Department of Epidemiology, School of Medicine, University of California Irvine, Irvine, CA, 92697-7550, USA.
Division of Biostatistics, Department of Public Health Sciences, University of California Davis, Davis, CA, USA.
Prev Sci. 2019 May;20(4):598-608. doi: 10.1007/s11121-019-0980-3.
Many community-based translations of evidence-based interventions are designed as one-arm studies due to ethical and other considerations. Evaluating the impacts of such programs is challenging. Here, we examine the effectiveness of the lifestyle intervention implemented by the Special Diabetes Program for Indians Diabetes Prevention (SDPI-DP) demonstration project, a translational lifestyle intervention among American Indian and Alaska Native communities. Data from the landmark Diabetes Prevention Program placebo group was used as a historical control. We compared the use of propensity score (PS) and disease risk score (DRS) matching to adjust for potential confounder imbalance between groups. The unadjusted hazard ratio (HR) for diabetes risk was 0.35 for SDPI-DP lifestyle intervention vs. control. However, when relevant diabetes risk factors were considered, the adjusted HR estimates were attenuated toward 1, ranging from 0.56 (95% CI 0.44-0.71) to 0.69 (95% CI 0.56-0.96). The differences in estimated HRs using the PS and DRS approaches were relatively small but DRS matching resulted in more participants being matched and smaller standard errors of effect estimates. Carefully employed, publicly available randomized clinical trial data can be used as a historical control to evaluate the intervention effectiveness of one-arm community translational initiatives. It is critical to use a proper statistical method to balance the distributions of potential confounders between comparison groups in this kind of evaluations.
许多基于社区的循证干预措施的翻译版本由于伦理和其他考虑因素而被设计为单臂研究。评估此类计划的影响具有挑战性。在这里，我们检查了印度裔美国人糖尿病预防特别计划 (SDPI-DP) 示范项目实施的生活方式干预的有效性，这是美国印第安人和阿拉斯加原住民社区的一项转化生活方式干预。使用具有里程碑意义的糖尿病预防计划安慰剂组的数据作为历史对照。我们比较了倾向评分 (PS) 和疾病风险评分 (DRS) 匹配的使用，以调整组间潜在混杂因素的不平衡。SDPI-DP 生活方式干预与对照组相比，糖尿病风险的未调整风险比 (HR) 为 0.35。然而，当考虑相关的糖尿病风险因素时，调整后的 HR 估计值趋于 1，范围从 0.56(95%CI 0.44-0.71)到 0.69(95%CI 0.56-0.96)。使用 PS 和 DRS 方法估计的 HR 差异相对较小，但 DRS 匹配导致更多参与者匹配，并且效果估计的标准误差更小。精心使用公开的随机临床试验数据可以作为历史对照来评估单臂社区转化计划的干预效果。在这种评估中，使用适当的统计方法来平衡比较组之间潜在混杂因素的分布至关重要。
