[1]
|
Turner, N. and Grose, R. (2010) Fibroblast Growth Factor Signalling: From Development to Cancer. Nature Reviews Cancer, 10, 116-129. https://doi.org/10.1038/nrc2780
|
[2]
|
Touat, M., Ileana, E., Postel-Vinay, S., et al. (2015) Targeting FGFR Signaling in Cancer. Clinical Cancer Research, 21, 2684-2694. https://doi.org/10.1158/1078-0432.CCR-14-2329
|
[3]
|
Wiedemann, M. and Trueb, B. (2000) Characterization of a Novel Protein (FGFRL1) from Human Cartilage Related to FGF Receptors. Genomics, 69, 275-279. https://doi.org/10.1006/geno.2000.6332
|
[4]
|
Pacini, L., Jenks, A.D., Lima, N.C., et al. (2021) Targeting the Fibro-blast Growth Factor Receptor (FGFR) Family in Lung Cancer. Cells, 10, 1154. https://doi.org/10.3390/cells10051154
|
[5]
|
Hallinan, N., Finn, S., Cuffe, S., et al. (2016) Targeting the Fibroblast Growth Factor Receptor Family in Cancer. Cancer Treatment Reviews, 46, 51-62. https://doi.org/10.1016/j.ctrv.2016.03.015
|
[6]
|
Helsten, T., Elkin, S., Arthur, E., et al. (2016) The FGFR Land-scape in Cancer: Analysis of 4,853 Tumors by Next- Generation Sequencing. Clinical Cancer Research, 22, 259-267.
https://doi.org/10.1158/1078-0432.CCR-14-3212
|
[7]
|
Babina, I.S. and Turner, N.C. (2017) Advances and Chal-lenges in Targeting FGFR Signalling in Cancer. Nature Reviews Cancer, 17, 318-332. https://doi.org/10.1038/nrc.2017.8
|
[8]
|
Weiss, J., Sos, M.L., Seidel, D., et al. (2010) Frequent and Focal FGFR1 Amplification Associates with Therapeutically Tractable FGFR1 Dependency in Squamous Cell Lung Cancer. Science Translational Medicine, 2, 62-93.
https://doi.org/10.1126/scitranslmed.3001451
|
[9]
|
Turner, N., Pearson, A., Sharpe, R., et al. (2010) FGFR1 Am-plification Drives Endocrine Therapy Resistance and Is a Therapeutic Target in Breast Cancer. Cancer Research, 70, 2085-2094.
https://doi.org/10.1158/0008-5472.CAN-09-3746
|
[10]
|
Fischbach, A., Rogler, A., Erber, R., et al. (2015) Fibro-blast Growth Factor Receptor (FGFR) Gene Amplifications Are Rare Events in Bladder Cancer. Histopathology, 66, 639-649. https://doi.org/10.1111/his.12473
|
[11]
|
Hart, K.C., Robertson, S.C., Kanemitsu, M.Y., et al. (2000) Transformation and Stat Activation by Derivatives of FGFR1, FGFR3, and FGFR4. Oncogene, 19, 3309-3320. https://doi.org/10.1038/sj.onc.1203650
|
[12]
|
Gallo, L.H., Nelson, K.N., Meyer, A.N., et al. (2015) Functions of Fi-broblast Growth Factor Receptors in Cancer Defined by Novel Translocations and Mutations. Cytokine & Growth Factor Reviews, 26, 425-449.
https://doi.org/10.1016/j.cytogfr.2015.03.003
|
[13]
|
Landberg, N., Dreimane, A., Rissler, M., et al. (2017) Primary Cells in BCR/FGFR1-Positive 8p11 Myeloproliferative Syndrome Are Sensitive to Dovitinib, Ponatinib, and Dasatinib. European Journal of Haematology, 99, 442-448.
https://doi.org/10.1111/ejh.12957
|
[14]
|
Andre, F. and Cortes, J. (2015) Rationale for Targeting Fibroblast Growth Factor Receptor Signaling in Breast Cancer. Breast Cancer Research and Treatment, 150, 1-8. https://doi.org/10.1007/s10549-015-3301-y
|
[15]
|
Su, X., Zhan, P., Gavine, P.R., et al. (2014) FGFR2 Amplifica-tion Has Prognostic Significance in Gastric Cancer: Results from a Large International Multicentre Study. British Journal of Cancer, 110, 967-975.
https://doi.org/10.1038/bjc.2013.802
|
[16]
|
Dutt, A., Salvesen, H.B., Chent, T.H., et al. (2008) Drug-Sensitive FGFR2 Mutations in Endometrial Carcinoma. Proceedings of the National Academy of Sciences of the United States of America, 105, 8713-8717.
https://doi.org/10.1073/pnas.0803379105
|
[17]
|
Akbay, E.A., Tchaicha, J.H., Altabef, A., et al. (2014) Kinase Do-main Activation of FGFR2 Yields High-Grade Lung Adenocarcinoma Sensitive to a Pan-FGFR Inhibitor in a Mouse Model of NSCLC. Cancer Research, 74, 4676-4684.
https://doi.org/10.1158/0008-5472.CAN-13-3218
|
[18]
|
Jung, E.J., Jung, E.J., Min, S.Y., et al. (2012) Fibroblast Growth Factor Receptor 2 Gene Amplification Status and Its Clinicopathologic Significance in Gastric Carcinoma. Hu-man Pathology, 43, 1559-1566.
https://doi.org/10.1016/j.humpath.2011.12.002
|
[19]
|
Baldia, P.H., Maurer, A., Heide, T., et al. (2016) Fibroblast Growth Factor Receptor (FGFR) Alterations in Squamous Differentiated Bladder Cancer: A Putative Therapeutic Target for a Small Subgroup. Oncotarget, 7, 1429-1439.
https://doi.org/10.18632/oncotarget.12198
|
[20]
|
Wu, Y.M., Su, F.Y., Kalyana-Sundaram, S., et al. (2013) Identifi-cation of Targetable FGFR Gene Fusions in Diverse Cancers. Cancer Discovery, 3, 636-647. https://doi.org/10.1158/2159-8290.CD-13-0050
|
[21]
|
Veekony, H., Ylstra, B., Wilting, S.M., et al. (2007) DNA Copy Number Gains at Loci of Growth Factors and Their Receptors in Salivary Gland Adenoid Cystic Carcinoma. Clin-ical Cancer Research, 13, 3133-3139.
https://doi.org/10.1158/1078-0432.CCR-06-2555
|
[22]
|
Liu, X., Zhang, W., Geng, D., et al. (2014) Clinical Signifi-cance of Fibroblast Growth Factor Receptor-3 Mutations in Bladder Cancer: A Systematic Review and Meta-Analysis. Genetics and Molecular Research, 13, 1109-1120.
https://doi.org/10.4238/2014.February.20.12
|
[23]
|
Rosty, C., Aubriot, M.H., Cappellen, D., et al. (2005) Clinical and Biological Characteristics of Cervical Neoplasias with FGFR3 Mutation. Molecular Cancer, 4, 15. https://doi.org/10.1186/1476-4598-4-15
|
[24]
|
Chesi, M., Nardini, E., Brents, L.A., et al. (1997) Frequent Translo-cation t(4;14)(p16.3;q32.3) in Multiple Myeloma Is Associated with Increased Expression and Activating Mutations of Fibroblast Growth Factor Receptor 3. Nature Genetics, 16, 260-264. https://doi.org/10.1038/ng0797-260
|
[25]
|
Zhang, Y., Hiraishi, Y., Wang, H., et al. (2005) Constitutive Activating Mutation of the FGFR3b in Oral Squamous Cell Carcinomas. International Journal of Cancer, 117, 166-168. https://doi.org/10.1002/ijc.21145
|
[26]
|
Seki, M., Nishimura, R., Yoshida, K., et al. (2015) Integrated Genetic and Epigenetic Analysis Defines Novel Molecular Subgroups in Rhabdomyosarcoma. Nature Communications, 6, Article No. 7557.
https://doi.org/10.1038/ncomms8557
|
[27]
|
Rizzo, A. and Brandi, G. (2021) Neoadjuvant Therapy for Cholangio-carcinoma: A Comprehensive Literature Review. Cancer Treatment and Research Communications, 27, 1003-1054. https://doi.org/10.1016/j.ctarc.2021.100354
|
[28]
|
Razumilava, N. and Gores, G.J. (2013) Classification, Diagnosis, and Management of Cholangiocarcinoma. Clinical Gastroenterology and Hepatology, 11, 13-43. https://doi.org/10.1016/j.cgh.2012.09.009
|
[29]
|
Wang, Y., Li, J., Xia, Y., et al. (2013) Prognostic Nomogram for Intrahepatic Cholangiocarcinoma after Partial Hepatectomy. Journal of Clinical Oncology, 31, 1188-1195. https://doi.org/10.1200/JCO.2012.41.5984
|
[30]
|
Tariq, N.U., Mcnamara, M.G. and Valle, J.W. (2019) Biliary Tract Cancers: Current Knowledge, Clinical Candidates and Future Challenges. Cancer Management and Research, 11, 2623-2642. https://doi.org/10.2147/CMAR.S157092
|
[31]
|
Subbiah, V., Lassen, U., Elez, E., et al. (2020) Dabraf-enib plus Trametinib in Patients with BRAF(V600E)-Mutated Biliary Tract Cancer (ROAR): A Phase 2, Open-Label, Single-Arm, Multicentre Basket Trial. The Lancet Oncology, 21, 1234-1243. https://doi.org/10.1016/S1470-2045(20)30321-1
|
[32]
|
Smyth, E.C., Babina, I.S. and Turner, N.C. (2017) Gate-keeper Mutations and Intratumoral Heterogeneity in FGFR2- Translocated Cholangiocarcinoma. Cancer Discovery, 7, 248-249.
https://doi.org/10.1158/2159-8290.CD-17-0057
|
[33]
|
Rizzo, A., Ricci, A.D., Tober, N., et al. (2020) Second-Line Treatment in Advanced Biliary Tract Cancer: Today and Tomorrow. Anticancer Research, 40, 3013-3030. https://doi.org/10.21873/anticanres.14282
|
[34]
|
Ueno, M., Ikeda, M., Sasaki, T., et al. (2020) Phase 2 Study of Lenvatinib Monotherapy as Second-Line Treatment in Unresectable Biliary Tract Cancer: Primary Analysis Results. BMC Cancer, 20, Article No. 1105.
https://doi.org/10.1186/s12885-020-07365-4
|
[35]
|
Plummer, R., Madi, A., Jeffels, M., et al. (2013) A Phase I Study of Pazopanib in Combination with Gemcitabine in Patients with Advanced Solid Tumors. Cancer Chemotherapy and Pharmacology, 71, 93-101.
https://doi.org/10.1007/s00280-012-1982-z
|
[36]
|
Shroff, R.T., Yarchoan, M., O’connor, A., et al. (2017) The Oral VEGF Receptor Tyrosine Kinase Inhibitor Pazopanib in Combination with the MEK Inhibitor Trametinib in Advanced Cholangiocarcinoma. British Journal of Cancer, 116, 1402-1407. https://doi.org/10.1038/bjc.2017.119
|
[37]
|
Mahipal, A., Tella, S.H., Kommalapati, A., et al. (2020) Prevention and Treatment of FGFR Inhibitor-Associated Toxicities. Critical Reviews in Oncology/Hematology, 155, 1030-1091. https://doi.org/10.1016/j.critrevonc.2020.103091
|
[38]
|
Cortellis. Clarivate Analytics Integrity. https://integrity.clarivate.com
|
[39]
|
Nogova, L., Sequist, L.V., Garcia, J.M.P., et al. (2017) Evaluation of BGJ398, a Fi-broblast Growth Factor Receptor 1-3 Kinase Inhibitor, in Patients with Advanced Solid Tumors Harboring Genetic Al-terations in Fibroblast Growth Factor Receptors: Results of a Global Phase I, Dose-Escalation and Dose-Expansion Study. Journal of Clinical Oncology, 35, 157-167. https://doi.org/10.1200/JCO.2016.67.2048
|
[40]
|
Javle, M.M., Roychowdhury, S., Kelley, R.K., et al. (2021) Final Results from a Phase II Study of Infigratinib (BGJ398), an FGFR-Selective Tyrosine Kinase Inhibitor, in Patients with Previously Treated Advanced Cholangiocarcinoma Harboring an FGFR2 Gene Fusion or Rearrangement. Journal of Clinical Oncology, 39, 345-350.
https://doi.org/10.1200/JCO.2021.39.3_suppl.265
|
[41]
|
Javle, M., Lowery, M., Shroff, R.T., et al. (2018) Phase II Study of BGJ398 in Patients with FGFR-Altered Advanced Cholangiocarcinoma. Journal of Clinical Oncology, 36, 276-281. https://doi.org/10.1200/JCO.2017.75.5009
|
[42]
|
Krook, M.A., Lenyo, A., Wilberding, M., et al. (2020) Efficacy of FGFR Inhibitors and Combination Therapies for Acquired Resistance in FGFR2-Fusion Cholangiocarcinoma. Molecular Cancer Therapeutics, 19, 847-857.
https://doi.org/10.1158/1535-7163.MCT-19-0631
|
[43]
|
Goyal, L., Shi, L., Liu, L.Y., et al. (2019) TAS-120 Over-comes Resistance to ATP-Competitive FGFR Inhibitors in Patients with FGFR2 Fusion-Positive Intrahepatic Cholangi-ocarcinoma. Cancer Discovery, 9, 1064-1079.
https://doi.org/10.1158/2159-8290.CD-19-0182
|
[44]
|
Rizzo, A. (2021) Novel Approaches for the Management of Biliary Tract Cancer: Today and Tomorrow. Expert Opinion on Investigational Drugs, 30, 295-297. https://doi.org/10.1080/13543784.2021.1896247
|
[45]
|
Hall, T.G., Yu, Y., Eathiraj, S., et al. (2016) Preclinical Ac-tivity of ARQ 087, a Novel Inhibitor Targeting FGFR Dysregulation. PLoS ONE, 11, 1625-1694. https://doi.org/10.1371/journal.pone.0162594
|
[46]
|
Mazzaferro, V., El-Rayes, B.F., Droz Dit Busset, M., et al. (2019) Derazantinib (ARQ 087) in Advanced or Inoperable FGFR2 Gene Fusion-Positive Intrahepatic Cholangiocarci-noma. British Journal of Cancer, 120, 165-171.
https://doi.org/10.1038/s41416-018-0334-0
|
[47]
|
Perera, T.P.S., Jovcheva, E., Mevellec, L., et al. (2017) Discovery and Pharmacological Characterization of JNJ-42756493 (Erdafitinib), a Functionally Selective Small-Molecule FGFR Family Inhibitor. Molecular Cancer Therapeutics, 16, 1010-1020. https://doi.org/10.1158/1535-7163.MCT-16-0589
|
[48]
|
Hanna, K.S. (2019) Erdafitinib to Treat Urothelial Carcino-ma. Drugs Today (Barc), 55, 495-501.
https://doi.org/10.1358/dot.2019.55.8.3010573
|
[49]
|
Park, J.O., Feng, Y.H., Chen, Y.Y., et al. (2019) Updated Results of a Phase IIa Study to Evaluate the Clinical Efficacy and Safety of Erdafitinib in Asian Advanced Cholangiocar-cinoma (CCA) Patients with FGFR Alterations. Journal of Clinical Oncology, 37, 4117. https://doi.org/10.1200/JCO.2019.37.15_suppl.4117
|
[50]
|
Rizzo, A. and Brandi, G. (2021) Novel Targeted Thera-pies for Advanced Cholangiocarcinoma. Medicina-Lithuania, 57, 212. https://doi.org/10.3390/medicina57030212
|
[51]
|
Rizzo, A. and Brandi, G. (2021) A Foreword on Biliary Tract Cancers: Emerging Treatments, Drug Targets, and Fundamental Knowledge Gaps. Expert Opinion on Investigational Drugs, 30, 279-284.
https://doi.org/10.1080/13543784.2021.1901192
|
[52]
|
Abou-Alfa, G.K., Sahai, V., Hollebecque, A., et al. (2020) Pemigatinib for Previously Treated, Locally Advanced or Metastatic Cholangiocarcinoma: A Multicentre, Open-Label, Phase 2 Study. The Lancet Oncology, 21, 671-684.
https://doi.org/10.1016/S1470-2045(20)30109-1
|
[53]
|
Romero, D. (2020) Benefit from Pemigatinib in Cholangio-carcinoma. Nature Reviews Clinical Oncology, 17, 337-341.
https://doi.org/10.1038/s41571-020-0369-z
|
[54]
|
Sootome, H., Fujita, H., Ito, K., et al. (2020) Futibatinib Is a Nov-el Irreversible FGFR 1-4 Inhibitor That Shows Selective Antitumor Activity against FGFR-Deregulated Tumors. Cancer Research, 80, 4986-4997.
https://doi.org/10.1158/0008-5472.CAN-19-2568
|
[55]
|
Bahleda, R., Meric-Bernstam, F., Goyal, L., et al. (2020) Phase I, First-in-Human Study of Futibatinib, a Highly Selective, Irreversible FGFR1-4 Inhibitor in Patients with Ad-vanced Solid Tumors. Annals of Oncology, 31, 1405-1412.
https://doi.org/10.1016/j.annonc.2020.06.018
|
[56]
|
Meric-Bernstam, F., Arkenau, H., Tran, B., et al. (2018) Effica-cy of TAS-120, an Irreversible Fibroblast Growth Factor Receptor (FGFR) Inhibitor, in Cholangiocarcinoma Patients with FGFR Pathway Alterations Who Were Previously Treated with Chemotherapy and Other FGFR Inhibitors. Annals of Oncology, 29, 119-121.
https://doi.org/10.1093/annonc/mdy149
|
[57]
|
Voss, M.H., Hierro, C., Heist, R.S., et al. (2019) A Phase I, Open-Label, Multicenter, Dose-Escalation Study of the Oral Selective FGFR Inhibitor Debio 1347 in Patients with Ad-vanced Solid Tumors Harboring FGFR Gene Alterations. Clinical Cancer Research, 25, 2699-2707. https://doi.org/10.1158/1078-0432.CCR-18-1959
|
[58]
|
Cleary, J.M., Iyer, G., Oh, D.Y., et al. (2020) Final Results from the Phase I Study Expansion Cohort of the Selective FGFR Inhibitor Debio 1,347 in Patients with Solid Tumors Harboring an FGFR Gene Fusion. Journal of Clinical Oncology, 38, 3603. https://doi.org/10.1200/JCO.2020.38.15_suppl.3603
|
[59]
|
Hyman, D.M., Goyal, L., Grivas, P., et al. (2019) FUZE Clinical Trial: A Phase 2 Study of Debio 1347 in FGFR Fusion-Positive Advanced Solid Tumors Irrespectively of Tu-mor Histology. Journal of Clinical Oncology, 37, 3157.
https://doi.org/10.1200/JCO.2019.37.15_suppl.TPS3157
|
[60]
|
Balasubramanian, B., Myint, K.Z., Yacqub-Usman, K., et al. (2021) FGF Signalling as a Therapeutic Target in Cholangiocarcinoma. Cancer Science, 112, 419-425.
|
[61]
|
Noble, M.E.M., Endicott, J.A. and Johnson, L.N. (2004) Protein Kinase Inhibitors: Insights into Drug Design from Structure. Science, 303, 1800-1805. https://doi.org/10.1126/science.1095920
|
[62]
|
Lamarca, A., Palm-er, D.H., Wasan, H.S., et al. (2019) ABC-06 Vertical Bar A Randomised Phase III, Multi-Centre, Open-Label Study of Active Symptom Control (ASC) Alone or ASC with Oxaliplatin/5-FU Chemotherapy (ASC plus mFOLFOX) for Pa-tients (pts) with Locally Advanced/Metastatic Biliary Tract Cancers (ABC) Previously-Treated with Cispla-tin/Gemcitabine (CisGem) Chemotherapy. Journal of Clinical Oncology, 37, 4003.
https://doi.org/10.1200/JCO.2019.37.15_suppl.4003
|
[63]
|
Loeuillard, E., Conboy, C.B., Gores, G.J., et al. (2019) Immunobiology of Cholangiocarcinoma. JHEP Reports, 1, 297-311. https://doi.org/10.1016/j.jhepr.2019.06.003
|