Children's Hospital London Health Sciences Centre, London, Canada.
Schulich School of Medicine & Dentistry, Western University, London, Ontario, Canada.
Neuropediatrics. 2024 Feb;55(1):42-48. doi: 10.1055/a-2101-7860. Epub 2023 May 26.
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.
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.
The results of regression modeling show baseline fatigue and baseline HRQoL ( = 0. 54 for child self-report and 0.51 for parent proxy report) and change in fatigue and HRQoL over 48 weeks ( = 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.
This study identified fatigue trajectories and risk factors associated with greater fatigue, helping clinicians and researchers identify the profile of fatigue in DMD children.
患有杜氏肌营养不良症(DMD)的儿童有疲劳的风险,这会对他们的健康相关生活质量(HRQoL)产生负面影响。本研究旨在通过检查 48 周的疲劳轨迹,并评估与这些疲劳轨迹相关的因素,来评估疲劳与 HRQoL 之间的关系。
本研究的样本包括 173 名年龄在 5 至 16 岁之间的患有 DMD 的受试者,他们参加了一项为期 48 周的新型治疗方法的 2 期临床试验(NCT00592553)。
回归模型的结果表明,基线疲劳和基线 HRQoL(儿童自我报告为 0.54,父母代理报告为 0.51)以及 48 周内疲劳和 HRQoL 的变化(儿童自我报告为 0.47,父母代理报告为 0.36)之间存在显著相关性。使用潜在类别增长模型识别出了儿童和父母代理报告的疲劳的三种独特的疲劳轨迹。与低疲劳组相比,高疲劳组的风险随着年龄的增加而增加 24%,并且儿童和父母代理分别报告的行走距离减少也会增加风险。
本研究确定了与更高疲劳相关的疲劳轨迹和危险因素,有助于临床医生和研究人员识别 DMD 儿童的疲劳特征。