Advertisement

Patient-Reported Outcomes in Children With Chronic Kidney Disease

  • Leonela Villegas
    Correspondence
    Address reprint requests to Leonela Villegas, MD, Division of Nephrology, Department of Pediatrics, Children's Hospital of Philadelphia, 3500 Civic Center Blvd, Philadelphia, PA 19104.
    Affiliations
    Division of Nephrology, Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, PA
    Search for articles by this author
  • Sandra Amaral
    Affiliations
    Division of Nephrology, Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, PA

    Division of Epidemiology, Department of Biostatistics, Epidemiology and Informatics, University of Pennsylvania, Philadelphia, PA
    Search for articles by this author
      Summary: Patient-reported outcomes (PROs) are quantitative assessments of a patient's perspective on their health and are derived directly from the patient, as opposed to clinician interpretation. PROs can serve as unique tools to improve health care providers’ understanding of the patient's daily lived experience and highlight salient domains that are specific to children with chronic kidney disease (CKD). As such, PROs fill an important gap in achieving optimal health and well-being for children with CKD. However, several knowledge gaps remain in the implementation of PROs within both the clinical and research realms. This review provides a broad overview of PRO development, implementation for children with CKD, and highlights future directions and challenges.

      Keywords

      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:

      Subscribe to Seminars in Nephrology
      Already a print subscriber? Claim online access
      Already an online subscriber? Sign in
      Institutional Access: Sign in to ScienceDirect

      REFERENCES

        • Bikbov B
        • Purcell CA
        • Levey AS
        • et al.
        Global, regional, and national burden of chronic kidney disease, 1990–2017: a systematic analysis for the Global Burden of Disease Study 2017.
        The Lancet. 2020; 395: 709-733
        • Harambat J
        • van Stralen KJ
        • Kim JJ
        • Tizard EJ.
        Epidemiology of chronic kidney disease in children.
        Pediatr Nephrol. 2012; 27: 363-373
        • Modi ZJ
        • Waldo A
        • Selewski DT
        • Troost JP
        • Gipson DS.
        Inpatient pediatric CKD health care utilization and mortality in the United States.
        Am J Kidney Dis. 2021; 77: 500-508
        • Selewski DT
        • Massengill SF
        • Troost JP
        • et al.
        Gaining the Patient Reported Outcomes Measurement Information System (PROMIS) perspective in chronic kidney disease: a Midwest Pediatric Nephrology Consortium study.
        Pediatr Nephrol. 2014; 29: 2347-2356
        • DeWalt DA
        • Gross HE
        • Gipson DS
        • et al.
        PROMIS(®) pediatric self-report scales distinguish subgroups of children within and across six common pediatric chronic health conditions.
        Qual Life Res. 2015; 24: 2195-2208
        • Heath J
        • Norman P
        • Christian M
        • Watson A.
        Measurement of quality of life and attitudes towards illness in children and young people with chronic kidney disease.
        Qual Life Res. 2017; 26: 2409-2419
        • Amaral S
        • Schuchard J
        • Bruno C
        • Furth S
        • Forrest C.
        The lived experiences of chronic kidney disease: cross-sectional findings from the MyKidneyHealth Study.
        Paper presented at Pediatric Academic Societies 2020, Philadelphia, PA2020 May 4
        • Selewski DT
        • Troost JP
        • Massengill SF
        • et al.
        The impact of disease duration on quality of life in children with nephrotic syndrome: a Midwest Pediatric Nephrology Consortium study.
        Pediatr Nephrol. 2015; 30: 1467-1476
        • Gutman T
        • Hanson CS
        • 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
        • Hanson CS
        • Craig JC
        • Tong A.
        In their own words: the value of qualitative research to improve the care of children with chronic kidney disease.
        Pediatr Nephrol. 2017; 32: 1501-1507
        • Glenn TW
        • Riekert KA
        • Roter D
        • et al.
        Engagement and affective communication during pediatric nephrology clinic visits: associations with medication adherence.
        Patient Educ Couns. 2021; 104: 578-584
        • Perrone RD
        • Coons SJ
        • Cavanaugh K
        • Finkelstein F
        • Meyer KB.
        Patient-reported outcomes in clinical trials of CKD-related therapies: report of a symposium sponsored by the National Kidney Foundation and the US Food and Drug Administration.
        Am J Kidney Dis. 2013; 62: 1046-1057
        • Aiyegbusi OL
        • Kyte D
        • Cockwell P
        • Anderson N
        • Calvert M.
        A patient-centred approach to measuring quality in kidney care: patient-reported outcome measures and patient-reported experience measures.
        Curr Opin Nephrol Hypertens. 2017; 26: 442-449
        • Schick-Makaroff K
        • Thummapol O
        • Thompson S
        • et al.
        Strategies for incorporating patient-reported outcomes in the care of people with chronic kidney disease (PRO kidney): a protocol for a realist synthesis.
        Syst Rev. 2019; 8: 20
        • Selewski DT
        • Thompson A
        • Kovacs S
        • et al.
        Patient-reported outcomes in glomerular disease.
        Clin J Am Soc Nephrol. 2017; 12: 140-148
        • Dobbels F
        • Decorte A
        • Roskams A
        • Van Damme-Lombaerts R.
        Health-related quality of life, treatment adherence, symptom experience and depression in adolescent renal transplant patients.
        Pediatr Transplant. 2010; 14: 216-223
        • Cella D
        • Hahn EA
        • Jensen SE
        • et al.
        Patient-reported outcomes in performance measurement.
        RTI Press, Research Triangle Park, NC2015
        • Jabrayilov R
        • Emons WHM
        • Sijtsma K.
        Comparison of classical test theory and item response theory in individual change assessment.
        Appl Psychol Meas. 2016; 40: 559-572
        • Porter I
        • Gonçalves-Bradley D
        • Ricci-Cabello I
        • Gibbons C
        • Gangannagaripalli J
        • Fitzpatrick R
        • et al.
        Framework and guidance for implementing patient-reported outcomes in clinical practice: evidence, challenges and opportunities.
        J Comp Eff Res. 2016; 5: 507-519
        • Wu AW
        • Predmore ZS.
        Patient-reported outcomes: toward better measurement of patient-centered care in CKD.
        J Am Soc Nephrol. 2019; 30: 523-525
        • Tractenberg RE
        • Garver A
        • Ljungberg IH
        • Schladen MM
        • Groah SL.
        Maintaining primacy of the patient perspective in the development of patient-centered patient reported outcomes.
        PLoS One. 2017; 12e0171114
        • Johnston BC
        • Patrick DL
        • Devji T
        • et al.
        Patient-reported outcomes.
        Cochrane handbook for systematic reviews of interventions. John Wiley & Sons, Ltd, Hoboken, NJ2019: 479-492
        • Troost JP
        • Waldo A
        • Carlozzi NE
        • et al.
        The longitudinal relationship between patient-reported outcomes and clinical characteristics among patients with focal segmental glomerulosclerosis in the Nephrotic Syndrome Study Network.
        Clin Kidney J. 2020; 13: 597-606
        • Selewski DT
        • Troost JP
        • Cummings D
        • et al.
        Responsiveness of the PROMIS® measures to changes in disease status among pediatric nephrotic syndrome patients: a Midwest pediatric nephrology consortium study.
        Health Qual Life Outcomes. 2017; 15: 166
        • Murphy SL
        • Mahan JD
        • Troost JP
        • et al.
        Longitudinal changes in health-related quality of life in primary glomerular disease: results from the CureGN study.
        Kidney Int Rep. 2020; 5: 1679-1689
        • Goldstein SL
        • Graham N
        • Warady BA
        • et al.
        Measuring health-related quality of life in children with ESRD: performance of the generic and ESRD-specific instrument of the Pediatric Quality of Life Inventory (PedsQL).
        Am J Kidney Dis. 2008; 51: 285-297
        • Splinter A
        • Tjaden LA
        • Haverman L
        • et al.
        Children on dialysis as well as renal transplanted children report severely impaired health-related quality of life.
        Qual Life Res. 2018; 27: 1445-1454
        • Díaz-González de Ferris ME
        • Pierce CB
        • Gipson DS
        • et al.
        Health-related quality of life in children with chronic kidney disease is affected by the number of medications.
        Pediatr Nephrol. 2021; 36: 1307-1310
        • Foster A
        • Croot L
        • Brazier J
        • Harris J
        • O'Cathain A
        The facilitators and barriers to implementing patient reported outcome measures in organisations delivering health related services: a systematic review of reviews.
        J Patient Rep Outcomes. 2018; 2: 46
        • Tang E
        • Bansal A
        • Novak M
        • Mucsi I.
        Patient-reported outcomes in patients with chronic kidney disease and kidney transplant—part 1.
        Front Med (Lausanne). 2018; 4: 254
        • Garimella PS
        • Weiner DE.
        Value-based kidney care: aligning metrics and incentives to improve the health of people with kidney disease.
        J Am Soc Nephrol. 2019; 30: 2282-2284
        • Bamford J
        • Wirz L.
        Piloting psychology annual reviews as a method of measuring psychological distress and quality of life in paediatric renal transplant patients.
        Biomed Res Int. 2016; 20161685362
        • Hind T
        • Lui S
        • Moon E
        • et al.
        Post-traumatic stress as a determinant of quality of life in pediatric solid-organ transplant recipients.
        Pediatr Transpl. 2021; 25: e14005
        • Akber A
        • Portale AA
        • Johansen KL.
        Use of pedometers to increase physical activity among children and adolescents with chronic kidney disease.
        Pediatr Nephrol. 2014; 29: 1395-1402
        • Wong C
        • Gerson A
        • Hooper SR
        • et al.
        Effect of elevated blood pressure on quality of life in children with chronic kidney disease.
        Pediatr Nephrol. 2016; 31: 1129-1136
        • Raja K
        • Parikh A
        • Webb H
        • Hothi D.
        Use of a low-dose prednisolone regimen to treat a relapse of steroid-sensitive nephrotic syndrome in children.
        Pediatr Nephrol. 2017; 32: 99-105
        • Baek HS
        • Kang HG
        • Choi HJ
        • et al.
        Health-related quality of life of children with pre-dialysis chronic kidney disease.
        Pediatr Nephrol. 2017; 32: 2097-2105
        • Warady BA
        • Barcia J
        • Benador N
        • et al.
        De novo weekly and biweekly darbepoetin alfa dosing in pediatric patients with chronic kidney disease.
        Pediatr Nephrol. 2018; 33: 125-137
        • Carlson J
        • Gerson AC
        • Matheson MB
        • et al.
        A longitudinal analysis of the effect of anemia on health-related quality of life in children with mild-to-moderate chronic kidney disease.
        Pediatr Nephrol. 2020; 35: 1659-1667
        • Khullar S
        • Banh T
        • Vasilevska-Ristovska J
        • et al.
        Impact of steroids and steroid-sparing agents on quality of life in children with nephrotic syndrome.
        Pediatr Nephrol. 2021; 36: 93-102
        • Wang L
        • Hua R
        • Zhu Y
        • et al.
        Single dose of rituximab in children with steroid-dependent /frequently relapsing nephrotic syndrome, clinical efficacy and evaluation of health-related quality of life.
        Iran J Kidney Dis. 2021; 15: 7
        • Imani PD
        • Aujo J
        • Kiguli S
        • Srivaths P
        • Brewer ED.
        Chronic kidney disease impacts health-related quality of life of children in Uganda, East Africa.
        Pediatr Nephrol. 2021; 36: 323-331
        • Forrest CB
        • Forrest KD
        • Clegg JL
        • et al.
        Establishing the content validity of PROMIS Pediatric pain interference, fatigue, sleep disturbance, and sleep-related impairment measures in children with chronic kidney disease and Crohn's disease.
        J Patient Rep Outcomes. 2020; 4: 11
        • Reeve BB
        • Edwards LJ
        • Jaeger BC
        • et al.
        Assessing responsiveness over time of the PROMIS® pediatric symptom and function measures in cancer, nephrotic syndrome, and sickle cell disease.
        Qual Life Res. 2018; 27: 249-257
        • Canetta PA
        • Troost JP
        • Mahoney S
        • et al.
        Health-related quality of life in glomerular disease.
        Kidney Int. 2019; 95: 1209-1224
        • Troost JP
        • Gipson DS
        • Carlozzi NE
        • et al.
        Using PROMIS® to create clinically meaningful profiles of nephrotic syndrome patients.
        Health Psychol. 2019; 38: 410-421