PDF(Pubmed)
Abstract：
Complex disorders usually affect multiple symptom domains measured by several outcomes. The importance of these outcomes is often different among patients. Current approaches integrate multiple outcomes without considering patient preferences at the individual level. In this paper, we propose a new composite Desirability of Outcome Ranking (DOOR) that integrates individual level ranking of outcome importance and define a winning probability measuring the overall treatment effect. Stratified randomization can be performed based on the participants\' baseline outcome rankings. A Wilcoxon-Mann-Whitney U-statistic is used to average the pairwise DOOR between one treated and one control patient, considering the difference in these patients\' ranking of outcome importance. We use both theoretical and empirical methods to examine the statistical properties of our method and to compare with conventional approaches. We conclude that the proposed composite DOOR properly reflects patient-level preferences and can be used in pivotal trials or comparative effectiveness trials for a patient-centered evaluation of overall treatment benefits.
摘要：
复杂的疾病通常影响多个症状领域，由几个结果衡量。这些结果的重要性在患者之间通常是不同的。当前的方法在不考虑个体水平上的患者偏好的情况下整合多种结果。在本文中,我们提出了一种新的综合期望结果排名（DOOR），它整合了个人对结果重要性的排名，并定义了衡量总体治疗效果的获胜概率。可以根据参与者的基线结果排名进行分层随机化。Wilcoxon-Mann-WhitneyU统计量用于平均一名治疗患者和一名对照患者之间的成对DOOR，考虑到这些患者结局重要性排名的差异。我们使用理论和经验方法来检查我们方法的统计特性，并与常规方法进行比较。我们得出的结论是，拟议的复合DOOR正确反映了患者水平的偏好，可用于关键试验或比较有效性试验，以患者为中心评估整体治疗益处。
