Ulbrich-Zürni Susanne, Teut Michael, Roll Stephanie, Mathie Robert T
Swiss Homeopathy Association, Zürich, Switzerland.
Institute for Social Medicine, Epidemiology and Health Economics, Charité - Universitätsmedizin Berlin, Berlin, Germany.
Homeopathy. 2018 Feb;107(1):10-18. doi: 10.1055/s-0037-1621731. Epub 2018 Jan 23.
The randomised controlled trial (RCT) is considered the 'gold standard' for establishing treatment efficacy or effectiveness of an intervention, but its data do not infer response in an individual patient. Individualised clinical care, a fundamental principle in complementary and alternative medicine (CAM), including homeopathy, seems well disposed in principle to being researched by single-patient (N-of-1) study design. Guidelines for reporting N-of-1 trials have recently been developed.
To overview the current status in the literature of the N-of-1 method and its application in medicine, including CAM. To consider whether the N-of-1 trial design offers an opportunity for novel research in homeopathy. N-OF-1 TRIAL DESIGN: The N-of-1 trial applies the principles of the conventional crossover, blinded, RCT design. The treatment under study and the comparator are repeated in a randomised order, and with suitable washout time, over a defined period. N-of-1 design is constrained for use in chronic stable conditions, and for interventions that have quick onset and cessation of effect, with modest or negligible carryover. Outcome data can be aggregated and interpreted for the individual subject; they can also be pooled with data from several similar N-of-1 trials, enabling more generalisable conclusions. THE N-OF-1 TRIAL IN CAM: The typical individualisation of patient care can be accommodated in N-of-1 study design if the patient and the specific therapeutic intervention are selected within the constraints of the method. Application of the N-of-1 method in CAM has been advocated but has been mainly limited, in practice, to a small number of studies in herbal and traditional Chinese medicine. THE N-OF-1 TRIAL IN HOMEOPATHY: Individualised homeopathy can be accommodated for investigation within the same methodological constraints; less in-depth homeopathic approaches to prescribing are also amendable to investigation using the N-of-1 method. No such studies have been published. We identify three main targets in its ready applicability to homeopathy: (1) to optimise clinical care in an individual patient; (2) to investigate whether the outcomes of treatment using homeopathy differ from those of placebo; (3) to aggregate data from a series of N-of-1 trials to enable broader conclusions about a group of patients or intervention.
The N-of-1 trial design offers important new investigative possibilities in homeopathy and should be explored as a means to optimise individualised health care or investigate effectiveness of the homeopathic intervention compared with placebo in individual subjects.
随机对照试验（RCT）被视为确立干预措施治疗效果或有效性的“金标准”，但其数据无法推断个体患者的反应。个性化临床护理是包括顺势疗法在内的补充和替代医学（CAM）的一项基本原则，从原则上讲，似乎很适合采用单病例（N-of-1）研究设计进行研究。最近已制定了报告N-of-1试验的指南。
概述N-of-1方法在医学文献中的现状及其在医学（包括CAM）中的应用。考虑N-of-1试验设计是否为顺势疗法的新研究提供了机会。
N-of-1试验设计：N-of-1试验应用传统交叉、盲法、RCT设计的原则。所研究的治疗方法和对照在规定的时间段内以随机顺序重复，并设置合适的洗脱期。N-of-1设计适用于慢性稳定疾病，以及起效快、作用终止快且残留效应适度或可忽略不计的干预措施。结局数据可针对个体受试者进行汇总和解释；也可与来自多个类似N-of-1试验的数据合并，从而得出更具普遍性的结论。
CAM中的N-of-1试验：如果在该方法的限制范围内选择患者和特定的治疗干预措施，N-of-1研究设计可以适应患者护理的典型个体化。N-of-1方法在CAM中的应用已得到提倡，但在实践中主要限于少数关于草药和中医的研究。
顺势疗法中的N-of-1试验：在相同的方法学限制范围内，可以对个性化顺势疗法进行研究；较不深入的顺势疗法开方方法也适合采用N-of-1方法进行研究。尚未发表此类研究。我们确定了其在顺势疗法中易于应用的三个主要目标：（1）优化个体患者的临床护理；（2）研究使用顺势疗法的治疗结果是否与安慰剂不同；（3）汇总一系列N-of-1试验的数据，以便对一组患者或干预措施得出更广泛的结论。
N-of-1试验设计为顺势疗法提供了重要的新研究可能性，应将其作为优化个体化医疗保健或研究顺势疗法干预措施与安慰剂在个体受试者中的有效性的一种手段进行探索。
