Immune Checkpoint Inhibitor Use in Advanced Hepatocellular Carcinoma: A Real-World Analysis of Efficacy and Toxicity.

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.