Evidence-based Assessment of Current and Emerging Bladder-sparing Therapies for Non–muscle-invasive Bladder Cancer After Bacillus Calmette-Guerin Therapy: A Systematic Review and Meta-analysis

Context: Currently, there is no standard of care for patients with non–muscle-invasive bladder cancer (NMIBC) who recur despite bacillus Calmette-Guerin (BCG) therapy. Although radical cystectomy is recommended, many patients decline to undergo or are ineligible to receive it. Multiple agents are being investigated for use in this patient population.


Objective: To systematically synthesize and describe the efficacy and safety of current and emerging treatments for NMIBC patients after treatment with BCG.

Evidence acquisition: A systematic literature search of MEDLINE, Embase, and the Cochrane Controlled Register of Trials (period limited to January 2007–June 2019) was performed. Abstracts and presentations from major conference proceedings were also reviewed. Randomized controlled trials were assessed using the Cochrane risk of bias tool. Data for single-arm trials were pooled using a random-effect meta-analysis with the proportions approach. Trials were grouped based on the minimum number of prior BCG courses required before enrollment and further stratified based on the proportion of patients with carcinoma in situ (CIS).

Evidence synthesis:  Thirty publications were identified with data from 23 trials for meta-analysis, of which 17 were single arm. Efficacy and safety outcomes varied widely across studies. Heterogeneity across trials was reduced in subgroup analyses. The pooled 12-mo response rates were 24% (95% confidence interval [CI]: 16–32%) for trials with two or more prior BCG courses and 36% (95% CI: 25–47%) for those with one or more prior BCG courses. In a subgroup analysis, the inclusion of ≥50% of patients with CIS was associated with a lower response.

Conclusions: The variability in efficacy and safety outcomes highlights the need for consistent endpoint reporting and patient population definitions. With promising emerging treatments currently in development, efficacious and safe therapeutic options are urgently needed for this difficult-to-treat patient population.

Patient summary: We examined the efficacy and safety outcomes of treatments for non–muscle-invasive bladder cancer after bacillus Calmette-Guerin therapy. Outcomes varied across studies and patient populations, but emerging treatments currently in development show promising efficacy.

Authors: Ashish M.Kamat1  Seth P.Lerner2 Michael O’Donnell3 Mihaela V.Georgieva4 Min Yang4 Brant A. Inman5 Wassim Kassouf6 Stephen A. Boorjian7 Mark D. Tyson8 Girish S. Kulkarni9 Sam S. Chang10 Badrinath R. Konety11 Robert   S. Svatek12 ArjunBalar13 J. Alfred Witjes14

1. University of Texas MD Anderson Cancer Center, Houston, TX, USA
2. Baylor College of Medicine, Houston, TX, USA
3. The University of Iowa, Iowa City, IA, USA
4. Analysis Group, Boston, MA, USA
5. Duke University, Durham, NC, USA
6. McGill University Health Center, Montreal, QC, Canada
7. Mayo Clinic, Rochester, MN, USA
9. University of Toronto, Toronto, ON, Canada
11. University of Minnesota, Minneapolis, MN, USA
12. The University of Texas Health, San Antonio, TX, USA
13. NYU Langone Health, New York, NY, US
14. Radboud University Nijmegen Medical Centre, Nijmegen, The Netherlands


Source: Kamat, Ashish M., Seth P. Lerner, Michael O’Donnell, Mihaela V. Georgieva, Min Yang, Brant A. Inman and Wassim Kassouf u. a. 2020. Evidence-based Assessment of Current and Emerging Bladder-sparing Therapies for Non–muscle-invasive Bladder Cancer After Bacillus Calmette-Guerin Therapy: A Systematic Review and Meta-analysis. European Urology Oncology. doi:10.1016/j.euo.2020.02.006, (Zugegriffen: 22. März 2020).

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