Dendrix Research Ltd, São Paulo, Brazil.
Am J Clin Oncol. 2013 Apr;36(2):143-5. doi: 10.1097/COC.0b013e3182436e6c.
With the growing number of new anticancer therapies, randomized phase II trials have been used more often in oncology. Although the primary objective of such trials is not to formally compare results between arms, this practice seems frequent. We sought to quantify the frequency of use of formal statistical testing or inference through the use of P values and confidence intervals (CIs) in randomized phase II trials.
We searched PubMed for randomized phase II trials assessing systemic cancer therapies published in the years 1995/1996 and 2005/2006. For each study, 2 reviewers independently abstracted data, including reporting of P values and CIs for the primary endpoint.
We retrieved 288 articles, 107 of which were eligible for analysis. The median number of patients per trial was 94, the primary endpoint was response rate in 71 (66.4%) cases, and a control arm was present in 55 (51.4%) trials. Either P values or CIs for the primary endpoint were reported in 85 (79.4%; 95% CI, 70.8%-86.1%) cases. Year of publication, source of funding, and use of a control group were not associated with this practice.
Formal statistical comparisons between arms of randomized phase II trials are frequently undertaken in medical oncology. The extent to which such a practice abrogates phase III testing is unknown.
随着越来越多的新型抗癌疗法的出现，随机二期临床试验在肿瘤学中被更频繁地应用。尽管这些试验的主要目的不是正式比较各治疗组之间的结果，但这种做法似乎很常见。我们试图通过使用 P 值和置信区间（CI）来量化在随机二期临床试验中正式统计检验或推断的使用频率。
我们在 PubMed 中搜索了 1995/1996 年和 2005/2006 年发表的评估全身癌症治疗的随机二期临床试验。对于每一项研究，两名评审员独立提取数据，包括主要终点的 P 值和 CI 的报告。
我们检索到 288 篇文章，其中 107 篇符合分析条件。每个试验的中位患者数为 94 例，主要终点为 71 例（66.4%）的反应率，55 例（51.4%）试验存在对照组。85 例（79.4%；95%CI，70.8%-86.1%）报告了主要终点的 P 值或 CI。发表年份、资金来源和对照组的使用与这种做法无关。
在肿瘤医学中，随机二期临床试验中各治疗组之间的正式统计比较经常进行。这种做法在多大程度上取代了三期试验尚不清楚。
