Comparative effectiveness of neoadjuvant chemotherapy in bladder and upper urinary tract urothelial carcinoma.

To assess the differential response to neoadjuvant chemotherapy (NAC) in patients with urothelial carcinoma of the bladder (UCB) compared to upper tract urothelial carcioma (UTUC) treated with radical surgery.

Data from 1299 patients with UCB and 276 with UTUC were obtained from multicentric collaborations. The association of disease location (UCB vs UTUC) with pathological complete response (pCR, defined as a post-treatment pathological stage ypT0N0) and pathological objective response (pOR, defined as ypT0-Ta-Tis-T1N0) after NAC was evaluated using logistic regression analyses. The association with overall (OS) and cancer-specific survival (CSS) was evaluated using Cox regression analyses.

A pCR was found in 250 (19.2%) patients with UCB and in 23 (8.3%) with UTUC (P < 0.01). A pOR was found in 523 (40.3%) patients with UCB and in 133 (48.2%) with UTUC (P = 0.02). On multivariable logistic regression analysis, patients with UTUC were less likely to have a pCR (odds ratio [OR] 0.45, 95% confidence interval [CI] 0.27-0.70; P < 0.01) and more likely to have a pOR (OR 1.57, 95% CI 1.89-2.08; P < 0.01). On univariable Cox regression analyses, UTUC was associated with better OS (hazard ratio [HR] 0.80, 95% CI 0.64-0.99, P = 0.04) and CSS (HR 0.63, 95% CI 0.49-0.83; P < 0.01). On multivariable Cox regression analyses, UTUC remained associated with CSS (HR 0.61, 95% CI 0.45-0.82; P < 0.01), but not with OS.

Our present findings suggest that the benefit of NAC in UTUC is similar to that found in UCB. These data can be used as a benchmark to contextualise survival outcomes and plan future trial design with NAC in urothelial cancer.

BJU international. 2020 Sep 27 [Epub ahead of print]

David D'Andrea, Surena Matin, Peter C Black, Firas G Petros, Homayoun Zargar, Colin P Dinney, Michael S Cookson, Wassim Kassouf, Marc A Dall'Era, John S McGrath, Jonathan L Wright, Andrew C Thorpe, Todd M Morgan, Jeffrey M Holzbeierlein, Trinity J Bivalacqua, Srikala S Sridhar, Scott North, Daniel A Barocas, Yair Lotan, Andrew J Stephenson, Bas W van Rhijn, Philippe E Spiess, Siamak Daneshmand, Shahrokh F Shariat

Department of Urology, Comprehensive Cancer Center, Medical University of Vienna, Vienna, Austria., Department of Urology, MD Anderson Cancer Center, Houston, TX, USA., Department of Urologic Sciences, University of British Columbia, Vancouver, BC, Canada., Department of Urology and Kidney Transplant, Eleanor N. Dana Cancer Center, The University of Toledo Medical Center, Toledo, OH, USA., Department of Urology, Center and The Stephenson Cancer Center, The University of Oklahoma Health Sciences, Oklahoma City, OK, USA., Division of Urology, Department of Surgery, McGill University Health Center, Montreal, QC, Canada., Department of Urology, Davis Medical Center, University of California at Davis, Sacramento, CA, USA., Department of Urology, University of Michigan Health System, Ann Arbor, MI, USA., Department of Urology, University of Washington, Seattle, WA, USA., Department of Urology, Freeman Hospital, Newcastle Upon Tyne, UK., Department of Urology, University of Kansas Medical Center, Kansas City, KS, USA., Department of Urology, The Johns Hopkins School of Medicine, The James Buchanan Brady Urological Institute, Baltimore, MD, USA., Department of Medical Oncology and Hematology, Princess Margaret Cancer Center, Toronto, ON, Canada., Department of Oncology, Cross Cancer Institute, University of Alberta, Edmonton, AB, Canada., Department of Urologic Surgery, Vanderbilt University Medical Center, Nashville, TN, USA., Department of Urology, University of Texas Southwestern Medical Center, Dallas, TX, USA., Glickman Urological and Kidney Institute, Cleveland Clinic, Cleveland, OH, USA., Department of Urology, The Netherlands Cancer Institute, Antoni van Leeuwenhoek Hospital, Amsterdam, The Netherlands., Department of Genitourinary Oncology, H Lee Moffitt Cancer Center and Research Institute, Tampa, FL, USA., Department of Urology, USC/Norris Comprehensive Cancer Center, University of Southern California, Los Angeles, CA, USA.

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