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Review
. 2023 Jan:179:1-14.
doi: 10.1016/j.ejca.2022.11.006. Epub 2022 Nov 12.

Cholangiocarcinoma: what are the options in all comers and how has the advent of molecular profiling opened the way to personalised medicine ?

Gael S Roth  1 , Cindy Neuzillet  2 , Matthieu Sarabi  3 , Julien Edeline  4 , David Malka  5 , Astrid Lièvre  6
Affiliations
Free article
Review

Cholangiocarcinoma: what are the options in all comers and how has the advent of molecular profiling opened the way to personalised medicine ?

Gael S Roth et al. Eur J Cancer. 2023 Jan.
Free article

Abstract

Cholangiocarcinoma is a deadly cancer comprising very heterogenous subtypes with a limited therapeutic arsenal in all comers. However, recent significant advances were made with immunotherapy in the first-line treatment of advanced cholangiocarcinoma, with the addition of durvalumab to cisplatin-gemcitabine chemotherapy showing a survival benefit. In the second line setting, only FOLFOX (5FU/folinic acid-oxaliplatin) is validated by a phase 3 trial, yet with a very modest benefit on survival; new options using 5FU with nanoliposomal-irinotecan may emerge in the next few years. The advent of molecular profiling in advanced cholangiocarcinoma in the last decade revealed frequent targetable alterations such as IDH1 mutations, FGFR2 fusions or rearrangements, HER2 amplification, BRAF V600E mutation and others. This strategy opened the way to personalised medicine for patients which are still fit after first-line treatment and the use of targeted inhibitors in first line constitutes a huge challenge with many ongoing trials to improve patients' care. This review exposes the recent clinical trial findings in non-molecularly selected advanced cholangiocarcinoma, offers a focus on how systematic molecular screening should be structured to allow patients to access to personalised medicine, and details which are the therapeutic options accessible in case of actionable alteration.

Keywords: Bile tract cancer; Cholangiocarcinoma; FGFR2 fusion; HER2 amplification; IDH1 mutation; Molecular profiling; Personnalized medicine.

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Conflict of interest statement

Conflict of interest statement Gael S Roth declares consulting or advisory role for Servier, AstraZeneca, Bristol Myers Squibb, MSD, Servier, Amgen, Ipsen, Viatris, Accord Healthcare, Sanofi; travel, accommodations or expenses from Servier, AstraZeneca, Bristol Myers Squibb, MSD, Roche, Amgen, Ipsen. Cindy Neuzillet declares consultancy/honoraria from Amgen, AstraZeneca, Baxter, Bristol-Myers Squibb, Fresenius Kabi, Incyte Biosciences, Merck, MSD, Mylan, Novartis, Nutricia, Pierre Fabre, Roche, Sanofi, Servier, Viatris; Research funding from Roche; clinical trials sponsored by OSE Immunotherapeutics, AstraZeneca, Bristol-Myers Squibb, Fresenius Kabi, Nutricia, Viatris. Matthieu Sarabi declares consultancy/honoraria AstraZeneca, Servier, Roche and accommodations or expenses from Ipsen. Julien Edeline declares consulting or advisory role for BTG, Bristol Myers Squibb, AstraZeneca, Bayer, Ipsen, AstraZeneca, MSD, Eisai, Boston Scientific, Roche, Basilea, Merck Serono, Servier; research funding from Bristol Myers Squibb (Inst), BeiGene (Inst); travel, accommodations or expenses from Amgen, Bristol Myers Squibb, Roche. David Malka declares honoraria for Roche, Amgen, Bayer, Merck Serono, Servier, Sanofi, Pierre Fabre, Viatris, Bristol Myers Squibb, MSD Oncology, LEO Pharma, Incyte, AstraZeneca; consulting or advisory role: Roche, Sanofi, Merck Serono, MSD, Servier, Bayer, Incyte, Amgen, Bristol Myers Squib, Taiho Oncology, AbbVie, AstraZeneca, Pierre Fabre; travel, accommodations or expenses from Roche, Bayer, Sanofi, Merck Serono, Amgen, Servier, Pierre Fabre, Bristol Myers Squibb/Pfizer, MSD, Viatris. Astrid Lièvre declares honoraria for lectures from AAA, Amgen, Bayer, BMS, Incyte, Ipsen, Leo-pharma, Mylan, Novartis, Pierre Fabre, Roche, Sandoz, Sanofi, Servier and Viatris; consulting/advisory relationship from AAA, Astellas, BMS, Incyte, Pierre Fabre and Servier; travel, accomodations or congress registration support from Bayer, Boehringer, Ipsen, Mylan, MSD, Pierre Fabre, Roche and Servier; research funding from Bayer (Inst), Lilly (Inst), Novartis (Inst). No other potential conflicts of interest were reported.

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