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Immune Checkpoint Inhibitor Use in Advanced Hepatocellular Carcinoma: A Real-World Analysis of Efficacy and Toxicity
Fode Tounkara1, Deepak Sherpally2, Khalid Mumtaz3
1Department of Biomedical Informatics, Center for Biostatistics, College of Medicine, The Ohio State University Wexner Medical Center, Columbus, OH 43210, USA.
Background:
While immune checkpoint inhibitors (ICIs) have redefined systemic therapy in hepatocellular carcinoma (HCC), pivotal trials have not yet included patients with advanced liver disease. Real-world data are needed to assess treatment outcomes in advanced liver disease populations.
Methods:
We conducted a retrospective analysis of 53 HCC patients treated with ICIs at a large single center between January 2017 and June 2023. Clinical characteristics, liver function scores [Child-Turcotte-Pugh (CTP) and albumin-bilirubin (ALBI)], treatment history, and survival outcomes were analyzed. Primary endpoints included progression-free survival (PFS), survival from ICI initiation (OS-ICI), and overall survival (OS). Secondary endpoints included incidence and predictors of immune-related adverse events (irAEs).
Results:
Among 53 HCC patients treated with ICIs, the median OS, OS-ICI, and PFS were 18.7 months (m), 7.4 m, and 4.6 m, respectively. On multivariable analysis, a higher ALBI grade and history of alcohol use were independently associated with worse PFS and OS-ICI, while prior locoregional therapy (LRT) significantly improved OS (HR: 0.43; p: 0.012). The ALBI grade outperformed the CTP score in predicting outcomes, highlighting its utility as a more objective liver function marker. Patients receiving atezolizumab-bevacizumab showed improved OS-ICI compared to other regimens (HR: 0.37; p = 0.021). irAEs occurred in 19% of patients, most commonly in those with CTP-A, and were generally manageable.
Conclusions:
These real-world insights into the efficacy and safety of ICI-based therapies across a more diverse HCC population are usually not represented in clinical trials.
