Bladder cancer is the fourth most common cancer in men and the tenth most common cancer in women. In 2017, over 77,000 Americans were diagnosed with bladder cancer, about three-quarters of whom had a type called non-muscle invasive bladder cancer (NMIBC), in which the cancer is limited to the inner layers of the bladder. NMIBC is usually treated with a combination of a procedure that removes the tumor while leaving the bladder in place and application of a medicine called Bacillus Calmette-Guérin (BCG) inserted directly into the bladder.
Unfortunately, NMIBC tumors frequently return or worsen. When this happens, patients face a difficult choice between undergoing radical cystectomy to remove their bladder entirely or trying additional therapy with medicine, such as intravesical treatment. This choice is not an easy one: selecting between these options involves weighing the risk of bladder cancer progression and the loss of a window of potential cure with intravesical therapy against the risk of morbidity and impact to quality of life with bladder removal.
Much remains unknown about the comparative effectiveness of these two treatment options. There is also very little information about how patient preferences and quality of life factor into the decision. The CISTO Study aims to fill in critical evidence gaps in order to facilitate personalized, patient-centered care. The results from CISTO may provide better evidence about the clinical and quality of life outcomes so that future patients, their caregivers, and their providers can make more informed treatment decisions.
Aim 1: To compare patient-reported and patient-centered clinical outcomes between patients undergoing radical cystectomy and those receiving bladder-sparing intravesical therapies for NMIBC that have failed first-line BCG.
Aim 2: To characterize the heterogeneity of treatments received and corresponding patient and caregiver preferences for NMIBC that have failed first-line BCG.
CISTO is a pragmatic observational study. What does this mean?
- The pragmatic trial approach looks at treatments and outcomes of the real-world patient experience.
- In an observational trial, the treatment option is selected by the patient.
- The CISTO Study includes three groups of participants:
- Patients who have selected radical cystectomy
- Patients who have selected intravesical therapy
CISTO will enroll 900 patient participants (at least 300 of whom have selected radical cystectomy) across 28 community- and university-based clinical sites. Participating sites will be asked to screen and enroll patients, to collect demographic and clinical data at the baseline study visit, and provide data on clinical outcomes for up to four years. The study will also enroll 25 caregiver participants. CISTO defines a caregiver as a person who partners with the patient in their care decisions. The Clinical Coordinating Center at the University of Washington will collect patient-reported outcomes during the follow-up period and perform qualitative interviews on a subset of these patients. Interviews will also be conducted with caregiver participants. The Data Coordinating Center will coordinate data operations for recruitment and reporting, and conduct data analyses. The Stakeholder Coordinating Center will work to ensure that the perspectives of patient partners and other stakeholders are incorporated throughout all phases of the study.
The Patient Voice
In developing this study the research team partnered with the Bladder Cancer Advocacy Network (BCAN), which is the only national advocacy organization devoted to advancing bladder cancer research and supporting those impacted by bladder cancer, to bring together a Patient Survey Network of over 1,300 bladder cancer patients. These bladder cancer patients identified questions that were most important to them. The top two questions for NMIBC patients were:
- How can patients and providers make decisions about the need for bladder removal, and what is the best timing?
- What are the best treatments for patients whose cancer returns or worsens after BCG treatment?
The CISTO Study has been designed with these critical questions at its core, ensuring that the patients’ voice has been incorporated throughout the development of this study. In addition, CISTO has a team of patients, caregivers, and external advisors who will continue to provide input about the implementation of the study and the sharing of results.