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量化替代终点韧性的函数类元分析框架AI 解读

A surrogate marker is a biomarker or other physical measurement used to replace a primary outcome in clinical trials to evaluate a treatment effect when the primary outcome of interest is costly, invasive, or takes a long time to observe. However, replacing a primary outcome with a surrogate can lead to the "surrogate paradox," in which a treatment appears beneficial based on the surrogate but is actually harmful with respect to the primary outcome. In this paper, we propose…

纵向结局与替代标志物联合评价的因果状态空间框架AI 解读

Surrogate markers offer the potential to reduce the burden of data collection by replacing costly or invasive primary outcomes with more accessible measurements, provided that they can faithfully indicate the effectiveness of a treatment. However, appropriate evaluation of a surrogate is particularly complex in longitudinal studies, where both outcomes and surrogates can evolve dynamically over time and interest lies not only in the treatment effect at one time, but rather…

临床试验替代标志物评价中的缺失数据校正AI 解读

Evaluating treatment effects is critical in clinical trials but sometimes involves lengthy, invasive, or costly follow-up procedures. In these cases, surrogate markers, which provide intermediate measures of the long-term treatment effect, allow clinicians to obtain results faster and more efficiently than would have otherwise been possible. Prior to adoption, it is vital that the utility of surrogate markers (i.e., their ability to capture the treatment effect on the…

替代标志物评价中秩方法的贝叶斯批评AI 解读

Surrogate markers are often employed in clinical trials to replace primary outcomes that may be difficult, expensive, or time-consuming to measure directly. These markers can accelerate the evaluation of new treatments, provided they reliably capture the causal relationship between treatment and true clinical benefit. Parast et al. (2024) recently proposed a rank-based approach for evaluating surrogate markers, characterized by its nonparametric nature and minimal…

利用替代指标信息进行高效检验AI 解读

In modern clinical trials, there is immense pressure to use surrogate markers in place of an expensive or long-term primary outcome to make more timely decisions about treatment effectiveness. However, using a surrogate marker to test for a treatment effect can be difficult and controversial. Existing methods tend to either rely on fully parametric methods where strict assumptions are made about the relationship between the surrogate and the outcome, or assume the surrogate…

以无模型方法评估删失中间结局对总生存期的替代作用AI 解读

Clinical trials or studies oftentimes require long-term and/or costly follow-up of participants to evaluate a novel treatment/drug/vaccine. There has been increasing interest in the past few decades in using short-term surrogate outcomes as a replacement of the primary outcome i.e., in using the surrogate outcome, which can potentially be observed sooner, to make inference about the treatment effect on the long-term primary outcome. Very few of the available statistical…

估计纵向替代结局治疗效应的半参数联合模型：慢性肾病试验应用AI 解读

In clinical trials where long follow-up is required to measure the primary outcome of interest, there is substantial interest in using an accepted surrogate outcome that can be measured earlier in time or with less cost to estimate a treatment effect. For example, in clinical trials of chronic kidney disease (CKD), the effect of a treatment is often demonstrated on a surrogate outcome, the longitudinal trajectory of glomerular filtration rate (GFR). However, estimating the…

利用替代指标对处理效应进行群序贯检验AI 解读

The identification of surrogate markers is motivated by their potential to make decisions sooner about a treatment effect. However, few methods have been developed to actually use a surrogate marker to test for a treatment effect in a future study. Most existing methods consider combining surrogate marker and primary outcome information to test for a treatment effect, rely on fully parametric methods where strict parametric assumptions are made about the relationship between…

删失数据下纵向替代标志物的稳健评估AI 解读

The development of statistical methods to evaluate surrogate markers is an active area of research. In many clinical settings, the surrogate marker is not simply a single measurement but is instead a longitudinal trajectory of measurements over time, e.g., fasting plasma glucose measured every 6 months for 3 years. In general, available methods developed for the single-surrogate setting cannot accommodate a longitudinal surrogate marker. Furthermore, many of the methods have…

优化替代标记的使用以提高检验功效AI 解读

Motivated by increasing pressure for decision makers to shorten the time required to evaluate the efficacy of a treatment such that treatments deemed safe and effective can be made publicly available, there has been substantial recent interest in using an earlier or easier to measure surrogate marker, $S$, in place of the primary outcome, $Y$. To validate the utility of a surrogate marker in these settings, a commonly advocated measure is the proportion of treatment effect…

利用效用异质的替代标记检验处理效应AI 解读

The primary benefit of identifying a valid surrogate marker is the ability to use it in a future trial to test for a treatment effect with shorter follow-up time or less cost. However, previous work has demonstrated potential heterogeneity in the utility of a surrogate marker. When such heterogeneity exists, existing methods that use the surrogate to test for a treatment effect while ignoring this heterogeneity may lead to inaccurate conclusions about the treatment effect,…