REFERENCES

1. Calderaro J, Ghaffari Laleh N, Zeng Q, et al. Deep learning-based phenotyping reclassifies combined hepatocellular-cholangiocarcinoma. Nat Commun 2023;14:8290.

2. Brindley PJ, Bachini M, Ilyas SI, et al. Cholangiocarcinoma. Nat Rev Dis Primers 2021;7:65.

3. Sung H, Ferlay J, Siegel RL, et al. Global cancer statistics 2020: GLOBOCAN estimates of incidence and mortality worldwide for 36 cancers in 185 countries. CA Cancer J Clin 2021;71:209-49.

4. Morgan E, Arnold M, Gini A, et al. Global burden of colorectal cancer in 2020 and 2040: incidence and mortality estimates from GLOBOCAN. Gut 2023;72:338-44.

5. Labib PL, Goodchild G, Pereira SP. Molecular pathogenesis of cholangiocarcinoma. BMC Cancer 2019;19:185.

6. Khan SA, Tavolari S, Brandi G. Cholangiocarcinoma: epidemiology and risk factors. Liver Int 2019;39 Suppl 1:19-31.

7. Endo I, Gonen M, Yopp AC, et al. Intrahepatic cholangiocarcinoma: rising frequency, improved survival, and determinants of outcome after resection. Ann Surg 2008;248:84-96.

8. Mavros MN, Economopoulos KP, Alexiou VG, Pawlik TM. Treatment and prognosis for patients with intrahepatic cholangiocarcinoma: systematic review and meta-analysis. JAMA Surg 2014;149:565-74.

9. Bertuccio P, Malvezzi M, Carioli G, et al. Global trends in mortality from intrahepatic and extrahepatic cholangiocarcinoma. J Hepatol 2019;71:104-14.

10. Woods E, Le D, Jakka BK, Manne A. Changing landscape of systemic therapy in biliary tract cancer. Cancers 2022;14:2137.

11. Moris D, Palta M, Kim C, Allen PJ, Morse MA, Lidsky ME. Advances in the treatment of intrahepatic cholangiocarcinoma: an overview of the current and future therapeutic landscape for clinicians. CA Cancer J Clin 2023;73:198-222.

12. Valle J, Wasan H, Palmer DH, et al. ABC-02 Trial Investigators. Cisplatin plus gemcitabine versus gemcitabine for biliary tract cancer. N Engl J Med 2010;362:1273-81.

13. Vogel A, Chen L, He AR, et al. Regional subgroup analysis of the phase 3 TOPAZ-1 study of durvalumab (D) plus gemcitabine and cisplatin (GC) in advanced biliary tract cancer (BTC). JCO 2022;40:4075.

14. Oh D, He AR, Qin S, et al. A phase 3 randomized, double-blind, placebo-controlled study of durvalumab in combination with gemcitabine plus cisplatin (GemCis) in patients (pts) with advanced biliary tract cancer (BTC): TOPAZ-1. JCO 2022;40:378.

15. Sia D, Tovar V, Moeini A, Llovet JM. Intrahepatic cholangiocarcinoma: pathogenesis and rationale for molecular therapies. Oncogene 2013;32:4861-70.

16. Saha SK, Gordan JD, Kleinstiver BP, et al. Isocitrate dehydrogenase mutations confer dasatinib hypersensitivity and SRC dependence in intrahepatic cholangiocarcinoma. Cancer Discov 2016;6:727-39.

17. Jusakul A, Cutcutache I, Yong CH, et al. Whole-genome and epigenomic landscapes of etiologically distinct subtypes of cholangiocarcinoma. Cancer Discov 2017;7:1116-35.

18. Desjonqueres E, Campani C, Marra F, Zucman-Rossi J, Nault JC. Preneoplastic lesions in the liver: molecular insights and relevance for clinical practice. Liver Int 2022;42:492-506.

19. van Dijk EL, Auger H, Jaszczyszyn Y, Thermes C. Ten years of next-generation sequencing technology. Trends Genet 2014;30:418-26.

20. De Luca A, Esposito Abate R, Rachiglio AM, et al. FGFR fusions in cancer: from diagnostic approaches to therapeutic intervention. Int J Mol Sci 2020;21:6856.

21. Vogel A, Segatto O, Stenzinger A, Saborowski A. FGFR2 inhibition in cholangiocarcinoma. Annu Rev Med 2023;74:293-306.

22. Abou-Alfa GK, Macarulla T, Javle MM, et al. Ivosidenib in IDH1-mutant, chemotherapy-refractory cholangiocarcinoma (ClarIDHy): a multicentre, randomised, double-blind, placebo-controlled, phase 3 study. Lancet Oncol 2020;21:796-807.

23. Lowery MA, Burris HA 3rd, Janku F, et al. Safety and activity of ivosidenib in patients with IDH1-mutant advanced cholangiocarcinoma: a phase 1 study. Lancet Gastroenterol Hepatol 2019;4:711-20.

24. Javle M, Borad MJ, Azad NS, et al. Pertuzumab and trastuzumab for HER2-positive, metastatic biliary tract cancer (MyPathway): a multicentre, open-label, phase 2a, multiple basket study. Lancet Oncol 2021;22:1290-300.

25. Harding J, Cleary J, Shapiro G, et al. Treating HER2-mutant advanced biliary tract cancer with neratinib: benefits of HER2-directed targeted therapy in the phase 2 SUMMIT ‘basket’ trial. Ann Oncol 2019;30:iv127.

26. Babina IS, Turner NC. Advances and challenges in targeting FGFR signalling in cancer. Nat Rev Cancer 2017;17:318-32.

27. Uson Junior PLS, Borad MJ. Precision approaches for cholangiocarcinoma: progress in clinical trials and beyond. Expert Opin Investig Drugs 2022;31:125-31.

28. Abou-Alfa GK, Sahai V, Hollebecque A, et al. Pemigatinib for previously treated, locally advanced or metastatic cholangiocarcinoma: a multicentre, open-label, phase 2 study. Lancet Oncol 2020;21:671-84.

29. Shi GM, Huang XY, Wen TF, et al. Pemigatinib in previously treated Chinese patients with locally advanced or metastatic cholangiocarcinoma carrying FGFR2 fusions or rearrangements: a phase II study. Cancer Med 2023;12:4137-46.

30. Bekaii-Saab TS, Valle JW, Van Cutsem E, et al. FIGHT-302: first-line pemigatinib vs gemcitabine plus cisplatin for advanced cholangiocarcinoma with FGFR2 rearrangements. Future Oncol 2020;16:2385-99.

31. Javle M, Lowery M, Shroff RT, et al. Phase II study of BGJ398 in patients with FGFR-altered advanced cholangiocarcinoma. J Clin Oncol 2018;36:276-82.

32. Javle M, Roychowdhury S, Kelley RK, et al. Infigratinib (BGJ398) in previously treated patients with advanced or metastatic cholangiocarcinoma with FGFR2 fusions or rearrangements: mature results from a multicentre, open-label, single-arm, phase 2 study. Lancet Gastroenterol 2021;6:803-15.

33. Makawita S, K Abou Alfa G, Roychowdhury S, et al. Infigratinib in patients with advanced cholangiocarcinoma with FGFR2 gene fusions/translocations: the PROOF 301 trial. Future Oncol 2020;16:2375-84.

34. Bahleda R, Meric-Bernstam F, Goyal L, et al. Phase I, first-in-human study of futibatinib, a highly selective, irreversible FGFR1-4 inhibitor in patients with advanced solid tumors. Ann Oncol 2020;31:1405-12.

35. Goyal L, Meric-Bernstam F, Hollebecque A, et al. Updated results of the FOENIX-CCA2 trial: efficacy and safety of futibatinib in intrahepatic cholangiocarcinoma (iCCA) harboring FGFR2 fusions/rearrangements. J Clin Oncol 2022;40:4009.

36. Borad MJ, Bridgewater JA, Morizane C, et al. A phase III study of futibatinib (TAS-120) versus gemcitabine-cisplatin (gem-cis) chemotherapy as first-line (1L) treatment for patients (pts) with advanced (adv) cholangiocarcinoma (CCA) harboring fibroblast growth factor receptor 2 ( FGFR2 ) gene rearrangements (FOENIX-CCA3). JCO 2020;38:TPS600.

37. Rizzo A, Ricci AD, Brandi G. IDH inhibitors in advanced cholangiocarcinoma: Another arrow in the quiver? Cancer Treat Res Commun 2021;27:100356.

38. Farshidfar F, Zheng S, Gingras MC, et al. Cancer Genome Atlas Network. Integrative genomic analysis of cholangiocarcinoma identifies distinct idh-mutant molecular profiles. Cell Rep 2017;18:2780-94.

39. Xiang X, Liu Z, Zhang C, et al. IDH mutation subgroup status associates with intratumor heterogeneity and the tumor microenvironment in intrahepatic cholangiocarcinoma. Adv Sci 2021;8:e2101230.

40. Lavacchi D, Caliman E, Rossi G, et al. Ivosidenib in IDH1-mutated cholangiocarcinoma: clinical evaluation and future directions. Pharmacol Ther 2022;237:108170.

41. Zhu AX, Macarulla T, Javle MM, et al. Final overall survival efficacy results of ivosidenib for patients with advanced cholangiocarcinoma with IDH1 mutation: the phase 3 randomized clinical ClarIDHy trial. JAMA Oncol 2021;7:1669-77.

42. Javle M, Bekaii-Saab T, Jain A, et al. Biliary cancer: utility of next-generation sequencing for clinical management. Cancer 2016;122:3838-47.

43. Li M, Zhang Z, Li X, et al. Whole-exome and targeted gene sequencing of gallbladder carcinoma identifies recurrent mutations in the ErbB pathway. Nat Genet 2014;46:872-6.

44. Ramanathan RK, Belani CP, Singh DA, et al. A phase II study of lapatinib in patients with advanced biliary tree and hepatocellular cancer. Cancer Chemother Pharmacol 2009;64:777-83.

45. Peck J, Wei L, Zalupski M, O'Neil B, Villalona Calero M, Bekaii-Saab T. HER2/neu may not be an interesting target in biliary cancers: results of an early phase II study with lapatinib. Oncology 2012;82:175-9.

46. Meric-Bernstam F, Beeram M, Hamilton E, et al. Zanidatamab, a novel bispecific antibody, for the treatment of locally advanced or metastatic HER2-expressing or HER2-amplified cancers: a phase 1, dose-escalation and expansion study. Lancet Oncol 2022;23:1558-70.

47. Ohba A, Morizane C, Ueno M, et al. Multicenter phase II trial of trastuzumab deruxtecan for HER2-positive unresectable or recurrent biliary tract cancer: HERB trial. Future Oncol 2022;18:2351-60.

48. Lee CK, Chon HJ, Cheon J, et al. 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). Lancet Gastroenterol 2023;8:56-65.

49. Xin HY, Sun RQ, Zou JX, et al. Association of BRAF variants with disease characteristics, prognosis, and targeted therapy response in intrahepatic cholangiocarcinoma. JAMA Netw Open 2023;6:e231476.

50. Subbiah V, Lassen U, Élez E, et al. Dabrafenib plus trametinib in patients with BRAFV600E-mutated biliary tract cancer (ROAR): a phase 2, open-label, single-arm, multicentre basket trial. Lancet Oncol 2020;21:1234-43.

51. Drilon A, Laetsch TW, Kummar S, et al. Efficacy of larotrectinib in TRK fusion-positive cancers in adults and children. N Engl J Med 2018;378:731-9.

52. Doebele RC, Drilon A, Paz-Ares L, et al. trial investigators. Entrectinib in patients with advanced or metastatic NTRK fusion-positive solid tumours: integrated analysis of three phase 1-2 trials. Lancet Oncol 2020;21:271-82.

53. Jakubowski CD, Azad NS. Immune checkpoint inhibitor therapy in biliary tract cancer (cholangiocarcinoma). Chin Clin Oncol 2020;9:2.

54. Fiste O, Ntanasis-Stathopoulos I, Gavriatopoulou M, et al. The emerging role of immunotherapy in intrahepatic cholangiocarcinoma. Vaccines 2021;9:422.

55. Jänne PA, Riely GJ, Gadgeel SM, et al. Adagrasib in non-small-cell lung cancer harboring a KRASG12C mutation. N Engl J Med 2022;387:120-31.

56. Awad MM, Liu S, Rybkin II, et al. Acquired resistance to KRASG12C Inhibition in cancer. N Engl J Med 2021;384:2382-93.

57. Sun J, Chen Q, Ma J. Notch-Sox9 axis mediates hepatocyte dedifferentiation in kras(G12V)-induced zebrafish hepatocellular carcinoma. Int J Mol Sci 2022;23:4705.

58. Zhou SL, Xin HY, Sun RQ, et al. Association of KRAS variant subtypes with survival and recurrence in patients with surgically treated intrahepatic cholangiocarcinoma. JAMA Surg 2022;157:59-65.

59. Cox AD, Fesik SW, Kimmelman AC, Luo J, Der CJ. Drugging the undruggable RAS: mission possible? Nat Rev Drug Discov 2014;13:828-51.

60. Pylayeva-Gupta Y, Grabocka E, Bar-Sagi D. RAS oncogenes: weaving a tumorigenic web. Nat Rev Cancer 2011;11:761-74.

61. Blair HA. Sotorasib: first approval. Drugs 2021;81:1573-9.

62. Skoulidis F, Li BT, Dy GK, et al. Sotorasib for lung cancers with KRAS p.G12C mutation. N Engl J Med 2021;384:2371-81.

63. Sharma P, Hu-Lieskovan S, Wargo JA, Ribas A. Primary, adaptive, and acquired resistance to cancer immunotherapy. Cell 2017;168:707-23.

64. Berchuck JE, Facchinetti F, DiToro DF, et al. The clinical landscape of cell-free DNA alterations in 1671 patients with advanced biliary tract cancer. Ann Oncol 2022;33:1269-83.

65. Uson Junior PLS, Majeed U, Yin J, et al. Cell-free tumor dna dominant clone allele frequency is associated with poor outcomes in advanced biliary cancers treated with platinum-based chemotherapy. JCO Precis Oncol 2022;6:e2100274.

66. Goyal L, Saha SK, Liu LY, et al. Polyclonal secondary FGFR2 mutations drive acquired resistance to FGFR inhibition in patients with FGFR2 fusion-positive cholangiocarcinoma. Cancer Discov 2017;7:252-63.

67. Silverman IM, Hollebecque A, Friboulet L, et al. Clinicogenomic analysis of FGFR2-rearranged cholangiocarcinoma identifies correlates of response and mechanisms of resistance to pemigatinib. Cancer Discov 2021;11:326-39.

68. Lau DK, Jenkins L, Weickhardt A. Mechanisms of acquired resistance to fibroblast growth factor receptor targeted therapy. Cancer Drug Resist 2019;2:568-79.

69. Lavacchi D, Pellegrini E, Palmieri VE, et al. Immune checkpoint inhibitors in the treatment of renal cancer: current state and future perspective. Int J Mol Sci 2020;21:4691.

70. Feng K, Liu Y, Zhao Y, et al. Efficacy and biomarker analysis of nivolumab plus gemcitabine and cisplatin in patients with unresectable or metastatic biliary tract cancers: results from a phase II study. J Immunother Cancer 2020;8:e000367.

71. Mohan A, Griffith KA, Wuchu F, et al. Devimistat in combination with gemcitabine and cisplatin in biliary tract cancer: preclinical evaluation and phase Ib multicenter clinical trial (BilT-04). Clin Cancer Res 2023;29:2394-400.

72. Zeng TM, Yang G, Lou C, et al. Clinical and biomarker analyses of sintilimab plus gemcitabine and cisplatin as first-line treatment for patients with advanced biliary tract cancer. Nat Commun 2023;14:1340.

73. Lee HS, Han DH, Cho K, et al. Integrative analysis of multiple genomic data from intrahepatic cholangiocarcinoma organoids enables tumor subtyping. Nat Commun 2023;14:237.

74. Bao X, Li Q, Chen J, et al. Molecular subgroups of intrahepatic cholangiocarcinoma discovered by single-cell RNA sequencing-assisted multiomics analysis. Cancer Immunol Res 2022;10:811-28.

75. Dong L, Lu D, Chen R, et al. Proteogenomic characterization identifies clinically relevant subgroups of intrahepatic cholangiocarcinoma. Cancer Cell 2022;40:70-87.e15.

Hepatoma Research
ISSN 2454-2520 (Online) 2394-5079 (Print)

Portico

All published articles are preserved here permanently:

https://www.portico.org/publishers/oae/

Portico

All published articles are preserved here permanently:

https://www.portico.org/publishers/oae/