Health-related quality of life functioning over a 2-year period in children with end-stage renal disease
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- Neul, S.K., Minard, C.G., Currier, H. et al. Pediatr Nephrol (2013) 28: 285. doi:10.1007/s00467-012-2313-7
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Optimal care of the pediatric chronic kidney disease/end stage renal disease (CKD/ESRD) patient must now incorporate health-related quality of life (HRQOL) assessment and management.
This study reports the first data on longitudinal change in global (PedsQLTM4.0) and disease-specific (PedsQLTM3.0 ESRD Module) HRQOL pediatric ESRD patient and proxy ratings over four assessment periods spanning approximately a 2-year period. General linear mixed modeling was used to analyze associations between patient demographics, medical variables, and patient and proxy HRQOL scores.
Self-reported and/or proxy data were available for at least two time-points for 53 patients (age 2–18 years; 60 % male), of whom 27 were receiving in-center hemodialysis.
Patient ratings on global health and physical activity, emotional, and social and disease-specific worry and communication domains were higher (i.e., better) than parent-proxy ratings, confirming the importance of obtaining both sources of information. Patients on dialysis longer, particularly females, reported worse emotional functioning; females also reported more physical appearance concerns. Parents rated older children and those on dialysis longer as functioning worse on multiple global and disease-specific (e.g., fatigue, relationship) domains. Parents also rated children as functioning increasingly worse in school over time. Further, patient ESRD history (acute onset vs. medically managed) impacted how parents viewed the burden of ESRD on their child over time.
KeywordsChildrenChronic kidney diseaseHealth-related quality of life
Children with chronic kidney disease (CKD) are now afforded improved long-term survival as a result of advances in technology and medical care . Such improvement in survival begs the question: “How well are we doing at addressing these patients’ health-related quality of life?” In earlier studies, children with end stage renal disease (ESRD) and their parents reported health-related quality of life (HRQOL) levels similar to that of a sample of newly diagnosed pediatric cancer patients undergoing chemotherapy and radiation [2, 3]. Children with ESRD are at-risk for underperforming academically, struggling socially, and experiencing adjustment difficulties and psychological stress [4–6]. Although their experiences can be similar to children with other chronic health conditions , children with ESRD must contend with restrictive dietary and fluid regimes and a lifetime cycle of recurrent dialysis and transplantation to sustain life . Further, dialysis treatments schedules are burdensome and interfere with school attendance and participation in peer-related activities, thereby compromising opportunities for attaining academic and psychosocial potential [9, 10]. As young adults, many struggle to complete their education, secure gainful employment, experience intimate relationships, and live independently [11–14]. To better understand how these challenges arise and to measure associations with interventions, standardized assessment using validated measures are needed to track an individual patient’s HRQOL strengths and needs . Furthermore, longitudinal assessment of HRQOL is necessary to identify patient factors (e.g., treatment modality, patient age, and gender) that may play a role in HRQOL trajectories, choice of intervention efforts at particular points in time, and impact on medical and psychosocial outcomes [16–19].
Assessment of HRQOL in pediatric dialysis patients is now mandated at least once per year by the United States Center for Medicaid and Medicare Services; however, there is little guidance regarding choice of measures, information management, and intervention development to improve and track HRQOL . In adult patients with ESRD, validated HRQOL assessment measures, such as the Short Form 36 Health Survey (SF-36), which measures more global aspects of quality of life, and the Kidney Disease Quality of Life instrument (KDQOLTM), which measures disease-specific aspects, are widely utilized to assess the impact of medical management on physical and emotional well-being . In pediatric patients with ESRD, validated global HRQOL assessment measures are available, such as the PedsQLTM4.0 Generic Core Scales, which assess physical, emotional, social, and school functioning . However, until recently no disease-specific measures were available. To address this gap, Goldstein et al. developed and validated the PedsQLTM3.0 ESRD Module to assess for HRQOL in children and adolescents on maintenance dialysis or with a renal transplant [3, 15]. The PedsQLTM3.0 ESRD Module was developed utilizing the methodology of the PedsQLTM instruments and can be used in tandem with the PedsQLTM4.0 to obtain both a global and disease-specific measure of HRQOL . Both measures include age-based forms to be completed by patients and their primary parent, yielding patient self-reports and parent proxy-reports assessing four global domains and seven disease-specific domains of HRQOL, respectively. Healthy cohort-based norms are available for the PedsQLTM4.0 measure with preliminary norms available for the ESRD Module based on a multi-center study of 193 pediatric ESRD patients and 190 parents .
To our knowledge, no long-term HRQOL study of children with ESRD has previously been performed [23, 24]. The purpose of our study was to describe longitudinally observed data examining both global and disease-specific aspects of HRQOL based on patient self- and parent proxy-reports in a cohort of dialysis-dependent children with ESRD and investigate potential associations with demographic and medical variables of interest.
This retrospective chart review study was approved by the Baylor College of Medicine Institutional Review Board. Data were obtained from a chart review of existing HRQOL surveys completed semi-annually by patients 5–17 years of age and the parents of patients aged 2–17 years as part of standard clinical care. Data were available for each patient for at least two of the four time-points observed approximately 6 months apart from 2009 through 2011. Records of patients undergoing maintenance in-center [hemodialysis (HD) three times per week], home-based nightly peritoneal dialysis, or home HD for at least 2 months at the time of their first survey and their parents were included in the study. Patients with known, significant cognitive delays or those who were unable to complete the surveys despite assistance from medical staff were excluded.
The HRQOL assessments were conducted utilizing the PedsQLTM4.0 Generic Core Scales and the PedsQLTM3.0 ESRD Module. Patients and their parents were asked to complete these paper-and-pencil surveys separately to avoid cross-informant bias. Spanish-language versions of the HRQOL measures and access to Spanish-language interpreters were provided to families who primarily speak or prefer to communicate in Spanish. American Sign Language (ASL) interpretation was provided for deaf and hard of hearing individuals. Surveys were completed during HD treatments, peritoneal dialysis clinic visits, or at home and brought back to the unit to return to a member of the Renal Quality of Life Team. This team consists of quality of life coordinators, certified child life specialists, renal social workers, a school liaison coordinator, and a pediatric health psychologist. Surveys were orally administered by members of the Renal Quality of Life Team for children aged 5–7 years or if a child was unable to read or write.
PedsQLTM4.0 generic core scales
The 23-item PedsQLTM4.0 Generic Core Scales contain four domains: (1) Health and Physical Functioning (8 items), (2) Emotional Functioning (5 items), (3) Social Functioning (5 items), and (4) School Functioning (5 items). Two summary scores (Psychosocial Health and Total Score) can be calculated. For the purposes of this study, only the Total Score was tabulated and will be referred to from herein as the Global Total Score. This measure has sound psychometric properties and is widely used in healthy school and community populations, as well as in pediatric populations with acute and chronic health conditions [22, 25].
PedsQLTM3.0 ESRD module
The 34-item PedsQLTM3.0 ESRD Module contains seven domains: (1) General Fatigue (4 items), (2) About My Kidney Disease (5 items), (3) Treatment Problems (4 items), (4) Family and Peer Interactions (3 items), (5) Worry (10 items), (6) Perceived Physical Appearance (3 items), and (7) Communication (5 items). A summary score (ESRD Total Score) may be calculated, and this measure has been validated in a multicenter study with preliminary norms available for children undergoing dialysis or with renal transplant .
Both instruments are relatively brief (approximately 5–10 min to complete), have parallel patient self-report and parent proxy-report forms, and use a Likert response scale, with higher scores indicating better HRQOL. Age-specific versions of the patient self-report and parent proxy-report [2–4 years (proxy only), 5–7, 8–12, or 13–18 years] are provided to ensure developmentally appropriate content and length. Further details and information on tabulating domain scores are available in references [25, 26] .
Baseline characteristics were summarized using means and standard deviations (SD) for continuously measured outcomes, and discrete variables are summarized by counts and percentages. This study consisted of three separate analyses for the global and disease-specific HRQOL measures: patient self-report scores, parent proxy-report scores, and the difference between respective patient and parent-proxy scores. Differences between scores were calculated by subtracting proxy scores from the patient scores. For each analysis, a general linear model with an unstructured covariance matrix of correlated errors was used to test for significant associations between repeatedly measured scores and independent variables. The primary outcomes of interest included an overall quality of life score (Global Total Score) and disease-specific quality of life score (ESRD Total Score). Secondary outcomes included the total scores for each of the four domains of the PedsQLTM4.0 and seven domains of the PedsQLTM3.0 ESRD Module. Independent variables consisted of baseline patient characteristics, including age, dialysis vintage, gender, ethnicity, ESRD history, treatment modality, and transplant history. Baseline age and dialysis vintage were calculated as of the date of the participating patient’s first survey, and ethnicity was defined by four groups: African-American, Hispanic, White, and Other. ESRD history was defined as cases medically managed prior to progressing to ESRD or incident cases based on a review of participant medical records. Treatment modality was defined as in-center HD or home peritoneal dialysis. One home HD participant was included in the peritoneal dialysis group based on similar clinic follow-up and support and being able to conduct dialysis in the comfort of home. Transplant history described whether or not the patient previously had a renal transplant. Statistical significance was assessed at the p ≤ 0.05 level.
For each analysis, the general linear model included a discrete, fixed effect for time period, and baseline characteristics were independently evaluated for significance. Statistically significant independent variables were included in a final, multivariable regression model adjusted for time period.
12.1 ± 4.9
ESRD history (%)
Transplant history (%)
Dialysis vintage (years)a
2.2 ± 3.1
General linear modeling results
Patient self-report results
Parent proxy-report results
Parent-proxy Global Total Scores (p = 0.13) and ESRD Total Scores (p = 0.36) were not statistically significantly associated with time (Fig. 1c, d). The average Global Total Score was 61.3 (95 % CI 54.0–68.6), and the average ESRD total score was 72.6 (95 % CI 63.3–81.8). Correlations between adjacent time-points were ≥0.50 for the Global Total Scores and ≥0.63 for the ESRD Total Scores. Adjusting for time, parent-proxy Global Total Scores (p = 0.003), ESRD Total Scores (p = 0.01), all four global domain scores (p ≤ 0.04), as well as General Fatigue (p = 0.01) and About My Kidney Disease (p = 0.0004) ESRD domain scores were inversely associated with baseline patient age. Parent-proxy Global Total Scores (p = 0.01), Health and Physical (p = 0.01), Social (p = 0.004), and School (p = 0.001) domain scores, as well as Family and Peer Interactions ESRD domain scores (p = 0.014) were inversely associated with dialysis vintage at baseline. Lower parent-proxy scores on these global and ESRD indices were associated with older patient age and greater time on dialysis at baseline. For example, Family and Peer Interactions domain scores declined by about 2.2 points for each vintage year. Age and vintage maintained statistically significant associations with Global Total Scores in a multivariable model with time, age, and dialysis vintage (p ≤ 0.02). However, age was no longer significantly associated with the Health & Physical, Social, or School domains (p ≥ 0.06) in a similar model.
Time was independently associated with the School Functioning domain scores (p = 0.03), even after adjusting for vintage and age (p < 0.001). Proxy scores decreased from 61.34 (95 % CI 54.6–68.0) at time-point 1 to 44.7 (95 % CI 38.4–51.1) at time-point 4. Otherwise, time was not associated with parent-proxy scores on the Health and Physical, Emotional, or Social Functioning domains (p ≥ 0.13).
Differences in patient and parent-proxy scores
Patient and parent-proxy scores on global and disease-specific aspects of health-related quality of life (HRQOL) over a 2-year period: respondent differences, range of scores, and a cohort comparison
Measures of HRQOL
Generic Core Scale
72.5 ± 16.2
67.0 ± 19.8
Health and Physical
75.0 ± 19.4
68.2 ± 24.1
72.5 ± 20.8
68.9 ± 21.1
77.8 ± 21.0
70.4 ± 23.8
63.6 ± 22.2
59.2 ± 25.0
72.4 ± 21.9
68.3 ± 24.0
About My Kidney Disease
70.4 ± 17.8
71.4 ± 19.4
74.2 ± 18.6
72.3 ± 20.0
Family and Peer Interactions
73.9 ± 25.2
72.2 ± 24.6
70.6 ± 21.6
74.6 ± 19.6
Perceived Physical Appearance
72.0 ± 26.2
74.4 ± 25.7
74.3 ± 24.9
73.2 ± 26.9
Optimal care for the children with ESRD now requires the assessment and management of HRQOL . These children and their families must adjust to and cope with living with a serious chronic illness which requires a burdensome, complex medical regimen [6, 27–29] and involves a lifetime of recurrent cycles of dialysis and transplant, all of which can be overwhelming . Current data on HRQOL for these children indicate suboptimal functioning socially, academically, emotionally, physically, and vocationally [14, 31].
Assessment of HRQOL in pediatric ESRD in the USA is now more sophisticated with the advent of disease-specific measures utilized in tandem with more global measures of functioning. We are now mandated to assess HRQOL in children with ESRD, but more attention must be paid to tracking HRQOL outcomes as part of this assessment process [17, 18]. At this time, however, no longitudinal data have yet been reported for either global or disease-specific aspects of HRQOL functioning in pediatric ESRD [24, 32]. We believe that our study is the first effort to longitudinally measure global and disease-specific aspects of HRQOL functioning in children with ESRD and to do so from the perspective of both patients and their parents. Further, our results provide preliminary data on patient factors associated with change in HRQOL over time.
Our findings demonstrate that the broad-based estimations of global and disease-related HRQOL functioning among the 53 patients reviewed remained fairly stable across the 2-year assessment period. When distinct aspects of HRQOL functioning were examined across time, relative strengths and vulnerabilities were revealed, and patients tended to have overall more positive appraisals of their functioning compared to their parents’ perceptions. Across assessment time-points, patients and their parents (to a lesser degree) felt that socially they were doing quite well, being close to or on par with healthy peers [2, 25] (Table 2). In general, however, on all domains assessed, patient and proxy rating scores over the course of the study period were comparable to scores obtained from a multicenter study examining HRQOL in a large cohort of pediatric ESRD patients and their caregivers . Of particular concern to our patients and their parents is the negative impact of ESRD on academic functioning. Both patients and their parents perceived academic issues to be a significant problem. Notably, parents indicated that over the 2-year assessment period these struggles significantly worsened even after taking into account the child’s length of time on dialysis. These concerns have been a focus of intervention efforts in our medical unit for a number of years. However, these efforts are now expanding beyond addressing individual patient needs on a piecemeal basis, regrettably sometimes provided on a “too little, too late” basis. As a result, school advocacy services are now available to facilitate patient/parent–school communication and education about ESRD and to recommend appropriate academic placement and services for patients.
Direct comparisons of patient and parent perceptions of HRQOL functioning revealed little agreement across the majority of the global and disease-specific domains assessed. Parents tended to view their children as doing more poorly than how the children viewed themselves. Discrepancies in child and parent perceptions on a variety of ratings are common in the literature [33, 34], further confirming the recommendation that both child and adult perspectives be assessed to yield a more comprehensive understanding of HRQOL functioning .
Indeed, as we examined which patient factors may be associated with changes in HRQOL outcomes over the 2-year study period, we found that having both patient and parent perspectives gave a more nuanced, complex picture. The longer patients were on dialysis, the worse they reported their emotional functioning to be. Compared to our male patients, our female patients indicated that they struggled more emotionally (regardless of length of time on dialysis) and had more concerns about their appearance being negatively affected by their disease. From our parents’ point of view, having a child who is older and who has been on dialysis longer negatively impacted most if not all aspects of global HRQOL functioning: physically, emotionally, socially, and academically. Further, children with these characteristics were also thought to experience more fatigue in daily life as a disease-related burden, as well as difficulties getting along with family and peers due to dealing with disease-related issues.
Finally, some of the most compelling findings were associated with the amount of discrepancy between patient and parent HRQOL perceptions. Discrepancies in patient and parent perceptions of emotional functioning and dealing with the burden of ESRD on daily life significantly changed over time. Relative to parent perceptions, our overall sample of patients felt that they were doing not only better emotionally but increasingly so over time. Given that the average age of our patients was in the early adolescent years, it may be that an increased desire for privacy and autonomy makes it more difficult for parents to know how well their child is doing emotionally. Limited knowledge coupled with parent tendencies toward worry and concern for a child with a serious chronic illness may account for such a discrepancy and the observed trend toward more divergent perceptions over time.
Although both patients and their parents identified disease-related burden as having a negative impact on HRQOL over the course of the study, their experiences when factors such as the manner in which ESRD was diagnosed appear to be linked to how patients and parents view the degree of burden on the child’s life. The subgroup of patients who were medically managed prior to progressing to ESRD continually indicated that the burden of ESRD was less of an issue than how their parents felt it might be for them. In contrast, for the group of patients who were diagnosed with ESRD upon presenting for acute medical care, the degree of disease-related burden fluctuated relative to how their parents felt they were being impacted. Receiving the unexpected news that you now have a serious health condition and will need to start dialysis is an upsetting, life-altering experience. In the role as our unit’s psychologist (SKN) and recently surveying patients and parents about dialysis-related traumatic stress, a handful of patients and family members recall this experience as being traumatic and disruptive to the adjustment and coping process. It may be that as the child and parent attempt to adjust to and cope with these new circumstances and demands, their perceptions of disease burden on daily life may fluctuate across time.
While this study is thought to be the first of its kind in presenting longitudinal data on HRQOL functioning in a relatively large, ethnically representative sample of children with ESRD, extrapolating these findings to the larger population of pediatric ESRD patients may be limited. Also, it was anticipated that more time-related change in HRQOL functioning would be detected and that other patient characteristics, such as ethnicity and dialysis treatment modality, would be associated with HRQOL outcomes. Multicenter studies are warranted to ensure adequate sample size and statistical power to better elucidate change over time in HRQOL outcomes, which patient factors may be related to such change, and which intervention targets would be most suitable.
Our study also had some methodological limitations. Completion of the HRQOL surveys typically occurred while patients were undergoing in-center HD treatments or attending peritoneal dialysis clinic visits. The side-effects of HD, such as cramping and nausea, and the distractions or stressors present in the clinic milieu may have influenced patient and/or parent responses. Minimizing such influences may involve administering surveys on non-dialysis/non-clinic days or consistently either before or after treatments/visits. However, these methods may be logistically impractical, may further burden patients and families, and may create additional unexpected influences on survey responses. Nonetheless, this methodological issue merits consideration in the planning of future studies.
As these efforts get underway and more data are amassed, an area of future direction should focus on tracking the impact of implemented interventions (e.g., school advocacy services) on subsequent assessments of HRQOL functioning. Such tracking can be done on the individual patient and parent levels via individual growth curve analyses. Another area of future study should focus on the survey responses of parents with children with known developmental delays. This group of patients and their parents and additional caregivers likely face additional and more complex medical and psychosocial challenges that impact HRQOL functioning. As more dialysis units begin utilizing global and ESRD specific measures of HRQOL functioning, data can be aggregated to examine HRQOL trends over time and which interventions make the greatest impact on improving HRQOL outcomes in children with ESRD and their families.
The authors acknowledge the members of the Texas Children’s Renal Quality of Life Program: Meredith Vela, Frida Wilson, Julie Guillen, Robin Davis, Ashley Fadul, and Lindsey Gregg who administer the PedsQLTM measures to our patients and who tirelessly coordinate efforts to provide much needed support to the children in our unit and their families. This work was supported, in part, by the Dan L. Duncan Institute for Clinical and Translational Research at Baylor College of Medicine, Houston, TX.