Vandervelde Laure, Van den Bergh Peter Y K, Goemans Nathalie, Thonnard Jean-Louis
Rehabilitation and Physical Medicine unit, Université catholique de Louvain, Unité de Réadaptation et de Médecine Physique (READ 5375), Tour Pasteur, 53, Av Mounier, 1200 Brussels, Belgium.
Neuromuscul Disord. 2009 Feb;19(2):99-103. doi: 10.1016/j.nmd.2008.11.004. Epub 2009 Jan 23.
Recently, a self-reported scale of activity limitations, the ACTIVLIM questionnaire, was developed and validated in patients with neuromuscular disorders (NMD). The purpose of this study was to investigate its sensitivity to change. One hundred thirty-two patients with NMD (mean age, range: 31, 6-80) were assessed twice, with 21+/-4 months in between, using the ACTIVLIM questionnaire. Mean score change, effect size, standardized response, mean paired t-test and an individual-level statistical approach were calculated for groups of patients according to their self-rated functional status evolution and for three main diagnostic groups (Ambulant and wheelchair-bound Duchenne muscular dystrophy patients, myotonic dystrophy patients, and patients with Charcot-Marie-Tooth neuropathy). The responsiveness indices showed that the change in activity measures was higher in patients who reported deteriorated functional status and in patients with Duchenne muscular dystrophy. The ACTIVLIM questionnaire showed a good sensitivity to change and could be useful in research settings to characterize the disease course of NMD.
最近，一种自我报告的活动受限量表——ACTIVLIM问卷，已在神经肌肉疾病（NMD）患者中开发并验证。本研究的目的是调查其对变化的敏感性。使用ACTIVLIM问卷对132例NMD患者（平均年龄，范围：31岁，6 - 80岁）进行了两次评估，两次评估间隔21±4个月。根据患者自我评定的功能状态演变，对患者组以及三个主要诊断组（能行走和需轮椅辅助的杜氏肌营养不良患者、强直性肌营养不良患者、腓骨肌萎缩症患者）计算了平均得分变化、效应量、标准化反应、平均配对t检验和个体水平统计方法。反应性指标表明，在报告功能状态恶化的患者以及杜氏肌营养不良患者中，活动指标的变化更大。ACTIVLIM问卷对变化具有良好的敏感性，可用于研究环境中以描述NMD的疾病进程。
