September 2022, Vol 3, No 3

For patients with localized intrahepatic cholangiocarcinoma (iCCA), surgical resection holds curative potential for only 30% to 35% of patients due to its high rate of recurrence.

Although gemcitabine plus platinum/fluorouracil combinations are the standard first-line treatment for advanced biliary tract cancers (BTCs), the overall outcomes are suboptimal.

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

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.

First-line treatment options are limited to gemcitabine/cisplatin (GemCis) for patients with advanced cholangiocarcinoma (CCA).

Surgery remains the only potentially curative modality for cholangiocarcinoma (CCA) in any site of origin.

On September 2, 2022, the US Food and Drug Administration approved durvalumab in combination with gemcitabine/cisplatin for advanced biliary tract cancers (BTCs) on the basis of the TOPAZ-1 study.