Cystoscopy and Systematic Bladder Tissue Sampling in Predicting pT0 Bladder Cancer: A Prospective Trial

Abstract

Purpose: Concern for discordance between clinical staging and final pathology drives current management of patients deemed appropriate candidates for radical cystectomy (RC). Thus, we set out to prospectively investigate reliability and shortcomings of cystoscopic evaluation in RC candidates. Materials and Methods: Patients undergoing RC for urothelial carcinoma (UC) were enrolled in a prospective single-arm study to evaluate reliability of Systematic Endoscopic Evaluation (SEE) in predicting pT0 UC (NCT02968732). SEE consisted of cystoscopy and tissue sampling at the time of RC. SEE results were compared to RC pathology. The primary endpoint was the negative predictive value (NPV) of SEE findings in predicting RC pathology. Results: 61 patients underwent SEE and RC. Indications included muscle-invasive bladder cancer (MIBC) in 42 (68.9%) and high-risk non-muscle invasive bladder cancer (NMIBC) in 19 (31.1%). Thirty-eight (62.3%, 90.5% of MIBC patients) received neoadjuvant chemotherapy (NAC). On SEE, 31 (50.8%) patients demonstrated no visual nor biopsy-based evidence of disease (seeT0), yet 16/31 (51.6%) harbored residual disease (>pT0), including eight (8/31, 25.8%) with residual ≥pT2 disease upon RC. The NPV of SEE predicting a pT0 bladder was 48.4% (CI: 30.2–66.9%), which was below our pre-specified hypothesis, and thus the trial was stopped for futility. Conclusions: Approximately 1 of 4 patients with seeT0 at the time of RC harbored residual MIBC. These prospective data definitively confirm major limitations of endoscopic assessment for pT0 bladder cancer. Future work should focus on novel imaging and biomarker strategies to optimize pre-RC evaluations for improved decision-making regarding bladder preservation.