The Lynx Group
Cholangiocarcinoma News

March 2022, Vol 3, No 1

The global, double-blind, placebo-controlled, phase 3 TOPAZ-1 clinical trial evaluated the first-line chemoimmunotherapy regimen of the PD-L1 inhibitor durvalumab plus gemcitabine and cisplatin in patients with advanced biliary tract cancer. Do-Youn Oh, MD, PhD, Division of Medical Oncology, Department of Internal Medicine, Seoul National University Hospital, South Korea, presented the results of the planned interim analysis for this study at the 2022 ASCO GI Cancers Symposium.
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The 2022 ASCO GI Cancers Symposium clearly highlighted the rapid trajectory of clinical research in biliary tract cancers. One of the most eagerly anticipated studies, TOPAZ-1, was presented by Do-Youn Oh, MD, PhD, from Seoul National University Hospital in South Korea. This phase 3 clinical trial enrolled 685 patients and investigated the addition of the PD-L1–directed agent durvalu­mab to gemcitabine and cisplatin chemotherapy backbone versus these chemotherapies alone. The results demonstrated a statistically significant improvement in overall survival (OS), overall response rate (ORR), and progression-free survival (PFS), without dose-limiting toxicities.
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The International Cholangiocarcinoma Research Network (ICRN) is a program initiated by the Cholangiocarcinoma Foundation (CCF). The core mission of this network of international experts is “to rapidly translate scientific discoveries into practice for cholangiocarcinoma patients through a collaborative, highly translational international research network.”
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At the 2022 Cholangiocarcinoma Foundation (CCF) Annual Conference on February 22-25, Melinda Bachini, Director of Advocacy of CCF, provided an update about the foundation’s advocacy and patient support initiatives. The goal of the CCF’s advocacy group is to provide educational and emotional support for the cholangiocarcinoma (CCA) community of patients and caregivers, and to empower them with the knowledge and guidance needed to self-advocate.
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On the second day of the 2022 Cholangiocarcinoma Foundation annual meeting, Lipika Goyal, MD, MPhil, Assistant Professor of Medicine, Harvard Medical School, and Lead, Liver Cancer Research Program, Massachusetts General Hospital Cancer Center, Boston, presented a clinical update on emerging systemic therapies in cholangiocarcinoma (CCA).
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The phase 2 FOENIX-CCA2 clinical trial evaluated the investigational FGFR1-4 inhibitor futibatinib in patients with advanced intrahepatic cholangiocarcinoma (CCA) harboring FGFR2 fusion or other rearrangements.
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Real-world data are limited regarding the natural history of patients with advanced cholangiocarcinoma (CCA) and FGFR2 fusion or other rearrangements who are receiving therapies for advanced disease. Rachna T. Shroff, MD, of the University of Arizona Cancer Center, Tucson, and colleagues conducted a retrospective, observational natural history study to compare real-world overall survival (OS) in patients with advanced CCA and FGFR2 fusion or other rearrangements versus patients with wild-type FGFR2 from the diagnosis of advanced CCA to death. Dr Shroff presented the study results at the 2022 ASCO GI Cancers Symposium.
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Mutations associated with homologous recombination deficiency (HRD) have been identified in up to 25% of patients with biliary tract cancers; however, the proportion of patients with germline versus somatic mutations has not been previously defined.
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The subtypes of cholangiocarcinoma (CCA), including intrahepatic CCA and extrahepatic CCA, have various underlying tumor microenvironments, pathobiology, epidemiologic features, and risk factors; however, the genetic underpinnings of the CCA subtypes have not been previously defined.
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Derazantinib is a potent oral inhibitor of FGFR1, FGFR2, and FGFR3 that is being investigated in the phase 2 FIDES-01 clinical trial of patients with advanced intrahepatic cholangiocarcinoma (CCA) harboring FGFR2 fusions or rearrangements.
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