A Phase II Study of Toripalimab in Combination with Gemcitabine and 5-Fluorouracil as First-Line Therapy for Advanced or Metastatic Biliary Tract Carcinoma.

ABSTRACT:

Background/objectives

The anti-PD-L1 antibody has been applied for use in first-line advanced biliary duct cancer patients. However, clinical evidence of toripalimab in combination with biweekly 5-fluorouracil (5-FU) is limited and predictive biomarkers of treatment benefits remain unclear.

Methods

This prospective study enrolled patients with unresectable or metastatic BTC who received toripalimab in combination with gemcitabine and biweekly 5-FU. The primary endpoints were progression-free survival (PFS) and objective response rate (ORR). Secondary endpoints included overall survival (OS) and safety. Exploratory analyses evaluated associations between programmed cell death-ligand 1 (PD-L1) expression or tumor mutational burden (TMB) and clinical outcomes.

Results

A

SUBMITTER: Lei F 

PROVIDER: S-EPMC12784805 | biostudies-literature | 2025 Dec

REPOSITORIES: biostudies-literature