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Multicenter Study
. 2025 Jun;23(3):102313.
doi: 10.1016/j.clgc.2025.102313. Epub 2025 Feb 4.

Real-World Treatment Patterns and Outcomes in Patients With Bacillus Calmette-Guérin-Unresponsive High-Risk Non-Muscle-Invasive Bladder Cancer: A Multicountry Medical Chart Review

Girish S Kulkarni  1 , Thomas Guzzo  2 , Philip H Abbosh  3 , William C Huang  4 , Neal Shore  5 , Zachary Smith  6 , Ho Kyung Seo  7 , Ja Hyeon Ku  8 , Jean-Benoit Paradis  9 , Romain Mathieu  10 , Mathieu Roumiguié  11 , Abhishek Srivastava  12 , Carly Rodriguez  13 , Claire M Fox  14 , Ekta Kapadia  14 , Mehmet Burcu  14 , Joost L Boormans  15
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Free article
Multicenter Study

Real-World Treatment Patterns and Outcomes in Patients With Bacillus Calmette-Guérin-Unresponsive High-Risk Non-Muscle-Invasive Bladder Cancer: A Multicountry Medical Chart Review

Girish S Kulkarni et al. Clin Genitourin Cancer. 2025 Jun.
Free article

Abstract

Introduction: Treatment patterns for patients with bacillus Calmette-Guérin (BCG)-unresponsive high-risk non-muscle-invasive bladder cancer (NMIBC) who are ineligible for or decline radical cystectomy (RC) are inconsistently reported. We retrospectively described demographic, clinical, and treatment characteristics for these patients and assessed their clinical outcomes.

Patients and methods: Medical charts of patients with BCG-unresponsive high-risk NMIBC (carcinoma in situ [cohort A] or T1/high-grade Ta [cohort B]) who were ineligible for or declined RC documented between January 1, 2011, and December 31, 2018, at 15 academic centers were reviewed. Primary objectives were to characterize demographic, clinical, and nonsurgical treatment characteristics. Secondary objectives included assessing real-world progression-free survival (rw-PFS) from muscle-invasive/metastatic disease, rw-PFS from worsening grade or stage, real-world complete response rate (rw-CRR) in cohort A, real-world event-free survival (rw-EFS) from high-risk NMIBC in cohort B, and overall survival.

Results: The study included 129 patients (cohort A, n = 57; cohort B, n = 72). Median age was 72.0 years (interquartile range, 64.0-80.0). Most patients were male (72.1%) and current/former smokers (69.8%). Median follow-up was 32.1 months (interquartile range, 20.7-47.6). BCG rechallenge with or without interferon-α (63.6%) was the most commonly utilized first nonsurgical therapy, followed by intravesical mitomycin C with or without electromotive drug administration or thermochemotherapy (15.5%), and intravesical valrubicin (10.9%); among those who received BCG rechallenge alone, 54.8% later received a non-BCG therapy in ≥ 2 subsequent treatments. 36-month rate for rw-PFS from muscle-invasive/metastatic disease was 73.5%, 66.8% for rw-PFS from worsening grade/stage, and 82.5% for overall survival. In cohort A, 6-month rw-CRR was 22.2%. In cohort B, 36-month rw-EFS rate from high-risk NMIBC was 50.2%.

Conclusion: After BCG-unresponsive disease, most patients with high-risk NMIBC received BCG rechallenge with or without other therapies, and > 25% experienced disease progression within the first 3 years. Effective bladder-sparing options for BCG-unresponsive NMIBC are needed.

Clinical trial registration: N/A.

Keywords: BCG; Bladder-sparing treatment; Nonsurgical treatment; Observational study; Urothelial carcinoma.

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Conflict of interest statement

Disclosure G. S. Kulkarni reports support for the present manuscript from MSD; research grants or contracts from Johnson and Johnson; fees for consulting and advisory board participation from AbbVie, Astellas, AstraZeneca, Bristol Myers Squibb, EMD Serono, enGene, Ferring, Johnson and Johnson, Knight Pharmaceuticals, Merck and Co., Inc., Pfizer, Photocure, TerSera, Theralase Technologies, Tolmar, and Verity; payment or honoraria for lectures, presentations, speakers bureaus, manuscript writing, or educational events from Ferring, Knight Pharmaceuticals, TerSera, and Tolmar; and leadership role as Vice Chair of Research of Bladder Cancer Canada. P. H. Abbosh reports research support for the present manuscript from MSD; research support or grants from Janssen, Natera, and the National Cancer Institute (U01 CA260369); honoraria for being a speaker from Great Debates and Updates; support for attending and being a speaker at the bladder cancer meeting 2024 from the American Association for Cancer Research; registration fee waived for being on a steering committee from the Bladder Cancer Advocacy Network; and patents planned, issued, or pending for urine biomarkers, neoantigen vaccines, and imidazolium salts. W. C. Huang reports payment to his institution for the present work from MSD. N. Shore reports consulting fees from AbbVie, Accord, Amgen, Antev, Arquer Diagnostics, Asieris Pharmaceuticals, Astellas, AstraZeneca, Aura Biosciences, Bayer, BioProtect, Bristol Myers Squibb, Clarity, Cold Genesys, Dendreon, Eli Lilly and Company, Exact Imaging, Ferring, Fize Medical, Invitae, Janssen, Lantheus, MDxHealth, Merck and Co., Inc., Minomic, Myriad, Novartis, Pfizer, PlatformQ, Preview, Promaxo, Propella, Protara Therapeutics, Sanofi Genzyme, Specialty Networks, Sumitomo, Telix, Tolmar, and UroGen Pharma; leadership or fiduciary role in other board, society, committee or advocacy group, paid or unpaid, for Alessa Therapeutics and Photocure; and stock options in Alessa Therapeutics and Photocure. R. Mathieu reports payment or honoraria for lectures, presentations, speakers’ bureaus, manuscript writing, or educational events from Ferring, Janssen, and Pfizer. M. Roumiguié reports consulting fees from Astellas Pharma, AstraZeneca, Bayer, Bristol Myers Squibb, Ferring, Janssen Oncology, Pfizer, and Pierre Fabre; payment or honoraria for lectures, presentations, speakers’ bureaus, manuscript writing, or educational events from Astellas Pharma, AstraZeneca, Bayer, Bristol Myers Squibb, Ferring, Janssen Oncology, Pfizer, and Pierre Fabre; and support for attending meetings and/or travel from Astellas Pharma, AstraZeneca, Bayer, Ferring, Janssen Oncology, and Pierre Fabre. C. Rodriguez reports payment made to IQVIA for services provided as contract research organization on the present manuscript from MSD. C. M. Fox, E. Kapadia, and M. Burcu are employees of Merck Sharp and Dohme LLC, a subsidiary of Merck and Co., Inc., Rahway, NJ, USA, and own stock in Merck and Co., Inc., Rahway, NJ, USA. J. L. Boormans reports support for the present manuscript from MSD; grants or contracts paid to his institution from Merck AG; consulting fees paid to his institution from Bayer, Bristol Myers Squibb, Janssen, Merck AG, and MSD; payment or honoraria to his institution for lectures, presentations, speakers bureaus, and manuscript writing or educational events from AstraZeneca and Ismar Healthcare; and payment to his institution for participation on a data safety monitoring board or advisory board of MSD. T. Guzzo, Z. Smith, H. K. Seo, J. H. Ku, J.-B. Paradis, and A. Srivastava declare no competing interests.

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