Treatment Patterns and Survival Outcomes Among Patients With Hepatocellular Carcinoma

Abstract

Importance The treatment landscape for hepatocellular carcinoma (HCC) is rapidly evolving, with the approval of multiple systemic therapies for patients with advanced disease since 2017. However, ideal treatment sequencing approaches remain unknown, and data on clinical practice patterns are limited. Objective To evaluate current treatment patterns, sequencing, and survival outcomes among patients receiving systemic therapy for HCC. Design, Setting, and Participants This retrospective cohort study used data from a nationwide electronic health record (EHR)–derived database of more than 280 oncology practices at more than 800 US cancer clinics to assess adult patients who were treated with systemic therapy for HCC from January 1, 2011, to December 31, 2023. Exposure First-line systemic therapy type. Main Outcomes and Measures Overall survival (OS) was defined as the time from first-line systemic therapy initiation to death, last clinical activity, or the censor date of December 31, 2023. Progression-free survival (PFS) was defined as the time from first-line systemic therapy initiation to the earliest of EHR-documented disease progression, death, last clinical encounter, or the censor date. Results Among 4198 patients (median [IQR] age, 67 [61-74] years; 3353 [79.9%] male), sorafenib was the most common first-line systemic therapy until it was overtaken by atezolizumab-bevacizumab in 2020; durvalumab-tremelimumab also became a common first-line therapy in 2023. Of the full study cohort, 871 (20.7%) received second-line therapy. Median OS was 8.1 months (95% CI, 7.7-8.6 months) but was not significantly associated with first-line systemic therapy type. Median PFS was 3.9 months (95% CI, 3.7-4.0 months); first-line atezolizumab-bevacizumab (hazard ratio, 0.84; 95% CI, 0.71-0.99) was associated with improved PFS compared with sorafenib. Conclusions and relevance In this cohort study of patients treated with systemic therapy for HCC between 2011 and 2023, atezolizumab-bevacizumab and durvalumab-tremelimumab emerged as the most common first-line systemic therapies administered for HCC, but relatively few patients received second-line therapy. Additional research is needed to determine optimal treatment sequencing and increase the likelihood that patients may benefit from second-line systemic therapy options.