Outcomes of Patients with Bacillus Calmette-Guérin (BCG)-Unresponsive Non-Muscle Invasive Bladder Cancer as Defined by the U.S. Food and Drug Administration.

Background: Limited data are available on the outcomes of patients with non-muscle invasive bladder cancer (NMIBC) unresponsive to intravesical bacillus Calmette-Guérin (BCG), as defined by the United States Food and Drug Administration.
Objective: To define the outcomes of patients with BCG-unresponsive NMIBC.
Methods: This was a retrospective, single-institution observational cohort study. Records of patients managed at our institution for BCG-unresponsive NMIBC between 2005 and 2020 were reviewed and clinical outcomes evaluated.
Results: The study included 149 patients. Management was with initial radical cystectomy in 60 patients (40%) and initial bladder-sparing therapy (BST) in 89 patients (60%). Overall survival was greater among patients undergoing RC than BST (HR 1.83, 95% CI 1.04-3.22, p  = 0.036), potentially due to patient selection, as no significant difference was noted for metastasis-free or cancer-specific survival. Patients opting for initial BST had high rates of treatment failure, with estimated 5-year cystectomy-free survival of only 42%. Patients who received additional lines of BST after a subsequent failure were at increased risk of having ≥pT3 or pN+ disease at cystectomy (42% for ≥2 lines BST, versus 18% for 1 line BST and 15% for initial cystectomy, p  = 0.038).
Conclusion: Among patients who underwent initial BST for BCG-unresponsive NMIBC, rates of treatment failure were very high. Patients who underwent delayed cystectomy after ≥2 lines of BST had elevated rates of extravesical disease. Our observations emphasize the importance of recent and ongoing clinical trials in this clinical space.
Competing Interests: Y.L. is an Editorial Board Member of this journal but was not involved in the peer-review process nor had access to any information regarding its peer-review. Y.L. reports relationships with the following entities: Abbott, Abbvie, Ambu, AstraZeneca, BioCanCell, CAPs Medical, Cepheid, Cleveland Diagnostics, C2I Genomics, Fergene, Ferring Research, FKD, GenomeDx Biosciences, Hitachi, Karl Storz, MDxHealth, Merck, Nanorobotics, Nucleix, Pacific Edge, Photocure, Seattle Genetics, Stimit, Verity Pharmaceuticals, Vessi Medical, Virtuoso Surgical. J.M.H., G.S.C., A.T., K.N., V.M. and S.L.W. report no conflicts.
(© 2022 – The authors. Published by IOS Press.)