Departments of Neurology and Pediatrics, University of Pennsylvania School of Medicine, and The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania 19104-4318, USA.
Pediatr Neurol. 2010 May;42(5):335-7. doi: 10.1016/j.pediatrneurol.2010.01.002.
The use of health-related quality of life scales as outcome measures in clinical trials is increasing. Although such measures have been validated in adults with Friedreich ataxia, they have not been studied in children with this disorder. The health-related quality of life of children with Friedreich ataxia was assessed using the PedsQL 4.0 Generic Core Module and Multidimensional Fatigue Scale. The scores from the Friedreich ataxia cohort were compared to those of control groups (children without a chronic disease). Minimal missing responses support the feasibility of using the PedsQL 4.0 in the Friedreich ataxia population. The scales demonstrated internal consistency, and concordance was observed between child and proxy scores. Children with Friedreich ataxia and their proxies reported lower health-related quality of life than did controls in the Core and Fatigue scales. A modest relationship was seen between markers of disease status and health-related quality of life, providing support for the idea that children with Friedreich ataxia have a lower health-related quality of life than those without a chronic disease. Additional studies are needed to examine the relationship between health-related quality of life and disease markers and to further establish the validity of the PedsQL 4.0 in this population.
健康相关生活质量量表在临床试验中的应用正在增加。尽管这些量表已经在弗里德里希共济失调的成年人中得到验证，但尚未在患有这种疾病的儿童中进行研究。使用 PedsQL 4.0 通用核心模块和多维疲劳量表评估了患有弗里德里希共济失调的儿童的健康相关生活质量。将弗里德里希共济失调队列的评分与对照组（无慢性病的儿童）的评分进行比较。最小的缺失反应支持在弗里德里希共济失调人群中使用 PedsQL 4.0 的可行性。这些量表表现出内部一致性，并且在儿童和代理评分之间观察到了一致性。在核心和疲劳量表中，患有弗里德里希共济失调的儿童及其代理人报告的健康相关生活质量低于对照组。疾病状态标志物与健康相关生活质量之间存在适度的关系，这支持了弗里德里希共济失调儿童的健康相关生活质量低于无慢性病儿童的观点。需要进一步的研究来检查健康相关生活质量与疾病标志物之间的关系，并进一步在该人群中确立 PedsQL 4.0 的有效性。
