% 0期刊文章% Adriaan D . de Jongh %鲁本P.A. van Eijk % %他答:里昂哈汤米·m·缩聚%一科妮van der Meijden Anita Beelen % %迈克尔·A·范·Es %一个以斯帖T . Kruitwagen Johanna Visser-Meily % % 1 H Veldink %伦纳德·H . van den Berg % T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ALS % D R 10.1212 / WNL 2023%。0000000000207483 % J半岛投注体育官网神经病学% P 10.1212 / WNL。0000000000207483 X %的背景和目的: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或“维度”成一个整体得分,可能不能准确地描述个体患者的疾病严重程度和预后。使用综合得分风险声明治疗无效如果不是所有维度的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: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。三个一维的分量表识别和多畴的规模是由7球,11汽车和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 < 0.001)。 Compared to 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 to the ALSFRS-R were 16.3% and 25.9% for 6- and 12- month clinical trials, respectively.Discussion: We developed the AIMS, consisting of unidimensional bulbar, motor and respiratory subscales that 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. %U //www.ebmtp.com/content/neurology/early/2023/06/16/WNL.0000000000207483.full.pdf