Subgroup analyses of a phase 1/2 study of lenvatinib (E7080), a multitargeted tyrosine kinase inhibitor, in patients (pts) with advanced hepatocellular carcinoma (HCC).

309 Background: Lenvatinib is an oral tyrosine kinase inhibitor of VEGFR1-3, FGFR1-4, PDGFRα, RET, and KIT. A phase 1/2 trial in pts (n = 46) with advanced HCC and Child-Pugh score A treated with lenvatinib 12 mg once daily showed a median overall survival (OS) of 18.7 months, and a median time to progression (TTP) of 12.8 months by investigator review and 7.4 months by independent radiologic review (IRR). Objective response rate was 37.0% as assessed by modified Response Evaluation Criteria in Solid Tumors (mRECIST). Based on these results, a phase 3 study comparing the efficacy and safety of lenvatinib versus sorafenib is ongoing. Here, we report subgroup analyses of the phase 2 part of this study. Methods: Subgroups were retrospectively analyzed by Barcelona clinic liver cancer (BCLC) staging, disease etiology, prior therapy for advanced HCC (including sorafenib), and baseline Alpha-fetoprotein (AFP) levels. Efficacy endpoints including OS and TTP by IRR were assessed for each subgroup. TTP based on mRECIST and OS were assessed from the date of study registration to progressive disease and to death, respectively. Results: Median TTP and OS for each of the relevant subgroups of baseline characteristics are shown in the table. Conclusions: Pts with advanced HCC treated with lenvatinib showed similar TTP regardless of subgroup. Although the number of patients was limited, lenvatinib treatment shows promising efficacy even in patients with hepatitis B virus. Clinical trial information: NCT00946153. [Table: see text]