Published online Oct 15, 2023. doi: 10.4251/wjgo.v15.i10.1796
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.
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.