Developing Consensus-Based Outcome Domains for Trials in Children and Adolescents With CKD: An International Delphi Survey

Charlotte Logeman; Chandana Guha; Martin Howell; Camilla S Hanson; Jonathan C Craig; Susan Samuel; Michael Zappitelli; Mina Matsuda-Abedini; Allison Dart; Susan Furth; Allison Eddy; Jaap Groothoff; Hui-Kim Yap; Detlef Bockenhauer; Aditi Sinha; Stephen I Alexander; Stuart L Goldstein; Debbie S Gipson; Mini Michael; Amanda Walker; Joshua Kausman; Segolene Gaillard; Justine Bacchetta; Michelle N Rheault; Bradley A Warady; Alicia Neu; Martin Christian; Steven McTaggart; Isaac Liu; Sharon Teo; Benedicte Sautenet; Talia Gutman; Simon Carter; Armando Teixeira-Pinto; Allison Tong

doi:10.1053/j.ajkd.2020.03.014

Developing Consensus-Based Outcome Domains for Trials in Children and Adolescents With CKD: An International Delphi Survey

Am J Kidney Dis. 2020 Oct;76(4):533-545. doi: 10.1053/j.ajkd.2020.03.014. Epub 2020 Jul 10.

Authors

Charlotte Logeman¹, Chandana Guha¹, Martin Howell¹, Camilla S Hanson¹, Jonathan C Craig², Susan Samuel³, Michael Zappitelli⁴, Mina Matsuda-Abedini⁴, Allison Dart⁵, Susan Furth⁶, Allison Eddy⁷, Jaap Groothoff⁸, Hui-Kim Yap⁹, Detlef Bockenhauer¹⁰, Aditi Sinha¹¹, Stephen I Alexander¹², Stuart L Goldstein¹³, Debbie S Gipson¹⁴, Mini Michael¹⁵, Amanda Walker¹⁶, Joshua Kausman¹⁶, Segolene Gaillard¹⁷, Justine Bacchetta¹⁸, Michelle N Rheault¹⁹, Bradley A Warady²⁰, Alicia Neu²¹, Martin Christian²², Steven McTaggart²³, Isaac Liu²⁴, Sharon Teo²⁴, Benedicte Sautenet²⁵, Talia Gutman¹, Simon Carter¹, Armando Teixeira-Pinto¹, Allison Tong²⁶

Affiliations

¹ Sydney School of Public Health, The University of Sydney, NSW, Australia; Centre for Kidney Research, The Children's Hospital at Westmead, Westmead, NSW, Australia.
² College of Medicine and Public Health, Flinders University, Adelaide, Australia.
³ Department of Pediatrics, Section of Nephrology, University of Calgary, Calgary, AB, Canada.
⁴ Department of Pediatrics, Division of Nephrology, Toronto Hospital for Sick Children, Toronto, Canada.
⁵ Department of Pediatrics and Child Health, The Children's Hospital Research Institute of Manitoba, University of Manitoba, Winnipeg, MB, Canada.
⁶ Department of Pediatrics, Perelman School of Medicine, Philadelphia, PA; Department of Epidemiology, Perelman School of Medicine, Philadelphia, PA; Division of Nephrology, The Children's Hospital of Philadelphia, Philadelphia, PA.
⁷ Department of Pediatrics, University of British Columbia, Vancouver, BC, Canada.
⁸ Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam University Medical Center, location AMC, Amsterdam, the Netherlands.
⁹ Department of Pediatrics, Yong Loo Lin School of Medicine, National University of Singapore, Singapore.
¹⁰ UCL Department of Renal Medicine and Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom.
¹¹ Division of Nephrology, Department of Pediatrics, All India Institute of Medical Sciences, New Delhi, India.
¹² Centre for Kidney Research, The Children's Hospital at Westmead, Westmead, NSW, Australia.
¹³ Center for Acute Care Nephrology, Cincinnati Children's Hospital, Cincinnati, OH.
¹⁴ Institute for Healthcare Policy and Innovation, University of Michigan, Ann Arbor, MI.
¹⁵ Renal Section, Department of Pediatrics, Baylor College of Medicine/Texas Children's Hospital, Houston, TX.
¹⁶ Department of Paediatrics, University of Melbourne, Melbourne, Victoria, Australia; Department of Nephrology and Murdoch Children's Research Institute, Royal Children's Hospital, Melbourne, Victoria, Australia.
¹⁷ Hospices Civils de Lyon, EPICIME-CIC 1407 de Lyon, Inserm, Service de Pharmacologie Clinique, Bron, France.
¹⁸ Department of Pediatric Nephrology, Reference Center for Rare Renal Diseases, Hospices civils de Lyon, Lyon, France.
¹⁹ Division of Nephrology, Department of Pediatrics, University of Minnesota Masonic Children's Hospital, Minneapolis, MN.
²⁰ Children's Mercy Hospital, Kansas City, MO.
²¹ Division of Pediatric Nephrology, The Johns Hopkins University School of Medicine, Baltimore, MD.
²² Nottingham Children's Hospital, Nottingham, United Kingdom.
²³ Child and Adolescent Renal Service, Queensland Children's Hospital and University of Queensland, Brisbane, Australia.
²⁴ Khoo Teck Puat-National University Children's Medical Institute, National University Hospital, Singapore.
²⁵ Department of Nephrology Hypertension, dialysis, kidney transplantation, Tours Hospital, SPHERE - INSERM 1246, University of Tours and Nantes, Tours, France.
²⁶ Sydney School of Public Health, The University of Sydney, NSW, Australia; Centre for Kidney Research, The Children's Hospital at Westmead, Westmead, NSW, Australia. Electronic address: [email protected].

PMID: 32654889
DOI: 10.1053/j.ajkd.2020.03.014

Abstract

Rationale & objective: The inconsistency in outcomes reported and lack of patient-reported outcomes across trials in children with chronic kidney disease (CKD) limits shared decision making. As part of the Standardized Outcomes in Nephrology (SONG)-Kids initiative, we aimed to generate a consensus-based prioritized list of critically important outcomes to be reported in all trials in children with CKD.

Study design: An online 2-round Delphi survey in English, French, and Hindi languages.

Settings & participants: Patients (aged 8-21 years), caregivers/family, and health care professionals (HCPs) rated the importance of outcomes using a 9-point Likert scale (7-9 indicating critical importance) and completed a Best-Worst Scale.

Analytical approach: We assessed the absolute and relative importance of outcomes. Comments were analyzed thematically.

Results: 557 participants (72 [13%] patients, 132 [24%] caregivers, and 353 [63%] HCPs) from 48 countries completed round 1 and 312 (56%) participants (28 [40%] patients, 64 [46%] caregivers, and 220 [56%] HCPs) completed round 2. Five outcomes were common in the top 10 for each group: mortality, kidney function, life participation, blood pressure, and infection. Caregivers and HCPs rated cardiovascular disease higher than patients. Patients gave lower ratings to all outcomes compared with caregivers/HCPs except they rated life participation (round 2 mean difference, 0.1), academic performance (0.1), mobility (0.4), and ability to travel (0.4) higher than caregivers and rated ability to travel (0.4) higher than HCPs. We identified 3 themes: alleviating disease and treatment burden, focusing on the whole child, and resolving fluctuating and conflicting goals.

Limitations: Most participants completed the survey in English.

Conclusions: Mortality, life participation, kidney function, and blood pressure were consistently highly prioritized by patients, caregivers, and HCPs. Patients gave higher priority to some lifestyle-related outcomes compared with caregivers/HCPs. Establishing critically important outcomes for all trials in children with CKD may improve consistent reporting of survival, kidney health, and clinical and life impact outcomes that are meaningful for decision making.

Keywords: Chronic kidney disease (CKD); Delphi survey; adolescents; blood pressure; caregivers; children; clinical trial design; consensus; core outcome set; kidney function; life participation; mortality; outcome measures; outcomes; patient-centered outcomes; pediatrics; priority ranking; treatment goals.

Publication types

Research Support, Non-U.S. Gov't

MeSH terms

Adolescent
Caregivers
Child
Consensus*
Delphi Technique*
Female
Health Personnel
Humans
International Cooperation
Male
Patient Outcome Assessment*
Renal Insufficiency, Chronic / therapy*
Treatment Outcome
Young Adult