Guillemin Francis
School of Public Health, Nancy University, and Clinical Epidemiology Centre Inserm CIE6, Nancy-University Hospital, France.
Nat Clin Pract Rheumatol. 2007 Jul;3(7):407-13. doi: 10.1038/ncprheum0528.
The identification of subgroups of patients from randomized clinical trials that are of specific interest for guiding clinical decisions can be an attractive idea; however, since such trials are designed for the comparison of groups of patients, performing subgroup analyses can result in misinterpretation of the data. Such analyses must, therefore, be performed and evaluated with caution: these should be pre-planned and included in the design of a suitably powered trial. Data obtained should be analyzed using formal statistical tests of interaction on proper subgroups rather than improper subgroups of patients, the results obtained should be delineated carefully, and details of how these analyses were performed, and how the data should be interpreted, should be reported in the trial paper. The caveats associated with this approach, such as the occurrence of false positive or false negative effects, chance differences in observed effects, lack of power to perform the analysis, floor or ceiling effects, issues relating to multiple statistical testing, and over-reporting and under-reporting are discussed in this review. Subgroup analyses can, however, provide valuable, albeit predominantly exploratory, information on which to base clinical decisions if they are performed in accordance with recommendations and guidelines, and do, therefore, have a legitimate place in rheumatology clinical trials.
从随机临床试验中识别出对指导临床决策具有特殊意义的患者亚组，可能是个颇具吸引力的想法；然而，由于此类试验旨在比较患者组，进行亚组分析可能会导致对数据的误解。因此，必须谨慎地进行和评估此类分析：这些分析应预先规划，并纳入具有足够效力的试验设计中。所获得的数据应使用针对适当患者亚组而非不适当亚组的正式交互作用统计检验进行分析，所获得的结果应仔细描述，并且应在试验论文中报告这些分析的执行方式以及数据的解释方式。本综述讨论了与这种方法相关的注意事项，例如出现假阳性或假阴性效应、观察到的效应中的偶然差异、进行分析的效力不足、下限或上限效应、与多重统计检验相关的问题以及报告过多和报告不足等。然而，如果按照建议和指南进行亚组分析，它们可以提供有价值的信息，尽管主要是探索性的，可作为临床决策的依据，因此，在风湿病临床试验中确实有其合理的地位。
