New and Emerging Systemic Therapeutic Options for Advanced Cholangiocarcinoma
Cholangiocarcinoma (CCA) represents a disease entity that comprises a heterogeneous group of biliary malignant neoplasms, with variable clinical presentation and severity. It may be classified according to its anatomical location and distinguished in intrahepatic (iCCA), perihilar (pCCA), or distal...
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MDPI AG
2020-03-01
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| Series: | Cells |
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| Online Access: | https://www.mdpi.com/2073-4409/9/3/688 |
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| author | Sara Massironi Lorenzo Pilla Alessandra Elvevi Raffaella Longarini Roberta Elisa Rossi Paolo Bidoli Pietro Invernizzi |
| author_facet | Sara Massironi Lorenzo Pilla Alessandra Elvevi Raffaella Longarini Roberta Elisa Rossi Paolo Bidoli Pietro Invernizzi |
| author_sort | Sara Massironi |
| collection | DOAJ |
| description | Cholangiocarcinoma (CCA) represents a disease entity that comprises a heterogeneous group of biliary malignant neoplasms, with variable clinical presentation and severity. It may be classified according to its anatomical location and distinguished in intrahepatic (iCCA), perihilar (pCCA), or distal (dCCA), each subtype implying distinct epidemiology, biology, prognosis, and strategy for clinical management. Its incidence has increased globally over the past few decades, and its mortality rate remains high due to both its biological aggressiveness and resistance to medical therapy. Surgery is the only potentially curative treatment and is the standard approach for resectable CCA; however, more than half of the patients have locally advanced or metastatic disease at presentation. For patients with unresectable CCA, the available systemic therapies are of limited effectiveness. However, the advances of the comprehension of the complex molecular landscape of CCA and its tumor microenvironment could provide new keys to better understand the pathogenesis, the mechanisms of resistance and ultimately to identify promising new therapeutic targets. Recently, clinical trials targeting isocitrate dehydrogenase (IDH)-1 mutations and fibroblast growth factor receptor (FGFR)-2 fusions, as well as immunotherapy showed promising results. All these new and emerging therapeutic options are herein discussed. |
| first_indexed | 2024-03-12T08:33:50Z |
| format | Article |
| id | doaj.art-6d06316879ad424582dc5836858a9d77 |
| institution | Directory Open Access Journal |
| issn | 2073-4409 |
| language | English |
| last_indexed | 2024-03-12T08:33:50Z |
| publishDate | 2020-03-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Cells |
| spelling | doaj.art-6d06316879ad424582dc5836858a9d772023-09-02T17:26:47ZengMDPI AGCells2073-44092020-03-019368810.3390/cells9030688cells9030688New and Emerging Systemic Therapeutic Options for Advanced CholangiocarcinomaSara Massironi0Lorenzo Pilla1Alessandra Elvevi2Raffaella Longarini3Roberta Elisa Rossi4Paolo Bidoli5Pietro Invernizzi6Division of Gastroenterology, San Gerardo Hospital, University of Milano-Bicocca School of Medicine, 20900 Monza, ItalyDivision of Medical Oncology, San Gerardo Hospital, University of Milano-Bicocca School of Medicine, 20900 Monza, ItalyDivision of Gastroenterology, San Gerardo Hospital, University of Milano-Bicocca School of Medicine, 20900 Monza, ItalyDivision of Medical Oncology, San Gerardo Hospital, University of Milano-Bicocca School of Medicine, 20900 Monza, ItalyGastrointestinal and Hepato-Pancreatic Surgery and Liver Transplantation Unit, Fondazione IRCCS Istituto Nazionale Tumori (INT, National Cancer Institute) - Università degli Studi di Milano, 20100 Milan, ItalyDivision of Medical Oncology, San Gerardo Hospital, University of Milano-Bicocca School of Medicine, 20900 Monza, ItalyDivision of Gastroenterology, San Gerardo Hospital, University of Milano-Bicocca School of Medicine, 20900 Monza, ItalyCholangiocarcinoma (CCA) represents a disease entity that comprises a heterogeneous group of biliary malignant neoplasms, with variable clinical presentation and severity. It may be classified according to its anatomical location and distinguished in intrahepatic (iCCA), perihilar (pCCA), or distal (dCCA), each subtype implying distinct epidemiology, biology, prognosis, and strategy for clinical management. Its incidence has increased globally over the past few decades, and its mortality rate remains high due to both its biological aggressiveness and resistance to medical therapy. Surgery is the only potentially curative treatment and is the standard approach for resectable CCA; however, more than half of the patients have locally advanced or metastatic disease at presentation. For patients with unresectable CCA, the available systemic therapies are of limited effectiveness. However, the advances of the comprehension of the complex molecular landscape of CCA and its tumor microenvironment could provide new keys to better understand the pathogenesis, the mechanisms of resistance and ultimately to identify promising new therapeutic targets. Recently, clinical trials targeting isocitrate dehydrogenase (IDH)-1 mutations and fibroblast growth factor receptor (FGFR)-2 fusions, as well as immunotherapy showed promising results. All these new and emerging therapeutic options are herein discussed.https://www.mdpi.com/2073-4409/9/3/688cholaniocarcinomamolecular landscapetargeted-therapyimmunotherapychemotherapy |
| spellingShingle | Sara Massironi Lorenzo Pilla Alessandra Elvevi Raffaella Longarini Roberta Elisa Rossi Paolo Bidoli Pietro Invernizzi New and Emerging Systemic Therapeutic Options for Advanced Cholangiocarcinoma Cells cholaniocarcinoma molecular landscape targeted-therapy immunotherapy chemotherapy |
| title | New and Emerging Systemic Therapeutic Options for Advanced Cholangiocarcinoma |
| title_full | New and Emerging Systemic Therapeutic Options for Advanced Cholangiocarcinoma |
| title_fullStr | New and Emerging Systemic Therapeutic Options for Advanced Cholangiocarcinoma |
| title_full_unstemmed | New and Emerging Systemic Therapeutic Options for Advanced Cholangiocarcinoma |
| title_short | New and Emerging Systemic Therapeutic Options for Advanced Cholangiocarcinoma |
| title_sort | new and emerging systemic therapeutic options for advanced cholangiocarcinoma |
| topic | cholaniocarcinoma molecular landscape targeted-therapy immunotherapy chemotherapy |
| url | https://www.mdpi.com/2073-4409/9/3/688 |
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