Progress Toward Improving Outcomes in Patients with Cholangiocarcinoma

Abstract

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.

Summary

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

Abbreviations

CCA:

Cholangiocarcinomas

iCCA:

Intrahepatic CCA

pCCA:

Perihilar CCA

dCCA:

Distal CCA

BTC:

Biliary tract cancers

PSC:

Primary sclerosing cholangitis

HCC:

Hepatocellular carcinoma

CT:

Computed tomography

MRI:

Magnetic resonance imaging

EUS:

Endoscopic ultrasound

ERC:

Endoscopic retrograde cholangiography

PTC:

Percutaneous transhepatic cholangiography

CA 19–9:

Carbohydrate antigen 19–9

IDH1:

Isocitrate dehydrogenase 1

ARID1A:

AT-rich interaction domain 1A

FGFR2:

Fibroblast growth factor receptor 2

EGFR:

Epidermal growth factor receptor

OS:

Overall survival

PVE:

Portal vein embolization

cHCC-CCA:

Combined hepatocellular-cholangiocarcinoma

ALPPS:

Associating liver partition and portal vein ligation for staged hepatectomy

NCCN:

National Comprehensive Cancer Network

AJCC:

American Joint Committee on Cancer

OLT:

Orthotopic liver transplantation

RFA:

Radiofrequency ablation

MWA:

Microwave ablation

TACE:

Transarterial chemoembolization

TARE:

Transarterial radioembolization

HAI:

Hepatic arterial infusion

SBRT:

Stereotactic body radiation therapy

RFS:

Recurrence-free survival

cTACE:

Conventional TACE

PDT:

Photodynamic therapy

PFS:

Progression free survival

BAP1:

BRCA1-associated protein 1

DDR:

DNA damage response

PARP:

The inhibitor poly (adenosine diphosphate-ribose) polymerase

PD-1:

Programmed-death-1

PD-L1:

Programmed-death ligand 1

ORR:

Objective response rate

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Funding

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 (2021). https://doi.org/10.1007/s11938-021-00333-2

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Keywords

  • Personalized medicine
  • Biliary cancer
  • Immunotherapy
  • Liver transplantation
  • FGFR2
  • IDH1/2