Futibatinib for <i>FGFR2</i> -Rearranged Intrahepatic Cholangiocarcinoma
Lipika Goyal, Funda Meric‐Bernstam, Antoine Hollebecque, Juan W. Valle, Chigusa Morizane, Thomas B. Karasic, Thomas A. Abrams, Junji Furuse, Robin Kate Kelley, Philippe A. Cassier, Heinz‐Josef Klümpen, Heung-Moon Chang, Li‐Tzong Chen, Josep Tabernero, Do‐Youn Oh, Amit Mahipal, Markus Moehler, Edith P. Mitchell, Yoshito Komatsu, Kunihiro Masuda, Daniel H. Ahn, Robert S. Epstein, Abdel‐Baset Halim, Yao Fu, Tehseen Salimi, Volker Wacheck, Yaohua He, Mei Liu, Karim A. Benhadji, John Bridgewater
Abstract
BACKGROUND: -altered tumors and strong preclinical activity against acquired resistance mutations associated with ATP-competitive FGFR inhibitors. METHODS: rearrangement-positive intrahepatic cholangiocarcinoma and disease progression after one or more previous lines of systemic therapy (excluding FGFR inhibitors). The patients received oral futibatinib at a dose of 20 mg once daily in a continuous regimen. The primary end point was objective response (partial or complete response), as assessed by independent central review. Secondary end points included the response duration, progression-free and overall survival, safety, and patient-reported outcomes. RESULTS: mutations. At a median follow-up of 17.1 months, the median progression-free survival was 9.0 months and overall survival was 21.7 months. Common treatment-related grade 3 adverse events were hyperphosphatemia (in 30% of the patients), an increased aspartate aminotransferase level (in 7%), stomatitis (in 6%), and fatigue (in 6%). Treatment-related adverse events led to permanent discontinuation of futibatinib in 2% of the patients. No treatment-related deaths occurred. Quality of life was maintained throughout treatment. CONCLUSIONS: fusion or rearrangement-positive intrahepatic cholangiocarcinoma, the use of futibatinib, a covalent FGFR inhibitor, led to measurable clinical benefit. (Funded by Taiho Oncology and Taiho Pharmaceutical; FOENIX-CCA2 ClinicalTrials.gov number, NCT02052778.).