Therapeutic Advances in Medical Oncology (Oct 2022)

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

  • 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

DOI
https://doi.org/10.1177/17588359221131526
Journal volume & issue
Vol. 14

Abstract

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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.