Liu C, Blumhardt L D
Division of Clinical Neurology, Faculty of Medicine, University Hospital, Queen's Medical Centre, Nottingham, UK.
Mult Scler. 1999 Feb;5(1):22-8. doi: 10.1177/135245859900500105.
Published Phase III immunomodulatory treatment trials in relapsing and remitting multiple sclerosis have demonstrated a modest decline in attack rates, but only a minor effect on disability. As genuine disability progression is difficult to ascertain in relatively short studies with the conventional rating scales available, the acquisition and analysis of relapse data are critical. However, there are as yet unresolved questions related to the latter. We will first discuss the problems associated with relapse definitions by trial investigators, the paucity of the data collected (especially on the magnitude and duration of exacerbations) and statistical issues in their analysis. We will then suggest practical points for obtaining more accurate information on relapses and evaluating them meaningfully. While there is still general consensus among neurologists that primary endpoints for therapeutic trials should be clinical, improvements for future protocols are essential.
已发表的关于复发缓解型多发性硬化症的III期免疫调节治疗试验表明，发作率有适度下降，但对残疾的影响很小。由于在使用现有传统评分量表的相对短期研究中，很难确定真正的残疾进展情况，因此复发数据的收集和分析至关重要。然而，与后者相关的问题尚未得到解决。我们将首先讨论试验研究者在复发定义方面存在的问题、所收集数据的匮乏（尤其是关于病情加重的程度和持续时间）以及分析中的统计问题。然后，我们将提出一些实用要点，以获取关于复发的更准确信息并进行有意义的评估。虽然神经科医生之间仍普遍达成共识，即治疗试验的主要终点应该是临床指标，但未来方案的改进至关重要。
