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随机效应元分析中治疗与亚组交互异质性的经验先验AI 解读

Subgroup analyses are central to the assessment of benefits and risks, where recommendations may depend on evidence that treatment effects differ across patient groups. Valid subgroup claims require evidence based on (within-trial) interaction estimates while accounting for the heterogeneity in those interaction effects. In the common case of only a few available studies, inference may benefit from the use of prior information on the expected amount of heterogeneity.…

考虑试验中期估计不确定性的盲态样本量重估AI 解读

For randomized controlled trials to be conclusive, it is important to set the target sample size accurately at the design stage. Comparing two normal populations, the sample size calculation requires specification of the variance other than the treatment effect and misspecification can lead to underpowered studies. Blinded sample size re-estimation is an approach to minimize the risk of inconclusive studies. Existing methods proposed to use the total (one-sample) variance…

元分析中研究内与跨研究的亚组比较AI 解读

Subgroup-specific meta-analysis synthesizes treatment effects for patient subgroups across randomized trials. Methods include joint or separate modeling of subgroup effects and treatment-by-subgroup interactions, but inconsistencies arise when subgroup prevalence differs between studies (e.g., proportion of non-smokers). A key distinction is between study-generated evidence within trials and synthesis-generated evidence obtained by contrasting results across trials. This…

个体参与者数据元分析中处理效应的学生化置换检验AI 解读

Meta-analysis is a well-established tool used to combine data from several independent studies, each of which usually compares the effect of an experimental treatment with a control group. While meta-analyses are often performed using aggregated study summaries, they may also be conducted using individual participant data (IPD). Classical meta-analysis models may be generalized to handle continuous IPD by formulating them within a linear mixed model framework. IPD…

用伪观测比较小样本临床试验的限制平均生存时间AI 解读

The widely used proportional hazard assumption cannot be assessed reliably in small-scale clinical trials and might often in fact be unjustified, e.g. due to delayed treatment effects. An alternative to the hazard ratio as effect measure is the difference in restricted mean survival time (RMST) that does not rely on model assumptions. Although an asymptotic test for two-sample comparisons of the RMST exists, it has been shown to suffer from an inflated type I error rate in…

非比例风险下生存分析统计方法的中立比较AI 解读

While well-established methods for time-to-event data are available when the proportional hazards assumption holds, there is no consensus on the best inferential approach under non-proportional hazards (NPH). However, a wide range of parametric and non-parametric methods for testing and estimation in this scenario have been proposed. To provide recommendations on the statistical analysis of clinical trials where non proportional hazards are expected, we conducted a…

通过贝叶斯优化改进自适应无缝试验设计AI 解读

We propose to use Bayesian optimization (BO) to improve the efficiency of the design selection process in clinical trials. BO is a method to optimize expensive black-box functions, by using a regression as a surrogate to guide the search. In clinical trials, planning test procedures and sample sizes is a crucial task. A common goal is to maximize the test power, given a set of treatments, corresponding effect sizes, and a total number of samples. From a wide range of…