Pain and Health-Related Quality of Life in ADPKD: Results from a National Patient-Powered Registry

Rationale and Objective ADPKD impacts health-related quality of life (HRQoL) including pain, discomfort, fatigue, emotional distress, and impaired mobility. Stakeholders prioritized kidney cyst-related pain as an important core outcome domain in clinical trials, leading to development of disease-specific assessment tools. Study Design The ADPKD Registry is hosted online with multiple disease-specific patient reported outcomes modules to characterize the patient experience in the United States. Setting and Participants The ADPKD Registry allows consented participants access to a Core Questionnaire which includes demographics, comorbid conditions, current symptoms, and kidney function. Participants complete subsequent modules on a 3-month schedule, including two validated HRQoL tools, the ADPKD Pain and Discomfort Scale (ADPKD-PDS), the ADPKD Impact Scale (ADPKD-IS) and a Healthcare Access and Utilization module. Exposures Patient-reported latest eGFR or creatinine used to calculate stage of chronic kidney disease. Outcomes Health-related quality of life, measured using validated ADPKD-specific tools; access to PKD-specific healthcare. Analytical Approach For the two HRQoL tools, scores were calculated for physical, emotional, and fatigue domains, pain severity, and pain interference (based on the licensed user manuals). Associations to health care access were also assessed. Results By July 2022, 1,086 individuals with ADPKD completed at least one of the HRQoL modules, 319 completed four over a year. Participants were an average age of 53, 71% women and 91% white, with all CKD stages represented. 2.5% reported being on dialysis and 23% had a kidney transplant. CKD Stage 4/5 participants reported the most dull kidney pain, while sharp kidney pain was evenly distributed across early CKD stages. Dull kidney pain had an impact on sleep regardless of CKD stage. There was a strong positive correlation between the ADPKD-PDS and ADPKD-IS. Patients with a neutral or positive HRQoL were less likely to have been denied access to imaging or other care. Limitations Currently all the information collected is patient-reported without health record validation of clinical variables. Conclusions Use of the HRQoL tools in the ADPKD Registry provided a broad cross-sectional assessment in the U.S. and provided granular information on the burden of pain across the CKD spectrum in ADPKD. The ADPKD Registry allowed assessment of ADPKD impact in a community that experiences decline in health and kidney function over decades.