Wolfe Gil I, Barohn Richard J, Sanders Donald B, McDermott Michel P
Department of Neurology, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, Texas 75390-8897, USA.
Department of Neurology, University of Kansas Medical Center, Kansas City, Kansas, USA.
Muscle Nerve. 2008 Nov;38(5):1429-1433. doi: 10.1002/mus.21142.
We determined the strength of correlation among, and responsiveness of, outcome measures used in a multicenter, double-blind, placebo-controlled trial of mycophenolate mofetil in combination with prednisone in myasthenia gravis (MG). The primary efficacy measure was the change from baseline in the Quantitative MG (QMG) score at week 12. Secondary outcome measures included the MG-Activities of Daily Living profile (MG-ADL) and MG Manual Muscle Test (MMT). The measures were collected at baseline and at weeks 4, 8, and 12 in the blinded study and at weeks 16, 20, 28, and 36 in an optional open-label extension. At baseline, the QMG was moderately correlated with the MG-ADL (r=0.55, P<0.0001) and the MMT (r=0.53, P<0.0001), but the correlation between the MG-ADL and the MMT was lower (r=0.30, P=0.007). These findings were similar at weeks 4, 8, and 12. Similar correlations were found among the changes in scores from baseline at weeks 12 and 36. The MMT and MG-ADL appeared to be the most sensitive measures for changes in MG status at weeks 12 and 36. Although a task force has recommended use of the QMG in prospective MG trials, the MMT and MG-ADL appear to be suitable alternatives and offer potential advantages. No special training or equipment is required, and they take less time.
我们确定了在一项多中心、双盲、安慰剂对照试验中，用于评估霉酚酸酯联合泼尼松治疗重症肌无力（MG）的各项结局指标之间的相关性强度及反应性。主要疗效指标是第12周时重症肌无力定量（QMG）评分相对于基线的变化。次要结局指标包括重症肌无力日常生活活动量表（MG-ADL）和重症肌无力徒手肌力测试（MMT）。在盲法研究的基线、第4周、第8周和第12周以及在一项可选的开放标签延长期的第16周、第20周、第28周和第36周收集这些指标。在基线时，QMG与MG-ADL（r=0.55，P<0.0001）和MMT（r=0.53，P<0.0001）中度相关，但MG-ADL与MMT之间的相关性较低（r=0.30，P=0.007）。在第4周、第8周和第12周时这些结果相似。在第12周和第36周时，从基线开始的评分变化之间也发现了类似的相关性。在第12周和第36周时，MMT和MG-ADL似乎是评估MG状态变化最敏感的指标。尽管一个特别工作组建议在前瞻性MG试验中使用QMG，但MMT和MG-ADL似乎是合适的替代指标，且具有潜在优势。它们不需要特殊培训或设备，而且耗时较少。
