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

Yeoungjee Cho, Bénédicte Sautenet, Gopala K. Rangan, Jonathan C. Craig, Albert Albert C M Ong, Arlene B. Chapman, Curie Ahn, Dongping Chen, Helen Coolican, Juliana Tze Kao, Ron T. Gansevoort, Ronald D. Perrone, Tess L. Harris, Vicente E. Torres, York P.C. Pei, Peter G. Kerr, Jessica Ryan, Talia Gutman, Martin R. Howell, Angela JuKarine E. Manera, Armando Teixeira-Pinto, Lorraine A. Hamiwka, Allison Tong

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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.

Original languageEnglish
Article number560 (2017)
Number of pages11
Issue number1
Publication statusPublished - 23 Nov 2017
Externally publishedYes

Bibliographical note

© The Author(s). 2017 Open Access. This article is distributed under the terms of the Creative Commons Attribution 4.0International License (, which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The Creative Commons Public Domain Dedication waiver ( applies to the data made available in this article, unless otherwise stated.


  • Core outcome set
  • Outcomes research
  • Patient-centred outcomes clinical trials,
  • Chronic kidney disease
  • Autosomal dominant polycystic kidney disease (ADPKD)


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