Pincus Theodore, Sokka Tuulikki
Division of Rheumatology and Immunology, Department of Medicine, Vanderbilt University, 203 Oxford House, Box 5, Nashville, TN 37232-4500, USA.
Rheum Dis Clin North Am. 2004 Nov;30(4):701-24, v-vi. doi: 10.1016/j.rdc.2004.08.002.
Randomized controlled clinical trials provide the best method to distinguish a drug from placebo without the inevitable selection biases that are seen in standard clinical care. This article reviews designs and limitations of clinical trials that are used in rheumatic diseases. The primary design in clinical trials is a parallel, in which patients are randomized in parallel to different therapies at different dosages or placebo. In recent years, other designs have been used increasingly, including "step-up," "step-down," and "cross-over" designs. Limitations of clinical trials in chronic diseases include a short time frame versus the long duration of disease, inclusion and exclusion criteria, use of surrogate markers that may not represent clinically relevant markers, statistical significance does not necessarily indicate clinical significance necessarily, and the fact that a control group does not assure the absence of bias. Therefore, long-term databases are needed to supplement clinical trials in analyzing results of therapy for rheumatoid arthritis.
随机对照临床试验提供了区分药物与安慰剂的最佳方法，而不会出现标准临床护理中不可避免的选择偏倚。本文综述了用于风湿性疾病的临床试验的设计和局限性。临床试验的主要设计是平行设计，即患者被平行随机分配到不同剂量的不同疗法或安慰剂组。近年来，其他设计越来越多地被使用，包括“逐步递增”、“逐步递减”和“交叉”设计。慢性病临床试验的局限性包括与疾病的长期病程相比时间框架较短、纳入和排除标准、使用可能不代表临床相关指标的替代指标、统计学显著性不一定必然表明临床显著性，以及对照组不能确保无偏倚这一事实。因此，需要长期数据库来补充临床试验，以分析类风湿关节炎的治疗结果。
