Efficacy and survival of nivolumab treatment for recurrent/unresectable esophageal squamous-cell carcinoma: real-world clinical data from a large multi-institutional cohort

Tomoki Makino; Shigeto Nakai; Kota Momose; Kotaro Yamashita; Koji Tanaka; Hiroshi Miyata; Sachiko Yamamoto; Masaaki Motoori; Yutaka Kimura; Yuki Ushimaru; Motohiro Hirao; Jin Matsuyama; Yusuke Akamaru; Yukinori Kurokawa; Hidetoshi Eguchi; Yuichiro Doki

doi:10.1007/s10388-024-01056-w

Efficacy and survival of nivolumab treatment for recurrent/unresectable esophageal squamous-cell carcinoma: real-world clinical data from a large multi-institutional cohort

Esophagus. 2024 May 8. doi: 10.1007/s10388-024-01056-w. Online ahead of print.

Authors

Affiliations

¹ Department of Gastroenterological Surgery, Graduate School of Medicine, Osaka University, 2-2-E2, Yamada-Oka, Suita, Osaka, 565-0871, Japan. tmakino@gesurg.med.osaka-u.ac.jp.
² Department of Gastroenterological Surgery, Graduate School of Medicine, Osaka University, 2-2-E2, Yamada-Oka, Suita, Osaka, 565-0871, Japan.
³ Department of Gastroenterological Surgery, Osaka International Cancer Institute, Osaka, Japan.
⁴ Department of Gastrointestinal Oncology, Osaka International Cancer Institute, Osaka, Osaka, Japan.
⁵ Department of Surgery, Osaka General Medical Center, Osaka, Japan.
⁶ Department of Gastroenterological Surgery, Kindai University Nara Hospital, Nara, Japan.
⁷ Department of Surgery, Sakai City Medical Center, Osaka, Japan.
⁸ Department of Surgery, National Hospital Organization Osaka National Hospital, Osaka, Japan.
⁹ Department of Gastroenterological Surgery, Higashiosaka City Medical Center, Osaka, Japan.
¹⁰ Department of Surgery, Osaka Rosai Hospital, Osaka, Japan.

PMID: 38717686
DOI: 10.1007/s10388-024-01056-w

Abstract

Background: Real-world clinical outcomes of and prognostic factors for nivolumab treatment for esophageal squamous-cell carcinoma (ESCC) remain unclear. This study aimed to evaluate real-world outcomes of nivolumab monotherapy in association with relevant clinical parameters in recurrent/unresectable advanced ESCC patients.

Methods: This population-based multicenter cohort study included a total of 282 patients from 15 institutions with recurrent/unresectable advanced ESCC who received nivolumab as a second-line or later therapy between 2014 and 2022. Data, including the best overall response, progression-free survival (PFS), and overall survival (OS), were retrospectively collected from these patients.

Results: Objective response and disease control rates were 17.0% and 47.9%, respectively. The clinical response to nivolumab treatment significantly correlated with development of overall immune-related adverse events (P < .0001), including rash (P < .0001), hypothyroidism (P = .03), and interstitial pneumonia (P = .004). Organ-specific best response rates were 20.6% in lymph nodes, 17.4% in lungs, 15.4% in pleural dissemination, and 13.6% in primary lesions. In terms of patient survival, the median OS and PFS was 10.9 and 2.4 months, respectively. Univariate analysis of OS revealed that performance status (PS; P < .0001), number of metastatic organs (P = .019), C-reactive protein-to-albumin ratio (CAR; P < .0001), neutrophil-lymphocyte ratio (P = .001), and PMI (P = .024) were significant. Multivariate analysis further identified CAR [hazard ratio (HR) = 1.61, 95% confidence interval (CI) 1.15-2.25, P = .0053)] in addition to PS (HR = 1.65, 95% CI 1.23-2.22, P = .0008) as independent prognostic parameters.

Conclusions: CAR and PS before nivolumab treatment are useful in predicting long-term survival in recurrent/unresectable advanced ESCC patients with second-line or later nivolumab treatment.

Trial registration: UMIN000040462.

Keywords: Esophageal cancer; Nivolumab; Response rate; Survival.