Retrospective Study
Copyright ©The Author(s) 2023. Published by Baishideng Publishing Group Inc. All rights reserved.
World J Gastroenterol. Nov 7, 2023; 29(41): 5641-5656
Published online Nov 7, 2023. doi: 10.3748/wjg.v29.i41.5641
Long-term efficacy and predictors of pembrolizumab-based regimens in patients with advanced esophageal cancer in the real world
Hong-Chi Wang, Xiang Huang, Jing Chen, Ye Li, Yang Cong, Bao-Lin Qu, Sheng-Qiang Feng, Fang Liu
Hong-Chi Wang, Xiang Huang, Jing Chen, Ye Li, Yang Cong, Bao-Lin Qu, Fang Liu, Department of Radiotherapy, The First Medical Center of Chinese PLA General Hospital, Beijing 100853, China
Sheng-Qiang Feng, Health Service, The Guard Bureau of Joint Staff Department of Chinese PLA, Beijing 100017, China
Author contributions: Liu F, Wang HC, and Huang X were involved in the study conception and design; Wang HC, Huang X, and Feng SQ drafted the article and interpreted the data; Chen J, Li Y, and Cong Y collected and analyzed the data; Qu BL supervised the report.
Institutional review board statement: The study was reviewed and approved by the Medical Ethics Committee of the General Hospital of the Chinese People’s Liberation Army, No. S2021-265-01.
Informed consent statement: All study participants or their legal guardian provided informed written consent about personal and medical data collection prior to study enrolment.
Conflict-of-interest statement: All the authors report no relevant conflicts of interest for this article.
Data sharing statement: No additional data are available.
Open-Access: This article is an open-access article that was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution NonCommercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: https://creativecommons.org/Licenses/by-nc/4.0/
Corresponding author: Fang Liu, MD, Chief Physician, Department of Radiotherapy, The First Medical Center of Chinese PLA General Hospital, No. 28 Fuxing Road, Haidian District, Beijing 100853, China. liufangfsq@163.com
Received: July 27, 2023
Peer-review started: July 27, 2023
First decision: September 26, 2023
Revised: October 7, 2023
Accepted: October 23, 2023
Article in press: October 23, 2023
Published online: November 7, 2023
Processing time: 103 Days and 3.3 Hours
Abstract
BACKGROUND

Pembrolizumab combined with chemotherapy has been proven effective as first-line therapy in patients with advanced esophageal cancer. Few trials have assessed the safety and efficacy of this treatment in patients with locally advanced disease.

AIM

To analyze long-term outcomes of pembrolizumab in locally advanced or metastatic esophageal squamous cell carcinoma (ESCC) in the real world.

METHODS

Patients with advanced ESCC admitted to our center from October 2019 to October 2021 were enrolled in this study. Clinical staging of the patients was based on the 8th edition of the American Joint Committee on Cancer TNM staging system. The patients received different treatments based on clinical stage. In brief, patients with locally advanced and resectable ESCC received neoadjuvant therapy combined with surgery. For those who were not candidates for resection, radical concurrent chemoradiotherapy plus pembrolizumab was more preferable. Patients with metastatic ESCC or who were unsuitable for radiotherapy underwent chemotherapy in combination with pembrolizumab. Long-term survival outcomes such as overall survival (OS), progression-free survival, disease-free survival, long-term adverse effects (AEs), immune maintenance therapy and predictors of immune checkpoint inhibitors (ICIs) efficacy were evaluated.

RESULTS

A total of 55 patients with advanced ESCC were enrolled in this retrospective, observational study. The median age was 61 years (range 44-74), with 47.3% (26/55) of the patients in stage IV and 45.5% of the patients had the tumor (25/55) located in the middle third of the esophagus. The median OS in all patients was not reached. The 12-mo OS rate among all patients was 78.8% and the 18-mo OS rate was 72.7%. 9 patients died due to tumor progression and 7 patients died due to treatment-related complications. The therapeutic effect evaluated at the interim evaluation was significantly reflected in the long-term outcome. Patients with complete response or partial response in all patients (P = 0.005) and in the chemoradiotherapy plus pembrolizumab group (P = 0.007) obtained a better prognosis than non-responders. A total of 20 patients (20/55, 36%) received immune maintenance therapy. Baseline peripheral blood biomarkers of the neutrophil-to-lymphocyte ratio, platelet-to-lymphocyte ratio, and neutrophil-to-(leukocyte-neutrophil) ratio did not predict the efficacy of ICIs.

CONCLUSION

Pembrolizumab combined with chemotherapy or radiotherapy resulted in favorable long-term survival in patients with locally advanced or metastatic ESCC, with safe and manageable long-term AEs.

Keywords: Esophageal cancer; Pembrolizumab; Radiotherapy; Long-term survival; Chemotherapy; Real-world

Core Tip: Pembrolizumab combined with chemotherapy has been proven effective as first-line therapy in patients with metastatic esophageal cancer. Few trials have assessed the safety and efficacy of this treatment in patients with locally advanced disease. Our study showed that this treatment in patients with locally advanced or metastatic esophageal squamous cell carcinoma resulted in favorable long-term survival and manageable long-term adverse effects. Randomized phase III trials should be carried out for further verification.