Retrospective Study
Copyright ©The Author(s) 2023. Published by Baishideng Publishing Group Inc. All rights reserved.
World J Gastrointest Oncol. Oct 15, 2023; 15(10): 1796-1806
Published online Oct 15, 2023. doi: 10.4251/wjgo.v15.i10.1796
Real-world clinical effectiveness of sorafenib among patients with unresectable hepatocellular carcinoma at two centers in the United States
Daneng Li, Stephen B Gruber, Shrividya Iyer, Sanjay Gupta, Mohamedtaki Tejani
Daneng Li, Department of Medical Oncology and Therapeutics Research, City of Hope Comprehensive Cancer Center, Duarte, CA 91010, United States
Stephen B Gruber, Center for Precision Medicine, City of Hope Comprehensive Cancer Center, Duarte, CA 91010, United States
Shrividya Iyer, Sanjay Gupta, Department of Oncology, Worldwide Real-World Evidence, Eisai Inc., Nutley, NJ 07110, United States
Mohamedtaki Tejani, Department of Oncology and Hematology, Advent Health Cancer Institute, Orlando, FL 32804, United States
Author contributions: Li D, Gruber SB, and Tejani M contributed to study design, data collection, data interpretation, and manuscript development; Iyer S and Gupta S contributed to study design, data interpretation, and manuscript development; all authors read and approved the final version.
Institutional review board statement: The study was reviewed and approved by the Ethics Committees of Advent Health Orlando and City of Hope.
Informed consent statement: Informed consent was not required for this study as it was a retrospective analysis and data were de-identified prior to analysis. Waivers for informed consent were provided by each site’s Institutional Review Board.
Conflict-of-interest statement: Dr.Li reports personal fees and other from AstraZeneca, other from Brooklyn ImmunoTherapeutics, personal fees from Adagene, personal fees from Coherus, personal fees from Delcath, personal fees from Eisai, personal fees from Exelixis, personal fees from Genentech, personal fees from Ipsen Biopharmaceuticals, personal fees from Merck, personal fees from Servier, personal fees from Sumitomo Pharma, and personal fees from TerSera Therapeutics, outside the submitted work.
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:
Corresponding author: Daneng Li, MD, Associate Professor, Department of Medical Oncology and Therapeutics Research, City of Hope Comprehensive Cancer Center, 1500 E. Duarte Road, Duarte, CA 91010, United States.
Received: May 31, 2023
Peer-review started: May 31, 2023
First decision: June 26, 2023
Revised: July 7, 2023
Accepted: September 1, 2023
Article in press: September 1, 2023
Published online: October 15, 2023

In the United States, sorafenib monotherapy was approved in 2007 for first-line (1L) treatment of patients with unresectable hepatocellular carcinoma (uHCC). As other therapies have been approved in recent years for hepatocellular carcinoma treatment in later lines, it is essential to assess clinical effectiveness of older therapies in actual clinical practice to inform healthcare practitioners’ decisions for better patient care.


To assess patient characteristics/clinical effectiveness of 1L sorafenib in uHCC patients treated in United States academic and community practice settings.


A retrospective observational study was conducted among adult patients (≥ 18 years) in the United States initiating sorafenib monotherapy as 1L systemic therapy for uHCC with Eastern Cooperative Oncology Group status of 0 or 1 between January 2016 and December 2019 at City of Hope and Advent Health. Data were extracted by trained abstractionists from individual patients’ electronic health records and captured in electronic case report forms. Institutional Review Board approvals were obtained prior to study initiation. Data were captured from the time of sorafenib initiation until death or the end of follow-up. All data were de-identified prior to analyses. Clinical outcomes assessed included provider-reported best response, progression-free survival (PFS), and overall survival (OS). PFS and OS were estimated using Kaplan-Meier methods.


Among 134 uHCC patients treated with 1L sorafenib, majority were male (75%), and most were Caucasian (62%) or Asian (19%). Median patient age was 64 years. The most common etiologies of liver disease were hepatitis C (54%), alcohol-related liver disease (16%), and hepatitis B (11%). Most patients were reported to have Barcelona Clinic Liver Cancer stage B (19%) or stage C (70%) disease. Of 134 patients, 110 (82%) were reported to have discontinued treatment or died during follow-up. Primary reasons for sorafenib discontinuation were reported as progression (35%) and toxicity (30%). Best overall response was reported for 124 patients, of which 7.3% reported complete or partial response. Median time to treatment discontinuation was 2.3 mo. Overall, 103 patients (77%) had disease progression or died during sorafenib therapy. Median PFS was estimated to be 2.9 mo. At the end of follow-up, 82 patients (61%) were deceased. Median OS was 8.5 mo.


Newer therapeutic options that have reported higher PFS and OS in real-world clinical practice should be considered to enhance patient outcomes.

Keywords: Retrospective observational study, Sorafenib, Hepatocellular carcinoma, Clinical effectiveness

Core Tip: As treatment options evolve for hepatocellular carcinoma (HCC) it is important to assess and understand the clinical outcomes with older treatment options in diverse real-world clinical practice settings to inform clinical decision making and identify the right patient for the right drug. The current study aimed to assess the patient characteristics and clinical effectiveness of sorafenib as first-line therapy in unresectable HCC patients treated in both academic and community practice settings in the United States.