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Published in: Trials 1/2017

Open Access 01-12-2017 | Study protocol

Standardised Outcomes in Nephrology—Polycystic Kidney Disease (SONG-PKD): study protocol for establishing a core outcome set in polycystic kidney disease

Authors: Yeoungjee Cho, Benedicte Sautenet, Gopala Rangan, Jonathan C. Craig, Albert C. M. Ong, Arlene Chapman, Curie Ahn, Dongping Chen, Helen Coolican, Juliana Tze-Wah Kao, Ron Gansevoort, Ronald Perrone, Tess Harris, Vicente Torres, York Pei, Peter G. Kerr, Jessica Ryan, Talia Gutman, Martin Howell, Angela Ju, Karine E. Manera, Armando Teixeira-Pinto, Lorraine A. Hamiwka, Allison Tong

Published in: Trials | Issue 1/2017

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Abstract

Background

Autosomal dominant polycystic kidney disease (ADPKD) is the most common potentially life threatening inherited kidney disease and is responsible for 5–10% of cases of end-stage kidney disease (ESKD). Cystic kidneys may enlarge up to 20 times the weight of a normal kidney due to the growth of renal cysts, and patients with ADPKD have an increased risk of morbidity, premature mortality, and other life-time complications including renal and hepatic cyst and urinary tract infection, intracranial aneurysm, diverticulosis, and kidney pain which impair quality of life. Despite some therapeutic advances and the growing number of clinical trials in ADPKD, the outcomes that are relevant to patients and clinicians, such as symptoms and quality of life, are infrequently and inconsistently reported. This potentially limits the contribution of trials to inform evidence-based decision-making. The Standardised Outcomes in Nephrology—Polycystic Kidney Disease (SONG-PKD) project aims to establish a consensus-based set of core outcomes for trials in PKD (with an initial focus on ADPKD but inclusive of all stages) that patients and health professionals identify as critically important.

Methods

The five phases of SONG-PKD are: a systematic review to identify outcomes that have been reported in existing PKD trials; focus groups with nominal group technique with patients and caregivers to identify, rank, and describe reasons for their choices; qualitative stakeholder interviews with health professionals to elicit individual values and perspectives on outcomes for trials involving patients with PKD; an international three-round Delphi survey with all stakeholder groups (including patients, caregivers, healthcare providers, policy makers, researchers, and industry) to gain consensus on critically important core outcome domains; and a consensus workshop to review and establish a set of core outcome domains and measures for trials in PKD.

Discussion

The SONG-PKD core outcome set is aimed at improving the consistency and completeness of outcome reporting across ADPKD trials, leading to improvements in the reliability and relevance of trial-based evidence to inform decisions about treatment and ultimately improve the care and outcomes for people with ADPKD.
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Metadata
Title
Standardised Outcomes in Nephrology—Polycystic Kidney Disease (SONG-PKD): study protocol for establishing a core outcome set in polycystic kidney disease
Authors
Yeoungjee Cho
Benedicte Sautenet
Gopala Rangan
Jonathan C. Craig
Albert C. M. Ong
Arlene Chapman
Curie Ahn
Dongping Chen
Helen Coolican
Juliana Tze-Wah Kao
Ron Gansevoort
Ronald Perrone
Tess Harris
Vicente Torres
York Pei
Peter G. Kerr
Jessica Ryan
Talia Gutman
Martin Howell
Angela Ju
Karine E. Manera
Armando Teixeira-Pinto
Lorraine A. Hamiwka
Allison Tong
Publication date
01-12-2017
Publisher
BioMed Central
Published in
Trials / Issue 1/2017
Electronic ISSN: 1745-6215
DOI
https://doi.org/10.1186/s13063-017-2298-4

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