{"entity":{"id":"paper-campani-pfmg2025-liver-cancer-j-hepatol-2026","kind":"paper","name":"Molecularly guided therapies for advanced primary liver cancers refractory to systemic treatment: results from the 2025 French genomic medicine initiative","aka":[],"tldr":"Whole-genome, exome and RNA sequencing through France's national genomics programme found a targetable change in about two-thirds of 103 interpretable liver cancers that had stopped responding to treatment; 31 patients got a matched drug and 10 had their disease controlled.","summary":"120 patients with primary liver cancer progressing on systemic therapy were enrolled across eight centres: 80 with hepatocellular carcinoma, 25 with cholangiocarcinoma, 9 with combined hepatocellular-cholangiocarcinoma, 4 with fibrolamellar carcinoma, 1 hepatic sarcoma and 1 epithelioid haemangioendothelioma. Tumour and blood were analysed by whole-genome, whole-exome and RNA sequencing through the PFMG2025 platforms, and results were reviewed by a molecular tumour board and graded on the ESCAT scale.\n\nOf 103 interpretable genomes, 67 patients had at least one actionable alteration (59 per cent of hepatocellular carcinomas, 80 per cent of cholangiocarcinomas, 78 per cent of combined tumours). 31 patients received matched therapy, 69 per cent of them after two or more previous lines. Disease control was achieved in 10 of the 31 (32.3 per cent), only among patients treated for ESCAT I to III alterations (41.7 per cent), with no benefit from ESCAT IV matches; median progression-free survival was 11.8 months with disease control against 2.4 months with progression.","asOf":"2026-11-03","links":[{"label":"J Hepatol 2026","url":"https://doi.org/10.1016/j.jhep.2026.03.019"},{"label":"PubMed","url":"https://pubmed.ncbi.nlm.nih.gov/41865801/"}],"tags":[],"related":[],"cancers":["hcc","cholangiocarcinoma"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":["ngs"],"trials":[],"people":["pierre-laurent-puig"],"bottlenecks":[],"keyPapers":["paper-pfmg2025-genomic-medicine-france-lancet-reg-health-eur-2025"],"journals":[],"dependsOn":[],"notes":[],"journal":"Journal of Hepatology","year":2026,"doi":"10.1016/j.jhep.2026.03.019","pmid":"41865801","authors":"Campani C, Laurent-Puig P, Villeret F, et al.","paperType":"observational","findings":["67 of 103 patients with an interpretable genome had at least one actionable alteration.","31 patients received a matched therapy; disease was controlled in 10 (32.3 per cent).","Disease control occurred only with ESCAT I to III matches (41.7 per cent), never with ESCAT IV."],"whatItMeans":"For liver cancers that have stopped responding, national genome sequencing found something to target in most patients, but only matches backed by stronger evidence (ESCAT I to III) helped. The authors say the combined sequencing found alterations beyond the reach of standard sequencing panels; the study did not test panels side by side.","caveats":["Single-arm, with no control group.","Only 31 of 120 enrolled patients received a matched therapy.","Most matches were ESCAT III, supported by evidence from other cancers rather than liver cancer."],"participants":120},"route":"/key-papers/paper-campani-pfmg2025-liver-cancer-j-hepatol-2026/","neighbours":{"cancer":[{"id":"cholangiocarcinoma","kind":"cancer","name":"Biliary tract cancer (cholangiocarcinoma)","route":"/cancers/cholangiocarcinoma/"},{"id":"hcc","kind":"cancer","name":"Hepatocellular carcinoma","route":"/cancers/hcc/"}],"term":[{"id":"ngs","kind":"term","name":"Next-generation sequencing (NGS)","route":"/terms/ngs/"}],"person":[{"id":"pierre-laurent-puig","kind":"person","name":"Pierre Laurent-Puig","route":"/people/pierre-laurent-puig/"}],"paper":[{"id":"paper-pfmg2025-genomic-medicine-france-lancet-reg-health-eur-2025","kind":"paper","name":"PFMG2025: integrating genomic medicine into the national healthcare system in France","route":"/key-papers/paper-pfmg2025-genomic-medicine-france-lancet-reg-health-eur-2025/"}]}}