Mayhew Jill E, Florence Julaine M, Mayhew Thomas P, Henricson Erik K, Leshner Robert T, McCarter Robert J, Escolar Diana M
Research Center for Genetic Medicine, Children's National Medical Center, 111 Michigan Avenue NW, Washington, DC 20010, USA.
Muscle Nerve. 2007 Jan;35(1):36-42. doi: 10.1002/mus.20654.
We studied the reliability of a series of endpoints in an evaluation of subjects with Duchenne muscular dystrophy (DMD). The endpoints included quantitative muscle tests (QMTs), timed function tests, forced vital capacity (FVC), and manual muscle tests (MMT). Thirty-one ambulatory subjects with DMD (mean age 8.9 years; range 5-16 years) were evaluated at eight sites by 15 newly trained evaluators as a test of interrater reliability of outcome measures. Both total QMT score [intraclass correlation coefficient (ICC) 0.96] and individual QMT assessments (ICC 0.85-0.96) were highly reliable. Forced vital capacity and all timed function tests were also highly reliable (ICC 0.97-0.99). MMT was the least reliable assessment method (ICC 0.61). These data suggest that primary surrogate outcome measures in large multicenter clinical trials in DMD should use QMT, FVC, or time function tests to obtain maximum power and greatest sensitivity.
我们在对杜氏肌营养不良症（DMD）患者的评估中研究了一系列终点指标的可靠性。这些终点指标包括定量肌肉测试（QMT）、定时功能测试、用力肺活量（FVC）和徒手肌力测试（MMT）。31名DMD门诊患者（平均年龄8.9岁；范围5 - 16岁）在8个地点由15名新培训的评估人员进行评估，以此作为对结果测量的评估者间可靠性的测试。总QMT评分[组内相关系数（ICC）0.96]和个体QMT评估（ICC 0.85 - 0.96）都具有高度可靠性。用力肺活量和所有定时功能测试也具有高度可靠性（ICC 0.97 - 0.99）。MMT是最不可靠的评估方法（ICC 0.61）。这些数据表明，在DMD的大型多中心临床试验中，主要替代结局指标应使用QMT、FVC或时间功能测试，以获得最大功效和最高敏感性。
