Department of Neurology, VU University Medical Center, De Boelelaan 1117, Amsterdam, The Netherlands.
Mult Scler. 2010 May;16(5):604-10. doi: 10.1177/1352458509359922. Epub 2010 Jan 19.
Because the development of new treatments in multiple sclerosis as well as the awareness of the importance of patient-oriented measures have become more important in the last two decades, new outcome measures have been developed with the aim of being more responsive to change and more clinically relevant to patients. The ability to detect improvement is sparsely studied. In the present study we evaluate the responsiveness of the Expanded Disability Status Scale and two quantitative tests (the timed 25-foot walk test and the nine-hole peg test) separately and in combination, to detect improvement after intravenous methylprednisolone. The Expanded Disability Status Scale, the timed 25-foot walk test and the nine-hole peg test were assessed in 112 multiple sclerosis patients before and 6 weeks after intravenous methylprednisolone. In addition patients were asked to rate their change as an anchor to evaluate the performance of the tests. Combining the timed 25-foot walk test and the nine-hole peg test turned out to be the optimal combination of measures to predict patient perceived improvement (positive predictive value of 67% and a negative predictive value of 59%, likelihood ratio of positive test 2.31 (95% confidence interval 1.08-4.95)). In the higher Expanded Disability Status Scale range (4.5 and higher), for all measures a significant change was more often perceived as clinically relevant than in the lower disability range. The Expanded Disability Status Scale seems not to be the preferred outcome of choice to detect patient perceived improvement in multiple sclerosis, especially in the lower Expanded Disability Status Scale range. Combining the timed walk test and the nine-hole peg test can improve the sensitivity to detect clinically relevant changes without conceding with respect to specificity.
由于在过去二十年中，多发性硬化症的新疗法的发展以及对患者导向措施重要性的认识变得更加重要，因此已经开发出了新的疗效评估方法，旨在更灵敏地反映变化并与患者的临床相关性更强。但是，关于检测改善的能力的研究还很少。在本研究中，我们分别评估了扩展残疾状况量表（Expanded Disability Status Scale，EDSS）和两种定量测试（定时 25 英尺步行测试和九孔钉测试）以及将它们组合在一起，以检测静脉内甲基强的松龙治疗后的改善情况。在静脉内甲基强的松龙治疗前和 6 周后，对 112 例多发性硬化症患者进行了 EDSS、定时 25 英尺步行测试和九孔钉测试的评估。此外，还要求患者对其改善情况进行评分，作为评估测试性能的锚定标准。结果表明，定时 25 英尺步行测试和九孔钉测试的组合是预测患者感知改善的最佳疗效评估方法（阳性预测值为 67%，阴性预测值为 59%，阳性测试的似然比为 2.31（95%置信区间为 1.08-4.95））。在更高的 EDSS 范围（4.5 及以上）中，对于所有措施，在更严重的残疾范围内，感知到的显著变化更常被认为具有临床相关性。EDSS 似乎不是检测多发性硬化症患者感知改善的首选疗效评估方法，尤其是在 EDSS 评分较低的范围内。定时步行测试和九孔钉测试的组合可以提高检测具有临床相关性变化的敏感性，而不会降低特异性。
