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For patients with metastatic cholangiocarcinoma, chemotherapy beyond first-line gemcitabine and cisplatin has limited benefit, including marginal survival benefit reported for second-line FOLFOX in a recent phase III trial (NEJM JW Oncol Hematol Aug 2019). Genomic profiling of these cancers indicates heterogeneity among intrahepatic, extrahepatic, and gallbladder cancer and also suggests potentially druggable targets.
Investigators now report results of an industry-sponsored, international, single-arm, open-label, phase II trial of pemigatinib — an oral tyrosine kinase inhibitor of fibroblast growth factor receptor (FGFR) 1, 2, and 3 — in patients with locally advanced or metastatic cholangiocarcinoma who had received prior chemotherapy. Of …