In the phase 2 basket ROAR study, patients with BRAF V600E mutation in 9 rare tumor types were enrolled and received dabrafenib plus trametinib as second- or later-line treatment. The results of biliary tract cancer cohort were recently published on ASCO Gastrointestinal Cancers Symposium 2019. Of the 557 prescreened biliary tract cancer patients, 49 harbored BRAF V600E mutation and 35 were enrolled. All of them had received gemcitabine and eighty percent patients had received at least 2 prior lines of systemic therapy. The objective response rate (ORR) and disease control rate (DCR), as assessed by BICR, was 12/33 (36.4%) and 25/33 (75.8%). The median PFS and median OS was 9.2 and 11.7 months, respectively. This study demonstrated that BRAF V600E is an actionable mutation and the combination of dabrafenib and trametinib is a potential therapeutic strategy for biliary tract cancer patients with this mutation.
Reference: J Clin Oncol 37, 2019 (suppl 4; abstr 187)