Their team conducted a web-based survey of the 1,300-member BCAN PSN and responses were collected between March 6, 2017, and April 6, 2017. The survey included questions about bladder cancer diagnosis and treatment history, willingness to participate in clinical trials with different study designs, and the relative importance of various outcomes.
A total of 291 individuals previously diagnosed with bladder cancer responded, 201 of whom reported having had NMIBC, and 104 of whom had experienced recurrent NMIBC after BCG treatment. Among all respondents and within the subgroup of patients who experienced recurrent NMIBC after BCG, only 11% reported being willing to consent to randomization for a study of radical cystectomy versus intravesical therapy. The majority (76%) of patients indicated a strong preference to keep their bladders. Based on these survey results, a randomized trial was deemed not feasible and thus a prospective observational cohort study was selected as the highest quality, most feasible and inclusive study design. Respondents recommended that the most relevant domains with which to compare our two treatments were survival and QOL, ranked in order of:
- Overall survival
- Risk of spread of the cancer to other parts of the body
- Overall quality of life
- Impact of the treatments on their caregiver or loved one
- Urinary quality of life
- The costs or financial distress these treatments can incur
The CISTO analysis plan is to use targeted maximum likelihood estimation (TMLE) in an attempt to account for unmeasured confounders and use propensity scores as a secondary analysis.
Dr. Gore concluded noting that patient input from the BCAN PSN informed the design of a patient-centered pragmatic trial for bladder cancer. These results highlight the importance of involving patients throughout the research process in order to construct feasible study designs whose results will be important to the affected patients.
Presented by: John L. Gore, MD, MS, FACS, is an Associate Professor in Urology, University of Washington, Seattle, Washington
Written By: Zachary Klaassen, MD, MSc – Assistant Professor of Urology, Georgia Cancer Center, Augusta University - Medical College of Georgia, @zklaassen_md at American Urological Association's 2019 Annual Meeting (AUA 2019), May 3 – 6, 2019 in Chicago, Illinois