Contemporary Outcomes of Patients with Non-Muscle Invasive Bladder Cancer Treated with BCG Therapy: Implications for Clinical Trial Design.

Recurrent disease after BCG presents a therapeutic challenge. To aid trial development, the US Food & Drug Administration defined 'adequate BCG' therapy and adopted the 'BCG unresponsive' disease state. Available data for efficacy benchmark comparison is outdated, leading to concerns about appropriate control arms and sample size calculations. We describe a contemporary cohort of non-muscle invasive bladder cancer patients treated with intravesical BCG and provide benchmark outcomes data.

This is a retrospective review of patients receiving adequate BCG therapy at a tertiary cancer center between January 2004 and August 2018. Unadjusted univariable analysis conducted using Pearson's chi-squared test. Kaplan-Meier estimates for RFS-HG, PFS-MIBC and OS used to create survival curves and compared using log-rank test.

Of the 542 patients who received adequate BCG, 518 (90%) had EAU high-risk disease with carcinoma in situ present in 175 (32%). With median follow-up of 47.8 months, freedom from high-grade recurrence at 1-, 3-, and 5-years was 81%, 76%, and 74%; and progression-free survival was 97%, 93%, and 92%, respectively. Progression to muscle invasion at 5-years was exclusively seen in patients with high-risk disease (PFS 91% high-risk, log-rank test p=0.024).

A contemporary cohort of NMIBC patients treated with adequate BCG demonstrated markedly better outcomes than seen in prior studies. These data could be used in the design of clinical trials, to guide power calculations, as well as serve as benchmarks for comparison to evaluate nonrandomized studies.

The Journal of urology. 2021 Jan 27 [Epub ahead of print]

Justin T Matulay, Roger Li, Patrick J Hensley, Nathan A Brooks, Vikram M Narayan, H Barton Grossman, Neema Navai, Colin P N Dinney, Ashish M Kamat

Department of Urology, Levine Cancer Institute/Atrium Health, Charlotte, North Carolina., Department of Genitourinary Oncology, Moffitt Cancer Center, Tampa, Florida., Department of Urology, University of Texas MD Anderson Cancer Center, Houston, Texas., Department of Urology, Emory University School of Medicine, Atlanta, Georgia., Urologic Oncology (Surgery), Wayne B. Duddlesten Professor of Cancer Research, University of Texas MD Anderson Cancer Center, Houston, Texas.

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