Lingjie Shen, Anja van Gestel, Peter Prinsen, Geraldine Vink, Felice N van Erning, Gijs Geleijnse, Maurits Kaptein
{"title":"真实世界证据对治疗选择的价值:结肠癌案例研究。","authors":"Lingjie Shen, Anja van Gestel, Peter Prinsen, Geraldine Vink, Felice N van Erning, Gijs Geleijnse, Maurits Kaptein","doi":"10.1200/CCI.23.00186","DOIUrl":null,"url":null,"abstract":"<p><strong>Purpose: </strong>Real-world evidence (RWE)-derived from analysis of real-world data (RWD)-has the potential to guide personalized treatment decisions. However, because of potential confounding, generating valid RWE is challenging. This study demonstrates how to responsibly generate RWE for treatment decisions. We validate our approach by demonstrating that we can uncover an existing adjuvant chemotherapy (ACT) guideline for stage II and III colon cancer (CC)-which came about using both data from randomized controlled trials and expert consensus-solely using RWD.</p><p><strong>Methods: </strong>Data from the population-based Netherlands Cancer Registry from a total of 27,056 patients with stage II and III CC who underwent curative surgery were analyzed to estimate the overall survival (OS) benefit of ACT. Focusing on 5-year OS, the benefit of ACT was estimated for each patient using G-computation methods by adjusting for patient and tumor characteristics and estimated propensity score. Subsequently, on the basis of these estimates, an ACT decision tree was constructed.</p><p><strong>Results: </strong>The constructed decision tree corresponds to the current Dutch guideline: patients with stage III or stage II with T stage 4 should receive surgery and ACT, whereas patients with stage II with T stage 3 should only receive surgery. Interestingly, we do not find sufficient RWE to conclude against ACT for stage II with T stage 4 and microsatellite instability-high (MSI-H), a recent addition to the current guideline.</p><p><strong>Conclusion: </strong>RWE, if used carefully, can provide a valuable addition to our construction of evidence on clinical decision making and therefore ultimately affect treatment guidelines. Next to validating the ACT decisions advised in the current Dutch guideline, this paper suggests additional attention should be paid to MSI-H in future iterations of the guideline.</p>","PeriodicalId":51626,"journal":{"name":"JCO Clinical Cancer Informatics","volume":null,"pages":null},"PeriodicalIF":3.3000,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Value of Real-World Evidence for Treatment Selection: A Case Study in Colon Cancer.\",\"authors\":\"Lingjie Shen, Anja van Gestel, Peter Prinsen, Geraldine Vink, Felice N van Erning, Gijs Geleijnse, Maurits Kaptein\",\"doi\":\"10.1200/CCI.23.00186\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Purpose: </strong>Real-world evidence (RWE)-derived from analysis of real-world data (RWD)-has the potential to guide personalized treatment decisions. However, because of potential confounding, generating valid RWE is challenging. This study demonstrates how to responsibly generate RWE for treatment decisions. We validate our approach by demonstrating that we can uncover an existing adjuvant chemotherapy (ACT) guideline for stage II and III colon cancer (CC)-which came about using both data from randomized controlled trials and expert consensus-solely using RWD.</p><p><strong>Methods: </strong>Data from the population-based Netherlands Cancer Registry from a total of 27,056 patients with stage II and III CC who underwent curative surgery were analyzed to estimate the overall survival (OS) benefit of ACT. Focusing on 5-year OS, the benefit of ACT was estimated for each patient using G-computation methods by adjusting for patient and tumor characteristics and estimated propensity score. Subsequently, on the basis of these estimates, an ACT decision tree was constructed.</p><p><strong>Results: </strong>The constructed decision tree corresponds to the current Dutch guideline: patients with stage III or stage II with T stage 4 should receive surgery and ACT, whereas patients with stage II with T stage 3 should only receive surgery. Interestingly, we do not find sufficient RWE to conclude against ACT for stage II with T stage 4 and microsatellite instability-high (MSI-H), a recent addition to the current guideline.</p><p><strong>Conclusion: </strong>RWE, if used carefully, can provide a valuable addition to our construction of evidence on clinical decision making and therefore ultimately affect treatment guidelines. Next to validating the ACT decisions advised in the current Dutch guideline, this paper suggests additional attention should be paid to MSI-H in future iterations of the guideline.</p>\",\"PeriodicalId\":51626,\"journal\":{\"name\":\"JCO Clinical Cancer Informatics\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":3.3000,\"publicationDate\":\"2024-05-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"JCO Clinical Cancer Informatics\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1200/CCI.23.00186\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"ONCOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"JCO Clinical Cancer Informatics","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1200/CCI.23.00186","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"ONCOLOGY","Score":null,"Total":0}
引用次数: 0
摘要
目的:真实世界证据(RWE)来自对真实世界数据(RWD)的分析,具有指导个性化治疗决策的潜力。然而,由于潜在的混杂因素,生成有效的真实世界证据具有挑战性。本研究展示了如何负责任地为治疗决策生成 RWE。我们验证了我们的方法,证明我们可以仅使用 RWD 来揭示现有的 II 期和 III 期结肠癌(CC)辅助化疗(ACT)指南,该指南是通过随机对照试验数据和专家共识产生的:方法:我们分析了荷兰癌症登记处(Netherlands Cancer Registry)以人口为基础的数据,该登记处共收集了 27056 名接受根治性手术的 II 期和 III 期结肠癌患者的数据,以估算 ACT 的总生存期(OS)。以5年OS为重点,通过调整患者和肿瘤特征以及估计倾向评分,使用G计算方法估算了每位患者的ACT获益。随后,根据这些估计值构建了ACT决策树:所构建的决策树符合荷兰现行指南:III 期或 II 期 T4 期患者应接受手术和 ACT 治疗,而 II 期 T3 期患者应仅接受手术治疗。有趣的是,我们没有发现足够的 RWE 来得出结论,反对对 T4 期 II 期和微卫星不稳定性高(MSI-H)患者进行 ACT,这也是当前指南中最新增加的一项内容:如果谨慎使用 RWE,可为我们构建临床决策证据提供有价值的补充,从而最终影响治疗指南。除了验证现行荷兰指南中建议的 ACT 决定外,本文还建议在今后的指南迭代中对 MSI-H 给予更多关注。
Value of Real-World Evidence for Treatment Selection: A Case Study in Colon Cancer.
Purpose: Real-world evidence (RWE)-derived from analysis of real-world data (RWD)-has the potential to guide personalized treatment decisions. However, because of potential confounding, generating valid RWE is challenging. This study demonstrates how to responsibly generate RWE for treatment decisions. We validate our approach by demonstrating that we can uncover an existing adjuvant chemotherapy (ACT) guideline for stage II and III colon cancer (CC)-which came about using both data from randomized controlled trials and expert consensus-solely using RWD.
Methods: Data from the population-based Netherlands Cancer Registry from a total of 27,056 patients with stage II and III CC who underwent curative surgery were analyzed to estimate the overall survival (OS) benefit of ACT. Focusing on 5-year OS, the benefit of ACT was estimated for each patient using G-computation methods by adjusting for patient and tumor characteristics and estimated propensity score. Subsequently, on the basis of these estimates, an ACT decision tree was constructed.
Results: The constructed decision tree corresponds to the current Dutch guideline: patients with stage III or stage II with T stage 4 should receive surgery and ACT, whereas patients with stage II with T stage 3 should only receive surgery. Interestingly, we do not find sufficient RWE to conclude against ACT for stage II with T stage 4 and microsatellite instability-high (MSI-H), a recent addition to the current guideline.
Conclusion: RWE, if used carefully, can provide a valuable addition to our construction of evidence on clinical decision making and therefore ultimately affect treatment guidelines. Next to validating the ACT decisions advised in the current Dutch guideline, this paper suggests additional attention should be paid to MSI-H in future iterations of the guideline.