桥接治疗比较：在HIV治疗中的示例应用
Bridged treatment comparisons: an illustrative application in HIV treatment
AI总结：
该研究提出一种跨研究治疗比较的融合方法，借助共享试验组和逆概率加权估计，结合两项ACTG试验数据实现HIV三联与单药治疗的风险比较，并给出融合诊断方法以验证假设有效性。
摘要：
治疗、干预或暴露因素的比较是流行病学领域的核心研究方向，但受实际条件或伦理限制，直接比较并非总能实现。本文详细阐述了一种用于跨研究比较治疗方案的融合方法。研究的驱动案例是：利用艾滋病临床试验组（AIDS Clinical Trial Group，ACTG）175试验（单药治疗vs双药治疗）和ACTG 320试验（双药治疗vs三药治疗）的数据，比较HIV感染者接受三联抗逆转录病毒治疗与单药治疗时，发生死亡、艾滋病或CD4细胞计数下降超过50%这一复合结局的风险。我们梳理了一组识别假设，并借助共享试验组（双药治疗组），采用逆概率加权估计量计算风险差。本文提出了一种基于共享组比较的融合诊断方法，可用于识别假设是否被违反。将数据融合估计量和诊断方法应用于ACTG试验的结果显示：对于基线CD4细胞计数在50-300个/立方毫米的个体，与单药治疗相比，三联治疗可降低复合结局风险。桥接治疗比较能够解决单一数据源无法回答的问题，但有效的融合推断需要审慎考量其基础假设。
英文摘要：
Comparisons of treatments, interventions, or exposures are of central interest in epidemiology, but direct comparisons are not always possible due to practical or ethical reasons. Here, we detail a fusion approach to compare treatments across studies. The motivating example entails comparing the risk of the composite outcome of death, AIDS, or greater than a 50% CD4 cell count decline in people with HIV when assigned triple versus mono antiretroviral therapy, using data from the AIDS Clinical Trial Group (ACTG) 175 (mono versus dual therapy) and ACTG 320 (dual versus triple therapy). We review a set of identification assumptions and estimate the risk difference using an inverse probability weighting estimator that leverages the shared trial arms (dual therapy). A fusion diagnostic based on comparing the shared arms is proposed that may indicate violation of the identification assumptions. Application of the data fusion estimator and diagnostic to the ACTG trials indicates triple therapy results in a reduction in risk compared to monotherapy in individuals with baseline CD4 counts between 50 and 300 cells/mm$^3$. Bridged treatment comparisons address questions that none of the constituent data sources could address alone, but valid fusion-based inference requires careful consideration of the underlying assumptions.
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