Neoadjuvant therapy in muscle-invasive bladder cancer: A model for rational accelerated drug development

Urologic Clinics of North America

Volumn 42, Issue 2, 2015, Pages 217-224

  Balar, Arjun V ^a   Milowsky, Matthew I ^b  

^a NEW YORK UNIVERSITY MEDICAL CENTER   (United States)

^b UNIVERSITY OF NORTH CAROLINA   (United States)

Author keywords

Bladder cancer; Cisplatin; Immunotherapy; Neoadjuvant chemotherapy; Predictive biomarkers; The Cancer Genome Atlas; Urothelial cancer

Indexed keywords

BIOLOGICAL MARKER; CABAZITAXEL; CISPLATIN; DASATINIB; DOXORUBICIN; GEMCITABINE; METHOTREXATE; PACLITAXEL; PANITUMUMAB; VINBLASTINE; ANTINEOPLASTIC AGENT;

ADJUVANT THERAPY; ARTICLE; BLADDER BIOPSY; CANCER SURVIVAL; CLINICAL EFFECTIVENESS; CYSTECTOMY; DRUG EFFICACY; HUMAN; INTERMETHOD COMPARISON; MUSCLE INVASIVE BLADDER CANCER; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL (TOPIC); TRANSURETHRAL RESECTION; TREATMENT INDICATION; ADJUVANT CHEMOTHERAPY; DRUG DEVELOPMENT; NEOADJUVANT THERAPY; PATHOLOGY; PROCEDURES; TREATMENT OUTCOME; TUMOR INVASION; URINARY BLADDER NEOPLASMS;

ANTINEOPLASTIC COMBINED CHEMOTHERAPY PROTOCOLS; CHEMOTHERAPY, ADJUVANT; CISPLATIN; CYSTECTOMY; DOXORUBICIN; DRUG DISCOVERY; HUMANS; METHOTREXATE; NEOADJUVANT THERAPY; NEOPLASM INVASIVENESS; TREATMENT OUTCOME; URINARY BLADDER NEOPLASMS; VINBLASTINE;

EID: 84955372451     PISSN: 00940143     EISSN: 1558318X     Source Type: Journal    
DOI: 10.1016/j.ucl.2015.02.004     Document Type: Review

References (50)

1. Foxtrot Collaborative Group. Feasibility of preoperative chemotherapy for locally advanced, operable colon cancer: the pilot phase of a randomised controlled trial. Lancet Oncol 2012;13:1152-60.
2. Song WA, Zhou NK, Wang W, et al. Survival benefit of neoadjuvant chemotherapy in non-small cell lung cancer: an updated meta-analysis of 13 randomized control trials. J Thorac Oncol 2010;5:510-6.
3. Robova H, Rob L, Halaska MJ, et al. High-dose density neoadjuvant chemotherapy in bulky IB cervical cancer. Gynecol Oncol 2013;128:49-53.
4. Tajima H, Ohta T, Kitagawa H, et al. Pilot study of neoadjuvant chemotherapy with gemcitabine and oral S-1 for resectable pancreatic cancer. Exp Ther Med 2012;3:787-92.
5. Fisher B, Bryant J, Wolmark N, et al. Effect of preoperative chemotherapy on the outcome of women with operable breast cancer. J Clin Oncol 1998;16: 2672-85.
6. Grossman HB, Natale RB, Tangen CM, et al. Neoadjuvant chemotherapy plus cystectomy compared with cystectomy alone for locally advanced bladder cancer. N Engl J Med 2003;349:859-66.
7. Stein JP, Lieskovsky G, Cote R, et al. Radical cystectomy in the treatment of invasive bladder cancer: long-term results in 1, 054 patients. J Clin Oncol 2001;19:666-75.
8. Donat SM, Shabsigh A, Savage C, et al. Potential impact of postoperative early complications on the timing of adjuvant chemotherapy in patients undergoing radical cystectomy: a high-volume tertiary cancer center experience. Eur Urol 2009;55: 177-85.
9. Sternberg CN, Donat SM, Bellmunt J, et al. Chemotherapy for bladder cancer: treatment guidelines for neoadjuvant chemotherapy, bladder preservation, adjuvant chemotherapy, and metastatic cancer. Urology 2007;69:62-79.
10. Skinner DG, Daniels JR, Russell CA, et al. The role of adjuvant chemotherapy following cystectomy for invasive bladder cancer: a prospective comparative trial. J Urol 1991;145:459-64 [discussion: 464-57].
11. Stockle M, Meyenburg W, Wellek S, et al. Advanced bladder cancer (stages pT3b, pT4a, pN1 and pN2): improved survival after radical cystectomy and 3 adjuvant cycles of chemotherapy. Results of a controlled prospective study. J Urol 1992;148:302-6 [discussion: 306-7].
12. Logothetis CJ, Johnson DE, Chong C, et al. Adjuvant cyclophosphamide, doxorubicin, and cisplatin chemotherapy for bladder cancer: an update. J Clin Oncol 1988;6:1590-6.
13. Logothetis CJ, Johnson DE, Chong C, et al. Adjuvant chemotherapy of bladder cancer: a preliminary report. J Urol 1988;139:1207-11.
14. Meeks JJ, Bellmunt J, Bochner BH, et al. A Systematic Review of Neoadjuvant and Adjuvant Chemotherapy for Muscle-invasive Bladder Cancer. Eur Urol 2012;62:523-33.
15. Svatek RS, Shariat SF, Lasky RE, et al. The effectiveness of off-protocol adjuvant chemotherapy for patients with urothelial carcinoma of the urinary bladder. Clin Cancer Res 2010;16:4461-7.
16. Sternberg CN, Kerst JM, Fossa SD, et al. Final results of EORTC intergroup randomized phase III trial comparing immediate versus deferred chemotherapy after radical cystectomy in patients with pT3T4 and/or N+ M0 transitional cell carcinoma (TCC) of the bladder. American Society of Clinical Oncology Annual Meeting. J Clin Oncol 2014; 32(Suppl):5s [abstr 4500].
17. Stadler WM, Lerner SP, Groshen S, et al. Phase III study of molecularly targeted adjuvant therapy in locally advanced urothelial cancer of the bladder based on p53 status. J Clin Oncol 2011;29:3443-9.
18. Paz-Ares LG, Solsona E, Esteban E, et al. Randomized phase III trial comparing adjuvant paclitaxel/gemcitabine/cisplatin (PGC) to observation in patients with resected invasive bladder cancer: results of the Spanish oncology genitourinary group (SOGUG) 99/01 study. J Clin Oncol 2010; 28(Suppl):18s [abstr LBA4518].
19. Leow JJ, Martin-Doyle W, Rajagopal PS, et al. Adjuvant chemotherapy for invasive bladder cancer: a 2013 updated systematic review and meta-analysis of randomized trials. Eur Urol 2014;66:42-54.
20. Burger M, Mulders P, Witjes W. Use of neoadjuvant chemotherapy for muscle-invasive bladder cancer is low among major European centres: results of a feasibility questionnaire. Eur Urol 2012; 61:1070-1.
21. Raj GV, Karavadia S, Schlomer B, et al. Contemporary use of perioperative cisplatin-based chemotherapy in patients with muscle-invasive bladder cancer. Cancer 2011;117:276-82.
22. Advanced Bladder Cancer (ABC) Meta-analysis Collaboration. Adjuvant chemotherapy in invasive bladder cancer: a systematic review and metaanalysis of individual patient data Advanced Bladder Cancer (ABC) Meta-analysis Collaboration. Eur Urol 2005;48:189-99 [discussion: 199-201].
23. Griffiths G, Hall R, Sylvester R, et al. International phase III trial assessing neoadjuvant cisplatin, methotrexate, and vinblastine chemotherapy for muscleinvasive bladder cancer: long-term results of the BA06 30894 trial. J Clin Oncol 2011;29:2171-7.
24. Advanced Bladder Cancer (ABC) Meta-analysis Collaboration. Neoadjuvant chemotherapy in invasive bladder cancer: a systematic review and metaanalysis. Lancet 2003;361:1927-34.
25. Kitamura H, Tsukamoto T, Masumori N, et al. Randomized phase III trial of neoadjuvant chemotherapy (NAC) with methotrexate, doxorubicin, vinblastine, and cisplatin (MVAC) followed by radical cystectomy (RC) compared with RC alone for invasive bladder cancer (BC): Japan Clinical Oncology Group Study, JCOG0209. J Clin Oncol 2013; 31(Suppl 6) [abstract: 249].
26. Sonpavde G, Goldman BH, Speights VO, et al. Quality of pathologic response and surgery correlate with survival for patients with completely resected bladder cancer after neoadjuvant chemotherapy. Cancer 2009;115:4104-9.
27. Splinter TA, Pavone-Macaluso M, Jacqmin D, et al. A European Organization for Research and Treatment of Cancer-Genitourinary Group phase 2 study of chemotherapy in stage T3-4N0-XM0 transitional cell cancer of the bladder: evaluation of clinical response. J Urol 1992;148:1793-6.
28. Plimack ER, Hoffman-Censits JH, Viterbo R, et al. Accelerated methotrexate, vinblastine, doxorubicin, and cisplatin is safe, effective, and efficient neoadjuvant treatment for muscle-invasive bladder cancer: results of a multicenter phase II study with molecular correlates of response and toxicity. J Clin Oncol 2014;32:1895-901.
29. Choueiri TK, Jacobus S, Bellmunt J, et al. Neoadjuvant dose-dense methotrexate, vinblastine, doxorubicin, and cisplatin with pegfilgrastim support in muscle-invasive urothelial cancer: pathologic, radiologic, and biomarker correlates. J Clin Oncol 2014; 32:1889-94.
30. Neoadjuvant dose dense gemcitabine and cisplatin (dd gc) in patients with muscle-invasive bladder cancer. Available at: http://clinicaltrials.gov/ct2/show/NCT01589094?term5dose+dense+gemcitabine+and1cisplatin&rank=1. Accessed August 20, 2012.
31. Plimack E, Hoffman-Censits J, Kutikov A, et al. Neoadjuvant dose-dense gemcitabine and cisplatin (DDGC) in patients (pts) with muscle-invasive bladder cancer (MIBC): final results of a multicenter phase II study. Chicago (IL): ASCO Annual Meeting. May 29-June 3 2014.
32. Skeldon SC, Semotiuk K, Aronson M, et al. Patients with Lynch syndrome mismatch repair gene mutations are at higher risk for not only upper tract urothelial cancer but also bladder cancer. Eur Urol 2013;63:379-85.
33. Herr HW, Faulkner JR, Grossman HB, et al. Surgical factors influence bladder cancer outcomes: a cooperative group report. J Clin Oncol 2004;22:2781-9.
34. Leissner J, Hohenfellner R, Thuroff JW, et al. Lymphadenectomy in patients with transitional cell carcinoma of the urinary bladder; significance for staging and prognosis. BJU Int 2000;85:817-23.
35. Koppie TM, Serio AM, Vickers AJ, et al. Age-adjusted Charlson comorbidity score is associated with treatment decisions and clinical outcomes for patients undergoing radical cystectomy for bladder cancer. Cancer 2008;112:2384-92.
36. Xu X, Hou Y, Yin X, et al. Single-cell exome sequencing reveals single-nucleotide mutation characteristics of a kidney tumor. Cell 2012;148:886-95.
37. Gerlinger M, Rowan AJ, Horswell S, et al. Intratumor heterogeneity and branched evolution revealed by multiregion sequencing. N Engl J Med 2012;366: 883-92.
38. Bajorin DF, Dodd PM, Mazumdar M, et al. Long-term survival in metastatic transitional-cell carcinoma and prognostic factors predicting outcome of therapy. J Clin Oncol 1999;17:3173-81.
39. Bellmunt J, Pons F, Orsola A. Molecular determinants of response to cisplatin-based neoadjuvant chemotherapy. Curr Opin Urol 2013;23:466-71.
40. Tripathy D, Harnden K, Blackwell K, et al. Next generation sequencing and tumor mutation profiling: are we ready for routine use in the oncology clinic? BMC Med 2014;12:140.
41. The Cancer Genome Atlas Research Network. Comprehensive molecular characterization of urothelial bladder carcinoma. Nature 2014;507: 315-22.
42. Iyer G, Al-Ahmadie H, Schultz N, et al. Prevalence and co-occurrence of actionable genomic alterations in high-grade bladder cancer. J Clin Oncol 2013;31:3133-40.
43. Iyer G, Hanrahan AJ, Milowsky MI, et al. Genome sequencing identifies a basis for everolimus sensitivity. Science 2012;338:221.
44. Carthon BC, Wolchok JD, Yuan J, et al. Preoperative CTLA-4 blockade: tolerability and immune monitoring in the setting of a presurgical clinical trial. Clin Cancer Res 2010;16:2861-71.
45. Ng Tang D, Shen Y, Sun J, et al. Increased frequency of ICOS+ CD4 T cells as a pharmacodynamic biomarker for anti-CTLA-4 therapy. Cancer Immunol Res 2013;1:229-34.
46. Powles T, Eder JP, Fine GD, et al. MPDL3280A (anti-PD-L1) treatment leads to clinical activity in metastatic bladder cancer. Nature 2014;515:558-62.
47. Galon J, Costes A, Sanchez-Cabo F, et al. Type, density, and location of immune cells within human colorectal tumors predict clinical outcome. Science 2006;313:1960-4.
48. Clay TM, Hobeika AC, Mosca PJ, et al. Assays for monitoring cellular immune responses to active immunotherapy of cancer. Clin Cancer Res 2001;7: 1127-35.
49. Horig H, Pullman W. From bench to clinic and back: Perspective on the 1st IQPC translational research conference. J Transl Med 2004;2:44.
50. Cowan NG, Chen Y, Downs TM, et al. Neoadjuvant chemotherapy use in bladder cancer: a survey of current practice and opinions. Adv Urol 2014;2014: 746298.