Can synthetic data be a proxy for real clinical trial data? A validation study.
Can synthetic data be a proxy for real clinical trial data? A validation study.
复制标题
合成数据能否作为真实的临床试验数据的替代？一项验证研究。
DOI：
10.1136/bmjopen-2020-043497
复制
发表时间：
2021-04-16
期刊：
影响因子：
2.9
通讯作者：
GOING-FWD Collaborators
中科院分区：
文献类型：
--
作者：
Azizi Z;Zheng C;Mosquera L;Pilote L;El Emam K;GOING-FWD Collaborators
越来越多的要求使研究数据，特别是临床试验数据，更广泛地用于二次分析。然而，由于复杂的隐私要求，数据可用性仍然是一个挑战。这一挑战可以通过使用合成数据来解决。使用机器学习方法生成的合成数据复制已发表的III期结肠癌试验二次分析。对照组中有1543例患者被纳入我们的分析。将发表在真实的数据集上的一项研究的分析复制到合成数据上，以研究肠梗阻和无事件生存率之间的关系。信息论度量被用来比较真实的和合成数据之间的单变量分布。使用CI重叠百分比评估双变量关系大小的相似性，对于来自两个数据集的多变量考克斯模型也是如此。真实的数据集与合成数据集的分析结果相似。单变量分布在信息理论度量的差异的1%以内。所有双变量关系在tau统计量上的CI重叠均超过50%。已发表研究的主要结论，即无肠梗阻对生存期有很大影响，被定向复制，总生存期的真实的和合成数据之间的HR CI重叠为61%（真实的数据：HR 1.56，95% CI 1.11 - 2.2;合成数据：HR 2.03，95% CI 1.44 - 2.87）和86%的无病生存期（真实的数据：HR 1.51，95% CI 1.18 - 1.95;合成数据：HR 1.63，95% CI 1.26 - 2.1）。分析结果与合成数据和真实的数据的结论之间的高度一致性表明，合成数据可用作真实的临床试验数据集的合理替代。 NCT 00079274。
There are increasing requirements to make research data, especially clinical trial data, more broadly available for secondary analyses. However, data availability remains a challenge due to complex privacy requirements. This challenge can potentially be addressed using synthetic data. Replication of a published stage III colon cancer trial secondary analysis using synthetic data generated by a machine learning method. There were 1543 patients in the control arm that were included in our analysis. Analyses from a study published on the real dataset were replicated on synthetic data to investigate the relationship between bowel obstruction and event-free survival. Information theoretic metrics were used to compare the univariate distributions between real and synthetic data. Percentage CI overlap was used to assess the similarity in the size of the bivariate relationships, and similarly for the multivariate Cox models derived from the two datasets. Analysis results were similar between the real and synthetic datasets. The univariate distributions were within 1% of difference on an information theoretic metric. All of the bivariate relationships had CI overlap on the tau statistic above 50%. The main conclusion from the published study, that lack of bowel obstruction has a strong impact on survival, was replicated directionally and the HR CI overlap between the real and synthetic data was 61% for overall survival (real data: HR 1.56, 95% CI 1.11 to 2.2; synthetic data: HR 2.03, 95% CI 1.44 to 2.87) and 86% for disease-free survival (real data: HR 1.51, 95% CI 1.18 to 1.95; synthetic data: HR 1.63, 95% CI 1.26 to 2.1). The high concordance between the analytical results and conclusions from synthetic and real data suggests that synthetic data can be used as a reasonable proxy for real clinical trial datasets. NCT00079274.
登录
查看更多内容
DOI：
10.1198/106186006x133933
发表时间：
2006-09-01
影响因子：
2.4
作者：
Hothorn, Torsten;Hornik, Kurt;Zeileis, Achim
通讯作者：
Zeileis, Achim
影响因子：
3.2
作者：
Benaim, Anat Reiner;Almog, Ronit;Beyar, Rafael
通讯作者：
Beyar, Rafael
影响因子：
4.6
作者：
de Montjoye, Yves-Alexandre;Hidalgo, Cesar A.;Verleysen, Michel;Blondel, Vincent D.
通讯作者：
Blondel, Vincent D.
影响因子：
7.4
作者：
El Emam K;Mosquera L;Bass J
通讯作者：
Bass J
影响因子：
3.8
作者：
Dandaleh, Fadi S.;Sherman, Scott K.;Turaga, Kiran K.
通讯作者：
Turaga, Kiran K.