Esserman L J, Alvarado M D, Howe R J, Mohan A J, Harrison B, Park C, O'Donoghue C, Ozanne E M
University of California, 1600 Divisadero, 2nd Floor, Box1710, San Francisco, CA, 94115, USA,
Breast Cancer Res Treat. 2014 Apr;144(2):371-8. doi: 10.1007/s10549-014-2881-2. Epub 2014 Mar 2.
The results from randomized clinical trials are often adopted slowly. This practice potentially prevents many people from benefiting from more effective care. Provide a framework for analyzing clinical trial results to determine whether and when early adoption of novel interventions is appropriate. The framework includes the evaluation of three components: confidence in trial results, impact of early, and late adoption if trial results are reversed or sustained. The adverse impact of early adoption, and the opportunity cost of late adoption are determined using Markov modeling to simulate the impact of early and late adoption in terms of quality of life years and resources gained or lost. We applied the framework to the TARGIT-A randomized clinical trial comparing intraoperative radiation (IORT) to standard external beam radiation (EBRT) and considered these results in the context of trials comparing endocrine therapy with and without radiation therapy in postmenopausal women. Confidence in the TARGIT-A trial 4 year results is high because the peak hazard for local recurrence in the trial is between 2 and 3 years. This is consistent with most trials, and no second peak has been observed in similar patient populations, suggesting that the TARGIT-A trial results are stable. The interventions offer approximately equivalent life expectancy. If IORT local recurrences rate were as high as 10 % at 10 years (which is higher than expected), we would project only 0.002 fewer expected life years (less than 1 day) compared to EBRT if IORT is adopted early. However, there is a $1.7 billion opportunity cost of waiting an additional 5 years to adopt IORT in low risk, hormone-receptor-positive, postmenopausal women. EBRT costs an additional $1467 in indirect costs per patient. Applying an evaluative framework for the adoption of clinical trial results to the TARGIT-A IORT therapy trial results in the assessment that the trial results are stable, early adoption would lead to minimal adverse impact, and substantially less resource use. Both IORT and no radiation are reasonable strategies to adopt.
随机临床试验的结果往往采用得很慢。这种做法可能会使许多人无法从更有效的治疗中受益。提供一个分析临床试验结果的框架,以确定早期采用新干预措施是否合适以及何时合适。该框架包括对三个组成部分的评估:对试验结果的信心、早期采用的影响以及如果试验结果被推翻或维持的晚期采用的影响。早期采用的不利影响以及晚期采用的机会成本是通过马尔可夫模型来确定的,该模型模拟早期和晚期采用在生活质量年数以及获得或损失的资源方面的影响。我们将该框架应用于TARGIT - A随机临床试验,该试验将术中放疗(IORT)与标准外照射放疗(EBRT)进行比较,并在比较绝经后妇女接受和不接受放疗的内分泌治疗的试验背景下考虑这些结果。对TARGIT - A试验4年结果的信心很高,因为该试验中局部复发的风险峰值在2至3年之间。这与大多数试验一致,并且在类似患者群体中未观察到第二个峰值,表明TARGIT - A试验结果是稳定的。这些干预措施提供的预期寿命大致相当。如果IORT在10年时的局部复发率高达10%(高于预期),那么如果早期采用IORT,与EBRT相比,我们预计预期寿命仅减少0.002年(不到1天)。然而,对于低风险、激素受体阳性的绝经后妇女,等待额外5年采用IORT存在17亿美元的机会成本。EBRT每位患者的间接成本额外增加1467美元。将临床试验结果采用的评估框架应用于TARGIT - A IORT治疗试验结果后评估得出,试验结果是稳定的,早期采用将导致最小的不利影响,并且资源使用大幅减少。IORT和不放疗都是合理的采用策略。