The last decade has evidenced a dramatic increase in the development and utilization of pediatric health-related quality of life (HRQOL) measures in an effort to improve pediatric patient health and well-being and determine the value of healthcare services. The emerging paradigm shift toward patient-reported outcomes (PROs) in clinical trials has provided the opportunity to further emphasize the value and essential need for pediatric patient self-reported outcomes measurement. Data from the PedsQL™ DatabaseSM were utilized to test the hypothesis that children as young as 5 years of age can reliably and validly report their HRQOL. The sample analyzed represented child self-report age data on 8,591 children ages 5 to 16 years from the PedsQL™ 4.0 Generic Core Scales DatabaseSM. Participants were recruited from general pediatric clinics, subspecialty clinics, and hospitals in which children were being seen for well-child checks, mild acute illness, or chronic illness care (n = 2,603, 30.3%), and from a State Children's Health Insurance Program (SCHIP) in California (n = 5,988, 69.7%). Items on the PedsQL™ 4.0 Generic Core Scales had minimal missing responses for children as young as 5 years old, supporting feasibility. The majority of the child self-report scales across the age subgroups, including for children as young as 5 years, exceeded the minimum internal consistency reliability standard of 0.70 required for group comparisons, while the Total Scale Scores across the age subgroups approached or exceeded the reliability criterion of 0.90 recommended for analyzing individual patient scale scores. Construct validity was demonstrated utilizing the known groups approach. For each PedsQL™ scale and summary score, across age subgroups, including children as young as 5 years, healthy children demonstrated a statistically significant difference in HRQOL (better HRQOL) than children with a known chronic health condition, with most effect sizes in the medium to large effect size range. The results demonstrate that children as young as the 5 year old age subgroup can reliably and validly self-report their HRQOL when given the opportunity to do so with an age-appropriate instrument. These analyses are consistent with recent FDA guidelines which require instrument development and validation testing for children and adolescents within fairly narrow age groupings and which determine the lower age limit at which children can provide reliable and valid responses across age categories.

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How young can children reliably and validly self-report their health-related quality of life?: An analysis of 8,591 children across age subgroups with the PedsQL™ 4.0 Generic Core Scales

Health-related quality of life (HRQOL) measurement has emerged as an important health outcome in clinical trials, clinical practice improvement strategies, and healthcare services research and evaluation While pediatric patient self-report should be considered the standard for measuring perceived HRQOL, there are circumstances when children are too young, too cognitively impaired, too ill or fatigued to complete a HRQOL instrument, and reliable and valid parent proxy-report instruments are needed in such cases Further, it is typically parents' perceptions of their children's HRQOL that influences healthcare utilization Data from the PedsQL™ DatabaseSM were utilized to test the reliability and validity of parent proxy-report at the individual age subgroup level for ages 2–16 years as recommended by recent FDA guidelines The sample analyzed represents parent proxy-report age data on 13,878 children ages 2 to 16 years from the PedsQL™ 40 Generic Core Scales DatabaseSM Parents were recruited from general pediatric clinics, subspecialty clinics, and hospitals in which their children were being seen for well-child checks, mild acute illness, or chronic illness care (n = 3,718, 268%), and from a State Children's Health Insurance Program (SCHIP) in California (n = 10,160, 732%) The percentage of missing item responses for the parent proxy-report sample as a whole was 21%, supporting feasibility The majority of the parent proxy-report scales across the age subgroups exceeded the minimum internal consistency reliability standard of 070 required for group comparisons, while the Total Scale Scores across the age subgroups approached or exceeded the reliability criterion of 090 recommended for analyzing individual patient scale scores Construct validity was demonstrated utilizing the known groups approach For each PedsQL™ scale and summary score, across age subgroups, healthy children demonstrated a statistically significant difference in HRQOL (better HRQOL) than children with a known chronic health condition, with most effect sizes in the medium to large effect size range The results demonstrate the feasibility, reliability, and validity of parent proxy-report at the individual age subgroup for ages 2–16 years These analyses are consistent with recent FDA guidelines which require instrument development and validation testing for children and adolescents within fairly narrow age groupings and which determine the lower age limit at which reliable and valid responses across age categories are achievable Even as pediatric patient self-report is advocated, there remains a fundamental role for parent proxy-report in pediatric clinical trials and health services research

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Parent proxy-report of their children's health-related quality of life: an analysis of 13,878 parents' reliability and validity across age subgroups using the PedsQL 4.0 Generic Core Scales.

The pediatric quality of life inventory: measuring pediatric health-related quality of life from the perspective of children and their parents.

Health-related quality of life (HRQOL) has been recognized as an important outcome, some contend the most important outcome for children's health care interventions. The PedsQL Measurement Model was designed as a modular approach to measuring pediatric health-related quality of life, developed to integrate the relative merits of generic and disease-specific approaches. We suggest that part of the process of improving the quality of health care includes measuring HRQOL outcomes from the perspective of children and their parents on a routine basis, consistent with a consumer-based health care system approach.

The Pediatric Quality of Life I nventory: Measuring Pediatric Health -R elated Quality of Life from the Perspectiv e of Children a nd Their Parents

pediatric oncology. Results Data demonstrate that children as young as 5 years of age can reliably and validly self-report their HRQOL when an age-appropriate instrument is utilized. Conclusions The evidence supports including pediatric patients’ perspectives in clinical trials. Parent proxy-report is recommended when pediatric patients are too young, too cognitively impaired, too ill or fatigued to complete a HRQOL instrument, but not as a substitute for child self-report when the child is willing and able to provide their perspective.

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Literature Review: Health-related Quality of Life Measurement in Pediatric Oncology: Hearing the Voices of the Children

The results of a 5-year longitudinal prospective study about the health-related quality of life (HRQL) of pediatric patients receiving allogeneic bone marrow or stem cell transplantation (BMT) are described. The patients' HRQL was assessed twice before, and five times after BMT, the end point being 1 year after BMT. For the measurement of HRQL, standardized questionnaires were completed by patients, parents and physicians. The final sample consisted of 68 patients aged 4-18 years, of which 19 were lost in the course of the study owing to relapse, transplant rejection and/or death. The worst HRQL was seen shortly after transplant and HRQL thereafter improved steadily, although the improvement was not always linear and not all patients drew benefit from this average positive evolution. Compromised emotional functioning, a high level of worry and reduced communication during the acute phase of treatment had a negative impact on HRQL 1 year after BMT. Nausea and pain during the acute phase of treatment did not have an effect on later HRQL. The interobserver agreement of HRQL reports between parents and their children was moderate to good, and generally better than child-physician and parent-physician agreement.

Health-related quality of life of pediatric patients receiving allogeneic stem cell or bone marrow transplantation: results of a longitudinal, multi-center study

P Norden

Papers

Health-related quality of life of pediatric patients receiving allogeneic stem cell or bone marrow transplantation: results of a longitudinal, multi-center study