TY -的T1 -发展Rasch-Built肌萎缩性脊髓侧索硬化症损害多畴的规模来衡量疾病进展在ALS JF -神经学乔-神经病学SP - e602 LP - - 10.1212 / WNL e612做。首页0000000000207483六世- 101 - 6盟Adriaan d . de Jongh AU -鲁本P.A. van Eijk盟盟——汤米他莱昂哈德·a . m .缩聚AU -安妮塔Beelen盟科妮van der Meijden AU -迈克尔·a·范Es盟Johanna硕士Visser-Meily AU - t . Kruitwagen盟以斯帖1月h . Veldink AU -伦纳德h . van den Berg Y1 - 2023/08/08 UR - //www.ez-admanager.com/content/101/6/e602.abstract N2 -背景和目标当前尺度用于肌萎缩性脊髓侧索硬化症(ALS)试图总结不同的功能域首页或“维度”1的总体成绩,可能不能准确地描述个体病人的疾病严重程度和预后。总分的使用风险声明治疗无效如果不是所有维度ALS疾病进展同样的影响。我们旨在开发ALS障碍多畴的规模(目标),全面描述疾病进展和增加的可能性识别有效的治疗方法。方法修改后的ALS功能评定量表(ALSFRS-R)和初步的问卷调查,基于文献综述和病人输入,完成在线从荷兰ALS患者注册中心每隔两月一次的在一段时间内的12个月。拉希两周两次试验法的因素分析,分析,和信噪比进行了优化策略来创建一个多畴的规模。可靠性、纵向下降和对生存进行评估。所需的样本量检测进展率减少35% / 6或12个月评估临床试验,定义了ALSFRS-R或目标分量表作为主要终点的家庭。结果初步调查问卷,包括110个问题,完成了367个病人。三个一维的分量表,和多畴的规模是由7球,11个马达,和5呼吸问题。分量表实现拉希模型需求,与优秀的0.91 - -0.94两次试验法的可靠性和强大与生存的关系(p & lt; 0.001). Compared with the ALSFRS-R, signal-to-noise ratios were higher as patients declined more uniformly per subscale. Consequently, the estimated sample size reductions achieved with the AIMS compared with those achieved with the ALSFRS-R were 16.3% and 25.9% for 6-month and 12-month clinical trials, respectively.Discussion We developed the AIMS, consisting of unidimensional bulbar, motor, and respiratory subscales, which may characterize disease severity better than a total score. AIMS subscales have high test-retest reliability, are optimized to measure disease progression, and are strongly related to survival time. The AIMS can be easily administered and may increase the likelihood of identifying effective treatments in ALS clinical trials.AIMS=ALS Impairment Multidomain Scale; ALS=amyotrophic lateral sclerosis; ALSFRS-R=Revised ALS Functional Rating Scale; ICC=intraclass coefficient; RMSEA=root mean square error of approximation; ROADS=Rasch-Built Overall ALS Disability Scale ER -