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Ref Type
Authors James M. Cleary, Srivatsan Raghavan, Yvonne Y. Li, Liam Spurr, Qibiao Wu, Lei Shi, Lauren K. Brais, Zunelly Odhiambo, Lipika Goyal, Anuj K. Patel, Atul B. Shinagare, Thomas E. Clancy, Geoffrey Shapiro, Ethan Cerami, William R. Sellers, William C. Hahn, Andrew D. Cherniack, Nabeel Bardeesy, Matthew Meyerson, Brian M. Wolpin
Title Therapeutic targeting of extracellular FGFR2 activating deletions in intrahepatic cholangiocarcinoma.
Abstract Text J Clin Oncol 38, 2020 (suppl 4; abstr 567)


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Molecular Profile Treatment Approach
Gene Name Source Synonyms Protein Domains Gene Description Gene Role
Therapy Name Drugs Efficacy Evidence Clinical Trials
Drug Name Trade Name Synonyms Drug Classes Drug Description
Gene Variant Impact Protein Effect Variant Description Associated with drug Resistance
FGFR2 H167_N173del deletion gain of function FGFR2 H167_N173del results in the deletion of seven amino acids in the Ig-like C2-type domain 2 of the Fgfr2 protein from amino acids 167 to 173 ( H167_N173del results in cell transformation (J Clin Oncol 38, 2020 (suppl 4; abstr 567), PMID: 33926920), anchorage-independent growth, constitutive activation of Fgfr2 kinase activity in culture, and tumor formation in an in vivo model (PMID: 33926920).
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
FGFR2 H167_N173del FGFR2 L617F intrahepatic cholangiocarcinoma predicted - resistant Debio 1347 Case Reports/Case Series Actionable In a clinical study, a patient with intrahepatic cholangiocarcinoma harboring FGFR2 H167_N173del developed resistance to Debio 1347 treatment after initial response, FGFR2 L617F was identified as an acquired mutation at disease progression (J Clin Oncol 38, 2020 (suppl 4; abstr 567)). detail...
FGFR2 H167_N173del intrahepatic cholangiocarcinoma predicted - sensitive Debio 1347 Case Reports/Case Series Actionable In a clinical study, Debio 1347 treatment resulted in durable partial response of 11 months in 2 patients with intrahepatic cholangiocarcinoma harboring FGFR2 H167_N173del (J Clin Oncol 38, 2020 (suppl 4; abstr 567)). detail...