International Bladder Cancer Group Consensus Statement on Clinical Trial Design for Patients with Bacillus Calmette-Guerin-exposed High-risk Non-muscle-invasive Bladder Cancer
Por:
Roumiguie, M, Kamat, AM, Bivalacqua, TJ, Lerner, SP, Kassouf, W, Bohle, A, Brausi, M, Buckley, R, Persad, R, Colombel, M, Lamm, D, Palou-Redorta, J, Soloway, M, Brothers, K, Steinberg, G, Lotan, Y, Sylvester, R, Witjes, JA, Black, PC
Publicada:
1 jul 2022
Ahead of Print:
1 jun 2022
Resumen:
Context: A large proportion of patients with non-muscle-invasive bladder cancer (NMIBC) fall in the gap between bacillus Calmette-Guerin (BCG)-naive and BCG-unresponsive disease. As multiple therapeutic agents move into this gray area, there is a critical need to define the disease state and establish recommendations for optimal trial design.
Objective: To develop a consensus on optimal trial design for patients with BCG-exposed NMIBC, defined as high-grade recurrence after BCG treatment that does not meet the criteria for BCG-unresponsive disease.
Evidence acquisition: We conducted a literature review using the Cochrane Library, Medline, and Embase and a review of clinical trials in ClinicalTrials.gov as a basis to generate consensus recommendations for clinical trial design in BCG-exposed NMIBC.
Evidence synthesis: BCG-exposed NMIBC encompasses BCG resistance (presence of high-grade Ta or carcinoma in situ [CIS] at 3-mo evaluation after induction BCG) and delayed relapse. Randomized controlled trials are required to compare experimental therapies to a control arm receiving additional BCG, although ongoing BCG shortages may impact our ability to follow an optimal trial design. A placebo should be used in combination with BCG if the treatment arm includes BCG plus a study drug. Trials will either need to separate patients with and without CIS into two cohorts, or stratify by the presence of CIS at the time of randomization. If two cohorts are used, the primary endpoint for CIS patients should be complete response within a predetermined time. The primary endpoint in a cohort with Ta/T1 only, or if a single combined cohort is used, should be the duration of event-free survival. Suggested efficacy thresholds and corresponding sample sizes are provided.
Conclusions: The International Bladder Cancer Group has developed recommendations regarding definitions, endpoints, and clinical trial design for BCG-exposed NMIBC to encourage uniformity among studies in this disease state.
Patient summary: Our consensus provides a precise definition of the disease state for bladder cancer not invading the bladder muscle and exposed to bacillus Calmette-Guerin (BCG) treatment. Clear guidance for conducting optimal clinical trials in this disease setting was established and we believe that this will promote further progress in this field. (C) 2021 European Association of Urology. Published by Elsevier B.V. All rights reserved.
Filiaciones:
Roumiguie, M:
Univ British Columbia, Vancouver Prostate Ctr, Dept Urol Sci, Vancouver, BC, Canada
Toulouse Canc Inst, Dept Urol, Toulouse, France
Toulouse Univ Hosp, Toulouse, France
Kamat, AM:
MD Anderson Canc Ctr, Dept Urol, Houston, TX USA
Bivalacqua, TJ:
Univ Penn, Dept Surg, Div Urol, Perelman Ctr Adv Med, Philadelphia, PA 19104 USA
Lerner, SP:
Baylor Coll Med, Scott Dept Urol, Dan L Duncan Canc Ctr, Houston, TX 77030 USA
Kassouf, W:
McGill Univ, Fac Med, Dept Surg Urol, Montreal, PQ, Canada
Bohle, A:
Univ Lubeck, Dept Urol, Lubeck, Germany
HELIOS Agnes Karll Krankenhaus, Bad Schwartau, Germany
Brausi, M:
Hesperia Hosp, Dept Urol, Modena, Italy
Buckley, R:
North York Gen Hosp, Toronto, ON, Canada
Persad, R:
North Bristol Hosp Trust, Southmead Hosp, Dept Urol, Bristol, Avon, England
Colombel, M:
Claude Bernard Univ, Hop Edouard Herriot, Lyon, France
Lamm, D:
Univ Arizona, Phoenix, AZ USA
BCG Oncol, Phoenix, AZ USA
Palou-Redorta, J:
Univ Autonoma Barcelona, Dept Urol, Fundacio Puigvert, Barcelona, Spain
Soloway, M:
Mem Hosp, Mem Canc Inst, Div Urol, Hollywood, FL USA
Brothers, K:
Natl Canc Inst Bladder Canc Task Force, Snowbasin, UT USA
Steinberg, G:
NYU Langone Hlth, Dept Urol, New York, NY USA
Lotan, Y:
UT Southwestern Med Ctr Dallas, Dept Urol, Dallas, TX USA
Sylvester, R:
European Assoc Urol Guidelines Off, Brussels, Belgium
Witjes, JA:
Radboud Univ Nijmegen, Dept Urol, Med Ctr, Nijmegen, Netherlands
Black, PC:
Univ British Columbia, Vancouver Prostate Ctr, Dept Urol Sci, Vancouver, BC, Canada
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