Goldberg J D, Weiss A I, Koury K J
J Clin Periodontol. 1986 May;13(5):411-7. doi: 10.1111/j.1600-051x.1986.tb01484.x.
In order to make effective use of the statistical theory of design of clinical trials for chronic diseases such as periodontal disease, certain issues must be considered. Any clinical trial requires that the disease definition be well-specified; that patient eligibility be explicit; that the observation times be explicit; that the duration and endpoint of therapy be specified; that the duration of subsequent followup observation be specified; and that the unit of observation (e.g., tooth, set of teeth, patient) be defined. In a chronic disease, the potential biases that can readily be introduced by self-selection of patients who enter the trial and/or who return for subsequent observation become more important, because subjects are required to remain on treatment and/or observation for prolonged periods. Further, the cyclical nature of some chronic diseases may require special attention to baseline definitions of active disease and disease outcome. These issues are illustrated with examples from clinical trials of hypertension, breast cancer screening, and Polycythemia Vera. Implications for periodontal disease are discussed.
为了有效利用针对诸如牙周病等慢性疾病的临床试验设计统计理论，必须考虑某些问题。任何临床试验都要求疾病定义明确；患者入选标准明确；观察时间明确；治疗的持续时间和终点明确；后续随访观察的持续时间明确；以及观察单位（例如，牙齿、牙齿组、患者）明确。在慢性疾病中，由于要求受试者长时间接受治疗和/或观察，由进入试验和/或返回接受后续观察的患者自我选择所容易引入的潜在偏倚变得更加重要。此外，一些慢性疾病的周期性可能需要特别关注活动性疾病和疾病转归的基线定义。这些问题通过高血压、乳腺癌筛查和真性红细胞增多症的临床试验实例进行说明。并讨论了对牙周病的影响。
