Real-world outcomes of systemic therapy in patients with hepatocellular carcinoma and decompensated Child-Pugh C cirrhosis.

Abstract

490 Background: Patients with hepatocellular carcinoma (HCC) and decompensated cirrhosis (Child-Pugh C) have a poor prognosis, due to underlying liver dysfunction. There is lack of data regarding systemic therapy in patients with HCC and CPC cirrhosis.. Therapeutic decisions in this setting are primarily guided by risk–benefit considerations, as systemic therapy may precipitate further hepatic decompensation. Consequently, most patients receive supportive care. Real-world data regarding systemic treatment outcomes in this population remain scarce. Methods: We utilized de-identified data from TriNetX, a global federated health research network, to identify patients with HCC and CPC cirrhosis using inclusion criteria requiring: bilirubin >2, albumin <3.5, INR >1.7, ascites, and encephalopathy two months prior to diagnosis. We analysed those who initiated systemic therapy with tyrosine kinase inhibitor (TKI) or an immune checkpoint inhibitor (ICI). Overall survival (OS) was assessed using Kaplan–Meier analysis, with group comparisons performed using log-rank tests. Propensity score matching was applied to adjust for age, sex, and comorbidities. Statistical significance was defined as two-sided P ≤ 0.05. Results: A total of 532 patients were included in the analysis. The median age was 63.9 years (range 55–73); 75.40% were male, 57.70% were Non-Hispanic White, 16.16% Black, 9.02% Asian, and 18.04% Hispanic. The Overall Survival (OS) rates for the entire cohort were 36.6% at 1 year, 25.3% at 2 years, 22.3% at 3 years, and 17.5% at 5 years. Among patients who initiated treatment within 3 months of diagnosis, 320 received frontline TKI therapy and 143 received ICI. Among the therapies used, sorafenib was most frequently used (42%), followed by lenvatinib, cabozantinib, regorafenib, atezolizumab plus bevacizumab, pembrolizumab, and durvalumab. After propensity score matching, 136 patients were included in each cohort. No significant difference in OS was observed between the TKI and ICI groups (Table 1). Conclusions: In this real-world cohort of HCC patients with CPC cirrhosis, OS was 36.6% at 1 year, 25.3% at 2 years, 22.3% at 3 years, and 17.5% at 5 years. Frontline TKI therapy showed numerically higher survival than ICI-based regimens, though the difference did not reach statistical significance. These findings highlight the need for prospective studies to better define treatment strategies in this high-risk population. Survival by treatment group. Time TKI OS% ICI OS% HR (95% CI) P Value 1 year 40.2 28 0.74 (0.54–1.03) 0.79 2 years 30.2 24.2 0.78 (0.57–1.06) 0.67 3 years 26.4 24.2 0.79 (0.58–1.08) 0.49