Real-world evidence of immunotherapy-associated outcomes in patients with hepatocellular carcinoma: Insights from TriNetX database.

Abstract

e16193 Background: Immunotherapy has become a cornerstone in the management of hepatocellular carcinoma (HCC), with immune checkpoint inhibitors (ICIs) and their combinations now established as preferred first-line systemic therapies. While ICIs have improved overall outcomes in HCC, only a subset of patients derive meaningful benefit. This underscores the critical need for understanding the clinical predictors to optimize treatment response in this population. Currently, large scale epidemiological studies are lacking. This investigation aims to explore the utilization of immune checkpoint inhibitors in treatment of HCC and associated treatment response and outcomes in real-world setting. Methods: For this cohort study, we utilized the US Collaborative Network within the TrinetX database, encompassing 111 healthcare organizations. We analyzed all participants aged 18 years and older diagnosed with hepatocellular carcinoma. Key clinical covariates including demographics, tumor characteristics, liver disease etiology, laboratory biomarkers, treatment, and outcomes were analyzed. We examined the lines of treatment and treatment pathways to assess the differences in survival probabilities using the Kaplan-Meier method. Cox proportional hazards regression modeling was utilized to identify clinical predictors and laboratory biomarkers for treatment response. P < 0.05 was considered significant. Results: We identified 206,159 patients diagnosed with HCC. The majority of the patients were male (69%) with a mean age of 63.9 years (standard deviation: 12.2 years). The cohort was 49% White, 9% Black, 17% Asian, and 8% Hispanic. Liver cirrhosis was present in 54%, while 20% had alcoholic liver disease, 33% had viral hepatitis, and 24% developed hepatic failure. Of these, 46.5% were treated with only Sorafenib, 12.1% were treated with Atezolimumab and Bevacizumab combination, 10.5% with Nivolumab, 6.5% with Pembrolizumab, and 4.6% with Durvalumab + Tremelimumb. After balancing for clinical covariates and year of HCC diagnosis, we identified different survival probabilities based on Kaplan-Meier curves (log rank P < 0.05). Further multivariable analyses are underway to account for time on treatment. Conclusions: We have identified a large cohort of patients with HCC, representative of the real-world setting. With the increased utilization of immune checkpoint inhibitors it is crucial to identify clinical characteristics and laboratory biomarkers associated with treatment response and outcomes. This study will allow us to fill this gap in knowledge.