Biliary Drainage Plus HAIC in Locally Advanced pCCA

Summary

Biliary drainage and stent placement remains to be the main palliative treatment choice for advanced perihiliar cholangiocarcinoma (pCCA), and the life expectancy is only 4-6 months. Previous single center prospective phase 2 trial showed that hepatic arterial infusion chemotherapy (HAIC) with oxaliplatin and 5-fluorouracil was an encouraging treatment choice for advanced pCCA due to its high tumor control, survival benefit, and low toxicity. Thus, the multicenter prospective controlled trial was designed to explore and confirm the survival benefit of biliary drainage plus hepatic arterial infusion chemotherapy with oxaliplatin and 5-fluorouracil compared with biliary drainage plus best support care treatment in locally advanced pCCA patients.

Timeline

Start

2021-11-01

Primary completion

2025-10-20

Completion

2025-10-20

Outcome

Met primary endpoint

paper Median OS was 17.63 months in the BD + HAIC group and 6.10 months in the BD + BSC group (P < 0.0001). PMID 42433279 ↗

Publications

• Background Ilyas SI, Khan SA, Hallemeier CL, Kelley RK, Gores GJ. Cholangiocarcinoma - evolving concepts and therapeutic strategies. Nat Rev Clin Oncol. 2018 Feb;15(2):95-111. doi: 10.1038/nrclinonc.2017.157. Epub 2017 Oct 10.
• Background Banales JM, Cardinale V, Carpino G, Marzioni M, Andersen JB, Invernizzi P, Lind GE, Folseraas T, Forbes SJ, Fouassier L, Geier A, Calvisi DF, Mertens JC, Trauner M, Benedetti A, Maroni L, Vaquero J, Macias RI, Raggi C, Perugorria MJ, Gaudio E, Boberg KM, Marin JJ, Alvaro D. Expert consensus document: Cholangiocarcinoma: current knowledge and future perspectives consensus statement from the European Network for the Study of Cholangiocarcinoma (ENS-CCA). Nat Rev Gastroenterol Hepatol. 2016 May;13(5):261-80. doi: 10.1038/nrgastro.2016.51. Epub 2016 Apr 20.
• Background Forner A, Vidili G, Rengo M, Bujanda L, Ponz-Sarvise M, Lamarca A. Clinical presentation, diagnosis and staging of cholangiocarcinoma. Liver Int. 2019 May;39 Suppl 1:98-107. doi: 10.1111/liv.14086. Epub 2019 Mar 25.
• Background Nath MC, Torbenson MS, Erickson LA. Perihilar Cholangiocarcinoma. Mayo Clin Proc. 2018 Mar;93(3):397-398. doi: 10.1016/j.mayocp.2018.01.017. No abstract available.
• Background Park J, Kim MH, Kim KP, Park DH, Moon SH, Song TJ, Eum J, Lee SS, Seo DW, Lee SK. Natural History and Prognostic Factors of Advanced Cholangiocarcinoma without Surgery, Chemotherapy, or Radiotherapy: A Large-Scale Observational Study. Gut Liver. 2009 Dec;3(4):298-305. doi: 10.5009/gnl.2009.3.4.298. Epub 2009 Dec 31.
• Background Valle J, Wasan H, Palmer DH, Cunningham D, Anthoney A, Maraveyas A, Madhusudan S, Iveson T, Hughes S, Pereira SP, Roughton M, Bridgewater J; ABC-02 Trial Investigators. Cisplatin plus gemcitabine versus gemcitabine for biliary tract cancer. N Engl J Med. 2010 Apr 8;362(14):1273-81. doi: 10.1056/NEJMoa0908721.
• Background Boehm LM, Jayakrishnan TT, Miura JT, Zacharias AJ, Johnston FM, Turaga KK, Gamblin TC. Comparative effectiveness of hepatic artery based therapies for unresectable intrahepatic cholangiocarcinoma. J Surg Oncol. 2015 Feb;111(2):213-20. doi: 10.1002/jso.23781. Epub 2014 Sep 1.
• Background Hu J, Zhu X, Wang X, Cao G, Wang X, Yang R. Evaluation of percutaneous unilateral trans-femoral implantation of side-hole port-catheter system with coil only fixed-catheter-tip for hepatic arterial infusion chemotherapy. Cancer Imaging. 2019 Mar 18;19(1):15. doi: 10.1186/s40644-019-0202-z.
• Background Wang X, Hu J, Cao G, Zhu X, Cui Y, Ji X, Li X, Yang R, Chen H, Xu H, Liu P, Li J, Li J, Hao C, Xing B, Shen L. Phase II Study of Hepatic Arterial Infusion Chemotherapy with Oxaliplatin and 5-Fluorouracil for Advanced Perihilar Cholangiocarcinoma. Radiology. 2017 May;283(2):580-589. doi: 10.1148/radiol.2016160572. Epub 2016 Nov 7.
• Dai Y, Wang S, Gao J, Wang Y, Yang X, Zhang S, Zhi X, Zheng K, Xu L, Cao G, Xing B, Wang K, Hao C, Zhu X, Zhang DY, Gao DK, Wang DX. Biliary drainage plus hepatic arterial infusion chemotherapy versus biliary drainage plus best supportive care for advanced perihilar cholangiocarcinoma in China: a prospective multicentre non-randomised controlled study. EClinicalMedicine. 2026 Jul 2;97:104040. doi: 10.1016/j.eclinm.2026.104040. eCollection 2026 Jul.

Drugs

Indications