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Identifying patient‐important outcomes in polycystic kidney disease: An international nominal group technique study
Author(s) -
Cho Yeoungjee,
Sautenet Benedicte,
Gutman Talia,
Rangan Gopala,
Craig Jonathan C,
Ong Albert C,
Chapman Arlene,
Ahn Curie,
Coolican Helen,
Kao Juliana TW,
Gansevoort Ron,
Perrone Ronald D,
Harris Tess,
Torres Vicente,
Pei York,
Kerr Peter G,
Ryan Jessica,
Johnson David W,
Viecelli Andrea K,
Geneste Claire,
Kim Hyunsuk,
Kim Yaerim,
Oh Yun Kyu,
TeixeiraPinto Armando,
Logeman Charlotte,
Howell Martin,
Ju Angela,
Manera Karine E,
Tong Allison
Publication year - 2019
Publication title -
nephrology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.752
H-Index - 61
eISSN - 1440-1797
pISSN - 1320-5358
DOI - 10.1111/nep.13566
Subject(s) - medicine , autosomal dominant polycystic kidney disease , quality of life (healthcare) , disease , kidney disease , normality , physical therapy , renal function , polycystic kidney disease , intensive care medicine , nursing , psychiatry
Aim Patients with autosomal dominant polycystic kidney disease (ADPKD) are at increased risk of premature mortality, morbidities and complications, which severely impair quality of life. However, patient‐centered outcomes are not consistently reported in trials in ADPKD, which can limit shared decision‐making. We aimed to identify outcomes important to patients and caregivers and the reasons for their priorities. Methods Nominal group technique was adopted involving patients with ADPKD and caregivers who were purposively selected from eight centres across Australia, France and the Republic of Korea. Participants identified, ranked and discussed outcomes for trials in ADPKD. We calculated an importance score (0–1) for each outcome and conducted thematic analyses. Results Across 17 groups, 154 participants (121 patients, 33 caregivers) aged 19 to 78 (mean 54.5 years) identified 55 outcomes. The 10 highest ranked outcomes were: kidney function (importance score 0.36), end‐stage kidney disease (0.32), survival (0.21), cyst size/growth (0.20), cyst pain/bleeding (0.18), blood pressure (0.17), ability to work (0.16), cerebral aneurysm/stroke (0.14), mobility/physical function (0.12), and fatigue (0.12). Three themes were identified: threatening semblance of normality, inability to control and making sense of diverse risks. Conclusion For patients with ADPKD and their caregivers, kidney function, delayed progression to end‐stage kidney disease and survival were the highest priorities, and were focused on achieving normality, and maintaining control over health and lifestyle. Implementing these patient‐important outcomes may improve the meaning and relevance of trials to inform clinical care in ADPKD.