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. 2024 Jan 21;16(1):e52656.
doi: 10.7759/cureus.52656. eCollection 2024 Jan.

Phase II Trial of FOLFIRINOX in Advanced Biliary Tract Cancer

Shouki Bazarbashi  1 , Mohamed Aseafan  2 , Mahmoud Elshenawy  1   3 , Ahmed Alzahrani  1 , Ali H Aljubran  1 , Fahad Almugbel  1 , Noura Alzannan  4 , Tusneem Elhassan  4
Affiliations

Phase II Trial of FOLFIRINOX in Advanced Biliary Tract Cancer

Shouki Bazarbashi et al. Cureus. .

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.

Keywords: advanced biliary tract cancer; cancer of gallbladder; cholangiocarcinoma; folfirinox chemotherapy; phase ii.

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Conflict of interest statement

The authors have declared that no competing interests exist.

Figures

Figure 1
Figure 1. Change in target lesion size in 13 patients treated with FOLFIRINOX
FOLFIRINOX: Fluorouracil, leucovorin, oxaliplatin, and irinotecan
Figure 2
Figure 2. Kaplan-Meier curve for PFS in 13 patients treated with first-line FOLFIRINOX
PFS: Progression-free survival, FOLFIRINOX: Fluorouracil, leucovorin, oxaliplatin, and irinotecan
Figure 3
Figure 3. Kaplan-Meier curve for OS in 13 patients treated with FOLFIRINOX
OS: Overall survival, FOLFIRINOX: Fluorouracil, leucovorin, oxaliplatin, and irinotecan
See this image and copyright information in PMC

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