Department of Surgery, University of Minnesota, Minneapolis, MN, USA.
J Am Coll Surg. 2013 Apr;216(4):774-80; discussion 780-1. doi: 10.1016/j.jamcollsurg.2012.12.036. Epub 2013 Feb 13.
Stakeholders derive many benefits from cancer clinical trials, including guidance for future oncologic treatment decisions. However, whether enrollment in cancer trials also improves patient survival independently of trial outcomes remains underinvestigated. We hypothesized that cancer trial enrollment is not associated with patient survival outcomes.
Using the 2002 to 2008 California Cancer Registry, we identified 555,469 patients with stage I to IV solid organ tumors. Baseline characteristics were compared by trial participation status. Logistic regression determined predictors of trial enrollment. Multivariate Cox proportional hazards regression examined the impact of trial participation on overall and cancer-specific mortality with adjustment for covariates.
Only 0.33% of our cohort was enrolled in clinical trials. Trial participants were likely to be younger than 65 (odds ratio [OR] 2.13; 95% CI 1.90 to 2.38), Hispanic rather than non-Hispanic white (OR 0.78; 95% CI 0.67 to 0.90), and have breast cancer (OR 3.14; 95% CI 2.62 to 3.77). Multivariate survival analyses demonstrated that enrollment in cancer trials predicted a lower hazard of death. However, when stratified by disease site, this survival benefit was observed only in lung, colon, and breast cancers. Sensitivity and interaction analyses confirmed these relationships.
In this first population-based study examining trial effect in solid organ cancers, enrollment into cancer trials predicted lower overall and cancer-specific mortality among common cancer sites. Although these findings may demonstrate a survival benefit due to trial enrollment, they likely also reflect the favorable attributes of trial enrollees. Once corroborated, stakeholders must consider broader cancer trial designs representative of the cancer burden treated in the real world.
利益相关者从癌症临床试验中获得了许多益处，包括为未来的肿瘤治疗决策提供指导。然而，参加癌症试验是否能独立于试验结果改善患者的生存仍未得到充分研究。我们假设癌症试验的参与与患者的生存结果无关。
我们使用 2002 年至 2008 年加利福尼亚癌症登记处的数据，确定了 555469 名 I 期至 IV 期实体器官肿瘤患者。通过试验参与状况比较了基线特征。逻辑回归确定了试验参与的预测因素。多变量 Cox 比例风险回归分析了试验参与对总生存率和癌症特异性生存率的影响，同时调整了协变量。
我们队列中只有 0.33%的患者参加了临床试验。试验参与者比 65 岁以下的人更年轻（比值比[OR] 2.13；95%置信区间[CI] 1.90 至 2.38），西班牙裔而非非西班牙裔白人（OR 0.78；95%CI 0.67 至 0.90），并且患有乳腺癌（OR 3.14；95%CI 2.62 至 3.77）。多变量生存分析表明，参加癌症试验预测死亡风险较低。然而，按疾病部位分层时，仅在肺癌、结肠癌和乳腺癌中观察到这种生存获益。敏感性和交互分析证实了这些关系。
在这项首次对实体器官癌进行的临床试验效果的基于人群的研究中，参加癌症试验预测了常见癌症部位的总生存率和癌症特异性生存率降低。尽管这些发现可能表明由于试验参与而获得了生存获益，但它们也可能反映了试验参与者的有利特征。一旦得到证实，利益相关者必须考虑更广泛的癌症试验设计，以代表在现实世界中治疗的癌症负担。
