Assessment of disease burden in the phase III CELESTIAL trial of cabozantinib (C) versus placebo (P) in advanced hepatocellular carcinoma (HCC)

Abstract

Background: Extrahepatic spread (EHS) and macrovascular invasion (MVI) are poor prognostic factors in HCC. In the CELESTIAL trial (NCT01908426), C improved overall survival (OS) and progression-free survival (PFS) vs P in patients (pts) with previously treated advanced HCC. Median OS was 10.2 mo with C vs 8.0 mo with P (HR, 0.76; 95% CI, 0.63-0.92; P=0.0049). Median PFS was 5.2 mo with C versus 1.9 mo with P (HR, 0.44; 95% CI, 0.36-0.52; P<0.0001). Here, we analyze OS and PFS based on (i) EHS, (ii)MVI, and (iii) the sum of target lesion diameters (SOD) at baseline. Method(s): A total of 707 pts, stratified by disease etiology, geographic region, and the presence of EHS and/or MVI, were randomized 2:1 to receive C, 60 mg once daily (N=470) or P (N=237). Eligible patients had a pathologic diagnosis of HCC, Child- Pugh score A, and ECOG performance status