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Background <p>Neoadjuvant immunochemotherapy is expected to become the standard treatment mode for locally advanced esophageal squamous cell carcinoma (ESCC). This study aims to analyze the clinical outcomes and long-term survival of neoadjuvant immunochemotherapy for locally advanced ESCC, and explore the feasibility of using major pathological response (MPR) as a surrogate endpoint.</p> Methods <p>This real-world retrospective study consecutively included eligible patients with stage II–IVA locally advanced ESCC who received neoadjuvant immunochemotherapy and surgery between 2019 and 2022 at the Department of Thoracic Surgery, the First Affiliated Hospital, Zhejiang University School of Medicine.</p> Results <p>This study collected a total of 166 patients, and ultimately included 126 patients after screening. The objective response rate (ORR) was 69.8% (88/126). The incidence of grade 3–4 adverse events (AEs) was 13.5% (17/126). MPR was observed in 49 (38.9%) patients, and 24 (19.0%) patients achieved a complete pathological response (pCR). The median progression-free survival (PFS) was 31.7&#xa0;months and the 3-year PFS rate was 56.3%. The median overall survival (OS) was not reached and the 3-year OS rate was 70.6%. The median PFS of the non-MPR group was 25.0&#xa0;months, with the MPR group not achieved (hazard ratio [HR], 2.503; 95% CI 1.359–4.610; <i>P</i> = 0.0022). The median OS in the non-MPR group was 31.7&#xa0;months and not reached in the MPR group (HR, 3.607; 95% CI 1.576–8.254; <i>P</i> = 0.0012). MPR is an independent prognostic factor affecting OS (HR, 2.522; 95% CI 1.018–6.401; <i>P</i> = 0.046).</p> Conclusions <p>Neoadjuvant immunochemotherapy is safe and effective for locally advanced ESCC, and can result in certain survival benefits. MPR can serve as a surrogate endpoint for predicting long-term OS.</p>

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Neoadjuvant immunochemotherapy for locally advanced esophageal squamous cell carcinoma in real-world practice: an analysis of the clinical outcomes and long-term survival, and the feasibility of using major pathological response as a surrogate endpoint

  • 期刊论文

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  • 发表日期：

    2025年4月29日

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  • Jiacong Liu,
  • Ziheng Wu,
  • Shihong Zhou,
  • Wang Lv,
  • Yiqing Wang,
  • Pinghui Xia,
  • Linhai Zhu,
  • Jian Hu

摘要

Background

Neoadjuvant immunochemotherapy is expected to become the standard treatment mode for locally advanced esophageal squamous cell carcinoma (ESCC). This study aims to analyze the clinical outcomes and long-term survival of neoadjuvant immunochemotherapy for locally advanced ESCC, and explore the feasibility of using major pathological response (MPR) as a surrogate endpoint.

Methods

This real-world retrospective study consecutively included eligible patients with stage II–IVA locally advanced ESCC who received neoadjuvant immunochemotherapy and surgery between 2019 and 2022 at the Department of Thoracic Surgery, the First Affiliated Hospital, Zhejiang University School of Medicine.

Results

This study collected a total of 166 patients, and ultimately included 126 patients after screening. The objective response rate (ORR) was 69.8% (88/126). The incidence of grade 3–4 adverse events (AEs) was 13.5% (17/126). MPR was observed in 49 (38.9%) patients, and 24 (19.0%) patients achieved a complete pathological response (pCR). The median progression-free survival (PFS) was 31.7 months and the 3-year PFS rate was 56.3%. The median overall survival (OS) was not reached and the 3-year OS rate was 70.6%. The median PFS of the non-MPR group was 25.0 months, with the MPR group not achieved (hazard ratio [HR], 2.503; 95% CI 1.359–4.610; P = 0.0022). The median OS in the non-MPR group was 31.7 months and not reached in the MPR group (HR, 3.607; 95% CI 1.576–8.254; P = 0.0012). MPR is an independent prognostic factor affecting OS (HR, 2.522; 95% CI 1.018–6.401; P = 0.046).

Conclusions

Neoadjuvant immunochemotherapy is safe and effective for locally advanced ESCC, and can result in certain survival benefits. MPR can serve as a surrogate endpoint for predicting long-term OS.