Emerging biomarkers in urothelial carcinoma: Challenges and opportunities
Tài liệu tham khảo
National Cancer Institute, Cancer Stat Facts: Bladder Cancer, (2019). https://seer.cancer.gov/statfacts/html/urinb.html (accessed April 12, 2020).
Smith, 2014, Muscle-invasive bladder cancer: evaluating treatment and survival in the national cancer data base, BJU Int., 114, 719, 10.1111/bju.12601
Burger, 2013, Epidemiology and risk factors of urothelial bladder cancer, Eur. Urol., 63, 234, 10.1016/j.eururo.2012.07.033
Chalasani, 2013, Histologic variants of urothelial bladder cancer and nonurothelial histology in bladder cancer, Can. Urol. Assoc. J., 3, 193, 10.5489/cuaj.1195
Gómez De Liaño, 2018, The continuing role of chemotherapy in the management of advanced urothelial cancer, Ther. Adv. Urol., 10, 455, 10.1177/1756287218814100
American Cancer Society, Survival Rates for Bladder Cancer, (2019). https://www.cancer.org/cancer/bladder-cancer/detection-diagnosis-staging/survival-rates.html (accessed April 12, 2020).
Nadal, 2019, Management of metastatic bladder cancer, Cancer Treat. Rev., 76, 10, 10.1016/j.ctrv.2019.04.002
Koshkin, 2018, Emerging role of Immunotherapy in advanced urothelial carcinoma, Curr. Oncol. Rep., 20, 48, 10.1007/s11912-018-0693-y
Gopalakrishnan, 2018, Immune checkpoint inhibitors in urothelial cancer: recent updates and future outlook, Ther. Clin. Risk Manag., 14, 1019, 10.2147/TCRM.S158753
Balar, 2019, Keynote 057: phase II trial of Pembrolizumab (pembro) for patients (pts) with high-risk (HR) nonmuscle invasive bladder cancer (NMIBC) unresponsive to bacillus calmette-guérin (BCG)., J. Clin. Oncol., 37, 10.1200/JCO.2019.37.7_suppl.350
U.S. Food and Drug Administration, FDA Approves Pembrolizumab for BCG-Unresponsive, High-Risk Non-Muscle Invasive Bladder Cancer, (2020). https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-pembrolizumab-bcg-unresponsive-high-risk-non-muscle-invasive-bladder-cancer (accessed January 24, 2020).
U.S Food and Drug Administration, FDA Grants Accelerated Approval to Erdafitinib for Metastatic Urothelial Carcinoma, (2019). https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-erdafitinib-metastatic-urothelial-carcinoma (accessed January 24, 2020).
Loriot, 2019, Erdafitinib in locally advanced or metastatic urothelial carcinoma, N. Engl. J. Med., 381, 338, 10.1056/NEJMoa1817323
Rosenberg, 2019, Pivotal trial of enfortumab vedotin in urothelial carcinoma after platinum and anti-programmed death 1/programmed death ligand 1 therapy, J. Clin. Oncol., 10.1200/JCO.19.01140
U.S. Food and Drug Administration, FDA Grants Accelerated Approval to Enfortumab Vedotin-EJFV for Metastatic Urothelial Cancer, (2019). https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-enfortumab-vedotin-ejfv-metastatic-urothelial-cancer (accessed January 24, 2020).
Mendiratta, 2018, Emerging biomarkers and targeted therapies in urothelial carcinoma, Ann. Transl. Med., 6, 10.21037/atm.2018.05.49
Ballman, 2015, Biomarker: predictive or prognostic?, J. Clin. Oncol., 33, 3968, 10.1200/JCO.2015.63.3651
Burke, 2016, Predicting clinical outcomes using molecular biomarkers, Biomark. Cancer, 8, 10.4137/BIC.S33380
Schiff, 2019, Precision therapy in advanced urothelial cancer, Expert Rev. Precis. Med. Drug Dev., 4, 81, 10.1080/23808993.2019.1582298
Tripathi, 2020, The utility of next generation sequencing in advanced urothelial carcinoma, Eur. Urol. Focus., 6, 41, 10.1016/j.euf.2019.08.016
Grivas, 2019, Conceptual framework for therapeutic development beyond anti-PD-1/PD-L1 in urothelial cancer, Am. Soc. Clin. Oncol. Educ. Book Am. Soc. Clin. Oncol. Annu. Meet., 39, 284, 10.1200/EDBK_237449
Lindgren, 2010, Combined gene expression and genomic profiling define two intrinsic molecular subtypes of urothelial carcinoma and gene signatures for molecular grading and outcome, Cancer Res., 70, 3463, 10.1158/0008-5472.CAN-09-4213
Sjödahl, 2012, A molecular taxonomy for urothelial carcinoma, Clin. Cancer Res., 18, 3377, 10.1158/1078-0432.CCR-12-0077-T
Marzouka, 2018, A validation and extended description of the Lund taxonomy for urothelial carcinoma using the TCGA cohort, Sci. Rep., 8
Damrauer, 2014, Intrinsic subtypes of high-grade bladder cancer reflect the hallmarks of breast cancer biology, Proc. Natl. Acad. Sci. U. S. A., 111, 3110, 10.1073/pnas.1318376111
Choi, 2014, Identification of distinct basal and luminal subtypes of muscle-invasive bladder cancer with different sensitivities to frontline chemotherapy, Cancer Cell, 25, 152, 10.1016/j.ccr.2014.01.009
Mo, 2018, Prognostic power of a tumor differentiation gene signature for bladder urothelial carcinomas, J. Natl. Cancer Inst., 110, 448, 10.1093/jnci/djx243
Weinstein, 2014, Comprehensive molecular characterization of urothelial bladder carcinoma, Nature, 507, 315, 10.1038/nature12965
Robertson, 2017, Comprehensive molecular characterization of muscle-invasive bladder cancer, Cell, 171, 540, 10.1016/j.cell.2017.09.007
Rebouissou, 2014, EGFR as a potential therapeutic target for a subset of muscle-invasive bladder cancers presenting a basal-like phenotype, Sci. Transl. Med., 6
Dadhania, 2016, Meta-analysis of the luminal and basal subtypes of bladder cancer and the identification of signature immunohistochemical markers for clinical use, EBioMedicine, 12, 105, 10.1016/j.ebiom.2016.08.036
Batista da Costa, 2019, Molecular characterization of neuroendocrine-like bladder cancer, Clin. Cancer Res., 25, 3908, 10.1158/1078-0432.CCR-18-3558
Grivas, 2019, Validation of a neuroendocrine-like classifier confirms poor outcomes in patients with bladder cancer treated with cisplatin-based neoadjuvant chemotherapy, Urol. Oncol. Semin. Orig. Investig.
Seiler, 2017, Impact of molecular subtypes in muscle-invasive bladder cancer on predicting response and survival after neoadjuvant chemotherapy [figure presented], Eur. Urol., 72, 544, 10.1016/j.eururo.2017.03.030
Seiler, 2017, Muscle-invasive bladder cancer: molecular subtypes and response to neoadjuvant chemotherapy, J. Clin. Oncol., 35, 10.1200/JCO.2017.35.6_suppl.281
Rosenberg, 2016, Atezolizumab in patients with locally advanced and metastatic urothelial carcinoma who have progressed following treatment with platinum-based chemotherapy: a single-arm, multicentre, phase 2 trial, Lancet, 387, 1909, 10.1016/S0140-6736(16)00561-4
Kamoun, 2019, A consensus molecular classification of muscle-invasive bladder cancer, Eur. Urol.
Mouw, 2017, DNA repair pathway alterations in bladder cancer, Cancers Basel, 9
Yoshida, 2019, Predictive biomarkers for drug response in bladder cancer, Int. J. Urol., 10.1111/iju.14082
Miyamoto, 2018, Molecular biomarkers in bladder preservation therapy for muscle-invasive bladder cancer, Lancet Oncol., 19, e683, 10.1016/S1470-2045(18)30693-4
Bellmunt, 2007, Gene expression of ERCC1 as a novel prognostic marker in advanced bladder cancer patients receiving cisplatin-based chemotherapy, Ann. Oncol. Off. J. Eur. Soc. Med. Oncol., 18, 522, 10.1093/annonc/mdl435
Mullane, 2016, Expression levels of DNA damage repair proteins are associated with overall survival in platinum-treated advanced urothelial carcinoma, Clin. Genitourin. Cancer, 14, 352, 10.1016/j.clgc.2015.12.029
Sakano, 2013, ERCC1 and XRCC1 expression predicts survival in bladder cancer patients receiving combined trimodality therapy, Mol. Clin. Oncol., 1, 403, 10.3892/mco.2013.85
Klatte, 2015, ERCC1 as a prognostic and predictive biomarker for urothelial carcinoma of the bladder following radical cystectomy, J. Urol., 194, 1456, 10.1016/j.juro.2015.06.099
Choueiri, 2014, Neoadjuvant dose-dense methotrexate, vinblastine, doxorubicin, and cisplatin with pegfilgrastim support in muscle-invasive urothelial cancer: pathologic, radiologic, and biomarker correlates, J. Clin. Oncol., 32, 1889, 10.1200/JCO.2013.52.4785
Urun, 2017, ERCC1 as a prognostic factor for survival in patients with advanced urothelial cancer treated with platinum based chemotherapy: a systematic review and meta-analysis, Crit. Rev. Oncol. Hematol., 120, 120, 10.1016/j.critrevonc.2017.10.012
Van Allen, 2014, Somatic ERCC2 mutations correlate with cisplatin sensitivity in muscle-invasive urothelial carcinoma, Cancer Discov., 4, 1140, 10.1158/2159-8290.CD-14-0623
Li, 2019, ERCC2 helicase domain mutations confer nucleotide excision repair deficiency and drive cisplatin sensitivity in muscle-invasive bladder cancer, Clin. Cancer Res., 25, 977, 10.1158/1078-0432.CCR-18-1001
Liu, 2016, Clinical validation of chemotherapy response biomarker ERCC2 in muscle-invasive urothelial bladder carcinoma, JAMA Oncol., 2, 1094, 10.1001/jamaoncol.2016.1056
Pietzak, 2019, Genomic differences between “primary” and “secondary” muscle-invasive bladder cancer as a basis for disparate outcomes to cisplatin-based neoadjuvant chemotherapy, Eur. Urol., 75, 231, 10.1016/j.eururo.2018.09.002
Iyer, 2018, Multicenter prospective phase ii trial of neoadjuvant dose-dense gemcitabine plus cisplatin in patients with muscle-invasive bladder cancer, J. Clin. Oncol., 36, 1949, 10.1200/JCO.2017.75.0158
Plimack, 2015, Defects in DNA repair genes predict response to neoadjuvant cisplatin-based chemotherapy in muscle-invasive bladder cancer, Eur. Urol., 68, 959, 10.1016/j.eururo.2015.07.009
Lorenzo-Romero, 2003, Prognostic implications of p53 gene mutations in bladder tumors., J. Urol., 169, 492, 10.1016/S0022-5347(05)63941-9
Kuczyk, 1995, p53 overexpression as a prognostic factor for advanced stage bladder cancer, Eur. J. Cancer, 31, 2243, 10.1016/0959-8049(95)00443-2
Edelman, 2000, Phase I/II study of paclitaxel, carboplatin, and methotrexate in advanced transitional cell carcinoma: a well-tolerated regimen with activity independent of p53 mutation, Urology, 55, 521, 10.1016/S0090-4295(99)00538-5
Sarkis, 1995, Prognostic value of p53 nuclear overexpression in patients with invasive bladder cancer treated with neoadjuvant MVAC., J. Clin. Oncol., 13, 1384, 10.1200/JCO.1995.13.6.1384
Cote, 1997, p53 and treatment of bladder cancer, Nature, 385, 123, 10.1038/385123b0
Seiler, 2015, Association of p53-ness with chemo-resistance in urothelial cancers treated with neoadjuvant gemcitabine plus cisplatin., J. Clin. Oncol., 33, 10.1200/jco.2015.33.15_suppl.4512
Choudhury, 2010, MRE11 expression is predictive of cause-specific survival following radical radiotherapy for muscle-invasive bladder cancer, Cancer Res., 70, 7017, 10.1158/0008-5472.CAN-10-1202
Laurberg, 2012, Expression of TIP60 (tat-interactive protein) and MRE11 (meiotic recombination 11 homolog) predict treatment-specific outcome of localised invasive bladder cancer, BJU Int., 110, E1228, 10.1111/j.1464-410X.2012.11564.x
Teo, 2017, DNA damage response and repair gene alterations are associated with improved survival in patients with platinum-treated advanced urothelial carcinoma, Clin. Cancer Res., 23, 3610, 10.1158/1078-0432.CCR-16-2520
Alliance for Clinical Trials in Oncology, Dose Dense Gemcitabine and Cisplatin Without Cystectomy for Patients With Muscle Invasive Bladder Urothelial Cancer and Select Genetic Alterations, (2018). https://clinicaltrials.gov/ct2/show/study/NCT03609216 (accessed April 12, 2020).
Geynisman, 2018, A phase II trial of risk-adapted treatment for muscle invasive bladder cancer after neoadjuvant accelerated MVAC., J. Clin. Oncol., 36, 10.1200/JCO.2018.36.6_suppl.TPS537
M. Galsky, Gemcitabine, Cisplatin, Plus Nivolumab in Patients With Muscle-invasive Bladder Cancer With Selective Bladder Sparing, (2018). https://clinicaltrials.gov/ct2/show/NCT03558087 (accessed January 25, 2020).
National Cancer Institute, Olaparib in Treating Patients With Metastatic or Advanced Urothelial Cancer With DNA-Repair Defects, (2017). https://clinicaltrials.gov/ct2/show/NCT03375307 (accessed January 25, 2020).
AstraZeneca, A Study of Durvalumab Alone and Durvalumab+Olaparib in Advanced, Platinum-Ineligible Bladder Cancer (BAYOU), (2018). https://clinicaltrials.gov/ct2/show/NCT03459846 (accessed January 25, 2020).
T.B. Powles, A. Balar, G. Gravis, R. Jones, A. Ravaud, J. Florence, P. Grivas, D. Petrylak, M. Galsky, J. Carles, S. Sridhar, H. Arkenau, D. Carroll, J. DeCesare, F. Mercier, D. Hodgson, J. Stone, J. Cosaert, D. Landers, An Adaptive, Biomarker Directed Platform Study In Metastatic Urothelial Cancer (Biscay) With Durvalumab In Combination With Targeted Therapies, (2019). https://oncologypro.esmo.org/meeting-resources/esmo-2019-congress/An-adaptive-biomarker-directed-platform-study-in-metastatic-urothelial-cancer-BISCAY-with-durvalumab-in-combination-with-targeted-therapies (accessed April 8, 2020).
Grivas, 2020, Rucaparib for recurrent, locally advanced, or metastatic urothelial carcinoma (mUC): results from ATLAS, a phase II open-label trial., J. Clin. Oncol., 38, 10.1200/JCO.2020.38.6_suppl.440
Helsten, 2016, The FGFR landscape in cancer: analysis of 4,853 tumors by next-generation sequencing, Clin. Cancer Res., 22, 259, 10.1158/1078-0432.CCR-14-3212
Moss, 2017, Comprehensive genomic characterization of upper tract urothelial carcinoma, Eur. Urol., 72, 641, 10.1016/j.eururo.2017.05.048
Hernández, 2006, Prospective study of FGFR3 mutations as a prognostic factor in nonmuscle invasive urothelial bladder carcinomas, J. Clin. Oncol., 24, 3664, 10.1200/JCO.2005.05.1771
Milowsky, 2014, Phase 2 trial of dovitinib in patients with progressive FGFR3-mutated or FGFR3 wild-type advanced urothelial carcinoma, Eur. J. Cancer, 50, 3145, 10.1016/j.ejca.2014.10.013
Janssen Research & Development LLC, A Study of Erdafitinib Compared With Vinflunine or Docetaxel or Pembrolizumab in Participants With Advanced Urothelial Cancer and Selected Fibroblast Growth Factor Receptor (FGFR) Gene Aberrations, (2018). https://clinicaltrials.gov/ct2/show/NCT03390504 (accessed January 26, 2020).
Pal, 2018, Efficacy of BGJ398, a fibroblast growth factor receptor 1–3 inhibitor, in patients with previously treated advanced urothelial carcinoma with FGFR3 alterations, Cancer Discov., 8, 812, 10.1158/2159-8290.CD-18-0229
Nogova, 2017, Evaluation of BGJ398, a Fibroblast growth factor receptor 1-3 kinase inhibitor, in patientswith advanced solid tumors harboring genetic alterations in fibroblast growth factor receptors: results of a global phase I, dose-escalation and dose-expansion study, J. Clin. Oncol., 35, 157, 10.1200/JCO.2016.67.2048
Kilgour, 2014, Exploratory biomarker analysis of a phase I study of AZD4547, an inhibitor of fibroblast growth factor receptor (FGFR), in patients with advanced solid tumors., J. Clin. Oncol., 32, 10.1200/jco.2014.32.15_suppl.11010
A. Necchi, D. Pouessel, R. Leibowitz-Amit, A. Flechon, S. Gupta, P. Barthelemy, M. Maio, X. Zhu, E. Asatiani, G. Serbest, H. Zhen, Y. Loriot, Interim Results of Fight-201, A Phase II, Open-Label, Multicenter Study of INCB054828 in Patients (PTS) with Metastatic or Surgically Unresectable Urothelial Carcinoma (UC) Harboring Fibroblast Growth Factor (FGF)/FGF Receptor (FGFR) Genetic Alterations, (2018). https://www.annalsofoncology.org/article/S0923-7534(19)49289-2/pdf (accessed April 12, 2020).
Schuler, 2019, Rogaratinib in patients with advanced cancers selected by FGFR mRNA expression: a phase 1 dose-escalation and dose-expansion study, Lancet Oncol., 10.1016/S1470-2045(19)30412-7
Voss, 2017, Debio 1347, an oral FGFR inhibitor: results from a first-in-human, phase I dose-escalation study in patients with FGFR genomically activated advanced solid tumors., J. Clin. Oncol., 35, 10.1200/JCO.2017.35.15_suppl.2500
Bellmunt, 2017, Safety and efficacy of docetaxel + b-701, a selective inhibitor of FGFR3, in subjects with advanced or metastatic urothelial carcinoma., J. Clin. Oncol., 35, 10.1200/JCO.2017.35.15_suppl.4540
ODonnell, 2012, 621 A Phase I Dose-escalation Study of MFGR1877S, a Human Monoclonal Anti-fibroblast Growth Factor Receptor 3 (FGFR3) Antibody, in Patients (pts) with Advanced Solid Tumors, Eur. J. Cancer, 48, 191, 10.1016/S0959-8049(12)72418-8
QED Therapeutics Inc., Study of Oral Infigratinib for the Adjuvant Treatment of Subjects With Invasive Urothelial Carcinoma With Susceptible FGFR3 Genetic Alterations, (2019). https://clinicaltrials.gov/ct2/show/NCT04197986 (accessed January 28, 2020).
Dai, 2019, Fibroblast growth factor receptors (FGFRs): structures and small molecule inhibitors, Cells, 8, 614, 10.3390/cells8060614
Dizman, 2019, Infigratinib in upper tract urothelial carcinoma vs urothelial carcinoma of the bladder and association with comprehensive genomic profiling/cell-free DNA results., J. Clin. Oncol., 37, 10.1200/JCO.2019.37.15_suppl.4510
Bellmunt, 2017, Antiangiogenesis to curb urothelial cancer, Lancet, 390, 2220, 10.1016/S0140-6736(17)32388-7
Shariat, 2010, Association of angiogenesis related markers with bladder cancer outcomes and other molecular markers, J. Urol., 183, 1744, 10.1016/j.juro.2010.01.018
Lautenschlaeger, 2013, Bladder preservation therapy for muscle-invading bladder cancers on radiation therapy oncology group trials 8802, 8903, 9506, and 9706: vascular endothelial growth factor B overexpression predicts for increased distant metastasis and shorter survival, Oncologist, 18, 685, 10.1634/theoncologist.2012-0461
Keck, 2015, Neuropilin-2 and its ligand VEGF-C predict treatment response after transurethral resection and radiochemotherapy in bladder cancer patients, Int. J. Cancer, 136, 443, 10.1002/ijc.28987
Petrylak, 2020, Ramucirumab plus docetaxel versus placebo plus docetaxel in patients with locally advanced or metastatic urothelial carcinoma after platinum-based therapy (RANGE): overall survival and updated results of a randomised, double-blind, phase 3 trial, Lancet Oncol., 21, 105, 10.1016/S1470-2045(19)30668-0
Rosenberg, 2019, CALGB 90601 (Alliance): randomized, double-blind, placebo-controlled phase III trial comparing gemcitabine and cisplatin with bevacizumab or placebo in patients with metastatic urothelial carcinoma., J. Clin. Oncol., 37, 10.1200/JCO.2019.37.15_suppl.4503
Choueiri, 2012, Double-blind, randomized trial of docetaxel plus vandetanib versus docetaxel plus placebo in platinum-pretreated metastatic urothelial cancer, J. Clin. Oncol., 30, 507, 10.1200/JCO.2011.37.7002
Dreicer, 2009, Phase 2 trial of sorafenib in patients with advanced urothelial cancer: a trial of the eastern cooperative oncology group, Cancer, 115, 4090, 10.1002/cncr.24467
Sridhar, 2011, A phase II trial of sorafenib in first-line metastatic urothelial cancer: a study of the PMH phase II consortium, Invest. New Drugs, 29, 1045, 10.1007/s10637-010-9408-4
Bellmunt, 2011, Phase II study of sunitinib as first-line treatment of urothelial cancer patients ineligible to receive cisplatin-based chemotherapy: baseline interleukin-8 and tumor contrast enhancement as potential predictive factors of activity, Ann. Oncol., 22, 2646, 10.1093/annonc/mdr023
Gallagher, 2011, Sunitinib in urothelial cancer: clinical, pharmacokinetic, and immunohistochemical study of predictors of response, Eur. Urol., 60, 344, 10.1016/j.eururo.2011.05.034
Jones, 2017, Randomized phase II study investigating pazopanib versus weekly paclitaxel in relapsed or progressive urothelial cancer, J. Clin. Oncol., 35, 1770, 10.1200/JCO.2016.70.7828
Geldart, 2015, SUCCINCT: an open-label, single-arm, non-randomised, phase 2 trial of gemcitabine and cisplatin chemotherapy in combination with sunitinib as first-line treatment for patients with advanced urothelial carcinoma, Eur. Urol., 67, 599, 10.1016/j.eururo.2014.11.003
Grivas, 2014, Double-blind, randomized, phase 2 trial of maintenance sunitinib versus placebo after response to chemotherapy in patients with advanced urothelial carcinoma, Cancer, 120, 692, 10.1002/cncr.28477
Apolo, 2017, A phase I study of cabozantinib plus nivolumab (CaboNivo) and ipilimumab (CaboNivoIpi) in patients (pts) with refractory metastatic urothelial carcinoma (mUC) and other genitourinary (GU) tumors., J. Clin. Oncol., 35
Herbst, 2019, Ramucirumab plus pembrolizumab in patients with previously treated advanced non-small-cell lung cancer, gastro-oesophageal cancer, or urothelial carcinomas (JVDF): a multicohort, non-randomised, open-label, phase 1a/b trial, Lancet Oncol., 20, 1109, 10.1016/S1470-2045(19)30458-9
Vogelzang, 2019, Phase Ib/II trial of lenvatinib plus pembrolizumab in urothelial cancer., J. Clin. Oncol., 37, 10.1200/JCO.2019.37.8_suppl.11
Mirati Therapeutics Inc., Mirati Therapeutics Announces Presentation Of Interim Phase 2 Sitravatinib Data In Urothelial Carcinoma And Oral Cavity Squamous Cell Carcinoma At The SITC 34th Annual Meeting, (2019). https://ir.mirati.com/news-releases/news-details/2019/Mirati-Therapeutics-Announces-Presentation-Of-Interim-Phase-2-Sitravatinib-Data-In-Urothelial-Carcinoma-And-Oral-Cavity-Squamous-Cell-Carcinoma-At-The-SITC-34th-Annual-Meeting/default.aspx (accessed January 28, 2020).
A. Balar, Atezolizumab With Bevacizumab in Previously Untreated Metastatic/Unresectable Urothelial Cancer, (2017). https://clinicaltrials.gov/ct2/show/NCT03272217 (accessed January 28, 2020).
Merck Sharp and Dohme Corp, Study of First-line Pembrolizumab (MK-3475) With Lenvatinib (MK-7902/E7080) in Urothelial Carcinoma Cisplatin-ineligible Participants Whose Tumors Express Programmed Cell Death-Ligand 1 and in Participants Ineligible for Platinum-containing Chemotherapy, (2019). https://clinicaltrials.gov/ct2/show/NCT03898180 (accessed April 8, 2020).
Hussain, 2014, A randomized phase 2 trial of gemcitabine/cisplatin with or without cetuximab in patients with advanced urothelial carcinoma, Cancer, 120, 2684, 10.1002/cncr.28767
Grivas, 2019, Role of targeted therapies in management of metastatic urothelial cancer in the era of immunotherapy, Curr. Treat. Options Oncol., 20, 67, 10.1007/s11864-019-0665-y
Hussain, 2007, National Cancer Institute, Trastuzumab, paclitaxel, carboplatin, and gemcitabine in advanced human epidermal growth factor receptor-2/neu-positive urothelial carcinoma: results of a multicenter phase II National Cancer Institute trial., J. Clin. Oncol., 25, 2218, 10.1200/JCO.2006.08.0994
Oudard, 2015, Multicentre randomised phase II trial of gemcitabine + platinum, with or without trastuzumab, in advanced or metastatic urothelial carcinoma overexpressing Her2, Eur. J. Cancer, 51, 45, 10.1016/j.ejca.2014.10.009
Ahn, 2019, HER2 status in breast cancer: changes in guidelines and complicating factors for interpretation, J. Pathol. Transl. Med., 0, 0
Bryce, 2017, Pertuzumab plus trastuzumab for HER2-positive metastatic urothelial cancer (mUC): preliminary data from MyPathway., J. Clin. Oncol., 35, 10.1200/JCO.2017.35.6_suppl.348
Sheng, 2019, A phase II study of RC48-ADC in HER2-positive patients with locally advanced or metastatic urothelial carcinoma., J. Clin. Oncol., 37, 10.1200/JCO.2019.37.15_suppl.4509
Daiichi Sankyo Inc., Trastuzumab Deruxtecan (DS-8201a) With Nivolumab in Advanced Breast and Urothelial Cancer, (2018). https://clinicaltrials.gov/ct2/show/NCT03523572 (accessed January 28, 2020).
Jhaveri, 2019, Ado-trastuzumab emtansine (T-DM1) in patients with HER2-amplified tumors excluding breast and gastric/gastroesophageal junction (GEJ) adenocarcinomas: results from the NCI-MATCH trial (EAY131) subprotocol Q, Ann. Oncol. Off. J. Eur. Soc. Med. Oncol., 30, 1821, 10.1093/annonc/mdz291
Wülfing, 2009, A single-arm, multicenter, open-label phase 2 study of lapatinib as the second-line treatment of patients with locally advanced or metastatic transitional cell carcinoma, Cancer, 115, 2881, 10.1002/cncr.24337
Powles, 2017, Phase III, double-blind, randomized trial that compared maintenance lapatinib versus placebo after first-line chemotherapy in patients with human epidermal growth factor receptor 1/2-positive metastatic bladder cancer., J. Clin. Oncol., 35, 48, 10.1200/JCO.2015.66.3468
Hyman, 2018, HER kinase inhibition in patients with HER2- and HER3-mutant cancers, Nature, 554, 189, 10.1038/nature25475
Bedard, 2019, Abstract CT139: NCI Molecular Analysis for Therapy Choice (NCI-MATCH EAY131) arm B: phase II study of afatinib in patients (pts) with HER2 (ERBB2) activating mutations, Clin. Trials, 10.1158/1538-7445.AM2019-CT139
Choudhury, 2016, Afatinib activity in platinum-refractory metastatic urothelial carcinoma in patients with ERBB alterations, J. Clin. Oncol., 34, 2165, 10.1200/JCO.2015.66.3047
Iyer, 2012, Supplementary materials for genome sequencing identifies a basis for everolimus sensitivity, Science, 80
Milowsky, 2013, Phase II study of everolimus in metastatic urothelial cancer, BJU Int., 112, 462, 10.1111/j.1464-410X.2012.11720.x
Bellmunt, 2018, Everolimus and pazopanib (E/P) benefit genomically selected patients with metastatic urothelial carcinoma, Br. J. Cancer, 119, 707, 10.1038/s41416-018-0261-0
National Cancer Institute, Targeted Therapy Directed by Genetic Testing in Treating Patients With Advanced Refractory Solid Tumors, Lymphomas, or Multiple Myeloma (The MATCH Screening Trial), (2015). https://clinicaltrials.gov/ct2/show/NCT02465060 (accessed January 24, 2020).
American Society of Clinical Oncology, TAPUR: testing the Use of Food and Drug Administration (FDA) Approved Drugs That Target a Specific Abnormality in a Tumor Gene in People With Advanced Stage Cancer, (2016). https://clinicaltrials.gov/ct2/show/NCT02693535 (accessed April 8, 2020).
ECOG-ACRIN, NCI-ComboMATCH Precision Medicine Cancer Trial, (2020). https://ecog-acrin.org/nci-combomatch (accessed April 8, 2020).
Rodriguez-Vida, 2018, Immunotherapy combinations and sequences in urothelial cancer: facts and hopes, Clin. Cancer Res., 24, 6115, 10.1158/1078-0432.CCR-17-3108
Shindo, 2019, Novel biomarkers for personalized cancer immunotherapy, Cancers Basel, 11, 1223, 10.3390/cancers11091223
van Dijk, 2019, The cancer immunogram as a framework for personalized immunotherapy in urothelial cancer, Eur. Urol., 75, 435, 10.1016/j.eururo.2018.09.022
Alifrangis, 2019, Molecular and histopathology directed therapy for advanced bladder cancer, Nat. Rev. Urol., 10.1038/s41585-019-0208-0
Powles, 2018, Atezolizumab versus chemotherapy in patients with platinum-treated locally advanced or metastatic urothelial carcinoma (IMvigor211): a multicentre, open-label, phase 3 randomised controlled trial, Lancet, 391, 748, 10.1016/S0140-6736(17)33297-X
Sharma, 2017, Nivolumab in metastatic urothelial carcinoma after platinum therapy (CheckMate 275): a multicentre, single-arm, phase 2 trial, Lancet Oncol, 18, 312, 10.1016/S1470-2045(17)30065-7
Patel, 2018, Avelumab in metastatic urothelial carcinoma after platinum failure (JAVELIN Solid Tumor): pooled results from two expansion cohorts of an open-label, phase 1 trial, Lancet Oncol., 19, 51, 10.1016/S1470-2045(17)30900-2
Powles, 2017, [Supplemental content] Efficacy and safety of durvalumab in locally advanced or metastatic urothelial carcinoma, JAMA Oncol., 3, 10.1001/jamaoncol.2017.2411
Bellmunt, 2017, Pembrolizumab as second-line therapy for advanced urothelial carcinoma, N. Engl. J. Med., 376, 1015, 10.1056/NEJMoa1613683
Fradet, 2019, Randomized phase III KEYNOTE-045 trial of pembrolizumab versus paclitaxel, docetaxel, or vinflunine in recurrent advanced urothelial cancer: results of > 2 years of follow-up, Ann. Oncol. Off. J. Eur. Soc. Med. Oncol., 10.1093/annonc/mdz127
Balar, 2017, First-line pembrolizumab in cisplatin-ineligible patients with locally advanced and unresectable or metastatic urothelial cancer (KEYNOTE-052): a multicentre, single-arm, phase 2 study, Lancet Oncol., 18, 1483, 10.1016/S1470-2045(17)30616-2
Balar, 2017, Atezolizumab as first-line treatment in cisplatin-ineligible patients with locally advanced and metastatic urothelial carcinoma: a single-arm, multicentre, phase 2 trial, Lancet, 389, 67, 10.1016/S0140-6736(16)32455-2
Suzman, 2019, FDA Approval summary: atezolizumab or pembrolizumab for the treatment of patients with advanced urothelial carcinoma ineligible for cisplatin-containing chemotherapy, Oncologist, 24, 563, 10.1634/theoncologist.2018-0084
European Medicines Agency, European Medicines Agency restricts use of Keytruda and Tecentriq in bladder cancer, (2018). https://www.ema.europa.eu/en/news/ema-restricts-use-keytruda-tecentriq-bladder-cancer (accessed January 26, 2020).
E. Grande, M. Galsky, J.A.A. Arija, M. De Santis, .D. Davis, U.F.F. De Giorgi, M. Mencinger, E. Kikuchi, X.G. del Muro, M. Gumus, M. Özgüroğlu, A.R. Kalebasty, S.H. Park, B.Y. Alekseev, F.A.B. Schutz, J.-.R. Li, A. Mecke, S. Mariathasan, A. Thastrom, A. Bamias, LBA14_PR - IMvigor130: Efficacy and Safety From a Phase III Study of Atezolizumab (atezo) as Monotherapy or in Combination With Platinum-Based Chemotherapy (PBC) vs Placebo + PBC in Previously Untreated Locally Advanced or Metastatic Urothelial Carcinoma, (2019). https://www.esmo.org/Press-Office/Press-Releases/ESMO-Congress-bladder-cancer-immunotherapy-imvigor130-grande (accessed January 26, 2020).
Merck Sharp and Dohme Corp, Study of Pembrolizumab With or Without Platinum-based Combination Chemotherapy Versus Chemotherapy Alone in Urothelial Carcinoma (MK-3475-361/KEYNOTE-361), (2016). https://clinicaltrials.gov/ct2/show/NCT02853305 (accessed April 13, 2020).
Bristol-Myers Squibb, Study of Nivolumab in Combination With Ipilimumab or Standard of Care Chemotherapy Compared to the Standard of Care Chemotherapy Alone in Treatment of Patients With Untreated Inoperable or Metastatic Urothelial Cancer, (2017). https://clinicaltrials.gov/ct2/show/NCT03036098 (accessed April 13, 2020).
Gandhi, 2018, Pembrolizumab plus chemotherapy in metastatic non-small-cell lung cancer, N. Engl. J. Med., 378, 2078, 10.1056/NEJMoa1801005
Arbour, 2019, Systemic therapy for locally advanced and metastatic non-small cell lung cancer: a review, J. Am. Med. Assoc., 322, 764, 10.1001/jama.2019.11058
AstraZeneca, Update on Phase III DANUBE Trial for Imfinzi and Tremelimumab in Unresectable, Stage IV Bladder Cancer, (2020). https://www.astrazeneca.com/media-centre/press-releases/2020/update-on-phase-iii-danube-trial-for-imfinzi-and-tremelimumab-in-unresectable-stage-iv-bladder-cancer-06032020.html (accessed April 8, 2020).
The ASCO Post, JAVELIN Bladder 100 Study of Avelumab for Urothelial Cancer Meets Primary Endpoint, (2020). https://www.ascopost.com/issues/january-25-2020/javelin-bladder-100-study-of-avelumab-for-urothelial-cancer-meets-primary-endpoint/ (accessed April 8, 2020).
Mariathasan, 2018, TGFβ attenuates tumour response to PD-L1 blockade by contributing to exclusion of T cells, Nature, 554, 544, 10.1038/nature25501
Funt, 2016, Correlation of peripheral and intratumoral T-cell receptor (TCR) clonality with clinical outcomes in patients with metastatic urothelial cancer (mUC) treated with atezolizumab., J. Clin. Oncol., 34, 10.1200/JCO.2016.34.15_suppl.3005
Grivas, 2019, Association between stromal/TGF-β/EMT gene expression signature and response to pembrolizumab monotherapy in cisplatin-ineligible patients with locally advanced (unresectable) or metastatic urothelial carcinoma., J. Clin. Oncol., 37, 10.1200/JCO.2019.37.7_suppl.433
Wang, 2018, EMT- and stroma-related gene expression and resistance to PD-1 blockade in urothelial cancer, Nat. Commun., 9
Mouw, 2017, DNA damage and repair biomarkers of immunotherapy response, Cancer Discov., 7, 675, 10.1158/2159-8290.CD-17-0226
Chan, 2019, Development of tumor mutation burden as an immunotherapy biomarker: utility for the oncology clinic, Ann. Oncol., 30, 44, 10.1093/annonc/mdy495
Iyer, 2017, Mismatch repair (MMR) detection in urothelial carcinoma (UC) and correlation with immune checkpoint blockade (ICB) response., J. Clin. Oncol., 35, 10.1200/JCO.2017.35.15_suppl.4511
Teo, 2018, Alterations in DNA damage response and repair genes as potential marker of clinical benefit from PD-1/PD-L1 blockade in advanced urothelial cancers, J. Clin. Oncol., 36, 1685, 10.1200/JCO.2017.75.7740
Galsky, 2018, Phase 2 trial of gemcitabine, cisplatin, plus ipilimumab in patients with metastatic urothelial cancer and impact of DNA damage response gene mutations on outcomes, Eur. Urol., 73, 751, 10.1016/j.eururo.2017.12.001
Necchi, 2020, Comprehensive assessment of immuno-oncology biomarkers in adenocarcinoma, urothelial carcinoma, and squamous-cell carcinoma of the bladder, Eur. Urol., 10.1016/j.eururo.2020.01.003
Zahoor, 2019, The cancer immunogram: a pledge for a comprehensive biomarker approach for personalized immunotherapy in urothelial cancer, Eur. Urol., 75, 445, 10.1016/j.eururo.2018.12.005
Powles, 2019, Clinical efficacy and biomarker analysis of neoadjuvant atezolizumab in operable urothelial carcinoma in the ABACUS trial, Nat. Med., 25, 1706, 10.1038/s41591-019-0628-7
Necchi, 2018, Pembrolizumab as neoadjuvant therapy before radical cystectomy in patients with muscle-invasive urothelial bladder carcinoma (PURE-01): an open-label, single-arm, phase II study, J. Clin. Oncol., 36, 3353, 10.1200/JCO.18.01148
Ayers, 2019, Molecular profiling of cohorts of tumor samples to guide clinical development of pembrolizumab as monotherapy, Clin. Cancer Res., 25, 1564, 10.1158/1078-0432.CCR-18-1316
Komiya, 2018, Updates in the clinical development of Epacadostat and other indoleamine 2,3-dioxygenase 1 inhibitors (IDO1) for human cancers, Front. Oncol., 8
Smith, 2017, Epacadostat plus pembrolizumab in patients with advanced urothelial carcinoma: preliminary phase I/II results of ECHO-202/KEYNOTE-037., J. Clin. Oncol., 35, 10.1200/JCO.2017.35.15_suppl.4503
Luke, 2019, BMS-986205, an indoleamine 2, 3-dioxygenase 1 inhibitor (IDO1i), in combination with nivolumab (nivo): updated safety across all tumor cohorts and efficacy in advanced bladder cancer (advBC)., J. Clin. Oncol., 37, 10.1200/JCO.2019.37.7_suppl.358
Long, 2019, Epacadostat plus pembrolizumab versus placebo plus pembrolizumab in patients with unresectable or metastatic melanoma (ECHO-301/KEYNOTE-252): a phase 3, randomised, double-blind study, Lancet Oncol., 20, 1083, 10.1016/S1470-2045(19)30274-8
Naing, 2018, Epacadostat plus durvalumab in patients with advanced solid tumors: preliminary results of the ongoing, open-label, phase I/II ECHO-203 study
Chu, 2020, Role of indoleamine-2,3-dioxygenase inhibitors in salvage therapy for non-muscle invasive bladder cancer, Urol. Clin. North Am., 47, 111, 10.1016/j.ucl.2019.09.013
Bristol-Myers Squibb, A Study of Chemo Only Versus Chemo Plus Nivo With or Without BMS-986205, Followed by Post- Surgery Therapy With Nivo or Nivo and BMS-986205 in Patients With MIBC, (2018). https://clinicaltrials.gov/ct2/show/NCT03661320 (accessed January 30, 2020).
Challita-Eid, 2016, Enfortumab vedotin antibody-drug conjugate targeting nectin-4 is a highly potent therapeutic agent in multiple preclinical cancer models, Cancer Res., 76, 3003, 10.1158/0008-5472.CAN-15-1313
Targeting Nectin-4 in Bladder Cancer, Cancer Discov. 7 (2017) OF3. doi: 10.1158/2159-8290.CD-NB2017-095.
Astellas Pharma Global Development Inc., A Study to Evaluate Enfortumab Vedotin Versus (vs) Chemotherapy in Subjects With Previously Treated Locally Advanced or Metastatic Urothelial Cancer (EV-301), (2018). https://clinicaltrials.gov/ct2/show/NCT03474107 (accessed April 12, 2020).
Petrylak, 2016, Anti-tumor activity, safety and pharmacokinetics (PK) of AGS15E (ASG-15ME) in a phase I dose escalation trial in patients (Pts) with metastatic urothelial cancer (mUC)., J. Clin. Oncol., 34, 10.1200/JCO.2016.34.15_suppl.4532
Tagawa, 2019, Sacituzumab govitecan (IMMU-132) in patients with previously treated metastatic urothelial cancer (mUC): results from a phase I/II study., J. Clin. Oncol., 37, 10.1200/JCO.2019.37.7_suppl.354
S.T. Tagawa, A. Balar, D.P. Petrylak, P. Grivas, N. Agarwal, C.N. Sternberg, Q. Hong, A. Gladden, C. Kanwal, P. Siemon-Hryczyk, T. Goswami, L.M. Itri, Y. Loriot, Initial Results From TROPHY-U-01: a Phase 2 Open-Label Study of Sacituzumab Govitecan in Patients (Pts) With Metastatic Urothelial Cancer (mUC) After Failure of Platinum-based Regimens (PLT) or Immunotherapy, (2019). https://oncologypro.esmo.org/Meeting-Resources/ESMO-2019-Congress/Initial-Results-From-TROPHY-U-01-A-Phase-2-Open-Label-Study-of-Sacituzumab-Govitecan-in-Patients-Pts-With-Metastatic-Urothelial-Cancer-mUC-After-Failure-of-Platinum-Based-Regimens-PLT-or-Im (accessed January 26, 2020).
Grivas, 2019, Mocetinostat for patients with previously treated, locally advanced/metastatic urothelial carcinoma and inactivating alterations of acetyltransferase genes, Cancer, 125, 533, 10.1002/cncr.31817
Ramakrishnan, 2019, Inhibition of EZH2 induces NK cell-mediated differentiation and death in muscle-invasive bladder cancer, Cell Death Differ., 10.1038/s41418-019-0278-9
Bitler, 2015, Synthetic lethality by targeting EZH2 methyltransferase activity in ARID1A-mutated cancers, Nat. Med., 21, 231, 10.1038/nm.3799
Martínez-Fernández, 2015, EZH2 in bladder cancer, a promising therapeutic target, Int. J. Mol. Sci., 16, 27107, 10.3390/ijms161126000
Christensen, 2019, Early detection of metastatic relapse and monitoring of therapeutic efficacy by ultra-deep sequencing of plasma cell-free DNA in patients with urothelial bladder carcinoma, J. Clin. Oncol., 37, 1547, 10.1200/JCO.18.02052
Grivas, 2019, Circulating tumor DNA alterations in advanced urothelial carcinoma and association with clinical outcomes: a pilot study, Eur. Urol. Oncol.
Kuderer, 2017, Comparison of 2 commercially available next-generation sequencing platforms in oncology, JAMA Oncol., 3, 996, 10.1001/jamaoncol.2016.4983
AstraZeneca, Prevalence of PD-L1 Expression in Patients With Advanced Urothelial Carcinoma (PREVAIL), (2018). https://clinicaltrials.gov/ct2/show/NCT03788746 (accessed January 26, 2020).
Caris Life Sciences, Caris Life Sciences Receives FDA Breakthrough Device Designation for MI TranscriptomeTM Companion Diagnostic Test, (2019). https://www.carislifesciences.com/news/caris-life-sciences-receives-fda-breakthrough-device-designation-for-mi-transcriptome-companion-diagnostic-test (accessed April 12, 2020).
Freedman, 2018, Use of next-generation sequencing tests to guide cancer treatment: results from a nationally representative survey of oncologists in the United States, JCO Precis. Oncol., 1
Klek, 2018, Genetic counseling (GC) and germline (GL) testing rates after adoption of an integrated clinical cancer genetics (CCG) approach to genomics tumor board (GTB), J. Clin. Oncol., 36, 10.1200/JCO.2018.36.15_suppl.1511
Nassar, 2019, Prevalence of pathogenic germline cancer risk variants in high-risk urothelial carcinoma, Genet. Med.
Carlo, 2017, Cancer predisposing germline mutations in patients (pts) with urothelial cancer (UC) of the renal pelvis (R-P), ureter (U) and bladder (B), J. Clin. Oncol., 35, 10.1200/JCO.2017.35.15_suppl.4510
Flaig, 2019, SWOG S1314: a randomized phase II study of co-expression extrapolation (COXEN) with neoadjuvant chemotherapy for localized, muscle-invasive bladder cancer, J. Clin. Oncol., 37, 10.1200/JCO.2019.37.15_suppl.4506
