两阶段单组试验很少得到充分报道
Two-stage single-arm trials are rarely reported adequately
摘要：
目的：两阶段单组试验设计通常用于II期肿瘤学，以推断二元主要结局的处理效应（例如，肿瘤反应）。必须有效地设计、分析和报告这些研究。然而，几乎没有证据表明情况是否如此，特别是在关键的统计考虑方面。因此，我们全面审查这些审判，特别是审查报告的质量。方法：确定并回顾了5年内使用“Simon两阶段设计”的已发表肿瘤学试验。评估文章是否报告了足够的设计详细信息（例如所需的样本量）和分析详细信息（例如置信区间（CI））。重新分析未调整其推断以纳入中期分析的文章，以评价对其报告的点估计值和CI的影响。结果：纳入了425篇报告单个治疗组结果的文章。其中，47.5%提供了确保设计再现性的五个组件。分别只有1.2%和2.1%的患者报告了调整后的点估计值或CI。只有55.3%的试验提供了最后阶段的拒绝界限，表明许多试验没有检验设计的假设。试验的再分析表明，报告的点估计值低估了处理效应，报告的CI太窄。结论：两阶段单组试验的关键设计细节往往未被报道。虽然定期进行推断，但很少以消除中期分析引入的偏倚的方式进行推断。为了最大限度地发挥其价值，未来的研究必须改进分析和报告的方式。
英文摘要：
Purpose: Two-stage single-arm trial designs are commonly used in phase II oncology to infer treatment effects for a binary primary outcome (e.g., tumour response). It is imperative that such studies be designed, analysed, and reported effectively. However, there is little available evidence on whether this is the case, particularly for key statistical considerations. We therefore comprehensively review such trials, examining in particular quality of reporting. Methods: Published oncology trials that utilised "Simon's two-stage design" over a 5 year period were identified and reviewed. Articles were evaluated on whether they reported sufficient design details, such as the required sample size, and analysis details, such as a confidence interval (CI). The articles that did not adjust their inference for the incorporation of an interim analysis were re-analysed to evaluate the impact on their reported point estimate and CI. Results: Four hundred and twenty five articles that reported the results of a single treatment arm were included. Of these, 47.5% provided the five components that ensure design reproducibility. Only 1.2% and 2.1% reported an adjusted point estimate or CI, respectively. Just 55.3% of trials provided the final stage rejection bound, indicating many trials did not test the hypothesis the design is constructed to assess. Re-analysis of the trials suggests that reported point estimates underestimated treatment effects and that reported CIs were too narrow. Conclusion: Key design details of two-stage single-arm trials are often unreported. Whilst inference is regularly performed, it is rarely done so in a way that removes the bias introduced by the interim analysis. In order to maximise their value, future studies must improve the way that they are analysed and reported.
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