Gocheva Vanya, Schmidt Simone, Orsini Anna-Lena, Hafner Patricia, Schaedelin Sabine, Rueedi Nicole, Weber Peter, Fischer Dirk
Division of Neuropediatrics and Developmental Medicine, University Children's Hospital of Basel (UKBB), University of Basel, Basel, Switzerland.
Department of Neurology, General Hospital Hietzing with Neurological Center Rosenhügel, Vienna, Austria.
J Child Neurol. 2019 Dec;34(14):873-885. doi: 10.1177/0883073819865681. Epub 2019 Aug 6.
This cross-sectional study assessed health-related quality of life (HRQOL) in ambulant and nonambulant patients with Duchenne muscular dystrophy, and explored the association between health-related quality of life and clinically assessed motor function. The Pediatric Quality of Life Inventory (PedsQL) Generic Core Scale and PedsQL Neuromuscular module were completed by 34 parent-child dyads. Association between PedsQL scores and overall motor abilities and the transfers and standing posture domain measured by the Motor Function Measure were examined. Child self-reported and parent proxy-reported mean PedsQL scores for children with Duchenne muscular dystrophy were lower than those for healthy children for the physical and psychosocial health-related quality of life. Fifty-six percent of patients reported clinically impaired psychosocial health-related quality of life scores. Several aspects of the generic and disease-specific health-related quality of life in patients with Duchenne muscular dystrophy were positively associated to overall motor function and transfers and standing posture domain. Associations remained stable when adjusted for age and corticosteroid use. The Motor Function Measure is clinically meaningful in the context of a patient's day-to-day life.
这项横断面研究评估了杜氏肌营养不良症能行走和不能行走患者的健康相关生活质量（HRQOL），并探讨了健康相关生活质量与临床评估的运动功能之间的关联。34对亲子完成了儿童生活质量量表（PedsQL）通用核心量表和PedsQL神经肌肉模块。研究了PedsQL评分与运动功能测量所评估的总体运动能力以及转移和站立姿势领域之间的关联。杜氏肌营养不良症患儿自我报告和家长代理报告的PedsQL平均得分在身体和心理社会健康相关生活质量方面低于健康儿童。56%的患者报告其心理社会健康相关生活质量得分存在临床受损情况。杜氏肌营养不良症患者通用和疾病特异性健康相关生活质量的几个方面与总体运动功能以及转移和站立姿势领域呈正相关。在对年龄和皮质类固醇使用情况进行调整后，这种关联仍然稳定。运动功能测量在患者的日常生活背景下具有临床意义。
