Establishing Meaningful Patient-Centered Outcomes with Relevance for Patients with Polycystic Kidney Disease: Patient, Caregiver, and Researcher Priorities for Research in Polycystic Kidney Disease.

KEY POINTS

Participants are less concerned about mortality than they are about kidney health. Psychological effect emerged as the most important theme regarding the effects of polycystic kidney disease on patients.

BACKGROUND

Patient involvement in research can help to ensure that the evidence generated aligns with their needs and priorities. In the Establishing Meaningful Patient-Centered Outcomes with Relevance for Patients with Polycystic Kidney Disease (PKD) project, we aimed to identify patient-important outcomes and discuss the effect of PKD on patients.

METHODS

Nine focus groups were held with adult patients with PKD, caregivers, and clinical or research experts in PKD. We used a nominal, multivote technique to rank patient-important outcomes to be prioritized by future PKD research. We conducted a thematic analysis of verbatim transcriptions to identify themes regarding the effect of PKD on their daily lives. Other focus group topics included insurability and patient engagement.

RESULTS

Ninety patients and/or caregivers and eight clinicians and/or researchers participated in the focus groups. Nine focus groups yielded 35 outcomes important to patients that were grouped into six categories, ranked in order of importance: kidney health, comorbidities, lifestyle, psychological effect, family and awareness, and mortality. Regarding the effect of PKD on the patient's daily lives, we identified five themes, listed in order of importance: psychological effect, effect on daily living, issues affecting decision making, health care-related issues, and PKD-specific testing dilemmas.

CONCLUSIONS

This study of stakeholder engagement in patients with PKD revealed important priorities and values that should be considered for future research and when caring for patients with PKD. Future research should focus on kidney health and managing comorbidities in patients with PKD. This will help to bridge the knowledge gap and develop meaningful comparative effectiveness research in PKD.