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Treatment options for metastatic cholangiocarcinoma beyond initial gemcitabine-based therapy are limited. Next-generation sequencing has identified potential targetable mutations as well as molecular heterogeneity, based on the primary tumor location in the gallbladder, intrahepatic, or extrahepatic bile ducts. Recent regulatory approval was obtained for the FGFR2 inhibitor pemigatinib in patients with cholangiocarcinoma harboring an FGFR2 gene fusion.
Investigators now report results of an industry-sponsored, international, open-label, single-arm, phase II basket trial (ROAR) of the BRAF inhibitor dabrafenib (150 mg twice daily) plus the MEK inhibitor trametinib (2 mg once daily) in 43 patients with cholangiocarcinoma harboring a BRAF V600E…