Molecular Features and Targeted Therapies in Cholangiocarcinoma

A special issue of Medicina (ISSN 1648-9144). This special issue belongs to the section "Oncology".

Deadline for manuscript submissions: closed (15 July 2021) | Viewed by 6760

Special Issue Editor


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Guest Editor
I.R.C.C.S. Istituto Tumori “Giovanni Paolo II”, Viale Orazio Flacco 65, 70124 Bari, Italy
Interests: biliary tract cancer; cholangiocarcinoma; gastric cancer; renal cell carcinoma; urothelial carcinoma; uterine leiomyosarcoma; prostate cancer
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Special Issue Information

Dear Colleagues,

Biliary tract cancer (BTC) represents the second most common primary liver tumor accounting for approximately 10–15% of all hepatobiliary malignancies and 3% of all gastrointestinal neoplasms. BTCs include a heterogeneous group of malignancies usually divided into intrahepatic cholangiocarcinoma, extrahepatic cholangiocarcinoma, gallbladder cancer, and ampulla of Vater cancer, according to anatomical location. Although traditionally considered rare tumors in Western countries, their incidence and mortality rate are on the whole rising worldwide. In particular, the incidence of intrahepatic cholangiocarcinoma is expected to further increase in the near future. 

Currently, radical surgery with microscopically negative resection margins is the only potentially curative therapy available, although most patients are diagnosed in late disease stages (locally advanced/unresectable or metastatic). Moreover, even after complete surgical resection, the recurrence rate is high, and the 5-year overall survival rate remains discouraging (20–35% at 5 years). As a result of the increasing availability of genomic sequencing data, many signaling pathways and new genetic aberrations involved in the carcinogenesis of BTC have recently been delineated, and IDH1 mutations and FGFR2 fusions have been positioned as the two main driver alterations in intrahepatic cholangiocarcinoma and are being actively explored with specific antitargeted agents. However, many other alterations as NTRK rearrangements or BRAF mutations are also emerging as new potential targets in BTC. On the other hand, the role of immunotherapy in BTC is currently under investigation, and checkpoint inhibitors are still looking for their niche in BTC. 

In this Special Issue, experts in this field will review the current targeted approaches to the management of patients with the spectrum of BTC.

Dr. Alessandro Rizzo
Guest Editor

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Keywords

  • intrahepatic cholangiocarcinoma
  • FGFR
  • IDH
  • gallbladder cancer
  • extrahepatic cholangiocarcinoma
  • pemigatinib
  • HER2
  • NTRK
  • BRAF

Published Papers (3 papers)

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Editorial

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3 pages, 245 KiB  
Editorial
Novel Targeted Therapies for Advanced Cholangiocarcinoma
by Alessandro Rizzo and Giovanni Brandi
Medicina 2021, 57(3), 212; https://doi.org/10.3390/medicina57030212 - 26 Feb 2021
Cited by 4 | Viewed by 1775
Abstract
Cholangiocarcinoma (CCA) includes a group of rare and aggressive hepatobiliary malignancies, including extrahepatic cholangiocarcinoma (eCCA) and intrahepatic cholangiocarcinoma (iCCA), with the former further subdivided into distal (dCCA) and perihilar cholangiocarcinoma (pCCA) [...] Full article
(This article belongs to the Special Issue Molecular Features and Targeted Therapies in Cholangiocarcinoma)

Review

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13 pages, 996 KiB  
Review
Targeted Therapies in Advanced Cholangiocarcinoma: A Focus on FGFR Inhibitors
by Alessandro Rizzo
Medicina 2021, 57(5), 458; https://doi.org/10.3390/medicina57050458 - 8 May 2021
Cited by 9 | Viewed by 3225
Abstract
Despite advanced diseases continuing to be associated with grim prognoses, the past decade has witnessed the advent of several novel treatment options for cholangiocarcinoma (CCA) patients. In fact, CCA has emerged as a heterogeneous group of malignancies harboring potentially druggable mutations in approximately [...] Read more.
Despite advanced diseases continuing to be associated with grim prognoses, the past decade has witnessed the advent of several novel treatment options for cholangiocarcinoma (CCA) patients. In fact, CCA has emerged as a heterogeneous group of malignancies harboring potentially druggable mutations in approximately 50% of cases, and thus, molecularly targeted therapies have been actively explored in this setting. Among these, fibroblast growth factor receptor (FGFR) inhibitors have reported important results, as witnessed by the FDA approval of pemigatinib in previously treated metastatic CCA patients harboring FGFR2 fusion or other rearrangements. Herein, we provide an overview of available evidence on FGFR inhibitors in CCA, especially focusing on the development, pitfalls and challenges of emerging treatments in this setting. Full article
(This article belongs to the Special Issue Molecular Features and Targeted Therapies in Cholangiocarcinoma)
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3 pages, 238 KiB  
Commentary
Dual HER2 Blockade: An Emerging Option in Metastatic Biliary Tract Cancer?
by Angela Dalia Ricci and Alessandro Rizzo
Medicina 2021, 57(12), 1301; https://doi.org/10.3390/medicina57121301 - 27 Nov 2021
Cited by 1 | Viewed by 1167
Abstract
Biliary tract cancer (BTC) includes a heterogeneous group of aggressive and rare hepatobiliary malignancies, including gallbladder cancer, ampullary carcinomas, intrahepatic cholangiocarcinoma (iCCA), and extrahepatic cholangiocarcinoma, further subclassified into distal (dCCA) and perihilar cholangiocarcinoma (pCCA) [...] Full article
(This article belongs to the Special Issue Molecular Features and Targeted Therapies in Cholangiocarcinoma)
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