Targeting BRAF-Mutant Biliary Tract Cancer: Recent Advances and Future Challenges.


Journal

Cancer control : journal of the Moffitt Cancer Center
ISSN: 1526-2359
Titre abrégé: Cancer Control
Pays: United States
ID NLM: 9438457

Informations de publication

Date de publication:
Historique:
entrez: 28 12 2020
pubmed: 29 12 2020
medline: 7 9 2021
Statut: ppublish

Résumé

Biliary tract cancers (BTCs) represent a heterogeneous group of aggressive solid tumors with limited therapeutic options, and include gallbladder cancer (GBC), ampulla of Vater cancer (AVC), intrahepatic cholangiocarcinoma (iCCA) and extrahepatic cholangiocarcinoma (eCCA). In the current review, we will discuss recent results of clinical trials testing targeted therapies in BRAF-mutant BTCs, with a particular focus on the recently published Phase II ROAR trial and ongoing active and recruiting clinical trials. Although the extended use of molecular profiling has paved the way toward a new era in BTC management, targeted therapies are limited to iCCA so far, and the prognosis of patients with metastatic disease has substantially not changed in the last decade. In this discouraging scenario, BRAF inhibition is currently emerging as a novel treatment option in patients harboring BRAF mutations.

Sections du résumé

BACKGROUND BACKGROUND
Biliary tract cancers (BTCs) represent a heterogeneous group of aggressive solid tumors with limited therapeutic options, and include gallbladder cancer (GBC), ampulla of Vater cancer (AVC), intrahepatic cholangiocarcinoma (iCCA) and extrahepatic cholangiocarcinoma (eCCA).
METHODS & RESULTS RESULTS
In the current review, we will discuss recent results of clinical trials testing targeted therapies in BRAF-mutant BTCs, with a particular focus on the recently published Phase II ROAR trial and ongoing active and recruiting clinical trials.
CONCLUSIONS CONCLUSIONS
Although the extended use of molecular profiling has paved the way toward a new era in BTC management, targeted therapies are limited to iCCA so far, and the prognosis of patients with metastatic disease has substantially not changed in the last decade. In this discouraging scenario, BRAF inhibition is currently emerging as a novel treatment option in patients harboring BRAF mutations.

Identifiants

pubmed: 33356500
doi: 10.1177/1073274820983013
pmc: PMC8642057
doi:

Substances chimiques

Protein Kinase Inhibitors 0
BRAF protein, human EC 2.7.11.1
Proto-Oncogene Proteins B-raf EC 2.7.11.1

Types de publication

Journal Article Review

Langues

eng

Sous-ensembles de citation

IM

Pagination

1073274820983013

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Auteurs

Alessandro Rizzo (A)

Department of Experimental, Diagnostic and Specialty Medicine, S. Orsola-Malpighi University Hospital, Bologna, Italy.
Division of Oncology, Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italy.

Alessandro Di Federico (AD)

Department of Experimental, Diagnostic and Specialty Medicine, S. Orsola-Malpighi University Hospital, Bologna, Italy.
Division of Oncology, Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italy.

Angela Dalia Ricci (AD)

Department of Experimental, Diagnostic and Specialty Medicine, S. Orsola-Malpighi University Hospital, Bologna, Italy.
Division of Oncology, Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italy.

Giorgio Frega (G)

Department of Experimental, Diagnostic and Specialty Medicine, S. Orsola-Malpighi University Hospital, Bologna, Italy.
Division of Oncology, Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italy.

Andrea Palloni (A)

Department of Experimental, Diagnostic and Specialty Medicine, S. Orsola-Malpighi University Hospital, Bologna, Italy.
Division of Oncology, Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italy.

Rachele Pagani (R)

Department of Experimental, Diagnostic and Specialty Medicine, S. Orsola-Malpighi University Hospital, Bologna, Italy.
Division of Oncology, Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italy.

Simona Tavolari (S)

Division of Oncology, Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italy.
Center of Applied Biomedical Research, S. Orsola-Malpighi University Hospital, Bologna, Italy.

Mariacristina Di Marco (MD)

Department of Experimental, Diagnostic and Specialty Medicine, S. Orsola-Malpighi University Hospital, Bologna, Italy.
Division of Oncology, Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italy.

Giovanni Brandi (G)

Department of Experimental, Diagnostic and Specialty Medicine, S. Orsola-Malpighi University Hospital, Bologna, Italy.
Division of Oncology, Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italy.

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Classifications MeSH