Core Outcome Domains for Trials in Autosomal Dominant Polycystic Kidney Disease: An International Delphi Survey

Am J Kidney Dis. 2020 Sep;76(3):361-373. doi: 10.1053/j.ajkd.2020.01.005. Epub 2020 Apr 28.

Abstract

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.

Keywords: Autosomal dominant polycystic kidney disease (ADPKD); Delphi; chronic kidney disease (CKD); chronic pain; clinical trial design; core outcome domains; patient priorities; patient-reported outcomes (PROs); qualitative research; quality of life (QoL).

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Africa / epidemiology
  • Aged
  • Asia / epidemiology
  • Caregivers / psychology
  • Child
  • Consensus
  • Delphi Technique
  • Female
  • Health Personnel / psychology
  • Humans
  • Intracranial Aneurysm / etiology
  • Kidney Failure, Chronic / epidemiology
  • Kidney Failure, Chronic / etiology
  • Male
  • Middle Aged
  • Pain / etiology
  • Patient Reported Outcome Measures
  • Patients / psychology
  • Polycystic Kidney, Autosomal Dominant / complications
  • Polycystic Kidney, Autosomal Dominant / epidemiology
  • Polycystic Kidney, Autosomal Dominant / psychology
  • Polycystic Kidney, Autosomal Dominant / therapy*
  • Qualitative Research
  • Quality of Life
  • Self Concept
  • Socioeconomic Factors
  • Stress, Psychological
  • Young Adult