Do Cancer Clinical Trial Populations Truly Represent Cancer Patients? A Comparison of Open Clinical Trials to the Cancer Genome Atlas
Do Cancer Clinical Trial Populations Truly Represent Cancer Patients? A Comparison of Open Clinical Trials to the Cancer Genome Atlas
复制标题
癌症临床试验人群是否真正代表癌症患者？
DOI：
10.1142/9789814749411_0029
复制
发表时间：
2016
影响因子：
--
通讯作者：
A. Butte
中科院分区：
文献类型：
--
作者：
N. Geifman;A. Butte
开放的临床试验数据为科学界提供了许多机会，以独立验证已发表的结果，评估新的假设和进行荟萃分析。这些数据为精准医学的科学进步提供了跳板，但问题是，癌症患者的临床试验数据总体上有多大代表性。在这里，我们提出了几个癌症临床试验数据的综合分析，并将这些数据与癌症基因组图谱（TCGA）的患者水平数据进行比较。癌症类型特异性生存率的比较显示，这些在试验受试者中总体较低。至少在某种程度上，这种影响可以通过试验受试者癌症的晚期阶段来解释。这项分析还表明，对于IV期癌症，结直肠癌患者的生存机会比乳腺癌患者更好。另一方面，对于所有其他阶段，乳腺癌患者的生存率高于结直肠癌患者。两个数据集之间不同疾病阶段的生存率比较显示，来自试验数据集的IV期癌症受试者的生存率低于来自TCGA的匹配IV期受试者。对这一观察结果的一个可能的解释是，IV期试验受试者的生存率较低，因为他们的癌症不太可能对治疗有反应。总之，我们在这里提出了一个新的临床试验数据集，它允许整合来自许多癌症临床试验的患者水平数据。我们的综合分析表明，癌症相关的临床试验并不能代表一般的癌症患者群体，主要是因为它们关注的是疾病的晚期阶段。临床试验数据的这些和其他局限性也许应该在医学研究和精准医学领域加以考虑。
Open clinical trial data offer many opportunities for the scientific community to independently verify published results, evaluate new hypotheses and conduct meta-analyses. These data provide a springboard for scientific advances in precision medicine but the question arises as to how representative clinical trials data are of cancer patients overall. Here we present the integrative analysis of data from several cancer clinical trials and compare these to patient-level data from The Cancer Genome Atlas (TCGA). Comparison of cancer type-specific survival rates reveals that these are overall lower in trial subjects. This effect, at least to some extent, can be explained by the more advanced stages of cancer of trial subjects. This analysis also reveals that for stage IV cancer, colorectal cancer patients have a better chance of survival than breast cancer patients. On the other hand, for all other stages, breast cancer patients have better survival than colorectal cancer patients. Comparison of survival in different stages of disease between the two datasets reveals that subjects with stage IV cancer from the trials dataset have a lower chance of survival than matching stage IV subjects from TCGA. One likely explanation for this observation is that stage IV trial subjects have lower survival rates since their cancer is less likely to respond to treatment. To conclude, we present here a newly available clinical trials dataset which allowed for the integration of patient-level data from many cancer clinical trials. Our comprehensive analysis reveals that cancer-related clinical trials are not representative of general cancer patient populations, mostly due to their focus on the more advanced stages of the disease. These and other limitations of clinical trials data should, perhaps, be taken into consideration in medical research and in the field of precision medicine.
影响因子：
45.3
作者：
Cooperberg, Matthew R.;Broering, Jeanette M.;Carroll, Peter R.
通讯作者：
Carroll, Peter R.