The past, present, and future of pT0 in bladder cancer clinical trials.
Journal
Current opinion in urology
ISSN: 1473-6586
Titre abrégé: Curr Opin Urol
Pays: United States
ID NLM: 9200621
Informations de publication
Date de publication:
01 09 2022
01 09 2022
Historique:
pubmed:
21
7
2022
medline:
4
8
2022
entrez:
20
7
2022
Statut:
ppublish
Résumé
Since the establishment of neoadjuvant chemotherapy as the standard of care for patients with muscle invasive bladder cancer, the pathologic absence of disease, denoted pT0, was found to be predictive of improved overall survival. Accordingly, it has been used in clinical trials as an optimal surrogate outcome measure, even in contemporary nonchemotherapeutic interventions. We review the role of pT0 as a catalyst for change in trial design and its suitability to facilitate more efficient and timely results. In addition, we explore the present and future of cT0, the clinical absence of disease, in defining treatment response and enabling bladder-sparing management options. The use of pT0 as a surrogate has provided initial results for the efficacy of immunotherapy in the neoadjuvant space. In combination with molecular markers, pT0 has improved our ability to identify treatment responders and its clinical counterpart, cT0, has been integrated into multiple trials to redefine postneoadjuvant chemotherapy management algorithms. The use of pT0 as a surrogate endpoint in bladder cancer trials has improved clinical trial design, defined efficacy of emerging therapeutics, and has the potential to redefine the postneoadjuvant treatment management for patients seeking bladder-sparing options.
Identifiants
pubmed: 35855573
doi: 10.1097/MOU.0000000000001022
pii: 00042307-202209000-00011
doi:
Types de publication
Journal Article
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
495-499Informations de copyright
Copyright © 2022 Wolters Kluwer Health, Inc. All rights reserved.
Références
Cancer of the urinary bladder - cancer stat facts. Cancer.gov. Retrieved 9 March 2022. Available at: https://seer.cancer.gov/statfacts/html/urinb.html . Accessed 9 March 2022.
Grossman HB, Natale RB, Tangen CM, et al. Neoadjuvant chemotherapy plus cystectomy compared with cystectomy alone for locally advanced bladder cancer. New Engl J Med 2003; 349:859–866.
Chism DD, Woods ME, Milowsky MI. Neoadjuvant paradigm for accelerated drug development: an ideal model in bladder cancer. Oncol 2013; 18:933–940.
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; 32:1895–1901.
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; 32:1889–1894.
Miron B, Hoffman-Censits JH, Anari F, et al. Defects in DNA repair genes confer improved long-term survival after cisplatin-based neoadjuvant chemotherapy for muscle-invasive bladder cancer. Eur Urol Oncol 2020; 3:544–547.
Pfister C, Gravis G, Fléchon A, et al. Dose-dense methotrexate, vinblastine, doxorubicin, and cisplatin or gemcitabine and cisplatin as perioperative chemotherapy for patients with nonmetastatic muscle-invasive bladder cancer: Results of the GETUG-AFU V05 VESPER Trial. J Clin Oncol 2022; 40:2013–2022.
Pfister C, Gravis G, Fléchon A, et al. Randomized phase III trial of dose-dense methotrexate, vinblastine, doxorubicin, and cisplatin, or gemcitabine and cisplatin as perioperative chemotherapy for patients with muscle-invasive bladder cancer. analysis of the GETUG/AFU V05 VESPER Trial Secondary Endpoints: Chemotherapy Toxicity and Pathological Responses. Eur Urol 2021; 79:214–221.
Plimack ER, Dunbrack RL, Brennan TA, et al. Defects in DNA repair genes predict response to neoadjuvant cisplatin-based chemotherapy in muscle-invasive bladder cancer. Eur Urol 2015; 68:959–967.
Flaig TW, Tangen CM, Daneshmand S, et al. A randomized phase II study of coexpression extrapolation (COXEN) with neoadjuvant chemotherapy for bladder cancer (SWOG S1314; NCT02177695). Clin Cancer Res 2021; 27:2435–2441.
Morera DS, Hasanali SL, Belew D, et al. Clinical parameters outperform molecular subtypes for predicting outcome in bladder cancer: results from multiple cohorts, including TCGA. J Urol 2020; 203:62–72.
Powles T, Kockx M, Rodriguez-Vida A, et al. Clinical efficacy and biomarker analysis of neoadjuvant atezolizumab in operable urothelial carcinoma in the ABACUS trial. Nat Med 2019; 25:1706–1714.
Bandini M, Ross JS, Raggi D, et al. Predicting the pathologic complete response after neoadjuvant pembrolizumab in muscle-invasive bladder cancer. Jnci J National Cancer Inst 2020; 113:48–53.
Bandini M, Gibb EA, Gallina A, et al. Does the administration of preoperative pembrolizumab lead to sustained remission postcystectomy? First survival outcomes from the PURE-01 study. Ann Oncol 2020; 31:1755–1763.
van Dijk N, Gil-Jimenez A, Silina K, et al. Preoperative ipilimumab plus nivolumab in locoregionally advanced urothelial cancer: the NABUCCO trial. Nat Med 2020; 26:1839–1844.
Gao J, Navai N, Alhalabi O, et al. Neoadjuvant PD-L1 plus CTLA-4 blockade in patients with cisplatin-ineligible operable high-risk urothelial carcinoma. Nat Med 2020; 26:1845–1851.
Gupta S, Sonpavde G, Weight CJ, et al. Results from BLASST-1 (Bladder Cancer Signal Seeking Trial) of nivolumab, gemcitabine, and cisplatin in muscle invasive bladder cancer (MIBC) undergoing cystectomy. J Clin Oncol 2020; 38: (6 Suppl): 439–439.
Grande E, Guerrero F, Puente J, et al. DUTRENEO trial: a randomized phase II trial of DUrvalumab and TREmelimumab versus chemotherapy as a NEOadjuvant approach to muscle-invasive urothelial bladder cancer (MIBC) patients (pts) prospectively selected by an interferon (INF)-gamma immune signature. J Clin Oncol 2020; 38: (15 Suppl): 5012–5012.
Geynisman DM, Abbosh P, Ross EA, et al. A phase II trial of risk enabled therapy after initiating neoadjuvant chemotherapy for bladder cancer (RETAIN BLADDER): interim analysis. J Clin Oncol 2021; 39: (6 Suppl): 397–397.
Galsky MD, Daneshmand S, Chan KG, et al. Phase 2 trial of gemcitabine, cisplatin, plus nivolumab with selective bladder sparing in patients with muscle- invasive bladder cancer (MIBC): HCRN GU 16-257. J Clin Oncol 2021; 39: (15 Suppl): 4503–4503.
Zibelman M, Asghar AM, Parker DC, et al. Cystoscopy and systematic bladder tissue sampling in predicting pT0 bladder cancer: a prospective trial. J Urol 2021; 205:1605–1611.
Asad D, Styrke J, Hagsheno M, et al. A prospective multicenter study of visual response-evaluation by cystoscopy in patients undergoing neoadjuvant chemotherapy for muscle invasive urinary bladder cancer. Scand J Urol 2021; 56:20–26.
Mansour AM, Singal R, Eldefrawy A, et al. 1747 Prognostic significance of cystoscopy findings following neoadjuvant chemotherapy for muscle-invasive bladder cancer. J Urol 2012; 187:e705.
Becker REN, Meyer AR, Brant A, et al. Clinical restaging and tumor sequencing are inaccurate indicators of response to neoadjuvant chemotherapy for muscle-invasive bladder cancer. Eur Urol 2021; 79:364–371.
Teo MY, Bambury RM, Zabor EC, et al. DNA damage response and repair gene alterations are associated with improved survival in patients with platinum-treated advanced urothelial carcinoma. Clin Cancer Res 2017; 23:3610–3618.
Christensen E, Birkenkamp-Demtröder K, Sethi H, et al. 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 2019; 37:1547–1557.
Panebianco V, Narumi Y, Altun E, et al. Multiparametric magnetic resonance imaging for bladder cancer: development of VI-RADS (Vesical Imaging-Reporting And Data System). Eur Urol 2018; 74:294–306.
Necchi A, Bandini M, Calareso G, et al. Multiparametric magnetic resonance imaging as a noninvasive assessment of tumor response to neoadjuvant pembrolizumab in muscle-invasive bladder cancer: preliminary findings from the PURE-01 Study. Eur Urol 2020; 77:636–643.