Duchenne 型肌营养不良症的功能变化：一项 12 个月的纵向队列研究。
Functional changes in Duchenne muscular dystrophy: a 12-month longitudinal cohort study.
机构信息
Department of Paediatric Neurology, Catholic University, Rome.
出版信息
Neurology. 2011 Jul 19;77(3):250-6. doi: 10.1212/WNL.0b013e318225ab2e. Epub 2011 Jul 6.
OBJECTIVE
The aim of the study was to assess different outcome measures in a cohort of ambulant boys with Duchenne muscular dystrophy (DMD) over 12 months in order to establish the spectrum of possible changes in relation to age and steroid treatment.
METHODS
The study is a longitudinal multicentric cohort study. A total of 106 ambulant patients with DMD were assessed using the 6-minute walk test (6MWT) and North Star Ambulatory Assessment (NSAA) at baseline and 12 months. Clinical data including age and steroid treatment were collected.
RESULTS
During the 12 months of the study, we observed a mean decline of 25.8 meters in the 6MWT with a SD of 74.3 meters. On NSAA, the mean decline was 2.2 points with a SD of 3.7. Not all the boys with DMD in our cohort showed a decline over the 12 months, with young boys showing some improvement in their 6MWT and NSAA scores up to the age of 7. NSAA and the 6MWT had the highest correlation (r = 0.52, p < 0.001).
CONCLUSIONS
This study provides longitudinal data of NSAA and 6MWT over a 12-month period. These data can be useful when designing a clinical trial.
目的
本研究旨在评估 12 个月内患有杜氏肌营养不良症（DMD）的门诊男孩队列中的不同结局测量指标，以确定与年龄和类固醇治疗相关的可能变化范围。
方法
本研究为一项纵向多中心队列研究。共对 106 名可走动的 DMD 患者在基线和 12 个月时使用 6 分钟步行测试（6MWT）和北美之星门诊评估（NSAA）进行评估。收集了包括年龄和类固醇治疗在内的临床数据。
结果
在研究的 12 个月期间，我们观察到 6MWT 的平均下降了 25.8 米，标准差为 74.3 米。在 NSAA 上，平均下降了 2.2 分，标准差为 3.7。我们队列中的并非所有 DMD 男孩在 12 个月内都有下降，年龄较小的男孩在 6MWT 和 NSAA 评分上直到 7 岁都有一定程度的改善。NSAA 和 6MWT 相关性最高（r=0.52，p<0.001）。
结论
本研究提供了 12 个月内 NSAA 和 6MWT 的纵向数据。这些数据在设计临床试验时可能会很有用。
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