Researchers have found that that FOLFIRINOX, a chemotherapy used for pancreatic cancer, may improve survival in advanced biliary tract cancer (BTC) patients. 

A new collaborative study from Pusan National University and Yonsei University suggests that the FOLFIRINOX regimen showed numerically improved survival outcomes compared to current standards such as FOLFOX, FOLFIRI, and nal-IRI/FL, while maintaining manageable toxicity levels.  

These findings suggest that FOLFIRINOX could serve as a promising second-line treatment option for patients with advanced BTC following first-line chemotherapy failure, warranting further validation through prospective clinical studies. 

Biliary tract cancers, including intrahepatic, perihilar, and extrahepatic cholangiocarcinoma and gallbladder cancer, are among the most aggressive gastrointestinal malignancies. Treatment options remain limited once the disease progresses after first-line chemotherapy, and survival rarely exceeds one year. 

The team of researchers led by Professor Yun Hak Kim from Pusan National University, analysed 12 years of clinical data from 54 patients treated at Yonsei Severance Hospital and combined the results with a systematic review and meta-analysis of 21 studies from around the world. The findings were published in the journal International Journal of Surgery. 

“We conducted a meta-analysis integrating 12 years of real-world data on the use of FOLFIRINOX or mFOLFIRINOX as salvage treatment in patients with advanced BTC treated at the Division of Gastroenterology, Department of Internal Medicine, Severance Hospital, along with all available published studies on second-line chemotherapy regimens for advanced BTC,” said Professor Kim. 

In this study, patients receiving FOLFIRINOX achieved a median progression-free survival of 4.2 months and an overall survival of 11.4 months. 

Still, the authors caution that toxicity remains significant. Nearly 40% of patients developed severe neutropenia, requiring dose adjustments or additional medical support. The team emphasises that FOLFIRINOX should be reserved for fit patients under close supervision until further prospective trials confirm its broader safety. 

Diana Turner, Deputy Editor, DDW