Liver Cancer (Hepatocellular)

    Actionable Target Abnormality Prevalence (%) Clinical Experience with Targeted Agent
    PIK3CA Mutation 6 Sirolimus (mTOR inhibitor):
    • Phase 2: RR 8%, SD 32%, PFS 15.3 wk (1)
    Everolimus (mTOR inhibitor):
    • Phase 1/2: RR 4%, PFS 3.8 mo (2)
    Temsirolimus (mTOR inhibitor):
    • Phase 1/2: RR 3%, SD 56%, PFS 2.8 mo, OS 8.9 mo (3)
    TSC2 Mutation 5
    PTEN Mutation 4
    BRAF Mutation 4 Sorafenib (VEGFR/RAF inhibitor):
    • Phase 3 vs. placebo: RR 2%, OS 10.7 vs. 7.9 mo with placebo (4)
    Sorafenib + erlotinib (EGFR inhibitor):
    • Phase 3 vs. 8.5 mo, PFS 3.2 vs. 4.0 mo (5)
    Other topics in Targets by Organ Site