American Journal of Kidney Diseases

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

      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.


      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.


      Most participants completed the survey in English.


      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.

      Index Words

      To read this article in full you will need to make a payment

      Purchase one-time access:

      Academic & Personal: 24 hour online accessCorporate R&D Professionals: 24 hour online access
      One-time access price info
      • For academic or personal research use, select 'Academic and Personal'
      • For corporate R&D use, select 'Corporate R&D Professionals'


      Subscribe to American Journal of Kidney Diseases
      Already a print subscriber? Claim online access
      Already an online subscriber? Sign in
      Institutional Access: Sign in to ScienceDirect


        • Chesnaye N.C.
        • Schaefer F.
        • Groothoff J.W.
        • et al.
        Mortality risk in European children with end-stage renal disease on dialysis.
        Kidney Int. 2016; 89: 1355-1362
        • Sethna C.B.
        • Merchant K.
        • Reyes A.
        Cardiovascular disease risk in children with kidney disease.
        Semin Nephrol. 2018; 38: 298-313
        • Tong A.
        • Wong G.
        • McTaggart S.
        • et al.
        Quality of life of young adults and adolescents with chronic kidney disease.
        J Pediatr. 2013; 163: 1179-1185 e1175
        • McDonald S.P.
        • Craig J.C.
        • Australian and New Zealand Paediatric Nephrology Association
        Long-term survival of children with end-stage renal disease.
        N Engl J Med. 2004; 350: 2654-2662
        • Tjaden L.A.
        • Vogelzang J.
        • Jager K.J.
        • et al.
        Long-term quality of life and social outcome of childhood end-stage renal disease.
        J Pediatr. 2014; 165: 336-342 e331
        • Tjaden L.A.
        • Maurice-Stam H.
        • Grootenhuis M.A.
        • Jager K.J.
        • Groothoff J.W.
        Impact of renal replacement therapy in childhood on long-term socioprofessional outcomes: a 30-year follow-up study.
        J Pediatr. 2016; 171 (189-195 e181-182)
        • Bertram J.F.
        • Goldstein S.L.
        • Pape L.
        • Schaefer F.
        • Shroff R.C.
        • Warady B.A.
        Kidney disease in children: latest advances and remaining challenges.
        Nat Rev Nephrol. 2016; 12: 182-191
        • Franke D.
        • Thomas L.
        • Steffens R.
        • et al.
        Patterns of growth after kidney transplantation among children with ESRD.
        Clin J Soc Am Nephrol. 2015; 10: 127-134
        • Medway M.
        • Tong A.
        • Craig J.C.
        • et al.
        Parental perspectives on the financial impact of caring for a child with CKD.
        Am J Kidney Dis. 2015; 65: 384-393
        • Tong A.
        • Lowe A.
        • Sainsbury P.
        • Craig J.C.
        Experiences of parents who have children with chronic kidney disease: a systematic review of qualitative studies.
        Pediatrics. 2008; 121: 349-360
        • Gutman T.
        • Hanson C.S.
        • Bernays S.
        • et al.
        Child and parental perspectives on communication and decision making in pediatric CKD: a focus group study.
        Am J Kidney Dis. 2018; 72: 547-559
        • Coyne I.
        • Amory A.
        • Kiernan G.
        • Gibson F.
        Children's participation in shared decision-making: children, adolescents, parents and healthcare professionals' perspectives and experiences.
        Eur J Oncol Nurs. 2014; 18: 273-280
        • Tong A.
        • Manns B.
        • Wang A.Y.M.
        • et al.
        Implementing core outcomes in kidney disease: report of the Standardized Outcomes in Nephrology (SONG) implementation workshop.
        Kidney Int. 2018; 94: 1053-1068
        • Chong L.S.H.
        • Sautenet B.
        • Tong A.
        • et al.
        Range and heterogeneity of outcomes in randomized trials of pediatric chronic kidney disease.
        J Pediatr. 2017; 186: 110-117
        • Joseph P.D.
        • Craig J.C.
        • Caldwell P.H.
        Clinical trials in children.
        Br J Clin Pharmacol. 2015; 79: 357-369
        • Williamson P.R.
        • Altman D.G.
        • Bagley H.
        • et al.
        The COMET Handbook: version 1.0.
        Trials. 2017; 18: 280
        • Manera K.E.
        • Tong A.
        • Craig J.C.
        • et al.
        An international Delphi survey helped develop consensus-based core outcome domains for trials in peritoneal dialysis.
        Kidney Int. 2019; 96: 699-710
        • Dalkey N.
        • Helmer O.
        An experimental application of the DELPHI method to the use of experts.
        Management Science. 1963; 9: 458-467
        • Cho Y.
        • Sautenet B.
        • Rangan G.
        • et al.
        Standardised Outcomes in Nephrology-Polycystic Kidney Disease (SONG-PKD): study protocol for establishing a core outcome set in polycystic kidney disease.
        Trials. 2017; 18: 560
        • Hamlet C.
        • Rumsey N.
        • Williamson H.
        • Johnson K.
        • Nduka C.
        Consensus research priorities for facial palsy: a Delphi survey of patients, carers, clinicians and researchers.
        J Plast Reconstr Aesthet Surg. 2018; 71: 1777-1784
        • Evangelidis N.
        • Tong A.
        • Manns B.
        • et al.
        Developing a set of core outcomes for trials in hemodialysis: an international Delphi survey.
        Am J Kidney Dis. 2017; 70: 464-475
        • Crudgington H.
        • Rogers M.
        • Bray L.
        • et al.
        Core Health Outcomes in Childhood Epilepsy (CHOICE): development of a core outcome set using systematic review methods and a Delphi survey consensus.
        Epilepsia. 2019; 60: 857-871
        • Hanson C.S.
        • Gutman T.
        • Craig J.C.
        • et al.
        Identifying important outcomes for young people with CKD and their caregivers: a nominal group technique study.
        Am J Kidney Dis. 2019; 74: 82-94
        • Schunemann H.
        • Brozek J.
        • Oxman A.
        GRADE Handbook for Grading Quality of Evidence and Strength of Recommendation.
        • Lee J.A.
        • Soutar G.
        • Louviere J.
        The best-worst scaling approach: an alternative to Schwartz's Values Survey.
        J Pers Assess. 2008; 90: 335-347
        • Bradley R.A.
        • Terry M.E.
        Rank analysis of incomplete block designs: I. the method of paired comparisons.
        Biometrika. 1952; 39: 324-345
        • Flynn T.N.
        • Louviere J.J.
        • Peters T.J.
        • Coast J.
        Best-worst scaling: what it can do for health care research and how to do it.
        J Health Econ. 2007; 26: 171-189
        • Giacomini M.K.
        • Cook D.J.
        Users' guides to the medical literature: XXIII. Qualitative research in health care A. Are the results of the study valid? Evidence-Based Medicine Working Group.
        JAMA. 2000; 284: 357-362
        • Modi Z.J.
        • Lu Y.
        • Ji N.
        • et al.
        Risk of cardiovascular disease and mortality in young adults with end-stage renal disease: an analysis of the US Renal Data System.
        JAMA Cardiol. 2019; 4: 353-362
        • Copelovitch L.
        • Warady B.A.
        • Furth S.L.
        Insights from the Chronic Kidney Disease in Children (CKiD) study.
        Clin J Am Soc Nephrol. 2011; 6: 2047-2053
        • Vogelzang J.L.
        • van Stralen K.J.
        • Jager K.J.
        • Groothoff J.W.
        Trend from cardiovascular to non-cardiovascular late mortality in patients with renal replacement therapy since childhood.
        Nephrol Dial Transplant. 2013; 28: 2082-2089
        • Mitsnefes M.M.
        • Laskin B.L.
        • Dahhou M.
        • Zhang X.
        • Foster B.J.
        Mortality risk among children initially treated with dialysis for end-stage kidney disease, 1990-2010.
        JAMA. 2013; 309: 1921-1929
        • Chavers B.M.
        • Solid C.A.
        • Gilbertson D.T.
        • Collins A.J.
        Infection-related hospitalization rates in pediatric versus adult patients with end-stage renal disease in the United States.
        J Am Soc Nephrol. 2007; 18: 952-959
        • Groothoff J.W.
        • Gruppen M.P.
        • Offringa M.
        • et al.
        Mortality and causes of death of end-stage renal disease in children: a Dutch cohort study.
        Kidney Int. 2002; 61: 621-629
        • Lofaro D.
        • Vogelzang J.L.
        • van Stralen K.J.
        • Jager K.J.
        • Groothoff J.W.
        Infection-related hospitalizations over 30 years of follow-up in patients starting renal replacement therapy at pediatric age.
        Pediatr Nephrol. 2016; 31: 315-323
        • Tjaden L.
        • Tong A.
        • Henning P.
        • Groothoff J.
        • Craig J.C.
        Children's experiences of dialysis: a systematic review of qualitative studies.
        Arch Dis Child. 2012; 97: 395-402
        • Bailey P.K.
        • Hamilton A.J.
        • Clissold R.L.
        • et al.
        Young adults' perspectives on living with kidney failure: a systematic review and thematic synthesis of qualitative studies.
        BMJ Open. 2018; 8e019926
        • Sautenet B.
        • Tong A.
        • Manera K.E.
        • et al.
        Developing consensus-based priority outcome domains for trials in kidney transplantation: a multinational Delphi survey with patients, caregivers, and health professionals.
        Transplantation. 2017; 101: 1875-1886
        • Cho Y.
        • Sautenet B.
        • Gutman T.
        • et al.
        Identifying patient-important outcomes in polycystic kidney disease: an international nominal group technique study.
        Nephrology. 2019; 24: 1214-1224
        • Amr M.
        • Bakr A.
        • El Gilany A.H.
        • Hammad A.
        • El-Refaey A.
        • El-Mougy A.
        Multi-method assessment of behavior adjustment in children with chronic kidney disease.
        Pediatr Nephrol. 2009; 24: 341-347
        • Lauth B.
        • Arnkelsson G.B.
        • Magnusson P.
        • Skarpheethinsson G.A.
        • Ferrari P.
        • Petursson H.
        Parent-youth agreement on symptoms and diagnosis: assessment with a diagnostic interview in an adolescent inpatient clinical population.
        J Physiol. 2010; 104: 315-322
        • Kilis-Pstrusinska K.
        • Medynska A.
        • Chmielewska I.B.
        • et al.
        Perception of health-related quality of life in children with chronic kidney disease by the patients and their caregivers: multicentre national study results.
        Qual Life Res. 2013; 22: 2889-2897
        • Masten A.S.
        • Best K.M.
        • Garmezy N.
        Resilience and development: contributions from the study of children who overcome adversity.
        Dev Psychopathol. 2008; 2: 425-444
        • Moreira J.M.
        • Bouissou Morais Soares C.M.
        • Teixeira A.L.
        • Simoes E.S.A.C.
        • Kummer A.M.
        Anxiety, depression, resilience and quality of life in children and adolescents with pre-dialysis chronic kidney disease.
        Pediatr Nephrol. 2015; 30: 2153-2162
        • Heath J.
        • Mackinlay D.
        • Watson A.R.
        • et al.
        Self-reported quality of life in children and young people with chronic kidney disease.
        Pediatr Nephrol. 2011; 26: 767-773
        • Leeman J.
        • Crandell J.L.
        • Lee A.
        • Bai J.
        • Sandelowski M.
        • Knafl K.
        Family functioning and the well-being of children with chronic conditions: a meta-analysis.
        Res Nurs Health. 2016; 39: 229-243
        • Block J.
        • Robins R.W.
        A longitudinal study of consistency and change in self-esteem from early adolescence to early adulthood.
        Child Dev. 1993; 64: 909-923
        • Akchurin O.M.
        • Kogon A.J.
        • Kumar J.
        • et al.
        Approach to growth hormone therapy in children with chronic kidney disease varies across North America: the Midwest Pediatric Nephrology Consortium report.
        BMC Nephrol. 2017; 18: 181
        • Ingulli E.G.
        • Mak R.H.
        Growth in children with chronic kidney disease: role of nutrition, growth hormone, dialysis, and steroids.
        Curr Opin Pediatr. 2014; 26: 187-192