{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,3]],"date-time":"2026-11-03T18:36:00Z","timestamp":1793730960566,"version":"4.6.1"},"reference-count":50,"publisher":"American Society of Clinical Oncology (ASCO)","issue":"9","content-domain":{"domain":["ascopubs.org"],"crossmark-restriction":true},"short-container-title":["JCO Precis Oncol"],"published-print":{"date-parts":[[2025,4]]},"abstract":"<jats:sec>\n            <jats:title>PURPOSE<\/jats:title>\n            <jats:p>\n              This study aimed to investigate the clinicomolecular profiles and the efficacy of human epidermal growth factor receptor 2 (HER2)-targeted therapy in\n              <jats:italic>HER2<\/jats:italic>\n              -amplified biliary tract cancer (BTC).\n            <\/jats:p>\n          <\/jats:sec>\n          <jats:sec>\n            <jats:title>METHODS<\/jats:title>\n            <jats:p>This study was an international collaboration that used combined data from the prospective SCRUM-Japan GOZILA and MONSTAR-SCREEN in Japan and retrospective reviews in the United States; patients with advanced BTC who had received systemic therapy were included. The clinicomolecular profiles were evaluated in an exploratory cohort, whereas the efficacy of HER2-targeted therapy was assessed in a biomarker-selected cohort.<\/jats:p>\n          <\/jats:sec>\n          <jats:sec>\n            <jats:title>RESULTS<\/jats:title>\n            <jats:p>\n              Of the 439 patients in the exploratory cohort, 43 (10%) had\n              <jats:italic>HER2<\/jats:italic>\n              amplification. The frequencies of coalterations were higher in patients with\n              <jats:italic>HER2<\/jats:italic>\n              amplification versus patients without\n              <jats:italic>HER2<\/jats:italic>\n              amplification including\n              <jats:italic>HER2<\/jats:italic>\n              mutations (26%\n              <jats:italic>v<\/jats:italic>\n              5%,\n              <jats:italic>P<\/jats:italic>\n              &lt; .001),\n              <jats:italic>TP53<\/jats:italic>\n              mutations (84%\n              <jats:italic>v<\/jats:italic>\n              61%,\n              <jats:italic>P<\/jats:italic>\n              = .003), and\n              <jats:italic>BRAF<\/jats:italic>\n              amplification (9%\n              <jats:italic>v<\/jats:italic>\n              2%,\n              <jats:italic>P<\/jats:italic>\n              = .030). There were no\n              <jats:italic>KRAS<\/jats:italic>\n              mutations identified in patients with\n              <jats:italic>HER2-<\/jats:italic>\n              amplified BTC. No significant difference in overall survival (OS) was observed between patients with and without\n              <jats:italic>HER2<\/jats:italic>\n              amplification (median, 17.7\n              <jats:italic>v<\/jats:italic>\n              16.9 months; hazard ratio [HR], 0.95 [95% CI, 0.65 to 1.40]). Of the 60 patients with\n              <jats:italic>HER2<\/jats:italic>\n              -amplified BTC in the biomarker-selected cohort (43 from Japan and 17 from the United States), the OS was significantly longer in 29 patients who received HER2-targeted therapy than in those who did not receive HER2-targeted therapy (median, 24.3\n              <jats:italic>v<\/jats:italic>\n              12.1 months; HR, 0.39 [95% CI, 0.23 to 0.82]). Multivariate analysis identified HER2-targeted therapy as an independent prognostic factor for OS (HR, 0.29 [95% CI, 0.14 to 0.58];\n              <jats:italic>P<\/jats:italic>\n              &lt; .001).\n            <\/jats:p>\n          <\/jats:sec>\n          <jats:sec>\n            <jats:title>CONCLUSION<\/jats:title>\n            <jats:p>\n              <jats:italic>HER2<\/jats:italic>\n              amplification was found in 10% of advanced BTC and was not identified as an independent prognostic factor for OS. Patients with\n              <jats:italic>HER2<\/jats:italic>\n              -amplified BTC derive significant benefit from HER2-targeted therapy.\n            <\/jats:p>\n          <\/jats:sec>","DOI":"10.1200\/po-24-00718","type":"journal-article","created":{"date-parts":[[2025,4,10]],"date-time":"2025-04-10T19:59:11Z","timestamp":1744315151000},"update-policy":"https:\/\/doi.org\/10.1200\/crossmark","source":"Crossref","is-referenced-by-count":5,"title":["Clinicomolecular Profile and Efficacy of Human Epidermal Growth Factor Receptor 2 (HER2)-Targeted Therapy for\n            <i>HER2<\/i>\n            -Amplified Advanced Biliary Tract Cancer"],"prefix":"10.1200","author":[{"ORCID":"https:\/\/orcid.org\/0009-0006-4570-6383","authenticated-orcid":true,"given":"Kanae","family":"Inoue","sequence":"first","affiliation":[{"name":"Department of Hepatobiliary and Pancreatic Oncology, National Cancer Center Hospital East, Kashiwa, 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