In the bladder cancer research arena there is growing effort to include patients in the design and implementation of studies in order to better identify outcomes that resonate most with patients. Patient reported outcomes (PROs), which include health related quality of life (HRQOL) domains as well as issues that impact financial well-being and decisional regret, offer us key insights from the patient perspective that can then guide us in the pursuit of patient centered outcomes. Given that bladder cancer patients appear to be at increased risk for depression and even suicide, these are critically important outcomes to study.¹

Understanding PROs requires the use of validated measures and longitudinal assessments, as well as the recognition that PROs are stage specific. There are several validated HRQOL instruments currently available integrating many of these end points, summarized in the Appendix. Comparisons of HRQOL scores between continent and incontinent diversion types among patients undergoing cystectomy is well characterized in the literature, the bulk of which is cross-sectional. In the nonmuscle invasive bladder cancer (NMIBC) space, while global health status scores appear satisfactory, sexual function domain scores are low. Patients who have undergone radical cystectomy consistently report lower HRQOL scores than patients with NMIBC with intact bladders. While HRQOL measures are well described for bladder cancer, there is a paucity of data regarding several meaningful patient centered end points.

It is increasingly recognized that financial toxicity, defined as the material as well as the psychological impact of navigating cancer care borne by patients and their caregivers and the associated coping mechanisms, has a detrimental impact on HRQOL, adherence and perhaps even survival. Despite bladder cancer being one of the most expensive cancers to treat on a per capita basis, financial toxicity is understudied in this group. Casilla-Lennon et al found that 24% of bladder cancer patients surveyed endorsed having “to pay more for medical care than you can afford,” with participants who were younger more likely to report this.² Financial toxicity was associated with lower general and cancer specific HRQOL and well-being scores, and almost twice the rate of delaying care due to costs. Further work using now available validated measures of financial toxicity is underway by our group.

Decisional regret, another understudied PRO, is defined as remorse related to a medical decision and has been associated with psychosocial distress and lower HRQOL. Studies comparing satisfaction and HRQOL between patients who undergo an orthotopic neobladder vs ileal conduit have shown mixed results. Given the difficulty in accounting for baseline differences in patients who undergo each type of diversion, the utility of these data is limited but overall suggests that patients who are appropriately counseled are typically satisfied with their diversion choice. Recently, Check et al specifically focused on decisional regret among 300 patients at 6 and 18 months after cystectomy.³ Patients completed validated measures of HRQOL, shared decision making, goal dissonance and the decision regret scale (DRS; 5 item). They found no differences in DRS scores over time, diversion type, clinical outcomes or demographics, but found that informed decision making predicted lower regret at both time points (b=–13.08, 95% CI –17.05 to –9.11, and b=–8.54, 95% CI –14.26 to –2.63).

With this in mind, we identify 3 key strategies to improving our understanding of patient centered outcomes that matter most to affected patients and how the use of PROs can facilitate this. First, incorporating patient engagement in research design vis-à-vis community based participatory research may be effective. For example, the Bladder Cancer Advocacy Network Patient Survey Network was created in 2014 and is a place for patients and caregivers to voice their opinions on bladder cancer research. This engagement platform has provided a successful example for PROs and research prioritization in bladder cancer. Hamad et al recently created an effective online learning module named PEER (Patient Empowerment through Engagement in Research) designed to train this community in bladder cancer disease specific research.⁴ Developing researcher training programs alike to better engage patients in research design is a novel area.

Second, supporting patient-centered trials that assess the breadth of patient centered outcomes is critically important. For instance, the Comparison of Intravesical Therapy and Surgery as Treatment Options for Bladder Cancer study evaluating patient reported HRQOL outcomes in patients with high risk NMIBC may serve as a landmark clinical trial in this regard. In this longitudinal and pragmatic study, a broad range of validated PRO outcomes along with qualitative investigation hold the promise of providing robust data to understand the experience of bacillus Calmette-Guérin nonresponsive patients as they make decisions to pursue further bladder sparing therapies vs cystectomy.

Third, it is imperative that we incorporate a diverse patient population across racial, gender and socioeconomic lines in our research. Disparities in outcomes and experiences are significant across Black, Medicaid and low socioeconomic status patients.⁵ Often, clinical trials and advocacy groups tend to be comprised of patients of higher socioeconomic status, higher education level and English speaking patients, and are less likely to include patients of color. How do we hear the voices of patients who do not fall into these categories? Patient navigation models may be key. Patient navigators are the central point of contact in coordinating communication between physicians and patients. This may address potential barriers in care, result in optimized utilization and matching of resources to patients, and enhance patient education regarding bladder cancer diagnosis, treatment and followup.

Overall, the PROs we measure should be shaped by 2 factors: end points that patients value and those that impact clinically meaningful outcomes. As we pay increasing attention to patient priorities beyond those of the surgeon and oncologist, such as survival and recurrence of disease, we may identify meaningful end points that may aid in treatment shared decision making in bladder cancer.