This exploratory study analysed plasma and tumour samples from patients with advanced HCC who received regorafenib in the RESORCE phase 3 trial to identify biomarkers associated with response to regorafenib. Decreased baseline plasma concentrations of 5 proteins (angiopoietin 1, cystatin B, the latency-associated peptide of transforming growth factor beta 1, oxidized low-density lipoprotein receptor 1, and C-C motif chemokine ligand 3; adjusted P ≤ .05) were significantly associated with increased overall survival after regorafenib treatment. Plasma levels of α-fetoprotein and c-MET were associated with poor outcome independently of regorafenib treatment. Levels of 9 plasma miRNAs (MIR30A, MIR122, MIR125B, MIR200A, MIR374B, MIR15B, MIR107, MIR320, and MIR645) were significantly associated with overall survival time with regorafenib (adjusted P ≤ .05). Next-generation sequencing analyses of tumour tissues identified mutations in CTNNB1 in 3 of 10 progressors and VEGFA amplification in 1 of 7 responders.
(Teufel M et al. – Gastroenterology, May 2019)