# Phase II Trial of FOLFIRINOX in Advanced Biliary Tract Cancer

## TL;DR

This study tested FOLFIRINOX as a first-line treatment for advanced biliary tract cancer and found promising response rates and survival outcomes.

## Contribution

The study provides new evidence on the efficacy and safety of FOLFIRINOX in treating advanced biliary tract cancer.

## Key findings

- The overall response rate was 54% with a disease control rate of 77%.
- Median progression-free survival was 6.8 months and median overall survival was 19.25 months.
- Grade 3/4 toxicities were mainly hematologic, with neutropenia being the most common.

## Abstract

Introduction: Biliary tract cancers (BTCs), characterized by poor prognosis and limited treatment options, are increasingly prevalent malignancies with a five-year survival rate of less than 20% for advanced-stage disease. The standard first-line chemotherapy combining gemcitabine and cisplatin offers modest survival benefits, necessitating the exploration of more effective therapies. This study reports the results of a single-arm, open-label, phase 2 trial assessing the efficacy and safety of fluorouracil, leucovorin, oxaliplatin, and irinotecan (FOLFIRINOX) as a first-line treatment for metastatic or locally advanced unresectable BTC.

Methods: Patients aged ≥18 with measurable disease and adequate organ function were enrolled, receiving biweekly FOLFIRINOX for up to 12 cycles with follow-up imaging every four cycles. The primary endpoint was the overall response rate (ORR), with progression-free survival (PFS), overall survival (OS), and safety as secondary endpoints.

Results: Thirteen patients were enrolled from December 2016 to September 2021 before early termination due to slow accrual and the emergence of immunotherapy. The ORR was 54%, with a disease control rate of 77%. Median PFS and OS were 6.8 and 19.25 months, respectively. Grade 3/4 toxicities were predominantly hematologic, with neutropenia being the most common severe adverse event.

Conclusion: The trial suggests that FOLFIRINOX is a potentially effective first-line therapy for unresectable or metastatic BTC with a manageable safety profile. However, the early termination of the study and the introduction of immunotherapy warrant further research to confirm these findings.