Molecularly guided therapies for advanced primary liver cancers refractory to systemic treatment: Results from the 2025 French Genomic Medicine Initiative - PubMed
5 hours ago
- #Liver Cancer
- #Genomic Profiling
- #Precision Medicine
- Study focused on molecularly guided therapies for advanced primary liver cancers (HCC, cHCC-CCA, CCA, FLC, HS, HEHE) refractory to systemic treatment.
- 120 patients enrolled (80 HCC, 25 CCA, 9 cHCC-CCA, 4 FLC, 1 HS, 1 HEHE) with genomic profiling via WGS, WES, and RNA sequencing.
- Actionable alterations found in 67 out of 103 interpretable genomes (HCC: 59%, CCA: 80%, cHCC-CCA: 78%).
- 31 patients received matched therapies, with disease control (DC) achieved in 10 patients (32.3%).
- DC observed exclusively in patients with ESCAT I-III alterations (41.7%), with no benefit for ESCAT IV alterations.
- Median progression-free survival was significantly longer in patients achieving DC (11.8 vs. 2.4 months; p=0.009).
- Findings support early, broad molecular profiling to optimize therapeutic matching and expand precision medicine access.
- Highlights transformative potential of genomics-guided strategies in improving outcomes for hepatobiliary malignancies.