Samson Pamela, Keogan Kathleen, Crabtree Traves, Colditz Graham, Broderick Stephen, Puri Varun, Meyers Bryan
Washington University in St. Louis, Division of Cardiothoracic Surgery, United States.
Washington University in St. Louis, Division of Biostatistics, United States.
Lung Cancer. 2017 Jan;103:6-10. doi: 10.1016/j.lungcan.2016.11.005. Epub 2016 Nov 9.
To identify the variability of short- and long-term survival outcomes among closed Phase III randomized controlled trials with small sample sizes comparing SBRT (stereotactic body radiation therapy) and surgical resection in operable clinical Stage I non-small cell lung cancer (NSCLC) patients.
Clinical Stage I NSCLC patients who underwent surgery at our institution meeting the inclusion/exclusion criteria for STARS (Randomized Study to Compare CyberKnife to Surgical Resection in Stage I Non-small Cell Lung Cancer), ROSEL (Trial of Either Surgery or Stereotactic Radiotherapy for Early Stage (IA) Lung Cancer), or both were identified. Bootstrapping analysis provided 10,000 iterations to depict 30-day mortality and three-year overall survival (OS) in cohorts of 16 patients (to simulate the STARS surgical arm), 27 patients (to simulate the pooled surgical arms of STARS and ROSEL), and 515 (to simulate the goal accrual for the surgical arm of STARS).
From 2000 to 2012, 749/873 (86%) of clinical Stage I NSCLC patients who underwent resection were eligible for STARS only, ROSEL only, or both studies. When patients eligible for STARS only were repeatedly sampled with a cohort size of 16, the 3-year OS rates ranged from 27 to 100%, and 30-day mortality varied from 0 to 25%. When patients eligible for ROSEL or for both STARS and ROSEL underwent bootstrapping with n=27, the 3-year OS ranged from 46 to 100%, while 30-day mortality varied from 0 to 15%. Finally, when patients eligible for STARS were repeatedly sampled in groups of 515, 3-year OS narrowed to 70-85%, with 30-day mortality varying from 0 to 4%.
Short- and long-term survival outcomes from trials with small sample sizes are extremely variable and unreliable for extrapolation.
在可手术的临床Ⅰ期非小细胞肺癌（NSCLC）患者中，比较立体定向体部放射治疗（SBRT）与手术切除的小样本Ⅲ期随机对照试验中，确定短期和长期生存结果的变异性。
确定在我们机构接受手术且符合STARS（Ⅰ期非小细胞肺癌中射波刀与手术切除比较的随机研究）、ROSEL（早期（IA期）肺癌手术或立体定向放射治疗试验）或两者纳入/排除标准的临床Ⅰ期NSCLC患者。自抽样分析进行了10000次迭代，以描述16例患者队列（模拟STARS手术组）、27例患者队列（模拟STARS和ROSEL的合并手术组）和515例患者队列（模拟STARS手术组的目标入组人数）的30天死亡率和三年总生存率（OS）。
2000年至2012年，873例接受切除的临床Ⅰ期NSCLC患者中有749例（86%）仅符合STARS、仅符合ROSEL或两者的研究标准。当仅符合STARS标准的患者以16例的队列大小重复抽样时，三年总生存率在27%至100%之间，30天死亡率在0至25%之间。当符合ROSEL或同时符合STARS和ROSEL标准的患者以n = 27进行自抽样时，三年总生存率在46%至100%之间，而30天死亡率在0至15%之间。最后，当符合STARS标准的患者以515例一组重复抽样时，三年总生存率缩小至70 - 85%，30天死亡率在0至4%之间。
小样本试验的短期和长期生存结果极具变异性，外推时不可靠。
