A review of molecularly targeted therapy in biliary tract carcinoma: what is the next step?

Patients with unresectable biliary tract carcinomas (BTCs) have a poor prognosis with a median overall survival of fewer than 12 months following systemic chemotherapy. In recent years, the identification of distinct molecular alterations with corresponding targeted therapies is modifying this thera...

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Autores principales:	Giacomo Aimar, Chiara Paratore, Clizia Zichi, Donatella Marino, Elisa Sperti, Andrea Caglio, Teresa Gamba, Francesca De Vita, Massimo Di Maio
Formato:	article
Lenguaje:	EN
Publicado:	Open Exploration Publishing Inc. 2021
Materias:	biliary tract carcinomas target therapy molecular alterations isocitrate dehydrogenase fibroblast growth factor receptor human epidermal growth factor receptor 2 multitarget therapy Internal medicine RC31-1245
Acceso en línea:	https://doaj.org/article/46affa1a81044e6ca6b2213bb2362c97
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spelling	oai:doaj.org-article:46affa1a81044e6ca6b2213bb2362c972021-11-08T09:58:50ZA review of molecularly targeted therapy in biliary tract carcinoma: what is the next step?10.37349/etat.2021.000562692-3114https://doaj.org/article/46affa1a81044e6ca6b2213bb2362c972021-10-01T00:00:00Zhttps://www.explorationpub.com/Journals/etat/Article/100256https://doaj.org/toc/2692-3114Patients with unresectable biliary tract carcinomas (BTCs) have a poor prognosis with a median overall survival of fewer than 12 months following systemic chemotherapy. In recent years, the identification of distinct molecular alterations with corresponding targeted therapies is modifying this therapeutic algorithm. The aim of this review is to present an overview of targeted therapy for BTCs, describing published available data and potential future challenges in ongoing trials. From clinicaltrials.gov online database all ongoing trials for BTCs (any stage) was examinated in July 2021, and data regarding study design, disease characteristics and type of treatments were registered. Oncogenic-driven therapy (targeted therapy) was investigated in 67 trials. According to research, 15 ongoing trials (22.4%) are investigating fibroblast growth factor (FGF) receptor (FGFR)-inhibitors in BTCs. Three (18.7%) are open-label randomized multicenter phase 3 trials, 8 (50%) are single-arm phase two trials, and 4 (25%) are phase one studies. Twelve (17.9%) clinical trials dealt with isocitrate dehydrogenase (IDH) 1/2 targeting therapy either in combination with cisplatin (Cis) and gemcitabine (Gem) as first-line treatment for BTCs or in monotherapy in patients with IDH1 mutant advanced malignancies, including cholangiocarcinoma (CCA). Nine (13.4%) clinical trials tested human epidermal growth factor receptor (HER) 2 targeting therapy. Four (44.4%) studies are phase I trials, two (22.2%) are phase I/II trials, and three (33.3%) phase II trials. Rare molecular alterations in BTCs, such as anaplastic lymphoma kinase (ALK), c-ros oncogene1 receptor tyrosine kinase (ROS1), and v-RAF murine sarcoma viral oncogene homologue B1 (BRAF), are also under investigation in a few trials. Forty-four clinical trials (17.2%) are investigating not oncogenic-driven multitarget therapy like multireceptor tyrosin kinase inhibitors and antiangiogenetic agents. In conclusion, this review shows that BTCs management is experiencing important innovations, especially in biomarker-based patient selection and in the new emerging therapeutic approach. Many ongoing trials could answer questions regarding the role of molecular inhibitors leading to new therapeutic frontiers for molecular subcategories of BTCs.Giacomo AimarChiara ParatoreClizia ZichiDonatella MarinoElisa SpertiAndrea CaglioTeresa GambaFrancesca De VitaMassimo Di MaioOpen Exploration Publishing Inc.articlebiliary tract carcinomastarget therapymolecular alterationsisocitrate dehydrogenasefibroblast growth factor receptorhuman epidermal growth factor receptor 2multitarget therapyInternal medicineRC31-1245ENExploration of Targeted Anti-tumor Therapy, Vol 2, Iss 5, Pp 448-464 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	biliary tract carcinomas target therapy molecular alterations isocitrate dehydrogenase fibroblast growth factor receptor human epidermal growth factor receptor 2 multitarget therapy Internal medicine RC31-1245
spellingShingle	biliary tract carcinomas target therapy molecular alterations isocitrate dehydrogenase fibroblast growth factor receptor human epidermal growth factor receptor 2 multitarget therapy Internal medicine RC31-1245 Giacomo Aimar Chiara Paratore Clizia Zichi Donatella Marino Elisa Sperti Andrea Caglio Teresa Gamba Francesca De Vita Massimo Di Maio A review of molecularly targeted therapy in biliary tract carcinoma: what is the next step?
description	Patients with unresectable biliary tract carcinomas (BTCs) have a poor prognosis with a median overall survival of fewer than 12 months following systemic chemotherapy. In recent years, the identification of distinct molecular alterations with corresponding targeted therapies is modifying this therapeutic algorithm. The aim of this review is to present an overview of targeted therapy for BTCs, describing published available data and potential future challenges in ongoing trials. From clinicaltrials.gov online database all ongoing trials for BTCs (any stage) was examinated in July 2021, and data regarding study design, disease characteristics and type of treatments were registered. Oncogenic-driven therapy (targeted therapy) was investigated in 67 trials. According to research, 15 ongoing trials (22.4%) are investigating fibroblast growth factor (FGF) receptor (FGFR)-inhibitors in BTCs. Three (18.7%) are open-label randomized multicenter phase 3 trials, 8 (50%) are single-arm phase two trials, and 4 (25%) are phase one studies. Twelve (17.9%) clinical trials dealt with isocitrate dehydrogenase (IDH) 1/2 targeting therapy either in combination with cisplatin (Cis) and gemcitabine (Gem) as first-line treatment for BTCs or in monotherapy in patients with IDH1 mutant advanced malignancies, including cholangiocarcinoma (CCA). Nine (13.4%) clinical trials tested human epidermal growth factor receptor (HER) 2 targeting therapy. Four (44.4%) studies are phase I trials, two (22.2%) are phase I/II trials, and three (33.3%) phase II trials. Rare molecular alterations in BTCs, such as anaplastic lymphoma kinase (ALK), c-ros oncogene1 receptor tyrosine kinase (ROS1), and v-RAF murine sarcoma viral oncogene homologue B1 (BRAF), are also under investigation in a few trials. Forty-four clinical trials (17.2%) are investigating not oncogenic-driven multitarget therapy like multireceptor tyrosin kinase inhibitors and antiangiogenetic agents. In conclusion, this review shows that BTCs management is experiencing important innovations, especially in biomarker-based patient selection and in the new emerging therapeutic approach. Many ongoing trials could answer questions regarding the role of molecular inhibitors leading to new therapeutic frontiers for molecular subcategories of BTCs.
format	article
author	Giacomo Aimar Chiara Paratore Clizia Zichi Donatella Marino Elisa Sperti Andrea Caglio Teresa Gamba Francesca De Vita Massimo Di Maio
author_facet	Giacomo Aimar Chiara Paratore Clizia Zichi Donatella Marino Elisa Sperti Andrea Caglio Teresa Gamba Francesca De Vita Massimo Di Maio
author_sort	Giacomo Aimar
title	A review of molecularly targeted therapy in biliary tract carcinoma: what is the next step?
title_short	A review of molecularly targeted therapy in biliary tract carcinoma: what is the next step?
title_full	A review of molecularly targeted therapy in biliary tract carcinoma: what is the next step?
title_fullStr	A review of molecularly targeted therapy in biliary tract carcinoma: what is the next step?
title_full_unstemmed	A review of molecularly targeted therapy in biliary tract carcinoma: what is the next step?
title_sort	review of molecularly targeted therapy in biliary tract carcinoma: what is the next step?
publisher	Open Exploration Publishing Inc.
publishDate	2021
url	https://doaj.org/article/46affa1a81044e6ca6b2213bb2362c97
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A review of molecularly targeted therapy in biliary tract carcinoma: what is the next step?

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