Contemporary Outcomes of Patients with Nonmuscle-Invasive Bladder Cancer Treated with bacillus Calmette-Guérin: Implications for Clinical Trial Design

No AccessJournal of UrologyAdult Urology1 Jun 2021Contemporary Outcomes of Patients with Nonmuscle-Invasive Bladder Cancer Treated with bacillus Calmette-Guérin: Implications for Clinical Trial DesignThis article is commented on by the following:Editorial CommentEditorial Comment Justin T. Matulay, Roger Li, Patrick J. Hensley, Nathan A. Brooks, Vikram M. Narayan, H. Barton Grossman, Neema Navai, Colin P. N. Dinney, and Ashish M. Kamat Justin T. MatulayJustin T. Matulay http://orcid.org/0000-0002-2467-5710 Department of Urology, Levine Cancer Institute/Atrium Health, Charlotte, North Carolina More articles by this author , Roger LiRoger Li Department of Genitourinary Oncology, Moffitt Cancer Center, Tampa, Florida More articles by this author , Patrick J. HensleyPatrick J. Hensley http://orcid.org/0000-0002-8098-901X Department of Urology, University of Texas MD Anderson Cancer Center, Houston, Texas Financial interest and/or other relationship with AUA Urology Care Foundation. More articles by this author , Nathan A. BrooksNathan A. Brooks Department of Urology, University of Texas MD Anderson Cancer Center, Houston, Texas More articles by this author , Vikram M. NarayanVikram M. Narayan http://orcid.org/0000-0003-3731-4209 Department of Urology, Emory University School of Medicine, Atlanta, Georgia More articles by this author , H. Barton GrossmanH. Barton Grossman Department of Urology, University of Texas MD Anderson Cancer Center, Houston, Texas More articles by this author , Neema NavaiNeema Navai http://orcid.org/0000-0001-8457-3786 Department of Urology, University of Texas MD Anderson Cancer Center, Houston, Texas More articles by this author , Colin P. N. DinneyColin P. N. Dinney http://orcid.org/0000-0002-8969-711X Department of Urology, University of Texas MD Anderson Cancer Center, Houston, Texas Financial interest and/or other relationship with NIH and FKD. More articles by this author , and Ashish M. KamatAshish M. Kamat §Correspondence: University of Texas MD Anderson Cancer Center, 1515 Holcombe Blvd., Unit 1373, Houston, Texas 77030 E-mail Address: [email protected] http://orcid.org/0000-0003-3546-9928 Department of Urology, University of Texas MD Anderson Cancer Center, Houston, Texas Urologic Oncology (Surgery), Wayne B. Duddlesten Professor of Cancer Research, University of Texas MD Anderson Cancer Center, Houston, Texas Financial interest and/or other relationship with FKD, Merck, BMS, Photocure, NIH, AIBCCR, Arquer, Theralase, Medac, Pfizer, Astra Zeneca, Imagin, Eisai/H3 Biomedicine, Cold Genesys, Sessen Bio, enGene, Janssen, ArTara, Seattle Genetics, FerGene More articles by this author View All Author Informationhttps://doi.org/10.1097/JU.0000000000001633AboutFull TextPDF ToolsAdd to favoritesDownload CitationsTrack CitationsPermissionsReprints ShareFacebookLinked InTwitterEmail Abstract Purpose: Recurrent disease after bacillus Calmette-Guérin treatment presents a therapeutic challenge. To aid trial development, the U.S. Food and Drug Administration defined “adequate bacillus Calmette-Guérin” therapy and adopted the “bacillus Calmette-Guérin unresponsive” disease state. Available data for efficacy benchmark comparison are outdated, leading to concerns about appropriate control arms and sample size calculations. We describe a contemporary cohort of patients with nonmuscle-invasive bladder cancer treated with intravesical bacillus Calmette-Guérin, and provide benchmark outcomes data. Materials and Methods: We retrospectively reviewed patients receiving adequate bacillus Calmette-Guérin therapy at a tertiary cancer center between January 2004 and August 2018. Unadjusted univariable analysis was conducted using the Pearson chi-square test. Kaplan-Meier estimates for recurrence-free survival—high grade, progression-free survival—muscle-invasive bladder cancer and overall survival were used to create survival curves and compared using the log-rank test. Results: Of the 542 patients who received adequate bacillus Calmette-Guérin, 518 (90%) had European Association Urology high risk disease, with carcinoma in situ present in 175 (32%). With a median followup of 47.8 months, freedom from high grade recurrence at 1, 3 and 5 years was 81%, 76% and 74%, respectively, and progression-free survival was 97%, 93% and 92%. Progression to muscle invasion at 5 years was exclusively seen in patients with high risk disease (progression-free survival 91%; log-rank test, p=0.024). Conclusions: A contemporary cohort of patients with nonmuscle-invasive bladder cancer treated with adequate bacillus Calmette-Guérin 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. References 1. : Cancer statistics, 2020. CA Cancer J Clin 2020; 70: 7. Google Scholar 2. : European Association of Urology Guidelines on non-muscle-invasive bladder cancer (TaT1 and carcinoma in situ)—2019 update. Eur Urol 2019; 76: 639. 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Duddlesten Professorship in Cancer Research, the Raymond and Maria Floyd Bladder Cancer Research Foundation Grant (AMK), and Grant P50CA091846 from the NIH/NCI UTMD Anderson SPORE in Genitourinary Cancer (Bladder) (CPND). © 2021 by American Urological Association Education and Research, Inc.FiguresReferencesRelatedDetailsCited ByHensley P and Kamat A (2021) Contemporary Outcomes of Patients with Nonmuscle-Invasive Bladder Cancer Treated with Bacillus Calmette-Guérin: Implications for Clinical Trial Design. Reply.Journal of Urology, VOL. 206, NO. 6, (1528-1528), Online publication date: 1-Dec-2021.Montorsi F, Moschini M, Necchi A, Deho F, Gandaglia G and Briganti A (2021) Contemporary Outcomes of Patients With Nonmuscle-Invasive Bladder Cancer Treated with Bacillus Calmette-Guérin: Implications for Clinical Trial Design. Letter.Journal of Urology, VOL. 206, NO. 6, (1528-1528), Online publication date: 1-Dec-2021.Related articlesJournal of UrologyMar 18, 2021, 12:00:00 AMEditorial CommentJournal of UrologyMar 18, 2021, 12:00:00 AMEditorial Comment Volume 205Issue 6June 2021Page: 1612-1621 Advertisement Copyright & Permissions© 2021 by American Urological Association Education and Research, Inc.KeywordsBCG vaccineurinary bladder neoplasmsimmunotherapyMetricsAuthor Information Justin T. Matulay Department of Urology, Levine Cancer Institute/Atrium Health, Charlotte, North Carolina More articles by this author Roger Li Department of Genitourinary Oncology, Moffitt Cancer Center, Tampa, Florida More articles by this author Patrick J. Hensley Department of Urology, University of Texas MD Anderson Cancer Center, Houston, Texas Financial interest and/or other relationship with AUA Urology Care Foundation. More articles by this author Nathan A. Brooks Department of Urology, University of Texas MD Anderson Cancer Center, Houston, Texas More articles by this author Vikram M. Narayan Department of Urology, Emory University School of Medicine, Atlanta, Georgia More articles by this author H. Barton Grossman Department of Urology, University of Texas MD Anderson Cancer Center, Houston, Texas More articles by this author Neema Navai Department of Urology, University of Texas MD Anderson Cancer Center, Houston, Texas More articles by this author Colin P. N. Dinney Department of Urology, University of Texas MD Anderson Cancer Center, Houston, Texas Financial interest and/or other relationship with NIH and FKD. More articles by this author Ashish M. Kamat Department of Urology, University of Texas MD Anderson Cancer Center, Houston, Texas Urologic Oncology (Surgery), Wayne B. Duddlesten Professor of Cancer Research, University of Texas MD Anderson Cancer Center, Houston, Texas §Correspondence: University of Texas MD Anderson Cancer Center, 1515 Holcombe Blvd., Unit 1373, Houston, Texas 77030 E-mail Address: [email protected] Financial interest and/or other relationship with FKD, Merck, BMS, Photocure, NIH, AIBCCR, Arquer, Theralase, Medac, Pfizer, Astra Zeneca, Imagin, Eisai/H3 Biomedicine, Cold Genesys, Sessen Bio, enGene, Janssen, ArTara, Seattle Genetics, FerGene More articles by this author Expand All Funding Source: This research was supported by the Wayne B. Duddlesten Professorship in Cancer Research, the Raymond and Maria Floyd Bladder Cancer Research Foundation Grant (AMK), and Grant P50CA091846 from the NIH/NCI UTMD Anderson SPORE in Genitourinary Cancer (Bladder) (CPND). Advertisement PDF DownloadLoading ...