Department of Medical Statistics, University Medical Center Göttingen, Göttingen, Germany.
Stat Methods Med Res. 2020 Jan;29(1):293-308. doi: 10.1177/0962280219833079. Epub 2019 Mar 1.
Meta-analytic methods may be used to combine evidence from different sources of information. Quite commonly, the normal-normal hierarchical model (NNHM) including a random-effect to account for between-study heterogeneity is utilized for such analyses. The same modeling framework may also be used to not only derive a combined estimate, but also to borrow strength for a particular study from another by deriving a . For instance, a small-scale randomized controlled trial could be supported by a non-randomized study, e.g. a clinical registry. This would be particularly attractive in the context of rare diseases. We demonstrate that a meta-analysis still makes sense in this extreme case, effectively based on a synthesis of only two studies, as illustrated using a recent trial and a clinical registry in Creutzfeld-Jakob disease. Derivation of a shrinkage estimate within a Bayesian random-effects meta-analysis may substantially improve a given estimate even based on only a single additional estimate while accounting for potential effect heterogeneity between the studies. Alternatively, inference may equivalently be motivated via a model specification that does not require a common overall mean parameter but considers the treatment effect in one study, and the difference in effects between the studies. The proposed approach is quite generally applicable to combine different types of evidence originating, e.g. from meta-analyses or individual studies. An application of this more general setup is provided in immunosuppression following liver transplantation in children.
元分析方法可用于合并来自不同信息源的证据。通常,采用包括随机效应以解释研究间异质性的正态-正态层次模型(NNHM)进行此类分析。同样的建模框架也可用于不仅得出综合估计值,而且还可以通过从另一个研究中得出 来为特定研究借用强度。例如,小型随机对照试验可以得到非随机研究的支持,例如临床登记。在罕见病的背景下,这将特别有吸引力。我们证明,即使在极端情况下,元分析仍然有意义,实际上是基于仅两项研究的综合,如使用最近的一项试验和克雅氏病的临床登记进行说明。即使仅基于单个额外的估计值,贝叶斯随机效应元分析中的收缩估计值的推导也可以大大改善给定的估计值,同时考虑到研究之间潜在的效果异质性。或者,也可以通过不需要共同总体均值参数但考虑一项研究中的治疗效果以及研究之间效果差异的模型规范来等效地进行推理。所提出的方法非常适用于合并不同类型的证据,例如来自荟萃分析或个体研究。在儿童肝移植后的免疫抑制中提供了这种更通用设置的应用。