Retrospective Study
Copyright ©The Author(s) 2022. Published by Baishideng Publishing Group Inc. All rights reserved.
World J Hepatol. Aug 27, 2022; 14(8): 1608-1620
Published online Aug 27, 2022. doi: 10.4254/wjh.v14.i8.1608
Real-life multi-center retrospective analysis on nivolumab in difficult-to-treat patients with advanced hepatocellular carcinoma
Nika De Wilde, Luisa Vonghia, Sven Francque, Thomas De Somer, Ali Bagdadi, Eva Staub, Jasper Lambrechts, Ana-Maria Bucalau, Gontran Verset, Christophe Van Steenkiste
Nika De Wilde, Department of Internal Medicine, University Hospital Ghent, Ghent 9000, Belgium
Luisa Vonghia, Sven Francque, Ali Bagdadi, Department of Gastroenterology and Hepatology, University Hospital Antwerp, Antwerp 2650, Belgium
Thomas De Somer, Department of Gastroenterology and Hepatology, Maria Middelares Hospital, Ghent 9000, Belgium
Eva Staub, Department of Psychiatry, Université Libre de Bruxelles, Brussels 1050, Belgium
Jasper Lambrechts, Department of Orthopedics, University of Ghent, Ghent 9000, Belgium
Ana-Maria Bucalau, Gontran Verset, Department of Gastroenterology, Hepatopancreatology and Digestive Oncology, CUB Erasme Hospital, Université Libre de Bruxelles, Brussels 1070, Belgium
Christophe Van Steenkiste, Department of Gastroenterology, AZ Maria Middelares, Ghent 9000, Belgium
Christophe Van Steenkiste, Department of Gastroenterology, University Antwerp, Antwerp 2650, Belgium
Author contributions: De Wilde N merged the dataset, executed the statistical analysis, wrote the manuscript and performed the revision; Verset G, Van Steenkiste C, Vonghia L were the supporting co-promotors in the entire process and provided the data; Francque S and De Somer T did a profound revision of the manuscript; Staub E and Bagdadi A helped with the partial composition of the dataset; Lambrechts J prepared the figures. All authors reviewed the manuscript.
Institutional review board statement: Approval of the ethics committee of the University Hospital of Antwerp, Belgium was obtained (EC number 21/06/080) and is attached. Our study conforms to the recognized standards of the Declaration of Helsinki. All participants were above 16 years of age.
Conflict-of-interest statement: There are no competing interests to declare for all authors.
Data sharing statement: Data available from the first author at nika.dewilde@ugent.be. Consent for data sharing was not obtained from the study participants, but the presented data are anonymized and risk of identification is low.
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: Gontran Verset, MMed, Doctor, Department of Gastroenterology, Hepatopancreatology and Digestive Oncology, CUB Erasme Hospital, Université Libre de Bruxelles, Route de Lennik 808, Brussels 1070, Belgium. gontran.verset@erasme.ulb.ac.be
Received: April 15, 2022
Peer-review started: April 15, 2022
First decision: May 12, 2022
Revised: May 27, 2022
Accepted: July 31, 2022
Article in press: July 31, 2022
Published online: August 27, 2022
Processing time: 132 Days and 20.5 Hours
ARTICLE HIGHLIGHTS
Research background

Hepatocellular carcinoma is one of the leading causes of cancer-related death worldwide. The landscape of the systemic treatment for advanced hepatocellular carcinoma (HCC) is changing quickly. However, frail patients with impaired liver function and poor performance status, who already received multiple treatment lines, are often excluded from randomized controlled trial’s and are ineligible for the standard of care. Nivolumab monotherapy has proven to be effective sometimes for advanced HCC, with a favorable safety profile, and hence nivolumab could be a valuable treatment option for these patients.

Research motivation

Given the recent fast evolutions in the systemic treatment of advanced HCC nowadays, this study is very topical. This article provides interesting information as a starting point for further research, e.g., on patient selection per treatment strategy.

Research objectives

We aimed in this study to evaluate the real-world effectiveness of nivolumab monotherapy in patients with advanced HCC who are not eligible for other treatment.

Research methods

We conducted a retrospective, multicentric study including 29 patients with advanced HCC. All patients had had prior chemotherapy or were intolerant or ineligible for treatments. Data were retrieved from patients’ medical records. The outcome parameters that we evaluated were radiological response according to RECIST criteria, the biological response through the evolution of the alpha-fetoprotein level, and clinical response considering both the Child–Pugh score and the World Health Organization performance status. A safety profile was also reported. Statistical analysis was performed using the SPSS Statistics 27 statistical software package.

Research results

The radiological overall response rate (ORR) to nivolumab monotherapy was 24.1%. The biological ORR was 20.7%. Radiological and biological responses were significantly associated both with each other and with overall survival. Overall survival was 14.5 mo (+/- 2.1), and progression-free survival was 10.9 mo (+/- 2.3). We confirmed the favorable safety profile of nivolumab. We showed notably better results than reported in literature. Hence nivolumab monotherapy should be considered as a valuable treatment option in selected patients otherwise not eligible for systemic treatment. Further research is warranted to confirm these findings.

Research conclusions

Nivolumab monotherapy is a good treatment choice in frail patients with HCC who are ineligible for the standard of care or other validated systemic treatments.

Research perspectives

This article provides interesting information as a starting point for further research. Future research is warranted to confirm the good treatment response to nivolumab in a subgroup of patients with advanced HCC, and furthermore to define this subgroup of patients, to facilitate patient selection per treatment strategy.