贝叶斯重新分析 III 期阿杜卡奴单抗（ADU）试验。
A Bayesian Reanalysis of the Phase III Aducanumab (ADU) Trial.
机构信息
GCS-fMRI, Koelliker Hospital and Department of Psychology, University of Turin, Turin, Italy.
FOCUS Lab, Department of Psychology, University of Turin, Turin, Italy.
出版信息
J Alzheimers Dis. 2022;87(3):1009-1012. doi: 10.3233/JAD-220132.
BACKGROUND
In December 2019, in light of additional blinded data, Biogen claimed efficacy of the drug Aducanumab (ADU).
OBJECTIVE
We conducted a reanalysis of the phase III ADU summary statistics, focusing in particular on the Clinical Dementia Rating-Sum of Boxes.
METHODS
We used a Bayesian framework to mitigate the problems of the null-hypothesis significance testing framework. In particular, we used Bayes Factor (BF) to analyze the summary statistics. The BF is the comparison of how well two hypotheses predict the data.
RESULTS
Our results showed that the evidence for ADU efficacy is very low. The results show that the only data with a BF value in favor of the alternative hypothesis (i.e., drug efficacy) is the high-dose condition in the EMERGE trial. However, the obtained BF falls within the range of values considered anecdotal, meaning a low level of evidence.
CONCLUSION
We provide a clearer interpretation of the results of the clinical trials based on the Bayesian framework, as this may be useful for future development and research in the field.
背景
2019 年 12 月，鉴于额外的盲法数据，Biogen 声称药物 Aducanumab（ADU）有效。
目的
我们对 III 期 ADU 汇总统计数据进行了重新分析，特别关注临床痴呆评定量表总和评分。
方法
我们使用贝叶斯框架来减轻零假设显著性检验框架的问题。具体来说，我们使用贝叶斯因子 (BF) 来分析汇总统计数据。BF 是比较两个假设对数据的预测能力。
结果
我们的结果表明，ADU 疗效的证据非常低。结果表明，唯一支持替代假设（即药物疗效）的 BF 值数据是 EMERGE 试验中的高剂量条件。然而，获得的 BF 值落在被认为是轶事证据的范围内，这意味着证据水平较低。
结论
我们基于贝叶斯框架对临床试验结果提供了更清晰的解释，这对于该领域的未来发展和研究可能是有用的。
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