Progress Toward Improving Outcomes in Patients with Cholangiocarcinoma


Purpose of review

To provide an update on the latest advances in treatment of cholangiocarcinoma.

Recent findings

Incidence of cholangiocarcinoma has been increasing over the past decade. A better understanding of the genetic landscape of cholangiocarcinoma and its risk factors resulted in earlier diagnosis and treatment option expansion to targeted therapy with FGFR inhibitors, and liver transplantation for early perihilar cholangiocarcinoma and early intrahepatic cholangiocarcinoma. IDH1/2 inhibition for intrahepatic cholangiocarcinoma is an emerging targeted therapy approach. Data supports benefits of adjuvant therapy for a subset of patients undergoing surgical resection. Approaches combining different treatment modalities such as chemotherapy, surgery, and radiation therapy appear to be promising.


Earlier diagnosis and genetic characterization provided additional treatment options for patients with previously incurable cholangiocarcinoma. A precision medicine approach with a focus on actionable genetic alterations and combination of treatment modalities are actively being explored and will further improve outcomes in our patients with cholangiocarcinoma.

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Fig. 1





Intrahepatic CCA


Perihilar CCA


Distal CCA


Biliary tract cancers


Primary sclerosing cholangitis


Hepatocellular carcinoma


Computed tomography


Magnetic resonance imaging


Endoscopic ultrasound


Endoscopic retrograde cholangiography


Percutaneous transhepatic cholangiography

CA 19–9:

Carbohydrate antigen 19–9


Isocitrate dehydrogenase 1


AT-rich interaction domain 1A


Fibroblast growth factor receptor 2


Epidermal growth factor receptor


Overall survival


Portal vein embolization


Combined hepatocellular-cholangiocarcinoma


Associating liver partition and portal vein ligation for staged hepatectomy


National Comprehensive Cancer Network


American Joint Committee on Cancer


Orthotopic liver transplantation


Radiofrequency ablation


Microwave ablation


Transarterial chemoembolization


Transarterial radioembolization


Hepatic arterial infusion


Stereotactic body radiation therapy


Recurrence-free survival


Conventional TACE


Photodynamic therapy


Progression free survival


BRCA1-associated protein 1


DNA damage response


The inhibitor poly (adenosine diphosphate-ribose) polymerase




Programmed-death ligand 1


Objective response rate


Papers of particular interest, published recently, have been highlighted as: • Of importance •• Of major importance

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This work was supported by NIDDK K08 (DK122114-01; NR); Gilead Research Scholar Award (NR).

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Correspondence to Nataliya Razumilava MD.

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Kawasaki, H., Akazawa, Y. & Razumilava, N. Progress Toward Improving Outcomes in Patients with Cholangiocarcinoma. Curr Treat Options Gastro 19, 153–168 (2021).

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