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Title: Phase II study of BGJ398 in patients with FGFR-Altered advanced cholangiocarcinoma
Authors: Milind Javle
Maeve Lowery
Rachna T. Shroff
Karl Heinz Weiss
Christoph Springfeld
Mitesh J. Borad
Ramesh K. Ramanathan
Lipika Goyal
Saeed Sadeghi
Teresa Macarulla
Anthony El-Khoueiry
Robin Kate Kelley
Ivan Borbath
Su Pin Choo
Do Youn Oh
Philip A. Philip
Li Tzong Chen
Thanyanan Reungwetwattana
Eric Van Cutsem
Kun Huei Yeh
Kristen Ciombor
Richard S. Finn
Anuradha Patel
Suman Sen
Dale Porter
Randi Isaacs
Andrew X. Zhu
Ghassan K. Abou-Alfa
Tanios Bekaii-Saab
National Cheng Kung University Hospital
Barbara Ann Karmanos Cancer Institute
National Cancer Centre, Singapore
KU Leuven– University Hospital Leuven
UCSF Helen Diller Family Comprehensive Cancer Center
Mayo Clinic Scottsdale-Phoenix, Arizona
National Taiwan University College of Medicine
Faculty of Medicine, Ramathibodi Hospital, Mahidol University
Keck School of Medicine of USC
University of Texas MD Anderson Cancer Center
Seoul National University
Cliniques Universitaires Saint-Luc, Brussels
Hospital Universitari Vall d'Hebron
Memorial Sloan-Kettering Cancer Center
David Geffen School of Medicine at UCLA
Ohio State University
Universitätsklinikum Heidelberg
Massachusetts General Hospital Cancer Center
Novartis Pharmaceuticals Corporation
Keywords: Biochemistry, Genetics and Molecular Biology;Medicine
Issue Date: 20-Jan-2018
Citation: Journal of Clinical Oncology. Vol.36, No.3 (2018), 276-282
Abstract: © 2017 by American Society of Clinical Oncology. Purpose No standard treatment exists for patients with cholangiocarcinoma for whom first-line gemcitabine-based therapy fails. Fibroblast growth factor receptor 2 (FGFR2) fusions/translocations are present in 13% to 17% of intrahepatic cholangiocarcinomas. BGJ398, an orally bioavailable, selective pan-FGFR kinase inhibitor, has shown preliminary clinical activity against tumors with FGFR alterations. Methods A multicenter, open-label, phase II study ( identifier: NCT02150967) evaluated BGJ398 antitumor activity in patients age $ 18 years with advanced or metastatic cholangiocarcinoma containing FGFR2 fusions or other FGFR alterations whose disease had progressed while receiving prior therapy. Patients received BGJ398 125 mg once daily for 21 days, then 7 days off (28-day cycles). The primary end point was investigator-assessed overall response rate. Results Sixty-one patients (35 women; median age, 57 years) with FGFR2 fusion (n = 48), mutation (n = 8), or amplification (n = 3) participated. At the prespecified data cutoff (June 30, 2016), 50 patients had discontinued treatment. All responsive tumors contained FGFR2 fusions. The overall response rate was 14.8% (18.8% FGFR2 fusions only), disease control rate was 75.4% (83.3% FGFR2 fusions only), and estimated median progression-free survival was 5.8 months (95% CI, 4.3 to 7.6 months). Adverse events included hyperphosphatemia (72.1% all grade), fatigue (36.1%), stomatitis (29.5%), and alopecia (26.2%). Grade 3 or 4 treatment-related adverse events occurred in 25 patients (41%) and included hyperphosphatemia (16.4%), stomatitis (6.6%), and palmar-plantar erythrodysesthesia (4.9%). Conclusion BGJ398 is a first-in-class FGFR kinase inhibitor with manageable toxicities that shows meaningful clinical activity against chemotherapy-refractory cholangiocarcinoma containing FGFR2 fusions. This promising antitumor activity supports continued development of BGJ398 in this highly selected patient population.
ISSN: 15277755
Appears in Collections:Scopus 2018

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