| Source: |
Cho, 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 2020, 'Core Outcome Domains for Trials in Autosomal Dominant Polycystic Kidney Disease : An International Delphi Survey', American Journal of Kidney Diseases, vol. 76, no. 3, pp. 361-373. https://doi.org/10.1053/j.ajkd.2020.01.005 |
| Description: |
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. |