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Reliability of the Charcot-Marie-Tooth functional outcome measure.
Journal of the Peripheral Nervous System ( IF 3.9 ) Pub Date : 2020-08-26 , DOI: 10.1111/jns.12406
Paula Bray 1 , Kayla M D Cornett 1 , Timothy Estilow 2, 3 , Davide Pareyson 4 , Riccardo Zuccarino 5, 6 , Mariola Skorupinska 7 , Menelaos Pipis 7 , Janet E Sowden 8 , Steven Scherer 3 , Mary M Reilly 7 , Michael E Shy 5 , David N Herrmann 8 , Joshua Burns 1 , Katy J Eichinger 8
Affiliation  

The CMT‐FOM is a 13‐item clinical outcome assessment (COA) that measures physical ability in adults with Charcot‐Marie‐Tooth disease (CMT). Test‐retest reliability, internal consistency and convergent validity have been established for the CMT‐FOM. This current study sought to establish inter‐rater reliability. Following an in‐person training of six international clinical evaluators we recruited 10 participants with genetically diagnosed CMT1A, (aged 18‐74 years, 6 female). Participants were evaluated using the CMT‐FOM over 2 days. Participants were given at least a 3 hour rest between evaluations, and were assessed twice each day. Following the provision of training by master trainers, all 13 items of the CMT‐FOM exhibited excellent inter‐rater reliability for raw scores (ICC1,1 0.825‐0.989) and z‐scores (ICC1,1 0.762‐0.969). Reliability of the CMT‐FOM total score was excellent (ICC1,1 0.983, 95% CI 0.958‐0.995). The CMT‐FOM is a reliable COA used by clinical evaluators internationally. The next steps are to establish further validation through psychometric evaluation of the CMT‐FOM in the Accelerate Clinical Trials in CMT (ACT‐CMT) study.

中文翻译:


Charcot-Marie-Tooth 功能结果测量的可靠性。



CMT-FOM 是一项 13 项临床结果评估 (COA),用于衡量患有腓骨肌萎缩症 (CMT) 的成人的身体能力。 CMT-FOM 已建立重测信度、内部一致性和收敛效度。目前的研究旨在建立评估者间的可靠性。在对 6 名国际临床评估员进行现场培训后,我们招募了 10 名基因诊断为 CMT1A 的参与者(年龄 18-74 岁,6 名女性)。使用 CMT-FOM 对参与者进行为期 2 天的评估。参与者在评估之间至少有 3 小时的休息时间,并且每天评估两次。经过大师级培训师的培训后,CMT-FOM 的所有 13 个项目在原始分数 (ICC 1,1 0.825-0.989) 和 z 分数 (ICC 1,1 0.762-0.969) 方面均表现出出色的评估者间信度。 CMT-FOM 总分的可靠性非常好(ICC 1,1 0.983,95% CI 0.958-0.995)。 CMT-FOM 是国际临床评估人员使用的可靠 COA。下一步是通过加速 CMT 临床试验 (ACT-CMT) 研究中的 CMT-FOM 心理测量评估来进一步验证。
更新日期:2020-08-26
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