Identifying patient-important outcomes in polycystic kidney disease: An international nominal group technique study

Yeoungjee Cho; Benedicte Sautenet; Talia Gutman; Gopala Rangan; Jonathan C Craig; Albert C Ong; Arlene Chapman; Curie Ahn; Helen Coolican; Juliana T-W Kao; Ron Gansevoort; Ronald D Perrone; Tess Harris; Vicente Torres; York Pei; Peter G Kerr; Jessica Ryan; David W Johnson; Andrea K Viecelli; Claire Geneste; Hyunsuk Kim; Yaerim Kim; Yun Kyu Oh; Armando Teixeira-Pinto; Charlotte Logeman; Martin Howell; Angela Ju; Karine E Manera; Allison Tong

doi:10.1111/nep.13566

Identifying patient-important outcomes in polycystic kidney disease: An international nominal group technique study

Nephrology (Carlton). 2019 Dec;24(12):1214-1224. doi: 10.1111/nep.13566. Epub 2019 May 2.

Authors

Yeoungjee Cho^{1

2

3}, Benedicte Sautenet⁴, Talia Gutman^{5

6}, Gopala Rangan^{7

8}, Jonathan C Craig⁹, Albert C Ong¹⁰, Arlene Chapman¹¹, Curie Ahn¹², Helen Coolican¹³, Juliana T-W Kao^{14

15}, Ron Gansevoort¹⁶, Ronald D Perrone¹⁷, Tess Harris¹⁸, Vicente Torres¹⁹, York Pei²⁰, Peter G Kerr²¹, Jessica Ryan²¹, David W Johnson^{1

2

3}, Andrea K Viecelli¹, Claire Geneste⁴, Hyunsuk Kim¹², Yaerim Kim¹², Yun Kyu Oh¹², Armando Teixeira-Pinto^{5

6}, Charlotte Logeman^{5

6}, Martin Howell^{5

6}, Angela Ju^{5

6}, Karine E Manera^{5

6}, Allison Tong^{5

6}

Affiliations

¹ Department of Nephrology, Princess Alexandra Hospital.
² Australasian Kidney Trials Network, University of Queensland.
³ Translational Research Institute, Brisbane, Queensland, Australia.
⁴ Department of Nephrology Hypertension, Dialysis, Kidney Transplantation, Tours Hospital, SPHERE - INSERM 1246, University of Tours and Nantes, Tours, France.
⁵ Sydney School of Public Health, The University of Sydney, Sydney, New South Wales, Australia.
⁶ Centre for Kidney Research, The Children's Hospital at Westmead.
⁷ Centre for Transplant and Renal Research, Westmead Institute for Medical Research, The University of Sydney.
⁸ Department of Renal Medicine, Westmead Hospital, Western Sydney Local Health District, Sydney, New South Wales, Australia.
⁹ College of Medicine and Public Health, Flinders University, Adelaide, South Australia, Australia.
¹⁰ Academic Nephrology Unit, Department of Infection Immunity & Cardiovascular Disease, University of Sheffield, Sheffield, UK.
¹¹ Department of Medicine, The University of Chicago, Chicago, Illinois, USA.
¹² Division of Nephrology, Seoul National University Hospital, Seoul, South Korea.
¹³ Polycystic Kidney Disease Foundation of Australia, Sydney, Australia.
¹⁴ School of Medicine, Fu Jen Catholic University and Fu Jen Catholic University Hospital.
¹⁵ Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan.
¹⁶ Faculty of Medical Sciences, University Medical Center Gronigen, Gronigen, the Netherlands.
¹⁷ Division of Nephrology, Tufts Medical Center, Tufts University School of Medicine, Boston, Massachusetts, US.
¹⁸ Polycystic Kidney Disease International, London, UK.
¹⁹ Department of Nephrology and Hypertension, Mayo Clinic, Rochester, Minnestota, USA.
²⁰ Division of Nephrology and Division of Genomic Medicine, University of Toronto, Toronto, Canada.
²¹ Department of Nephrology, Monash Medical Centre and Monash University, Melbourne, Victoria, Australia.

PMID: 30663163
DOI: 10.1111/nep.13566

Abstract

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.

Keywords: cyst; kidney function; outcomes; patient; polycystic kidney disease; qualitative.

MeSH terms

Attitude to Health
Australia
Caregivers / psychology
Cost of Illness*
Disease Progression
Evaluation Studies as Topic
Female
France
Humans
Kidney Failure, Chronic* / etiology
Kidney Failure, Chronic* / mortality
Kidney Function Tests / psychology
Life Style*
Male
Middle Aged
Patient Outcome Assessment*
Polycystic Kidney, Autosomal Dominant* / diagnosis
Polycystic Kidney, Autosomal Dominant* / epidemiology
Polycystic Kidney, Autosomal Dominant* / physiopathology
Polycystic Kidney, Autosomal Dominant* / psychology
Progression-Free Survival
Quality of Life*
Republic of Korea