Abou-Alfa GK et al. Ivosidenib in IDH1-mutant, chemotherapy-refractory cholangiocarcinoma (ClarIDHy): A multicentre, randomised, double-blind, placebo-controlled, phase 3 study. Lancet Oncol 2020;21(6):796-807. Abstract
Abou-Alfa GK et al. Pemigatinib for previously treated, locally advanced or metastatic cholangiocarcinoma: A multicentre, open-label, phase 2 study. Lancet Oncol 2020;21(5):671-84. Abstract
Bekaii-Saab TS et al. FIGHT-302: First-line pemigatinib vs gemcitabine plus cisplatin for advanced cholangiocarcinoma with FGFR2 rearrangements. Future Oncol 2020;16(30):2385-99. Abstract
Berchuck JE et al. The clinical landscape of cell-free DNA alterations in 1671 patients with advanced biliary tract cancer. Ann Oncol 2022;33(12):1269-83. Abstract
Borad MJ et al. A phase III study of futibatinib (TAS-120) versus gemcitabine-cisplatin (gem-cis) chemotherapy as first-line (1L) treatment for patients (pts) with advanced (adv) cholangiocarcinoma (CCA) harboring fibroblast growth factor receptor 2 (FGFR2) gene rearrangements (FOENIX-CCA3). Gastrointestinal Cancers Symposium 2020;Abstract TPS600.
Chang W-H et al. MTAP deficiency contributes to immune landscape remodelling and tumour evasion. Immunology 2023;168(2):331-45. Abstract
Doki Y et al. Tolerability and efficacy of durvalumab, either as monotherapy or in combination with tremelimumab, in patients from Asia with advanced biliary tract, esophageal, or head-and-neck cancer. Cancer Med 2022;11(13):2550-60. Abstract
Finn RS et al. Three-year follow-up data from KEYNOTE-966: Pembrolizumab (pembro) plus gemcitabine and cisplatin (gem/cis) compared with gem/cis alone for patients (pts) with advanced biliary tract cancer (BTC). ASCO 2024;Abstract 4093.
Galluzzi L et al. Immunostimulation with chemotherapy in the era of immune checkpoint inhibitors. Nat Rev Clin Oncol 2020;17(12):725-41. Abstract
Goyal L et al. Futibatinib for FGFR2-rearranged intrahepatic cholangiocarcinoma. N Engl J Med 2023;19;388(3):228-39. Abstract
Goyal L et al. Updated results of the FOENIX-CCA2 trial: Efficacy and safety of futibatinib in intrahepatic cholangiocarcinoma (iCCA) harboring FGFR2 fusions/rearrangements. ASCO 2022;Abstract 4009.
Goyal L et al. Case 8-2021: A 34-year-old woman with cholangiocarcinoma. N Engl J Med 2021;18;384(11):1054-64. Abstract
Goyal L et al. Targeting FGFR inhibition in cholangiocarcinoma. Cancer Treat Rev 2021;95:102170. Abstract
Gravett AM et al. Gemcitabine alters the proteasome composition and immunopeptidome of tumour cells. Oncoimmunology 2018;7(6):e1438107. Abstract
Harding JJ et al. Zanidatamab for HER2-amplified, unresectable, locally advanced or metastatic biliary tract cancer (HERIZON-BTC-01): A multicentre, single-arm, phase 2b study. Lancet Oncol 2023;24(7):772-82. Abstract
Javle M et al. Pertuzumab and trastuzumab for HER2-positive, metastatic biliary tract cancer (MyPathway): A multicentre, open-label, phase 2a, multiple basket study. Lancet Oncol 2021;22(9):1290-1300. Abstract
Job S et al. Identification of four immune subtypes characterized by distinct composition and functions of tumor microenvironment in intrahepatic cholangiocarcinoma. Hepatology 2020;72(3):965-81. Abstract
Ju JY et al. Mismatch repair protein deficiency/microsatellite instability is rare in cholangiocarcinomas and associated with distinctive morphologies. Am J Clin Pathol 2020;153(5):598-604. Abstract
Kato S et al. Analysis of MDM2 Amplification: Next-generation sequencing of patients with diverse malignancies. JCO Precis Oncol 2018;2018:PO.17.00235. Abstract
Kendre G et al. Charting co-mutation patterns associated with actionable drivers in intrahepatic cholangiocarcinoma. J Hepatol 2023;78(3):614-26. Abstract
Kim RD et al. A phase 2 multi-institutional study of nivolumab for patients with advanced refractory biliary tract cancer. JAMA Oncol 2020;1;6(6):888-94. Abstract
Lamarca A et al. Molecular targeted therapies: Ready for “prime time” in biliary tract cancer. J Hepatol 2020;73(1):170-85. Abstract
Le DT et al. Mismatch repair deficiency predicts response of solid tumors to PD-1 blockade. Science 2017;357(6349):409-13. Abstract
LoRusso P et al. The MDM2-p53 antagonist brigimadlin (BI 907828) in patients with advanced or metastatic solid tumors: Results of a phase Ia, first-in-human, dose-escalation study. Cancer Discov 2023;13(8):1802-13. Abstract
Macarulla T et al. Efficacy and safety of brigimadlin (BI 907828), an MDM2–p53 antagonist, in patients (pts) with advanced biliary tract cancer: Data from two phase Ia/Ib dose-escalation/expansion trials. Gastrointestinal Cancers Symposium 2024;Abstract 487.
Marjon K et al. MTAP deletions in cancer create vulnerability to targeting of the MAT2A/PRMT5/RIOK1 axis. Cell Rep 2016;15(3):574-87. Abstract
Meric-Bernstam F et al. Safety profile and adverse event management for futibatinib, an irreversible FGFR1-4 inhibitor: Pooled safety analysis of 469 patients. Clin Cancer Res 2024;30(8):1466-77. Abstract
Nakamura Y et al. Tucatinib and trastuzumab for previously treated human epidermal growth factor receptor 2-positive metastatic biliary tract cancer (SGNTUC-019): A phase II basket study. J Clin Oncol 2023;41(36):5569-78. Abstract
Ngoi NYL et al. Methylthioadenosine phosphorylase genomic loss in advanced gastrointestinal cancers. Oncologist 2024;29(6):493-503. Abstract
Oh D-Y et al. Durvalumab or placebo plus gemcitabine and cisplatin in participants with advanced biliary tract cancer (TOPAZ-1): Updated overall survival from a randomised phase 3 study. Lancet Gastroenterol Hepatol 2024;9(8):694-704. Abstract
Oh D-Y et al. A phase 3 randomized, double-blind, placebo-controlled study of durvalumab in combination with gemcitabine plus cisplatin (GemCis) in patients (pts) with advanced biliary tract cancer (BTC): TOPAZ-1. Gastrointestinal Cancers Symposium 2022;Abstract 378.
Oh D-Y et al. Durvalumab plus gemcitabine and cisplatin in advanced biliary tract cancer. NEJM Evid 2022;1(8). Abstract
Ohba A et al. Trastuzumab deruxtecan (T-DXd; DS-8201) in patients (pts) with HER2-expressing unresectable or recurrent biliary tract cancer (BTC): An investigator-initiated multicenter phase 2 study (HERB trial). ASCO 2022;Abstract 4006.
Ostwal V et al. Trastuzumab plus gemcitabine-cisplatin for treatment-naïve human epidermal growth factor receptor 2-positive biliary tract adenocarcinoma: A multicenter, open-label, phase II study (TAB). J Clin Oncol 2024;42(7):800-7. Abstract
Piha-Paul SA et al. Efficacy and safety of pembrolizumab for the treatment of advanced biliary cancer: Results from the KEYNOTE-158 and KEYNOTE-028 studies. Int J Cancer 2020;147(8):2190-8. Abstract
Staudacher AH, Brown MP. Antibody drug conjugates and bystander killing: Is antigen-dependent internalisation required? Br J Cancer 2017;117(12):1736-42. Abstract
Tang T-Y et al. Genomic profile of intrahepatic cholangiocarcinoma with MTAP loss. ASCO 2022;Abstract 4102.
Valle JW et al. Impact of mutation status on efficacy outcomes in TOPAZ-1: A phase III study of durvalumab (D) or placebo (PBO) plus gemcitabine and cisplatin (+ GC) in advanced biliary tract cancer (BTC). ESMO Asia 2022;Abstract 68O.
Vogel A et al. An open-label study of pemigatinib in cholangiocarcinoma: Final results from FIGHT-202. ESMO Open 2024;9(6):103488. Abstract
Vogel A et al. TOURMALINE: A phase 3b study of durvalumab with gemcitabine-based chemotherapy regimens in advanced biliary tract cancer. ASCO 2024;Abstract TPS4188.
Vogel A et al. Regional subgroup analysis of the phase 3 TOPAZ-1 study of durvalumab (D) plus gemcitabine and cisplatin (GC) in advanced biliary tract cancer (BTC). ASCO 2022;Abstract 4075.
Wu Q et al. Protein arginine methylation: From enigmatic functions to therapeutic targeting. Nat Rev Drug Discov 2021;20(7):509-30. Abstract
Yarchoan M et al. PD-L1 expression and tumor mutational burden are independent biomarkers in most cancers. JCI Insight 2019;4(6):e126908. Abstract
Yoo C et al. Brightline-2: A phase IIa/IIb trial of brigimadlin (BI 907828) in advanced biliary tract cancer, pancreatic ductal adenocarcinoma or other solid tumors. Future Oncol 2024;20(16):1069-77. Abstract
Zhou Z-X et al. Antifungal drugs and drug-induced liver injury: A real-world study leveraging the FDA adverse event reporting system database. Front Pharmacol 2022;13:891336. Abstract
Zhu AX et al. Final overall survival efficacy results of ivosidenib for patients with advanced cholangiocarcinoma with IDH1 mutation: The phase 3 randomized clinical ClarIDHy trial. JAMA Oncol 2021;7(11):1669-77. Abstract
Zhu AX et al. Final results from ClarIDHy, a global, phase III, randomized, double-blind study of ivosidenib (IVO) versus placebo (PBO) in patients (pts) with previously treated cholangiocarcinoma (CCA) and an isocitrate dehydrogenase 1 (IDH1) mutation. Gastrointestinal Cancers Symposium 2021;Abstract 266.
Zhu H et al. Targeting p53-MDM2 interaction by small-molecule inhibitors: Learning from MDM2 inhibitors in clinical trials. J Hematol Oncol 2022;15(1):91. Abstract