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

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 JuKarine E. Manera, Armando Teixeira-Pinto, Lorraine A. Hamiwka, Allison Tong

Research output: Contribution to journalArticle

1 Citation (Scopus)

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.

Original languageEnglish (US)
Article number560
JournalTrials
Volume18
Issue number1
DOIs
StatePublished - Nov 23 2017

Fingerprint

Autosomal Dominant Polycystic Kidney
Polycystic Kidney Diseases
Nephrology
Kidney
Caregivers
Cysts
Consensus
Consensus Development Conferences
Quality of Life
Cystic Kidney Diseases
Premature Mortality
Diverticulum
Health
Kidney Diseases
Intracranial Aneurysm
Focus Groups
Administrative Personnel
Urinary Tract Infections
Health Personnel
Chronic Kidney Failure

Keywords

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

ASJC Scopus subject areas

  • Medicine (miscellaneous)
  • Pharmacology (medical)

Cite this

Standardised Outcomes in Nephrology-Polycystic Kidney Disease (SONG-PKD) : Study protocol for establishing a core outcome set in polycystic kidney disease. / Cho, Yeoungjee; Sautenet, Benedicte; Rangan, Gopala; Craig, Jonathan C.; Ong, Albert C.M.; Chapman, Arlene; Ahn, Curie; Chen, Dongping; Coolican, Helen; Kao, Juliana Tze Wah; Gansevoort, Ron; Perrone, Ronald; Harris, Tess; Torres, Vicente; Pei, York; Kerr, Peter G.; Ryan, Jessica; Gutman, Talia; Howell, Martin; Ju, Angela; Manera, Karine E.; Teixeira-Pinto, Armando; Hamiwka, Lorraine A.; Tong, Allison.

In: Trials, Vol. 18, No. 1, 560, 23.11.2017.

Research output: Contribution to journalArticle

Cho, Y, Sautenet, B, Rangan, G, Craig, JC, Ong, ACM, Chapman, A, Ahn, C, Chen, D, Coolican, H, Kao, JTW, Gansevoort, R, Perrone, R, Harris, T, Torres, V, Pei, Y, Kerr, PG, Ryan, J, Gutman, T, Howell, M, Ju, A, Manera, KE, Teixeira-Pinto, A, Hamiwka, LA & Tong, A 2017, 'Standardised Outcomes in Nephrology-Polycystic Kidney Disease (SONG-PKD): Study protocol for establishing a core outcome set in polycystic kidney disease', Trials, vol. 18, no. 1, 560. https://doi.org/10.1186/s13063-017-2298-4
Cho, Yeoungjee ; Sautenet, Benedicte ; Rangan, Gopala ; Craig, Jonathan C. ; Ong, Albert C.M. ; Chapman, Arlene ; Ahn, Curie ; Chen, Dongping ; Coolican, Helen ; Kao, Juliana Tze Wah ; Gansevoort, Ron ; Perrone, Ronald ; Harris, Tess ; Torres, Vicente ; Pei, York ; Kerr, Peter G. ; Ryan, Jessica ; Gutman, Talia ; Howell, Martin ; Ju, Angela ; Manera, Karine E. ; Teixeira-Pinto, Armando ; Hamiwka, Lorraine A. ; Tong, Allison. / Standardised Outcomes in Nephrology-Polycystic Kidney Disease (SONG-PKD) : Study protocol for establishing a core outcome set in polycystic kidney disease. In: Trials. 2017 ; Vol. 18, No. 1.
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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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T1 - Standardised Outcomes in Nephrology-Polycystic Kidney Disease (SONG-PKD)

T2 - Study protocol for establishing a core outcome set in polycystic kidney disease

AU - Cho, Yeoungjee

AU - Sautenet, Benedicte

AU - Rangan, Gopala

AU - Craig, Jonathan C.

AU - Ong, Albert C.M.

AU - Chapman, Arlene

AU - Ahn, Curie

AU - Chen, Dongping

AU - Coolican, Helen

AU - Kao, Juliana Tze Wah

AU - Gansevoort, Ron

AU - Perrone, Ronald

AU - Harris, Tess

AU - Torres, Vicente

AU - Pei, York

AU - Kerr, Peter G.

AU - Ryan, Jessica

AU - Gutman, Talia

AU - Howell, Martin

AU - Ju, Angela

AU - Manera, Karine E.

AU - Teixeira-Pinto, Armando

AU - Hamiwka, Lorraine A.

AU - Tong, Allison

PY - 2017/11/23

Y1 - 2017/11/23

N2 - 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.

AB - 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.

KW - Autosomal dominant polycystic kidney disease

KW - Chronic kidney disease

KW - Core outcome set

KW - Outcomes research

KW - Patient-centred outcomes clinical trials

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