HCC Treatment Shifts Toward Immunotherapy, but Survival Gains Remain Limited: JAMA Study Shows
USA: A large real-world analysis published in JAMA Network Open offers new insights into how systemic treatment strategies for hepatocellular carcinoma (HCC) have evolved over the past decade, while also highlighting persistent gaps in treatment sequencing and survival outcomes.
The study, led by Kelsey S. Lau-Min from the Division of Hematology/Oncology, Department of Medicine, Massachusetts General Hospital, Boston, examined contemporary treatment patterns and outcomes among patients receiving systemic therapy for advanced HCC across the United States.
The therapeutic landscape for HCC has expanded rapidly since 2017, with several targeted agents and immunotherapy-based regimens gaining regulatory approval. Despite this progress, uncertainty persists regarding the optimal sequencing of available therapies and their impact on patient survival in routine clinical practice. To address this, the investigators analyzed data from a nationwide electronic health record–derived database encompassing more than 280 oncology practices and over 800 cancer clinics.
The retrospective cohort included 4,198 adult patients treated with systemic therapy for HCC between January 2011 and December 2023. The median age of the cohort was 67 years, and nearly 80% of patients were male. First-line systemic therapy type served as the primary exposure, while overall survival (OS) and progression-free survival (PFS) were the main outcomes of interest.
The study led to the following findings:
- First-line treatment patterns for hepatocellular carcinoma shifted markedly over time, with sorafenib initially being the most commonly used therapy before being overtaken by atezolizumab–bevacizumab starting in 2020.
- The immunotherapy combination of durvalumab and tremelimumab emerged as a commonly prescribed first-line option by 2023, highlighting increased adoption of immunotherapy-based regimens in HCC care.
- Despite the availability of multiple systemic therapies, progression to subsequent lines of treatment was limited, with only around 20% of patients receiving second-line systemic therapy.
- Overall survival outcomes remained limited, with a median overall survival of 8.1 months and no significant differences observed according to first-line treatment type.
- Median progression-free survival was 3.9 months, with first-line atezolizumab–bevacizumab associated with a modest but statistically significant improvement in progression-free survival compared with sorafenib.
The authors note several limitations, including reliance on EHR-derived data largely from community oncology settings, potential undercapture of care delivered outside participating practices, and missing information on tumor burden and liver-specific disease severity. However, the inclusion of patients across all adult age groups and insurance types, along with access to recent treatment data, strengthens the relevance of the findings.
Overall, the study highlights substantial evolution in first-line HCC treatment over the past decade but emphasizes that many patients fail to access subsequent therapies. The authors conclude that further research—leveraging integrated datasets and prospective trials—is needed to refine treatment sequencing strategies and improve the likelihood that patients with HCC can benefit from multiple lines of systemic therapy.
Reference:
Lau-Min KS, Tramontano AC, Iheanacho F, Abrams TA, Manz CR. Treatment Patterns and Survival Outcomes Among Patients With Hepatocellular Carcinoma. JAMA Netw Open. 2025;8(12):e2551665. doi:10.1001/jamanetworkopen.2025.51665
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