Core Outcome Domains for Trials in Autosomal Dominant Polycystic Kidney Disease: An International Delphi Survey.
Am J Kidney Dis
; 76(3): 361-373, 2020 09.
Article
em En
| MEDLINE
| ID: mdl-32359822
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. STUDYDESIGN:
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. ANALYTICALAPPROACH:
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 ofoutcomes:
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.Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Rim Policístico Autossômico Dominante
Tipo de estudo:
Etiology_studies
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Guideline
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Prognostic_studies
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Qualitative_research
Limite:
Adolescent
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Adult
/
Aged
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Child
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Female
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Humans
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Male
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Middle aged
País/Região como assunto:
Africa
/
Asia
Idioma:
En
Ano de publicação:
2020
Tipo de documento:
Article