Phase 2 trial of first-line lenvatinib plus toripalimab and FOLFOX chemotherapy for hepatocellular carcinoma with extrahepatic metastasis

The phase 2 LTSC trial at Sun Yat-sen University (Guangzhou, China) evaluated the combination of first-line lenvatinib with toripalimab and FOLFOX chemotherapy among patients with hepatocellular carcinoma (HCC) and extrahepatic metastasis. The combination induced objective response in 43% of patients. The median progression-free survival was 9.73 months (95% CI 2.89-16.58) and the median overall survival was 14.63 months (11.77-17.5), while among patients with high-risk features median PFS was 8.3 months (3.02-13.58) and median OS was 13.7 months (9.24-18.16). The most common adverse events were neutropenia and elevated transaminase levels. Exploratory single-nucleus RNA sequencing analysis suggested that patients with higher abundance of tumor-infiltrating immune cells were more likely to benefit from this combination. The authors conclude that among patients with HCC and extrahepatic metastases, the combination of lenvatinib with toripalimab and FOLFOX chemotherapy showed promising antitumor activity with manageable toxicity.