{"title":"为什么在癌症药物试验中,无进展生存期的效应大小系统性地大于总生存期?预后评分是前进的方向。","authors":"Luca Locher , Miquel Serra-Burriel , Dario Trapani , Emanuel Nussli , Kerstin N. Vokinger","doi":"10.1016/j.ejca.2024.115106","DOIUrl":null,"url":null,"abstract":"<div><div>Cancer drugs have accumulated the most approvals over the past years. Overall survival (OS) is considered the gold standard for cancer trial outcomes. However, its use has declined over the past years, in favor of surrogate endpoints, such as progression-free survival (PFS). PFS allows to assess outcomes earlier and, thus, accelerates approval of cancer drugs. Previous studies have demonstrated a poor correlation between PFS and OS. Using simulation models, we examined why PFS usually overestimates survival benefit. We created a publicly accessible <span><span>web application</span><svg><path></path></svg></span> that allows users to run the simulations with different parameter settings. Based on the findings, we propose that assessment of preliminary evidence should be based on a combination of OS result and prognostic scores that reflect the health status of surviving patients.</div></div>","PeriodicalId":11980,"journal":{"name":"European Journal of Cancer","volume":"213 ","pages":"Article 115106"},"PeriodicalIF":7.6000,"publicationDate":"2024-10-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Why effect sizes are systematically larger for progression-free survival than overall survival in cancer drug trials: Prognostic scores as a way forward\",\"authors\":\"Luca Locher , Miquel Serra-Burriel , Dario Trapani , Emanuel Nussli , Kerstin N. Vokinger\",\"doi\":\"10.1016/j.ejca.2024.115106\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><div>Cancer drugs have accumulated the most approvals over the past years. Overall survival (OS) is considered the gold standard for cancer trial outcomes. However, its use has declined over the past years, in favor of surrogate endpoints, such as progression-free survival (PFS). PFS allows to assess outcomes earlier and, thus, accelerates approval of cancer drugs. Previous studies have demonstrated a poor correlation between PFS and OS. Using simulation models, we examined why PFS usually overestimates survival benefit. We created a publicly accessible <span><span>web application</span><svg><path></path></svg></span> that allows users to run the simulations with different parameter settings. Based on the findings, we propose that assessment of preliminary evidence should be based on a combination of OS result and prognostic scores that reflect the health status of surviving patients.</div></div>\",\"PeriodicalId\":11980,\"journal\":{\"name\":\"European Journal of Cancer\",\"volume\":\"213 \",\"pages\":\"Article 115106\"},\"PeriodicalIF\":7.6000,\"publicationDate\":\"2024-10-30\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"European Journal of Cancer\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://www.sciencedirect.com/science/article/pii/S0959804924017131\",\"RegionNum\":1,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"ONCOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"European Journal of Cancer","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S0959804924017131","RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"ONCOLOGY","Score":null,"Total":0}
引用次数: 0
摘要
在过去几年中,癌症药物累计获批数量最多。总生存期(OS)被认为是癌症试验结果的黄金标准。然而,在过去几年中,它的使用率有所下降,取而代之的是无进展生存期(PFS)等替代终点。无进展生存期可以更早地评估结果,从而加速癌症药物的审批。以往的研究表明,无进展生存期与手术生存期之间的相关性较差。我们利用模拟模型研究了 PFS 通常会高估生存获益的原因。我们创建了一个可公开访问的网络应用程序,用户可以使用不同的参数设置运行模拟。根据研究结果,我们建议应结合 OS 结果和反映存活患者健康状况的预后评分来评估初步证据。
Why effect sizes are systematically larger for progression-free survival than overall survival in cancer drug trials: Prognostic scores as a way forward
Cancer drugs have accumulated the most approvals over the past years. Overall survival (OS) is considered the gold standard for cancer trial outcomes. However, its use has declined over the past years, in favor of surrogate endpoints, such as progression-free survival (PFS). PFS allows to assess outcomes earlier and, thus, accelerates approval of cancer drugs. Previous studies have demonstrated a poor correlation between PFS and OS. Using simulation models, we examined why PFS usually overestimates survival benefit. We created a publicly accessible web application that allows users to run the simulations with different parameter settings. Based on the findings, we propose that assessment of preliminary evidence should be based on a combination of OS result and prognostic scores that reflect the health status of surviving patients.
期刊介绍:
The European Journal of Cancer (EJC) serves as a comprehensive platform integrating preclinical, digital, translational, and clinical research across the spectrum of cancer. From epidemiology, carcinogenesis, and biology to groundbreaking innovations in cancer treatment and patient care, the journal covers a wide array of topics. We publish original research, reviews, previews, editorial comments, and correspondence, fostering dialogue and advancement in the fight against cancer. Join us in our mission to drive progress and improve outcomes in cancer research and patient care.