New systemic treatment paradigms in advanced biliary tract cancer and variations in patient access across Europe

被引：5

作者：

Rimassa, Lorenza ^{[1
,2
]}

Lamarca, Angela ^{[3
]}

O'Kane, Grainne M. ^{[4
,5
]}

Edeline, Julien ^{[6
]}

Mcnamara, Mairead G. ^{[7
]}

Vogel, Arndt ^{[8
,9
,10
]}

Fassan, Matteo ^{[11
,12
]}

Forner, Alejandro ^{[13
,14
]}

Kendall, Timothy ^{[15
,16
,17
]}

Adeva, Jorge ^{[18
]}

Casadei-Gardini, Andrea ^{[19
]}

Fornaro, Lorenzo ^{[20
]}

Hollebecque, Antoine ^{[21
]}

Lowery, Maeve A. ^{[22
]}

Macarulla, Teresa ^{[23
]}

Malka, David ^{[24
]}

Mariamidze, Elene ^{[25
]}

Niger, Monica ^{[26
]}

Ustav, Anu ^{[27
]}

Bridgewater, John ^{[28
]}

Macias, Rocio I. R. ^{[14
,29
]}

Braconi, Chiara ^{[17
,30
,31
]}

机构：

[1] Humanitas Univ, Dept Biomed Sci, Via Rita Levi Montalcini 4, I-20072 Milan, Italy

[2] IRCCS Humanitas Res Hosp, Humanitas Canc Ctr, Med Oncol & Hematol Unit, Via A Manzoni 56, I-20089 Milan, Italy

[3] Hosp Univ Fdn Jimenez Diaz, Inst Invest Sanitaria, Oncohlth Inst, Dept Med Oncol,Fdn Jimenez Diaz, Avda Reyes Catolicos 2, E-28040 Madrid, Spain

[4] Univ Coll Dublin, Dublin, Ireland

[5] St Vincents Univ Hosp, Dept Med Oncol, Elm Pk, Dublin, Ireland

[6] Univ Rennes, INSERM, COSS Chem Oncogenesis Stress Signaling UMR S 1242, CLCC Eugene Marquis, F-35000 Rennes, France

[7] Christie NHS Fdn Trust, Dept Med Oncol, Manchester M20 4BX, England

[8] UHN, Toronto Gen Hosp, 200 Elizabeth St, Toronto, ON M5G 2C4, Canada

[9] Princess Margaret Canc Ctr, 610 Univ Ave, Toronto, ON M5G 2M9, Canada

[10] Hannover Med Sch, Carl Neuberg Str 1, D-30659 Hannover, Germany

[11] Univ Padua, Dept Med DIMED, Via Gabelli 61, I-35121 Padua, Italy

[12] IRCCS, Veneto Inst Oncol IOV, Via Gattamelata 64, I-35128 Padua, Italy

[13] Univ Barcelona, Barcelona Clin, Liver Unit, Hosp Clin ,IDIBAPS,Liver Canc BCLC Grp, Villarroel 170, Barcelona 08036, Spain

[14] Ctr Invest Biomed Red Enfermedades Cardiovasc CIBE, Ave Monforte de Lemos 3-5, Madrid 28029, Spain

[15] Univ Edinburgh, Inst Regenerat & Repair, Ctr Inflammat Res, 5 Little France Dr, Edinburgh EH16 4UU, Scotland

[16] Univ Edinburgh, 51 Little France Crescent, Edinburgh EH16 4SA, Midlothian, Scotland

[17] Canc Res UK Beatson Inst, Switchback Rd, Glasgow G61 1BD, Scotland

[18] Hosp Univ 12 Octubre, Dept Med Oncol, Ave Cordoba S-N, Madrid 28041, Spain

[19] Univ Vita Salute San Raffaele, Osped San Raffaele Sci Inst, Dept Ophthalmol, Via Olgettina 60, I-20132 Milan, Italy

[20] Azienda Osped Univ Pisana, Med Oncol Unit 2, Via Roma 67, I-56126 Pisa, Italy

[21] Gustave Roussy, Dept Med Oncol, 114 Rue Edouard Vaillant, F-94805 Villejuif, France

[22] Trinity Coll Dublin, Trinity St James Canc Inst, Dublin, Ireland

[23] Vall dHebron Inst Oncol VHIO, Ctr Cellex, Carrer Natzaret 115-117, Barcelona 08035, Spain

[24] Inst Mutualiste Montsouris, Dept Oncol Med, 42 Blvd Jourdan, F-75014 Paris, France

[25] Todua Clin, Dept Oncol & Hematol, GE-0112 Tbilisi, Georgia

[26] Fdn IRCCS Ist Nazl Tumori Milano, Med Oncol Dept, Via Venezian 1, I-20133 Milan, Italy

[27] North Estonian Med Ctr, Sutiste Rd 19, EE-13419 Tallinn, Estonia

[28] UCL, Canc Inst, 72 Huntley St, London WC1E 6DD, England

[29] Univ Salamanca, Expt Hepatol & Drug Targeting HEVEPHARM Grp, IBSAL, CIBERehd, Campus M Unamuno S-N, Salamanca 37007, Spain

[30] Univ Glasgow, Sch Canc Sci, Switchback Rd, Glasgow G61 1QH, Scotland

[31] Beatson West Scotland Canc Ctr, 1053 Great Western Rd, Glasgow G12 0YN, Scotland

来源：

LANCET REGIONAL HEALTH-EUROPE | 2025年 / 50卷

关键词：

Biliary tract cancer; Cholangiocarcinoma; Gallbladder cancer; Immunotherapy; Molecularly targeted therapy; NGS; Access to therapy; Access to NGS; ACTIVE-SYMPTOM-CONTROL; POSITIVE SOLID TUMORS; PEMBRO PLUS GEMCITABINE; RANDOMIZED PHASE-III; PRIMARY LIVER-CANCER; OPEN-LABEL; INTRAHEPATIC CHOLANGIOCARCINOMA; METASTATIC CHOLANGIOCARCINOMA; CISPLATIN GEM/CIS; FGFR2; FUSIONS;

D O I：

10.1016/j.lanepe.2024.101170

中图分类号：

R19 [保健组织与事业（卫生事业管理）];

学科分类号：

摘要：

In recent years, treatment options for patients with advanced biliary tract cancer (BTC) have increased significantly due to the positive results from phase 2/3 clinical trials of immune checkpoint inhibitors, combined with chemotherapy, and molecularly targeted agents. These advances have led to the need for molecular testing to identify actionable alterations and patients amenable to targeted therapies. However, these improvements have brought with them many questions and challenges, including the identification of resistance mechanisms and therapeutic sequences. In this Series paper we aim to provide an overview of the current systemic treatment options for patients with BTC, highlighting disparities in access to innovative treatments and molecular testing across European countries, which lead to inequalities in the possibilities of treating patients with advanced BTC. We also discuss how ongoing European collaborative projects, such as the COST Action Precision-BTC-Network CA22125, supported by COST (European Cooperation in Science and Technology), linked to the European Network for the Study of Cholangiocarcinoma (ENSCCA), can help overcome these disparities and improve the current scenario. Copyright (c) 2024 The Author(s). Published by Elsevier Ltd. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/).

引用

页数：17

共 118 条

[1] PROOF 301: Results of an early discontinued randomized phase 3 trial of the oral FGFR inhibitor infigratinib vs. gemcitabine plus cisplatin in patients with advanced cholangiocarcinoma (CCA) with an FGFR2 gene fusion/rearrangement. [J].

Abou-Alfa, Ghassan K. ;

Borbath, Ivan ;

Roychowdhury, Sameek ;

Goyal, Lipika ;

Lamarca, Angela ;

Macarulla, Teresa ;

Shroff, Rachna T. ;

Oh, Do-Youn ;

Javle, Milind M. ;

Tamas, Catalin ;

Savastano, David M. ;

Van Veenhuyzen, David Friedrich ;

Xu, Cindy ;

Solanas, Jacki ;

Freas, Elizabeth .

JOURNAL OF CLINICAL ONCOLOGY, 2024, 42 (3_SUPPL) :516-516

[2] Ivosidenib in IDH1-mutant, chemotherapy-refractory Croatia& cholangiocarcinoma (ClarlDHy): a multicentre, randomised, double-blind, placebo-controlled, phase 3 study [J].

Abou-Alfa, Ghassan K. ;

Macarulla, Teresa ;

Javle, Milind M. ;

Kelley, Robin K. ;

Lubner, Sam J. ;

Adeva, Jorge ;

Cleary, James M. ;

Catenacci, Daniel V. ;

Borad, Mitesh J. ;

Bridgewater, John ;

Harris, William P. ;

Murphy, Adrian G. ;

Oh, Do-Youn ;

Whisenant, Jonathan ;

Lowery, Maeve A. ;

Goyal, Lipika ;

Shroff, Rachna T. ;

El-Khoueiry, Anthony B. ;

Fan, Bin ;

Wu, Bin ;

Chamberlain, Christina X. ;

Jiang, Liewen ;

Gliser, Camelia ;

Pandya, Shuchi S. ;

Valle, Juan W. ;

Zhu, Andrew X. .

LANCET ONCOLOGY, 2020, 21 (06) :796-807

[3] Pemigatinib for previously treated, locally advanced or metastatic cholangiocarcinoma: a multicentre, open-label, phase 2 study [J].

Abou-Alfa, Ghassan K. ;

Sahai, Vaibhav ;

Hollebecque, Antoine ;

Vaccaro, Gina ;

Melisi, Davide ;

Al-Rajabi, Raed ;

Paulson, Andrew S. ;

Borad, Mitesh J. ;

Gallinson, David ;

Murphy, Adrian G. ;

Oh, Do-Youn ;

Dotan, Efrat ;

Catenacci, Daniel, V ;

Van Cutsem, Eric ;

Ji, Tao ;

Lihou, Christine F. ;

Zhen, Huiling ;

Feliz, Luis ;

Vogel, Arndt .

LANCET ONCOLOGY, 2020, 21 (05) :671-684

[4]

[Anonymous], EFPIA the root cause of unavailability and delay to innovative medicines

[5] ESMO study on the availability and accessibility of biomolecular technologies in oncology in Europe [J].

Bayle, A. ;

Bonastre, J. ;

Chaltiel, D. ;

Latino, N. ;

Rouleau, E. ;

Peters, S. ;

Galotti, M. ;

Bricalli, G. ;

Besse, B. ;

Giuliani, R. .

ANNALS OF ONCOLOGY, 2023, 34 (10) :934-945

[6] Practical considerations in screening for genetic alterations in cholangiocarcinoma [J].

Bekaii-Saab, T. S. ;

Bridgewater, J. ;

Normanno, N. .

ANNALS OF ONCOLOGY, 2021, 32 (09) :1111-1126

[7] Adagrasib in Advanced Solid Tumors Harboring a KRASG12C Mutation [J].

Bekaii-Saab, Tanios S. ;

Yaeger, Rona ;

Spira, Alexander I. ;

Pelster, Meredith S. ;

Sabari, Joshua K. ;

Hafez, Navid ;

Barve, Minal ;

Velastegui, Karen ;

Yan, Xiaohong ;

Shetty, Aditya ;

Der-Torossian, Hirak ;

Pant, Shubham .

JOURNAL OF CLINICAL ONCOLOGY, 2023, 41 (25) :4097-+

[8] FIGHT-302: first-line pemigatinib vs gemcitabine plus cisplatin for advanced cholangiocarcinoma with FGFR2 rearrangements [J].

Bekaii-Saab, Tanios S. ;

Valle, Juan W. ;

Van Cutsem, Eric ;

Rimassa, Lorenza ;

Furuse, Junji ;

Ioka, Tatsuya ;

Melisi, Davide ;

Macarulla, Teresa ;

Bridgewater, John ;

Wasan, Harpreet ;

Borad, Mitesh J. ;

Abou-Alfa, Ghassan K. ;

Jiang, Ping ;

Lihou, Christine F. ;

Zhen, Huiling ;

Asatiani, Ekaterine ;

Feliz, Luis ;

Vogel, Arndt .

FUTURE ONCOLOGY, 2020, 16 (30) :2385-2399

[9] The clinical landscape of cell-free DNA alterations in 1671 patients with advanced biliary tract cancer [J].

Berchuck, J. E. ;

Facchinetti, F. ;

DiToro, D. F. ;

Baiev, I ;

Majeed, U. ;

Reyes, S. ;

Chen, C. ;

Zhang, K. ;

Sharman, R. ;

Uson, P. L. S., Jr. ;

Maurer, J. ;

Shroff, R. T. ;

Pritchard, C. C. ;

Wu, M-J ;

Catenacci, D. V. T. ;

Javle, M. ;

Friboulet, L. ;

Hollebecque, A. ;

Bardeesy, N. ;

Zhu, A. X. ;

Lennerz, J. K. ;

Tan, B. ;

Borad, M. ;

Parikh, A. R. ;

Kiedrowski, L. A. ;

Kelley, R. K. ;

Mody, K. ;

Juric, D. ;

Goyal, L. .

ANNALS OF ONCOLOGY, 2022, 33 (12) :1269-1283

[10]

Biospace, 2023, Relay Therapeutics Pauses Lirafugratinib Rare Cancer Plans due to IRA

← 1 2 3 4 5 6 7 8 9 10 →