Core Outcome Domains for Trials in Autosomal Dominant Polycystic Kidney Disease: An International Delphi SurveyCho, Y., Rangan, G., Logeman, C., Ryu, H., Sautenet, B., Perrone, R. D., Nadeau-Fredette, A-C., Mustafa, R. A., Htay, H., Chonchol, M., Harris, T., Gutman, T., Craig, J. C., Ong, A. C. M., Chapman, A., Ahn, C., Coolican, H., Kao, J. T-W., Gansevoort, R. T., Torres, V., Pei, Y., Johnson, D. W., Viecelli, A. K., Teixeira-Pinto, A., Howell, M., Ju, A., Manera, K. E. & Tong, A., Sep-2020, In : American Journal of Kidney Diseases. 76, 3, p. 361-373 13 p.
Research output: Contribution to journal › Article › Academic › peer-review
RATIONALE & OBJECTIVE: Outcomes reported in trials involving patients with autosomal dominant polycystic kidney disease (ADPKD) are heterogeneous and rarely include patient-reported outcomes. We aimed to identify critically important consensus-based core outcome domains to be reported in trials in ADPKD.
STUDY DESIGN: An international 2-round online Delphi survey was conducted in English, French, and Korean languages.
SETTING & PARTICIPANTS: Patients/caregivers and health professionals completed a 9-point Likert scale (7-9 indicating critical importance) and a Best-Worst Scale.
ANALYTICAL APPROACH: The absolute and relative importance of outcomes were assessed. Comments were analyzed thematically.
RESULTS: 1,014 participants (603 [60%] patients/caregivers, 411 [40%] health professionals) from 56 countries completed round 1, and 713 (70%) completed round 2. The prioritized outcomes were kidney function (importance score, 8.6), end-stage kidney disease (8.6), death (7.9), blood pressure (7.9), kidney cyst size/growth (7.8), and cerebral aneurysm (7.7). Kidney cyst-related pain was the highest rated patient-reported outcome by both stakeholder groups. Seven themes explained the prioritization of outcomes: protecting life and health, directly encountering life-threatening and debilitating consequences, specificity to ADPKD, optimizing and extending quality of life, hidden suffering, destroying self-confidence, and lost opportunities.
LIMITATIONS: Study design precluded involvement from those without access to internet or limited computer literacy.
CONCLUSIONS: Kidney function, end-stage kidney disease, and death were the most important outcomes to patients, caregivers, and health professionals. Kidney cyst-related pain was the highest rated patient-reported outcome. Consistent reporting of these top prioritized outcomes may strengthen the value of trials in ADPKD for decision making.
|Number of pages||13|
|Journal||American Journal of Kidney Diseases|
|Early online date||28-Apr-2020|
|Publication status||Published - Sep-2020|