{"entity":{"id":"paper-hayashi-site-specific-vs-empirical-chemotherapy-cup-jco-2019","kind":"paper","name":"Randomised phase 2 trial of site-specific treatment based on gene expression profiling versus carboplatin and paclitaxel in cancer of unknown primary","aka":[],"tldr":"Using a gene expression test to guess where a cancer of unknown primary came from, and treating it as that cancer, did not help patients live longer than standard carboplatin and paclitaxel.","summary":"Japanese multicentre randomised phase 2 trial of 130 patients with cancer of unknown primary, assigned to site-specific chemotherapy chosen from a 2,000-gene expression tissue-of-origin assay or to empirical carboplatin plus paclitaxel.\n\nOne-year survival was 44.2 percent with site-specific treatment against 54.9 percent with empirical chemotherapy, and progression-free survival did not differ. The trial, like the earlier GEFCAPI 04 study, argued against routine tissue-of-origin testing to direct chemotherapy.","asOf":"2026-09-18","links":[{"label":"J Clin Oncol 2019","url":"https://doi.org/10.1200/JCO.18.00771"},{"label":"PubMed","url":"https://pubmed.ncbi.nlm.nih.gov/30653423/"}],"tags":[],"related":[],"cancers":["cup-favourable-subsets","cup-unfavourable"],"sections":[],"technologies":[],"targets":[],"drugs":["carboplatin","paclitaxel"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":["jco"],"dependsOn":[],"notes":[],"journal":"Journal of Clinical Oncology","year":2019,"doi":"10.1200/JCO.18.00771","pmid":"30653423","authors":"Hayashi H, Kurata T, Takiguchi Y, et al.","paperType":"rct","findings":["One-year survival 44.2 percent with site-specific therapy versus 54.9 percent with carboplatin and paclitaxel; no improvement."],"whatItMeans":"Predicting the primary site by gene expression and treating accordingly is not better than empirical chemotherapy, so guidelines do not recommend it; the field moved to genomic profiling for actionable targets instead.","caveats":["Randomised phase 2 with survival as the primary endpoint; underpowered for small differences.","Some predicted primaries received chemotherapy of limited efficacy, which may have blunted any benefit."],"changedPractice":true,"participants":130},"route":"/key-papers/paper-hayashi-site-specific-vs-empirical-chemotherapy-cup-jco-2019/","neighbours":{"cancer":[{"id":"cup-favourable-subsets","kind":"cancer","name":"Cancer of unknown primary, favourable subsets","route":"/cancers/cup-favourable-subsets/"},{"id":"cup-unfavourable","kind":"cancer","name":"Cancer of unknown primary, unfavourable (adenocarcinoma and poorly differentiated carcinoma)","route":"/cancers/cup-unfavourable/"}],"drug":[{"id":"carboplatin","kind":"drug","name":"Carboplatin","route":"/drugs/carboplatin/"},{"id":"paclitaxel","kind":"drug","name":"Paclitaxel / nab-paclitaxel","route":"/drugs/paclitaxel/"}],"journal":[{"id":"jco","kind":"journal","name":"Journal of Clinical Oncology","route":"/journals/jco/"}]}}