Recent advances in systemic therapy for advanced intrahepatic cholangiocarcinoma

Background The incidence of intrahepatic cholangiocarcinoma (IHCCA) is rising around the world. The disease is becoming a major global health issue. Conventionally, most patients with cholangiocarcinoma present with advanced disease and systemic therapy is the mainstay of treatment. This review disc...

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Main Authors: Changhoon Yoo, Jaewon Hyung, Stephen L. Chan
Format: Article
Language:English
Published: Karger Publishers 2023-06-01
Series:Liver Cancer
Online Access:https://beta.karger.com/Article/FullText/531458
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author Changhoon Yoo
Jaewon Hyung
Stephen L. Chan
author_facet Changhoon Yoo
Jaewon Hyung
Stephen L. Chan
author_sort Changhoon Yoo
collection DOAJ
description Background The incidence of intrahepatic cholangiocarcinoma (IHCCA) is rising around the world. The disease is becoming a major global health issue. Conventionally, most patients with cholangiocarcinoma present with advanced disease and systemic therapy is the mainstay of treatment. This review discusses recent advances in systemic treatments for patients with IHCCA. Summary The addition of durvalumab to a gemcitabine plus cisplatin (GemCis) regimen has significantly improved overall survival (OS) in the phase 3 TOPAZ-1 trial and is currently recommended as a standard first-line treatment. The phase 3 ABC-06 and phase 2b NIFTY trials have shown the benefit of second-line fluoropyrimidine plus oxaliplatin, and fluoropyrimidine plus nanoliposomal irinotecan, respectively. They have provided a treatment option for patients without actionable alterations who progressed to first-line therapy. For patients with actionable genomic alterations, including FGFR2 rearrangement, IDH1 mutation, BRAF mutation, and ERBB2 amplification, targeted agents have shown encouraging efficacy in several phase 2-3 trials, and are recommended as subsequent treatments. Immune checkpoint inhibitors are being investigated for the treatment of previously treated patients, although only a small proportion of patients showed durable responses. Key Messages Recent advances in systemic treatments have improved clinical outcomes in patients with advanced IHCCA. However, most patients eventually show resistance to the treatment and tumor progression occurs within a year. Indeed, there should be further efforts to improve the outcomes of patients with advanced IHCCA.
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spelling doaj.art-f6244b003b5e423abb3f32c392c179f02023-07-06T12:16:43ZengKarger PublishersLiver Cancer2235-17951664-55532023-06-011110.1159/000531458531458Recent advances in systemic therapy for advanced intrahepatic cholangiocarcinomaChanghoon Yoohttps://orcid.org/0000-0002-1451-8455Jaewon HyungStephen L. Chanhttps://orcid.org/0000-0001-8998-5480Background The incidence of intrahepatic cholangiocarcinoma (IHCCA) is rising around the world. The disease is becoming a major global health issue. Conventionally, most patients with cholangiocarcinoma present with advanced disease and systemic therapy is the mainstay of treatment. This review discusses recent advances in systemic treatments for patients with IHCCA. Summary The addition of durvalumab to a gemcitabine plus cisplatin (GemCis) regimen has significantly improved overall survival (OS) in the phase 3 TOPAZ-1 trial and is currently recommended as a standard first-line treatment. The phase 3 ABC-06 and phase 2b NIFTY trials have shown the benefit of second-line fluoropyrimidine plus oxaliplatin, and fluoropyrimidine plus nanoliposomal irinotecan, respectively. They have provided a treatment option for patients without actionable alterations who progressed to first-line therapy. For patients with actionable genomic alterations, including FGFR2 rearrangement, IDH1 mutation, BRAF mutation, and ERBB2 amplification, targeted agents have shown encouraging efficacy in several phase 2-3 trials, and are recommended as subsequent treatments. Immune checkpoint inhibitors are being investigated for the treatment of previously treated patients, although only a small proportion of patients showed durable responses. Key Messages Recent advances in systemic treatments have improved clinical outcomes in patients with advanced IHCCA. However, most patients eventually show resistance to the treatment and tumor progression occurs within a year. Indeed, there should be further efforts to improve the outcomes of patients with advanced IHCCA.https://beta.karger.com/Article/FullText/531458
spellingShingle Changhoon Yoo
Jaewon Hyung
Stephen L. Chan
Recent advances in systemic therapy for advanced intrahepatic cholangiocarcinoma
Liver Cancer
title Recent advances in systemic therapy for advanced intrahepatic cholangiocarcinoma
title_full Recent advances in systemic therapy for advanced intrahepatic cholangiocarcinoma
title_fullStr Recent advances in systemic therapy for advanced intrahepatic cholangiocarcinoma
title_full_unstemmed Recent advances in systemic therapy for advanced intrahepatic cholangiocarcinoma
title_short Recent advances in systemic therapy for advanced intrahepatic cholangiocarcinoma
title_sort recent advances in systemic therapy for advanced intrahepatic cholangiocarcinoma
url https://beta.karger.com/Article/FullText/531458
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