Progression-free survival as surrogate endpoint of overall survival in esophageal squamous cell carcinoma: a real-world data and literature-based analysis

Background: The surrogacy of progression-free survival (PFS) for overall survival (OS) in esophageal squamous cell carcinoma (ESCC) remains unelucidated. This study aimed to determine the validity of PFS as a surrogate endpoint for OS in ESCC patients treated with definitive radiotherapy or definiti...

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Main Authors: Weiming Han, Lan Wang, Chen Li, Junqiang Chen, Wencheng Zhang, Xin Wang, Qingsong Pang, Yidian Zhao, Xinchen Sun, Kaixian Zhang, Gaofeng Li, Ling Li, Xueying Qiao, Miaoling Liu, Yadi Wang, Lei Deng, Wenqing Wang, Nan Bi, Tao Zhang, Wei Deng, Wenjie Ni, Xiao Chang, Zongmei Zhou, Jun Liang, Qinfu Feng, Lvhua Wang, Dongfu Chen, Jima Lv, Shuchai Zhu, Chun Han, Zefen Xiao
Format: Article
Language:English
Published: SAGE Publishing 2022-10-01
Series:Therapeutic Advances in Medical Oncology
Online Access:https://doi.org/10.1177/17588359221131526
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author Weiming Han
Lan Wang
Chen Li
Junqiang Chen
Wencheng Zhang
Xin Wang
Qingsong Pang
Yidian Zhao
Xinchen Sun
Kaixian Zhang
Gaofeng Li
Ling Li
Xueying Qiao
Miaoling Liu
Yadi Wang
Lei Deng
Wenqing Wang
Nan Bi
Tao Zhang
Wei Deng
Wenjie Ni
Xiao Chang
Zongmei Zhou
Jun Liang
Qinfu Feng
Lvhua Wang
Dongfu Chen
Jima Lv
Shuchai Zhu
Chun Han
Zefen Xiao
author_facet Weiming Han
Lan Wang
Chen Li
Junqiang Chen
Wencheng Zhang
Xin Wang
Qingsong Pang
Yidian Zhao
Xinchen Sun
Kaixian Zhang
Gaofeng Li
Ling Li
Xueying Qiao
Miaoling Liu
Yadi Wang
Lei Deng
Wenqing Wang
Nan Bi
Tao Zhang
Wei Deng
Wenjie Ni
Xiao Chang
Zongmei Zhou
Jun Liang
Qinfu Feng
Lvhua Wang
Dongfu Chen
Jima Lv
Shuchai Zhu
Chun Han
Zefen Xiao
author_sort Weiming Han
collection DOAJ
description Background: The surrogacy of progression-free survival (PFS) for overall survival (OS) in esophageal squamous cell carcinoma (ESCC) remains unelucidated. This study aimed to determine the validity of PFS as a surrogate endpoint for OS in ESCC patients treated with definitive radiotherapy or definitive chemoradiotherapy (dRT/dCRT), as well as characterize the prognostic factors and survival of such patients. Methods: A total of 3662 patients from 10 cancer centers were enrolled. One-, 2-, and 3-year PFS (PFS12, PFS24, and PSF36, respectively) were used as time points for analysis. At each time point, ESCC-specific mortality and OS were characterized using competing risk and conditional survival models, while correlation between PFS and OS was evaluated by linear regression. Results: At PFS12, PFS24, and PFS36, a progressive decrease in 5-year ESCC-specific mortality (35.2%–13.4%) and increase in 5-year OS (46.6%–62.9%) were observed. Regardless, the OS of patients remained markedly lower than those of the age- and sex-matched Chinese general population. TNM stage remained a significant prognostic factor at PFS36. Strong correlation was found between 3-year PFS and 5-year OS, which was further externally validated. Conclusions: Three-year PFS may act as a potential surrogate endpoint for 5-year OS. TNM stage was considered a significant prognostic factor for OS, and may represent the optimal prognostic tool to guide clinical decision-making and post-treatment follow-up.
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spelling doaj.art-1e122a2995cf43e3b80ec255001bd2112022-12-22T02:38:03ZengSAGE PublishingTherapeutic Advances in Medical Oncology1758-83592022-10-011410.1177/17588359221131526Progression-free survival as surrogate endpoint of overall survival in esophageal squamous cell carcinoma: a real-world data and literature-based analysisWeiming HanLan WangChen LiJunqiang ChenWencheng ZhangXin WangQingsong PangYidian ZhaoXinchen SunKaixian ZhangGaofeng LiLing LiXueying QiaoMiaoling LiuYadi WangLei DengWenqing WangNan BiTao ZhangWei DengWenjie NiXiao ChangZongmei ZhouJun LiangQinfu FengLvhua WangDongfu ChenJima LvShuchai ZhuChun HanZefen XiaoBackground: The surrogacy of progression-free survival (PFS) for overall survival (OS) in esophageal squamous cell carcinoma (ESCC) remains unelucidated. This study aimed to determine the validity of PFS as a surrogate endpoint for OS in ESCC patients treated with definitive radiotherapy or definitive chemoradiotherapy (dRT/dCRT), as well as characterize the prognostic factors and survival of such patients. Methods: A total of 3662 patients from 10 cancer centers were enrolled. One-, 2-, and 3-year PFS (PFS12, PFS24, and PSF36, respectively) were used as time points for analysis. At each time point, ESCC-specific mortality and OS were characterized using competing risk and conditional survival models, while correlation between PFS and OS was evaluated by linear regression. Results: At PFS12, PFS24, and PFS36, a progressive decrease in 5-year ESCC-specific mortality (35.2%–13.4%) and increase in 5-year OS (46.6%–62.9%) were observed. Regardless, the OS of patients remained markedly lower than those of the age- and sex-matched Chinese general population. TNM stage remained a significant prognostic factor at PFS36. Strong correlation was found between 3-year PFS and 5-year OS, which was further externally validated. Conclusions: Three-year PFS may act as a potential surrogate endpoint for 5-year OS. TNM stage was considered a significant prognostic factor for OS, and may represent the optimal prognostic tool to guide clinical decision-making and post-treatment follow-up.https://doi.org/10.1177/17588359221131526
spellingShingle Weiming Han
Lan Wang
Chen Li
Junqiang Chen
Wencheng Zhang
Xin Wang
Qingsong Pang
Yidian Zhao
Xinchen Sun
Kaixian Zhang
Gaofeng Li
Ling Li
Xueying Qiao
Miaoling Liu
Yadi Wang
Lei Deng
Wenqing Wang
Nan Bi
Tao Zhang
Wei Deng
Wenjie Ni
Xiao Chang
Zongmei Zhou
Jun Liang
Qinfu Feng
Lvhua Wang
Dongfu Chen
Jima Lv
Shuchai Zhu
Chun Han
Zefen Xiao
Progression-free survival as surrogate endpoint of overall survival in esophageal squamous cell carcinoma: a real-world data and literature-based analysis
Therapeutic Advances in Medical Oncology
title Progression-free survival as surrogate endpoint of overall survival in esophageal squamous cell carcinoma: a real-world data and literature-based analysis
title_full Progression-free survival as surrogate endpoint of overall survival in esophageal squamous cell carcinoma: a real-world data and literature-based analysis
title_fullStr Progression-free survival as surrogate endpoint of overall survival in esophageal squamous cell carcinoma: a real-world data and literature-based analysis
title_full_unstemmed Progression-free survival as surrogate endpoint of overall survival in esophageal squamous cell carcinoma: a real-world data and literature-based analysis
title_short Progression-free survival as surrogate endpoint of overall survival in esophageal squamous cell carcinoma: a real-world data and literature-based analysis
title_sort progression free survival as surrogate endpoint of overall survival in esophageal squamous cell carcinoma a real world data and literature based analysis
url https://doi.org/10.1177/17588359221131526
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