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Refractory Bile Duct Carcinoma

MedGen UID:
1652770
•Concept ID:
C4727192
•
Neoplastic Process

Definition

Bile duct carcinoma that is resistant to treatment. [from NCI]

Professional guidelines

PubMed

Practical review for diagnosis and clinical management of perihilar cholangiocarcinoma.
Dondossola D, Ghidini M, Grossi F, Rossi G, Foschi D
World J Gastroenterol 2020 Jul 7;26(25):3542-3561. doi: 10.3748/wjg.v26.i25.3542. PMID: 32742125Free PMC Article
Cholangiocarcinoma 2020: the next horizon in mechanisms and management.
Banales JM, Marin JJG, Lamarca A, Rodrigues PM, Khan SA, Roberts LR, Cardinale V, Carpino G, Andersen JB, Braconi C, Calvisi DF, Perugorria MJ, Fabris L, Boulter L, Macias RIR, Gaudio E, Alvaro D, Gradilone SA, Strazzabosco M, Marzioni M, Coulouarn C, Fouassier L, Raggi C, Invernizzi P, Mertens JC, Moncsek A, Rizvi S, Heimbach J, Koerkamp BG, Bruix J, Forner A, Bridgewater J, Valle JW, Gores GJ
Nat Rev Gastroenterol Hepatol 2020 Sep;17(9):557-588. Epub 2020 Jun 30 doi: 10.1038/s41575-020-0310-z. PMID: 32606456Free PMC Article
Expert consensus document: Cholangiocarcinoma: current knowledge and future perspectives consensus statement from the European Network for the Study of Cholangiocarcinoma (ENS-CCA).
Banales JM, Cardinale V, Carpino G, Marzioni M, Andersen JB, Invernizzi P, Lind GE, Folseraas T, Forbes SJ, Fouassier L, Geier A, Calvisi DF, Mertens JC, Trauner M, Benedetti A, Maroni L, Vaquero J, Macias RI, Raggi C, Perugorria MJ, Gaudio E, Boberg KM, Marin JJ, Alvaro D
Nat Rev Gastroenterol Hepatol 2016 May;13(5):261-80. Epub 2016 Apr 20 doi: 10.1038/nrgastro.2016.51. PMID: 27095655

Recent clinical studies

Etiology

Zanidatamab for HER2-amplified, unresectable, locally advanced or metastatic biliary tract cancer (HERIZON-BTC-01): a multicentre, single-arm, phase 2b study.
Harding JJ, Fan J, Oh DY, Choi HJ, Kim JW, Chang HM, Bao L, Sun HC, Macarulla T, Xie F, Metges JP, Ying J, Bridgewater J, Lee MA, Tejani MA, Chen EY, Kim DU, Wasan H, Ducreux M, Bao Y, Boyken L, Ma J, Garfin P, Pant S; HERIZON-BTC-01 study group
Lancet Oncol 2023 Jul;24(7):772-782. Epub 2023 Jun 2 doi: 10.1016/S1470-2045(23)00242-5. PMID: 37276871
Targeted therapies in advanced biliary tract cancers-a narrative review.
LaPelusa M, Heumann T, Goff L, Agarwal R
Chin Clin Oncol 2023 Apr;12(2):14. Epub 2023 Mar 13 doi: 10.21037/cco-22-93. PMID: 36946186Free PMC Article
Trastuzumab plus FOLFOX for HER2-positive biliary tract cancer refractory to gemcitabine and cisplatin: a multi-institutional phase 2 trial of the Korean Cancer Study Group (KCSG-HB19-14).
Lee CK, Chon HJ, Cheon J, Lee MA, Im HS, Jang JS, Kim MH, Park S, Kang B, Hong M, Kim JW, Park HS, Kang MJ, Park YN, Choi HJ
Lancet Gastroenterol Hepatol 2023 Jan;8(1):56-65. Epub 2022 Oct 31 doi: 10.1016/S2468-1253(22)00335-1. PMID: 36328033
Ivosidenib in IDH1-mutant, chemotherapy-refractory cholangiocarcinoma (ClarIDHy): a multicentre, randomised, double-blind, placebo-controlled, phase 3 study.
Abou-Alfa GK, Macarulla T, Javle MM, Kelley RK, Lubner SJ, Adeva J, Cleary JM, Catenacci DV, Borad MJ, Bridgewater J, Harris WP, Murphy AG, Oh DY, Whisenant J, Lowery MA, Goyal L, Shroff RT, El-Khoueiry AB, Fan B, Wu B, Chamberlain CX, Jiang L, Gliser C, Pandya SS, Valle JW, Zhu AX
Lancet Oncol 2020 Jun;21(6):796-807. Epub 2020 May 13 doi: 10.1016/S1470-2045(20)30157-1. PMID: 32416072Free PMC Article
Expert consensus document: Cholangiocarcinoma: current knowledge and future perspectives consensus statement from the European Network for the Study of Cholangiocarcinoma (ENS-CCA).
Banales JM, Cardinale V, Carpino G, Marzioni M, Andersen JB, Invernizzi P, Lind GE, Folseraas T, Forbes SJ, Fouassier L, Geier A, Calvisi DF, Mertens JC, Trauner M, Benedetti A, Maroni L, Vaquero J, Macias RI, Raggi C, Perugorria MJ, Gaudio E, Boberg KM, Marin JJ, Alvaro D
Nat Rev Gastroenterol Hepatol 2016 May;13(5):261-80. Epub 2016 Apr 20 doi: 10.1038/nrgastro.2016.51. PMID: 27095655

Diagnosis

Intrahepatic cholangiocarcinoma: Evolving strategies in management and treatment.
Bragazzi MC, Venere R, Ribichini E, Covotta F, Cardinale V, Alvaro D
Dig Liver Dis 2024 Mar;56(3):383-393. Epub 2023 Sep 16 doi: 10.1016/j.dld.2023.08.052. PMID: 37722960
Practical review for diagnosis and clinical management of perihilar cholangiocarcinoma.
Dondossola D, Ghidini M, Grossi F, Rossi G, Foschi D
World J Gastroenterol 2020 Jul 7;26(25):3542-3561. doi: 10.3748/wjg.v26.i25.3542. PMID: 32742125Free PMC Article
Cholangiocarcinoma 2020: the next horizon in mechanisms and management.
Banales JM, Marin JJG, Lamarca A, Rodrigues PM, Khan SA, Roberts LR, Cardinale V, Carpino G, Andersen JB, Braconi C, Calvisi DF, Perugorria MJ, Fabris L, Boulter L, Macias RIR, Gaudio E, Alvaro D, Gradilone SA, Strazzabosco M, Marzioni M, Coulouarn C, Fouassier L, Raggi C, Invernizzi P, Mertens JC, Moncsek A, Rizvi S, Heimbach J, Koerkamp BG, Bruix J, Forner A, Bridgewater J, Valle JW, Gores GJ
Nat Rev Gastroenterol Hepatol 2020 Sep;17(9):557-588. Epub 2020 Jun 30 doi: 10.1038/s41575-020-0310-z. PMID: 32606456Free PMC Article
Expert consensus document: Cholangiocarcinoma: current knowledge and future perspectives consensus statement from the European Network for the Study of Cholangiocarcinoma (ENS-CCA).
Banales JM, Cardinale V, Carpino G, Marzioni M, Andersen JB, Invernizzi P, Lind GE, Folseraas T, Forbes SJ, Fouassier L, Geier A, Calvisi DF, Mertens JC, Trauner M, Benedetti A, Maroni L, Vaquero J, Macias RI, Raggi C, Perugorria MJ, Gaudio E, Boberg KM, Marin JJ, Alvaro D
Nat Rev Gastroenterol Hepatol 2016 May;13(5):261-80. Epub 2016 Apr 20 doi: 10.1038/nrgastro.2016.51. PMID: 27095655
Genomic and transcriptional alterations of cholangiocarcinoma.
Ito T, Sakurai-Yageta M, Goto A, Pairojkul C, Yongvanit P, Murakami Y
J Hepatobiliary Pancreat Sci 2014 Jun;21(6):380-7. Epub 2014 Feb 13 doi: 10.1002/jhbp.67. PMID: 24532422

Therapy

Molecular-based targeted therapies in patients with hepatocellular carcinoma and hepato-cholangiocarcinoma refractory to atezolizumab/bevacizumab.
Limousin W, Laurent-Puig P, Ziol M, Ganne-Carrié N, Nahon P, Ait-Omar A, Seror O, Sidali S, Campani C, Blanc P, Lermine A, Marisa L, Zucman-Rossi J, Nault JC
J Hepatol 2023 Dec;79(6):1450-1458. Epub 2023 Aug 28 doi: 10.1016/j.jhep.2023.08.017. PMID: 37647991
FIGHT-101, a first-in-human study of potent and selective FGFR 1-3 inhibitor pemigatinib in pan-cancer patients with FGF/FGFR alterations and advanced malignancies.
Subbiah V, Iannotti NO, Gutierrez M, Smith DC, Féliz L, Lihou CF, Tian C, Silverman IM, Ji T, Saleh M
Ann Oncol 2022 May;33(5):522-533. Epub 2022 Feb 14 doi: 10.1016/j.annonc.2022.02.001. PMID: 35176457
CAFs shape myeloid-derived suppressor cells to promote stemness of intrahepatic cholangiocarcinoma through 5-lipoxygenase.
Lin Y, Cai Q, Chen Y, Shi T, Liu W, Mao L, Deng B, Ying Z, Gao Y, Luo H, Yang X, Huang X, Shi Y, He R
Hepatology 2022 Jan;75(1):28-42. Epub 2021 Dec 5 doi: 10.1002/hep.32099. PMID: 34387870
Ivosidenib in IDH1-mutant, chemotherapy-refractory cholangiocarcinoma (ClarIDHy): a multicentre, randomised, double-blind, placebo-controlled, phase 3 study.
Abou-Alfa GK, Macarulla T, Javle MM, Kelley RK, Lubner SJ, Adeva J, Cleary JM, Catenacci DV, Borad MJ, Bridgewater J, Harris WP, Murphy AG, Oh DY, Whisenant J, Lowery MA, Goyal L, Shroff RT, El-Khoueiry AB, Fan B, Wu B, Chamberlain CX, Jiang L, Gliser C, Pandya SS, Valle JW, Zhu AX
Lancet Oncol 2020 Jun;21(6):796-807. Epub 2020 May 13 doi: 10.1016/S1470-2045(20)30157-1. PMID: 32416072Free PMC Article
Expert consensus document: Cholangiocarcinoma: current knowledge and future perspectives consensus statement from the European Network for the Study of Cholangiocarcinoma (ENS-CCA).
Banales JM, Cardinale V, Carpino G, Marzioni M, Andersen JB, Invernizzi P, Lind GE, Folseraas T, Forbes SJ, Fouassier L, Geier A, Calvisi DF, Mertens JC, Trauner M, Benedetti A, Maroni L, Vaquero J, Macias RI, Raggi C, Perugorria MJ, Gaudio E, Boberg KM, Marin JJ, Alvaro D
Nat Rev Gastroenterol Hepatol 2016 May;13(5):261-80. Epub 2016 Apr 20 doi: 10.1038/nrgastro.2016.51. PMID: 27095655

Prognosis

Targeted therapies in advanced biliary tract cancers-a narrative review.
LaPelusa M, Heumann T, Goff L, Agarwal R
Chin Clin Oncol 2023 Apr;12(2):14. Epub 2023 Mar 13 doi: 10.21037/cco-22-93. PMID: 36946186Free PMC Article
Vanishing bile duct syndrome following pembrolizumab infusion: case report and review of the literature.
Gemelli M, Carbone M, Abbate MI, Mancin M, Zucchini N, Colonese F, Invernizzi P, Bidoli P, Cortinovis D
Immunotherapy 2022 Mar;14(4):175-181. Epub 2021 Dec 7 doi: 10.2217/imt-2021-0078. PMID: 34873918
Mutational spectrum and precision oncology for biliary tract carcinoma.
Lin J, Cao Y, Yang X, Li G, Shi Y, Wang D, Long J, Song Y, Mao J, Xie F, Bai Y, Zhang L, Yang X, Wan X, Wang A, Guan M, Zhao L, Hu K, Pan J, Huo L, Lu X, Mao Y, Sang X, Zhang H, Wang K, Wang X, Zhao H
Theranostics 2021;11(10):4585-4598. Epub 2021 Mar 4 doi: 10.7150/thno.56539. PMID: 33754015Free PMC Article
The role of photodynamic therapy for hilar cholangiocarcinoma.
Cheon YK
Korean J Intern Med 2010 Dec;25(4):345-52. Epub 2010 Nov 27 doi: 10.3904/kjim.2010.25.4.345. PMID: 21179270Free PMC Article
Photodynamic therapy for cholangiocarcinoma.
Berr F
Semin Liver Dis 2004 May;24(2):177-87. doi: 10.1055/s-2004-828894. PMID: 15192790

Clinical prediction guides

Molecular-based targeted therapies in patients with hepatocellular carcinoma and hepato-cholangiocarcinoma refractory to atezolizumab/bevacizumab.
Limousin W, Laurent-Puig P, Ziol M, Ganne-Carrié N, Nahon P, Ait-Omar A, Seror O, Sidali S, Campani C, Blanc P, Lermine A, Marisa L, Zucman-Rossi J, Nault JC
J Hepatol 2023 Dec;79(6):1450-1458. Epub 2023 Aug 28 doi: 10.1016/j.jhep.2023.08.017. PMID: 37647991
Zanidatamab for HER2-amplified, unresectable, locally advanced or metastatic biliary tract cancer (HERIZON-BTC-01): a multicentre, single-arm, phase 2b study.
Harding JJ, Fan J, Oh DY, Choi HJ, Kim JW, Chang HM, Bao L, Sun HC, Macarulla T, Xie F, Metges JP, Ying J, Bridgewater J, Lee MA, Tejani MA, Chen EY, Kim DU, Wasan H, Ducreux M, Bao Y, Boyken L, Ma J, Garfin P, Pant S; HERIZON-BTC-01 study group
Lancet Oncol 2023 Jul;24(7):772-782. Epub 2023 Jun 2 doi: 10.1016/S1470-2045(23)00242-5. PMID: 37276871
Trastuzumab plus FOLFOX for HER2-positive biliary tract cancer refractory to gemcitabine and cisplatin: a multi-institutional phase 2 trial of the Korean Cancer Study Group (KCSG-HB19-14).
Lee CK, Chon HJ, Cheon J, Lee MA, Im HS, Jang JS, Kim MH, Park S, Kang B, Hong M, Kim JW, Park HS, Kang MJ, Park YN, Choi HJ
Lancet Gastroenterol Hepatol 2023 Jan;8(1):56-65. Epub 2022 Oct 31 doi: 10.1016/S2468-1253(22)00335-1. PMID: 36328033
Mutational spectrum and precision oncology for biliary tract carcinoma.
Lin J, Cao Y, Yang X, Li G, Shi Y, Wang D, Long J, Song Y, Mao J, Xie F, Bai Y, Zhang L, Yang X, Wan X, Wang A, Guan M, Zhao L, Hu K, Pan J, Huo L, Lu X, Mao Y, Sang X, Zhang H, Wang K, Wang X, Zhao H
Theranostics 2021;11(10):4585-4598. Epub 2021 Mar 4 doi: 10.7150/thno.56539. PMID: 33754015Free PMC Article
Ivosidenib in IDH1-mutant, chemotherapy-refractory cholangiocarcinoma (ClarIDHy): a multicentre, randomised, double-blind, placebo-controlled, phase 3 study.
Abou-Alfa GK, Macarulla T, Javle MM, Kelley RK, Lubner SJ, Adeva J, Cleary JM, Catenacci DV, Borad MJ, Bridgewater J, Harris WP, Murphy AG, Oh DY, Whisenant J, Lowery MA, Goyal L, Shroff RT, El-Khoueiry AB, Fan B, Wu B, Chamberlain CX, Jiang L, Gliser C, Pandya SS, Valle JW, Zhu AX
Lancet Oncol 2020 Jun;21(6):796-807. Epub 2020 May 13 doi: 10.1016/S1470-2045(20)30157-1. PMID: 32416072Free PMC Article

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