Generalizability of cancer clinical trial results
Generalizability of cancer clinical trial results
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DOI：
10.1002/cncr.21907
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发表时间：
2006-06-01
期刊：
影响因子：
6.2
通讯作者：
Bodurka, DC
中科院分区：
文献类型：
--
作者：
Elting, LS;Cooksley, C;Bodurka, DC
背景临床试验结果的普遍性是值得怀疑的，因为只有不到5%的癌症患者参与。作者研究了临床试验参与者和非参与者的可比性以及差异的潜在影响。回顾性队列研究了1990年1月至1997年12月期间诊断的19，340例癌症患者，其特征是参加了试验。将临床重要因素在试验参与者中的分布与非参与者和在监测、流行病学和最终结果人群中同一时期诊断的患者人群中的分布进行比较，通过使用考克斯比例风险分析来检查这些因素对生存率的影响。与非参与者相比，试验参与者更年轻，表现状态更好，合并症更少。然而，参与者更可能患有局部晚期疾病，阳性淋巴结状态，低分化肿瘤，肝转移和多个转移部位。前者与生存期显著延长相关，而后者与生存期显著缩短相关。试验参与者和非参与者之间缺乏可比性，这使临床试验结果的普遍性受到质疑。尽管临床试验的选择性招募是合理的，但作者鼓励在“所有参与者”中使用基于人群的有效性试验。"
BACKGROUND. The generalizability of clinical trial results is questionable, because fewer than 5% of cancer patients participate. The authors examined the comparability of clinical trial participants and nonparticipants and the potential impact of differences.METHODS. A retrospective cohort of 19,340 cancer patients who were diagnosed between January 1990 and December 1997 was characterized by trial participation. The distributions of prognostically important factors among trial participants were compared with the distributions among nonparticipants and the population of patients diagnosed during the same period in the Surveillance, Epidemiology, and End Results population, The impact of these factors on survival was examined by using a Cox proportional hazards analysis.RESULTS. Trial participants were younger and had better performance status and fewer comorbid conditions compared with nonparticipants. However, participants were more likely to have locally advanced disease, positive lymph node status, poorly differentiated tumors, liver metastases, and multiple metastatic sites. The former factors were associated with significantly longer survival, whereas the later factors were associated with significantly shorter survival.CONCLUSIONS. The lack of comparability between trial participants and nonparticipants called into question the generalizability of clinical trial results. Although selective recruitment for clinical trials is justified, the authors encourage the use of population-based trials of effectiveness in "all comers."