Brown J R, Beebe G W, Kurtzke J F, Loewenson R B, Silberberg D H, Tourtellotte W W
Neurology. 1979 Sep;29(9 pt. 2):3-23. doi: 10.1212/wnl.29.9_part_2.3.
Poorly designed trials of therapy for multiple sclerosis (MS) can waste time and money, and may lead either to false hopes or to the overlooking of a potentially effective treatment. A well-designed trial may well develop useful scientific information even if the putative therapy fails to show any therapeutic effect. The diagnosis, clinical course, and definitions of the stages of MS are discussed as they relate to trials of therapy. The goals of such trials include favorable modification of an exacerbation, favorable modification or prevention of future exacerbations, effective treatment of the progressive stage, and improvement of function in the stable-deficit stage. There should be an orderly progression from a small preliminary trial to a modest pilot trial and, when indicated, a full trial. All types of trials require careful organization and management, appropriate selection of patients, and properly planned and recorded observations. The treatment contrast--how the new treatment will be evaluated--provides the essential structure of the trial. The hypothesis being examined, the treatment contrast, and the observations being made in the designed clinical trial will govern the form of the analysis and the nature of the interpretations. Each goal requires that specific strategies and design considerations be applied to preliminary, pilot, and full trials.
设计不佳的多发性硬化症（MS）治疗试验可能会浪费时间和金钱，并且可能导致错误的希望或忽视一种潜在有效的治疗方法。即使假定的治疗方法未能显示出任何治疗效果，精心设计的试验也很可能会产生有用的科学信息。本文将讨论MS的诊断、临床病程以及各阶段的定义，因为它们与治疗试验相关。此类试验的目标包括有利地改善病情加重情况、有利地改善或预防未来的病情加重、有效治疗进展期以及改善稳定缺损期的功能。应该从小型初步试验有序推进到适度的试点试验，并在适当时进行全面试验。所有类型的试验都需要精心组织和管理、合理选择患者以及进行妥善规划和记录的观察。治疗对照——即如何评估新治疗方法——为试验提供了基本结构。在设计的临床试验中所检验的假设、治疗对照以及所进行的观察将决定分析的形式和解释的性质。每个目标都要求将特定的策略和设计考虑因素应用于初步试验、试点试验和全面试验。
