Sylvia Lawry Centre for Multiple Sclerosis Research, Munich, Germany.
J Neurol Sci. 2009 Dec;287 Suppl 1:S50-5. doi: 10.1016/S0022-510X(09)71301-2.
There is no gold standard in monitoring disease activity for clinical trials in multiple sclerosis. Various outcome measures, including relapses, disability and magnetic resonance imaging (MRI) measures have been used to demonstrate the efficacy of the different available therapies for multiple sclerosis. Recently, the potential limitations of these measures have received increasing attention, and these have stimulated research into more appropriate and sensitive outcome measures for clinical trials. For example, it has been shown that widely-used MRI measures add little, if any, independent information to that provided by more clinically relevant measures such as relapses and disability. Similarly, the Expanded Disability status Scale (EDSS), which is the most widely-used measure of disability related to multiple sclerosis, is insufficiently sensitive to detect robust changes in disability over the timeframes usually used in clinical trials. An alternative to the EDSS is the Multiple Sclerosis Severity Score (MSSS), a severity scale which relates clinical disability to disease duration. The MSSS was originally developed from a database of nearly ten thousand patients from eleven European countries and Australia and has since been reproduced in an independent dataset of 1134 patients from the placebo arms of randomised clinical trials. Based on the MSSS score, disease severity can be defined, which shows stability over time and may provide evidence-based decision support for patient management. Another alternative to measure disability is the objective quantification of physical activity. There is evidence that recent developments in pervasive computing using tiny accelerometers may have the potential to increase the reliability and precision of motor assessment, especially in the mid-range of the EDSS. The outcome measures discussed have potential use as online tools for evidence-based decision support which are increasingly being used in medical research and clinical decision-making.
在多发性硬化症的临床试验中，目前并没有监测疾病活动的金标准。各种结果指标，包括复发、残疾和磁共振成像（MRI）测量，都已被用于证明各种现有多发性硬化症治疗方法的疗效。最近，这些措施的潜在局限性受到了越来越多的关注，这也促使人们研究更合适和敏感的临床试验结果指标。例如，已经表明，广泛使用的 MRI 测量指标除了提供更多与临床相关的指标（如复发和残疾）之外，几乎没有提供任何独立信息。同样，扩展残疾状况量表（EDSS）是最广泛用于测量与多发性硬化症相关的残疾的指标，但它对临床试验中通常使用的时间框架内的残疾变化检测不够敏感。EDSS 的替代方法是多发性硬化症严重程度评分（MSSS），这是一种将临床残疾与疾病持续时间相关联的严重程度量表。MSSS 最初是从来自 11 个欧洲国家和澳大利亚的近 10000 名患者的数据库中开发的，此后在来自随机临床试验安慰剂组的 1134 名患者的独立数据集得到了重现。基于 MSSS 评分，可以定义疾病严重程度，其在时间上具有稳定性，并且可能为患者管理提供基于证据的决策支持。另一种测量残疾的替代方法是对身体活动进行客观量化。有证据表明，使用微型加速度计的普及计算的最新进展可能具有提高运动评估的可靠性和精确性的潜力，特别是在 EDSS 的中值范围内。讨论的这些结果指标具有作为基于证据的决策支持的在线工具的潜在用途，它们越来越多地用于医学研究和临床决策制定。
