ASCO Highlights

The randomized, double-blind, global, phase 3 TOPAZ-1 trial demonstrated that the first-line PD-L1 inhibitor durvalumab plus gemcitabine/cisplatin (GemCis; median follow-up, 16.8 months) as first-line treatment significantly improved overall survival (OS) versus placebo plus GemCis (median follow-up, 15.9 months) in patients with advanced biliary tract cancers (BTCs).¹

Tumor-related liver failure (TRLF) is the most common cause of death in patients with metastatic intrahepatic cholangiocarcinoma (iCCA), accounting for up to 72% of deaths in patients treated with systemic therapy alone.¹

Primary analysis of the pivotal, single-arm, phase 2 FOENIX-CCA2 study demonstrated that the FGFR1-4 inhibitor futibatinib as second-line treatment yielded durable objective responses in patients with intrahepatic cholangiocarcinoma (iCCA) harboring FGFR2 fusion/rearrangements.

Adjuvant capecitabine (CAP) is the current standard of care as adjuvant therapy for patients with resected cholangiocarcinoma (CCA); however, patient outcomes are still suboptimal.

At the CCA Summit during the 2021 Annual Meeting of the American Society of Clinical Oncology (ASCO), Mitesh J. Borad, MD, Associate Professor of Medicine, Mayo Clinic, Phoenix, AZ, reviewed some of the key topics presented at ASCO 2021 related to cholangiocarcinoma (CCA) and other biliary tract cancers. Before turning to the specific presentations, Dr Borad welcomed the May 28, 2021, FDA approval of infigratinib (Truseltiq) for patients with advanced or metastatic CCA and FGFR2 fusions or rearrangements. Infigratinib follows pemigatinib (Pemazyre) as the second targeted therapy now available for this patient population.

At the CCA Summit held during the 2021 ASCO Gastrointestinal (GI) Cancers Symposium, Rachna T. Shroff, MD, MS, Chief, Section of GI Medical Oncology, University of Arizona Cancer Center, Tucson, discussed 15 clinical trials that were presented at the ASCO GI Cancers Symposium on cholangiocarcinoma (CCA) and hepatobiliary diseases. She highlighted key advances related to chemotherapy, targeted therapies, and biomarkers in the management of biliary tract cancers, including CCA.

Final results from the phase 3 clinical trial ClarIDHy showed that ivosidenib (Tibsovo), a first-in-class oral inhibitor of isocitrate dehydrogenase 1 (IDH1) mutation, prolonged the median overall survival (OS) in patients with previously treated advanced cholangiocarcinoma (CCA) and IDH1 mutation. Although this improvement did not reach statistical significance, after adjusting for crossovers from the placebo to the ivosidenib group, the difference in median OS was statistically significant.