Duchenne Muscular Dystrophy Fatigue Trajectories.

IF 1.1 4区 医学 Q4 CLINICAL NEUROLOGY Neuropediatrics Pub Date : 2024-02-01 Epub Date: 2023-05-26 DOI:10.1055/a-2101-7860
Yi Sally Wei, Mona Hnaini, Basmah ElAloul, Eugenio Zapata, Craig Campbell
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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.

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杜兴氏肌肉萎缩症疲劳轨迹。
导言:患有杜氏肌营养不良症(DMD)的儿童有可能出现疲劳,从而对其健康相关生活质量(HRQoL)产生负面影响。本研究旨在通过观察 48 周内的疲劳轨迹,评估疲劳与 HRQoL 之间的关联,并评估与这些疲劳轨迹相关的因素:研究样本包括173名参加一项为期48周的新型疗法2期临床试验(NCT00592553)的DMD受试者,他们的年龄在5岁至16岁之间:回归模型的结果显示,基线疲劳和基线 HRQoL(儿童自我报告的 R 2 = 0.54,家长代理报告的 R 2 = 0.51)以及 48 周内疲劳和 HRQoL 的变化(儿童自我报告的 R 2 = 0.47,家长代理报告的 R 2 = 0.36)彼此显著相关。利用潜类增长模型,为儿童和家长代理报告的疲劳确定了三种独特的疲劳轨迹。根据儿童和家长代理的报告,与低疲劳组相比,高疲劳组的风险随着年龄的增加和步行距离的减少而增加了 24%:本研究确定了疲劳轨迹以及与疲劳程度相关的风险因素,有助于临床医生和研究人员识别 DMD 儿童的疲劳特征。
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来源期刊
Neuropediatrics
Neuropediatrics 医学-临床神经学
CiteScore
2.80
自引率
0.00%
发文量
94
审稿时长
>12 weeks
期刊介绍: For key insights into today''s practice of pediatric neurology, Neuropediatrics is the worldwide journal of choice. Original articles, case reports and panel discussions are the distinctive features of a journal that always keeps abreast of current developments and trends - the reason it has developed into an internationally recognized forum for specialists throughout the world. Pediatricians, neurologists, neurosurgeons, and neurobiologists will find it essential reading.
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