Most people with spina bifida (SB) suffer from urinary incontinence (UI), necessitating life-long urological care.
Health professionals have assumed that continence, achieved by medical or surgical management, leads to
better health-related quality of life (HRQOL) in people with SB. However, studies have failed to demonstrate
this association. Because SB results in a multisystem disability, UI may not be the only, or even primary,
determinant of HRQOL in people with SB. To respond to individuals’ preferences and needs, clinicians must
focus on patients’ individual health goals across several dimensions (e.g., symptoms; physical and social
function) and determine how well they are met. Only by identifying patients interested in UI treatments, and
helping them define their therapeutic goals, will we be able to determine if improving UI increases HRQOL. My
long-term goal is to use my clinical and research expertise to optimize children’s SB care, which is currently
disconnected from patient-centered goals and evidence-based practice. My short-term goal is to develop a tool
(MyGoal) to improve how we approach UI decisions with SB patients. This tool would offer many advantages,
including simplifying decision making by helping patients articulate what is important to them, framing the
discussion, and aligning treatments toward those goals. Finally, this approach should allow for effective shared
decision making (SDM) by letting patients select health outcomes of highest priority, and clinicians determine
treatments most likely to achieve them. By using the tool to achieve goal setting and attainment, we
hypothesize that patients will experience greater SDM, satisfaction with patient-provider communication, and
HRQOL. Therefore, the specific aims of this career development award are to: (1) Engage children with SB to
better understand their experience with UI in the context of other competing care issues; (2) Employ a mixed-
methods approach to develop and refine the MyGoal tool to identify SB patients interested in addressing UI
and establish their continence goals; and (3) Employ a ‘before-after’ prospective study design at a single site to
measure the impact of the MyGoal tool on SDM, children’s satisfaction with patient-provider communication,
and their HRQOL. The work proposed in this application will have a significant impact by improving SB patient
care through the use of patient-centered tools. In the future, we anticipate applying for R03-level funding to
develop the MyGoal tool for adults. We also anticipate applying for R01-level funding to evaluate interventions
targeting patient-centered goals in SB across several healthcare systems and to monitor renal function
prospectively, using the patient cohort from the K23 award. This application will serve to advance my expertise
in qualitative research, including design-thinking methods, patient communication and SDM, patient-reported
outcome evaluation, and cutting-edge quantitative study design and analysis. Receiving this award is essential
to my career development, as it will provide me with further practical research experience and didactic training,
within a team of experienced and encouraging mentors in a patient-centered research environment.
