Charcot-Marie-Tooth 功能结局测量量表（CMT-FOM）。
The Charcot-Marie-Tooth Functional Outcome Measure (CMT-FOM).
机构信息
From the Department of Neurology (K.E., J.M., J.S., D.N.H.), University of Rochester, NY; Faculty of Health Sciences & Children's Hospital at Westmead (J.B., K.C.), University of Sydney, Australia; and Departments of Neurology (C.B., M.E.S.), Physical Therapy (M.L.S.), and Pediatrics (R.S.), University of Iowa, Carver College of Medicine, Iowa City.
出版信息
Neurology. 2018 Oct 9;91(15):e1381-e1384. doi: 10.1212/WNL.0000000000006323. Epub 2018 Sep 19.
OBJECTIVE
The purpose of this study was to examine the feasibility, reliability, and convergent validity of the Charcot-Marie-Tooth Functional Outcome Measure (CMT-FOM), a new performance-based measure assessing functional ability in adults with CMT disease.
METHODS
Adults with CMT type 1A (CMT1A) were recruited at the Universities of Rochester and Iowa. Participants were assessed using the CMT-FOM, CMT Exam Score (CMTES), and a symptom report. Test-retest reliability was examined using intraclass correlation coefficients, internal consistency using Cronbach α, and convergent and known-groups validity using Spearman rank analysis and the Mann-Whitney test.
RESULTS
Forty-three individuals (70% women; mean age 41, SD 14.9 years) participated. The CMT-FOM (mean 25.3 ± 8.7, range 12-44/52) was moderately correlated with the CMTES (ρ = 0.62; < 0.0001) and exhibited acceptable reliability (intraclass correlation coefficient = 0.92) and internal consistency (Cronbach α = 0.81). The CMT-FOM discriminated between participants with clinically mild vs moderate-severe CMT1A. Participants with the mildest CMT1A who demonstrated a floor effect on the CMTES showed functional limitations on the CMT-FOM.
CONCLUSIONS
The CMT-FOM is well tolerated and showed no floor/ceiling effects in an adult CMT1A cohort matching those likely to enter upcoming clinical trials. It appears to be reliable, and our data support convergent and known-groups validity in adults with CMT1A. Longitudinal studies further examining the psychometric properties of the CMT-FOM and its responsiveness to change before its application in therapeutic trials are necessary.
目的
本研究旨在检验 Charcot-Marie-Tooth 功能评定量表（CMT-FOM）的可行性、可靠性和聚合效度，该量表是一种新的基于表现的测量工具，用于评估 CMT 疾病成人的功能能力。
方法
在罗切斯特大学和爱荷华大学招募了 CMT 1A 型（CMT1A）的成年人。参与者使用 CMT-FOM、CMT 检查评分（CMTES）和症状报告进行评估。使用组内相关系数检验测试-重测信度，使用 Cronbach α 检验内部一致性，使用 Spearman 秩分析和 Mann-Whitney 检验检验聚合和已知组有效性。
结果
共有 43 人（70%为女性；平均年龄 41 岁，SD 为 14.9 岁）参与了研究。CMT-FOM（平均 25.3±8.7，范围 12-44/52）与 CMTES 呈中度相关（ρ=0.62；<0.0001），具有可接受的可靠性（组内相关系数=0.92）和内部一致性（Cronbach α=0.81）。CMT-FOM 可区分临床轻度与中度-重度 CMT1A 参与者。在 CMTES 上表现出地板效应的最轻度 CMT1A 参与者在 CMT-FOM 上表现出功能限制。
结论
CMT-FOM 在匹配即将进入临床试验的患者的 CMT1A 成人队列中具有良好的耐受性，且没有出现地板/天花板效应。它似乎具有可靠性，我们的数据支持 CMT1A 成人的聚合和已知组有效性。在其在治疗试验中的应用之前，有必要进行进一步研究以检验 CMT-FOM 的心理测量特性及其对变化的反应能力的纵向研究。
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