The impact of novel systemic treatment options on the outcome of patients with hepatocellular carcinoma

Background The approval of effective novel therapeutic options, including targeted therapies (lenvatinib, cabozantinib, regorafenib, ramucirumab) as well as immunotherapy has increased our therapeutic armamentarium for hepatocellular carcinoma (HCC). The aim of this study was to compare two HCC cohorts receiving systemic treatment: a recent cohort of patients diagnosed and treated in the era of novel therapeutic options and a historic cohort of patients treated with sorafenib only.

Methods Comparison of two cohorts of patients with Child-Pugh stage (CPS) A and B cirrhosis and HCC who received systemic therapy at the Medical University of Vienna. Patients with CPS C were excluded as prognosis in these patients is mainly determined by underlying liver disease. Cohort 1 (including patients who received at least one novel systemic therapy) were treated between 07/2013 - 07/2019 and cohort 2 (received sorafenib only) between 05/2006 - 07/2013.

Results Thirty patients (25 male, 5 female) were included in the recent cohort 1, while 60 patients (46 male, 14 female) were included in the historic control cohort 2. Mean age was 65 ± 9 years, and 79 % were male. The proportion of patients with vascular invasion (70 % vs. 30 %; p < 0.001) and symptomatic tumors (ECOG 1: 53 % vs. 30 %; p = 0.036) was higher in cohort 2, while the proportion of patients with intermediate stage HCC (BCLC B: 33 % vs. 13 %; p = 0.025) was higher in cohort 1. Overall survival (calculated from the time of systemic treatment initiation) was significantly longer in cohort 1: 28 (95 %CI: 26-30) months vs. 10 (95 %CI:7-12) months (p < 0.001).

Conclusions The improved survival observed with the newly approved systemic therapies may have two main reasons. First, patients received systemic treatment earlier in the course of their disease as reflected by a higher proportion of BCLC B patients. Second, the availability of several systemic treatment options allowed for effective treatment sequencing.

Publication History

Article published online:
26 May 2020