was supported by an investigator-initiated grant from PTC Therapeutics.
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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

Disclosure

Craig Campbell has been a site investigator for ataluren clinical trials and served as Chair of the Advisory Committee for PTC Therapeutics.


Supplementary Material



Publication History

Received: 14 February 2022

Accepted: 23 May 2023

Accepted Manuscript online:
26 May 2023

Article published online:
04 July 2023

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