Real-world clinical impact of first-line immune checkpoint inhibitor-based therapy in advanced esophageal squamous cell carcinoma

Background: Chemotherapy (chemo) combined with an immune checkpoint inhibitor (ICI) or dual ICI therapy with nivolumab and ipilimumab (nivo + ipi) is the standard first-line treatment for patients with advanced esophageal squamous cell carcinoma (ESCC). In this study, we evaluated real-world clinica...

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Main Authors: M. Tamba, K. Chin, H. Osumi, M. Ogura, S. Fukuoka, S. Udagawa, K. Shimozaki, K. Yoshino, T. Wakatsuki, E. Shinozaki, K. Yamaguchi, A. Ooki
Format: Article
Language:English
Published: Elsevier 2025-06-01
Series:ESMO Gastrointestinal Oncology
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Online Access:http://www.sciencedirect.com/science/article/pii/S2949819825000408
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author M. Tamba
K. Chin
H. Osumi
M. Ogura
S. Fukuoka
S. Udagawa
K. Shimozaki
K. Yoshino
T. Wakatsuki
E. Shinozaki
K. Yamaguchi
A. Ooki
author_facet M. Tamba
K. Chin
H. Osumi
M. Ogura
S. Fukuoka
S. Udagawa
K. Shimozaki
K. Yoshino
T. Wakatsuki
E. Shinozaki
K. Yamaguchi
A. Ooki
author_sort M. Tamba
collection DOAJ
description Background: Chemotherapy (chemo) combined with an immune checkpoint inhibitor (ICI) or dual ICI therapy with nivolumab and ipilimumab (nivo + ipi) is the standard first-line treatment for patients with advanced esophageal squamous cell carcinoma (ESCC). In this study, we evaluated real-world clinical outcomes for first-line ICI-based therapy and explored its prognostic factors. Patients and methods: This single-center retrospective study included patients with ESCC who received ICI-based therapy between January 2021 and July 2024. Results: In total, 92 patients received either ICI + chemo (n = 60) or nivo + ipi (n = 32). The median progression-free survival and overall survival (OS) were 5.0 and 16.0 months for ICI + chemo and 3.5 and 16.9 months for nivo + ipi, respectively. Of the 70 patients with measurable lesions, early tumor shrinkage (ETS) was achieved in 37% for ICI + chemo and 33% for nivo + ipi. ETS was significantly associated with a lower performance status and neutrophil-to-lymphocyte ratios, but not with the treatment regimen or programmed death-ligand 1 (PD-L1) status. Patients who achieved ETS showed significant tumor reduction and a durable response. ETS was an independent predictor of favorable OS (hazard ratio 0.34, 95% confidence interval 0.11-0.88, P = 0.04), whereas neither the treatment regimen nor the PD-L1 status influenced OS. Immune-related adverse events of grade ≥3 occurred in 12% of patients. Conclusions: First-line immunotherapy is effective and safe for the treatment of patients with ESCC. Rapid and deep tumor shrinkage may serve as an early predictive biomarker for longer survival.
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spelling doaj-art-1374028e68314a2bb7eb85b94a1ce74c2025-08-20T03:21:32ZengElsevierESMO Gastrointestinal Oncology2949-81982025-06-01810017110.1016/j.esmogo.2025.100171Real-world clinical impact of first-line immune checkpoint inhibitor-based therapy in advanced esophageal squamous cell carcinomaM. Tamba0K. Chin1H. Osumi2M. Ogura3S. Fukuoka4S. Udagawa5K. Shimozaki6K. Yoshino7T. Wakatsuki8E. Shinozaki9K. Yamaguchi10A. Ooki11Department of Gastroenterological Chemotherapy, Cancer Institute Hospital, Japanese Foundation for Cancer Research, Tokyo, JapanDepartment of Gastroenterological Chemotherapy, Cancer Institute Hospital, Japanese Foundation for Cancer Research, Tokyo, JapanDepartment of Gastroenterological Chemotherapy, Cancer Institute Hospital, Japanese Foundation for Cancer Research, Tokyo, JapanDepartment of Gastroenterological Chemotherapy, Cancer Institute Hospital, Japanese Foundation for Cancer Research, Tokyo, JapanDepartment of Gastroenterological Chemotherapy, Cancer Institute Hospital, Japanese Foundation for Cancer Research, Tokyo, JapanDepartment of Gastroenterological Chemotherapy, Cancer Institute Hospital, Japanese Foundation for Cancer Research, Tokyo, JapanDepartment of Gastroenterological Chemotherapy, Cancer Institute Hospital, Japanese Foundation for Cancer Research, Tokyo, JapanDepartment of Gastroenterological Chemotherapy, Cancer Institute Hospital, Japanese Foundation for Cancer Research, Tokyo, JapanDepartment of Gastroenterological Chemotherapy, Cancer Institute Hospital, Japanese Foundation for Cancer Research, Tokyo, JapanDepartment of Gastroenterological Chemotherapy, Cancer Institute Hospital, Japanese Foundation for Cancer Research, Tokyo, JapanDepartment of Gastroenterological Chemotherapy, Cancer Institute Hospital, Japanese Foundation for Cancer Research, Tokyo, JapanDepartment of Gastroenterological Chemotherapy, Cancer Institute Hospital, Japanese Foundation for Cancer Research, Tokyo, Japan; Correspondence to: Dr Akira Ooki, Department of Gastroenterological Chemotherapy, Cancer Institute Hospital, Japanese Foundation for Cancer Research, Tokyo 135-8550, Japan. Tel: +81-3-3520-0111Background: Chemotherapy (chemo) combined with an immune checkpoint inhibitor (ICI) or dual ICI therapy with nivolumab and ipilimumab (nivo + ipi) is the standard first-line treatment for patients with advanced esophageal squamous cell carcinoma (ESCC). In this study, we evaluated real-world clinical outcomes for first-line ICI-based therapy and explored its prognostic factors. Patients and methods: This single-center retrospective study included patients with ESCC who received ICI-based therapy between January 2021 and July 2024. Results: In total, 92 patients received either ICI + chemo (n = 60) or nivo + ipi (n = 32). The median progression-free survival and overall survival (OS) were 5.0 and 16.0 months for ICI + chemo and 3.5 and 16.9 months for nivo + ipi, respectively. Of the 70 patients with measurable lesions, early tumor shrinkage (ETS) was achieved in 37% for ICI + chemo and 33% for nivo + ipi. ETS was significantly associated with a lower performance status and neutrophil-to-lymphocyte ratios, but not with the treatment regimen or programmed death-ligand 1 (PD-L1) status. Patients who achieved ETS showed significant tumor reduction and a durable response. ETS was an independent predictor of favorable OS (hazard ratio 0.34, 95% confidence interval 0.11-0.88, P = 0.04), whereas neither the treatment regimen nor the PD-L1 status influenced OS. Immune-related adverse events of grade ≥3 occurred in 12% of patients. Conclusions: First-line immunotherapy is effective and safe for the treatment of patients with ESCC. Rapid and deep tumor shrinkage may serve as an early predictive biomarker for longer survival.http://www.sciencedirect.com/science/article/pii/S2949819825000408esophageal canceresophageal squamous cell carcinomachemotherapyimmunotherapyearly tumor shrinkagedepth of response
spellingShingle M. Tamba
K. Chin
H. Osumi
M. Ogura
S. Fukuoka
S. Udagawa
K. Shimozaki
K. Yoshino
T. Wakatsuki
E. Shinozaki
K. Yamaguchi
A. Ooki
Real-world clinical impact of first-line immune checkpoint inhibitor-based therapy in advanced esophageal squamous cell carcinoma
ESMO Gastrointestinal Oncology
esophageal cancer
esophageal squamous cell carcinoma
chemotherapy
immunotherapy
early tumor shrinkage
depth of response
title Real-world clinical impact of first-line immune checkpoint inhibitor-based therapy in advanced esophageal squamous cell carcinoma
title_full Real-world clinical impact of first-line immune checkpoint inhibitor-based therapy in advanced esophageal squamous cell carcinoma
title_fullStr Real-world clinical impact of first-line immune checkpoint inhibitor-based therapy in advanced esophageal squamous cell carcinoma
title_full_unstemmed Real-world clinical impact of first-line immune checkpoint inhibitor-based therapy in advanced esophageal squamous cell carcinoma
title_short Real-world clinical impact of first-line immune checkpoint inhibitor-based therapy in advanced esophageal squamous cell carcinoma
title_sort real world clinical impact of first line immune checkpoint inhibitor based therapy in advanced esophageal squamous cell carcinoma
topic esophageal cancer
esophageal squamous cell carcinoma
chemotherapy
immunotherapy
early tumor shrinkage
depth of response
url http://www.sciencedirect.com/science/article/pii/S2949819825000408
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