At what age can children reliably and validly self-report their health-related quality of life? An investigation using the PedsQL(tm) 4.0 Generic Core Scales Database

Health-related quality of life (HRQOL) assessment has emerged as a vital health outcome measure in clinical trials, healthcare services and evaluation, and population health outcomes research. Reliability, validity, and parent-child agreement of the PedsQL™ 4.0 Generic Core Scales were examined using child self-report and parent proxy-report age subgroup data on over 8,000 children ages 5-16 years from the PedsQL 4.0 Generic Core Scales DatabaseSM. The PedsQL™ 4.0 Generic Core Scales demonstrated good internal consistency reliability for children as young as 5 years; healthy children across the age subgroups demonstrated a statistically significant difference in HRQOL (better HRQOL) than children with a known chronic health condition. Confirmatory factor analysis demonstrated that a 5-factor model fit almost identically across the age subgroups, providing further evidence that children as young as 5 years are reliable and valid self-reporters of their HRQOL. Parent-child agreement was in the moderate-to-good range, with parents reporting significantly higher PedsQL™ 4.0 scores across the age subgroups. In conclusion, the analyses support the reliability and validity of child self-report in children as young as 5 years old

The PedsQL™ as a patient-reported outcome in children and adolescents with fibromyalgia: an analysis of OMERACT domains

BACKGROUND: Fibromyalgia is a chronic health condition characterized by widespread musculoskeletal pain, multiple tender points on physical examination, generalized muscular aching, stiffness, fatigue, nonrestorative sleep pattern, cognitive dysfunction, and mood disturbance. Recently, the Outcome Measures in Rheumatoid Arthritis Clinical Trials (OMERACT) Fibromyalgia Syndrome Workshop ranked and prioritized the domains that should be consistently measured in fibromyalgia clinical trials, specifically, pain, generic health-related quality of life, fatigue, sleep quality, and physical function. The focus of these deliberations was exclusively on adult patients, and to our knowledge, these domains have not been previously tested within a multidimensional framework in children and adolescents with fibromyalgia. METHODS: An analysis to determine the feasibility, reliability, and validity of the PedsQL™ 4.0 (Pediatric Quality of Life Inventory™) Generic Core Scales, PedsQL™ Multidimensional Fatigue Scale, and PedsQL™ Rheumatology Module Pain and Hurt Scale as patient-reported outcome (PRO) measures for pediatric patients with fibromyalgia. The PedsQL™ Scales were completed by 59 families in a pediatric rheumatology clinic in a large children's hospital. RESULTS: The PedsQL™ evidenced minimal missing responses (0.53% patient self-report, 0.70% parent proxy-report), achieved excellent reliability for the Generic Core Scales Total Scale Score (α = 0.88 patient self-report, 0.87 parent proxy-report), the Multidimensional Fatigue Scale Total Scale Score (α = 0.94 patient self-report, 0.94 parent proxy-report), and acceptable reliability for the 4-item Rheumatology Module Pain and Hurt Scale (α = 0.68 patient self-report, 0.75 parent proxy-report). The PedsQL™ Generic Core Scales and Multidimensional Fatigue Scale significantly distinguished between pediatric patients with fibromyalgia and healthy children. Pediatric patients with fibromyalgia self-reported severely impaired physical and psychosocial functioning, significantly lower on most dimensions when compared to pediatric cancer patients receiving cancer treatment, and significantly lower on all dimensions than pediatric patients with other rheumatologic diseases. Patients with fibromyalgia self-reported significantly greater pain and fatigue than pediatric patients with other rheumatologic conditions, and generally more fatigue than pediatric patients receiving treatment for cancer. CONCLUSION: The results demonstrate the excellent measurement properties of the PedsQL™ Scales in fibromyalgia. These PedsQL™ Scales measure constructs consistent with the recommended OMERACT Fibromyalgia Syndrome Workshop domains. The findings highlight the severely impaired HRQOL of pediatric patients with fibromyalgia. Regular monitoring of pediatric patients with fibromyalgia will help identify children and adolescents at risk for severely impaired HRQOL. These PedsQL™ Scales are appropriate outcome measures for clinical trials and health services research for pediatric patients with fibromyalgia

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

BACKGROUND: 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™ Database(SM )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. METHODS: The sample analyzed represents parent proxy-report age data on 13,878 children ages 2 to 16 years from the PedsQL™ 4.0 Generic Core Scales Database(SM). 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, 26.8%), and from a State Children's Health Insurance Program (SCHIP) in California (n = 10,160, 73.2%). RESULTS: The percentage of missing item responses for the parent proxy-report sample as a whole was 2.1%, supporting feasibility. The majority of the parent proxy-report scales across the age subgroups 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, 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. CONCLUSION: 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

Impaired health-related quality of life in children and adolescents with chronic conditions: a comparative analysis of 10 disease clusters and 33 disease categories/severities utilizing the PedsQL™ 4.0 Generic Core Scales

<p>Abstract</p> <p>Background</p> <p>Advances in biomedical science and technology have resulted in dramatic improvements in the healthcare of pediatric chronic conditions. With enhanced survival, health-related quality of life (HRQOL) issues have become more salient. The objectives of this study were to compare generic HRQOL across ten chronic disease clusters and 33 disease categories/severities from the perspectives of patients and parents. Comparisons were also benchmarked with healthy children data.</p> <p>Methods</p> <p>The analyses were based on over 2,500 pediatric patients from 10 physician-diagnosed disease clusters and 33 disease categories/severities and over 9,500 healthy children utilizing the PedsQL™ 4.0 Generic Core Scales. Patients were recruited from general pediatric clinics, subspecialty clinics, and hospitals.</p> <p>Results</p> <p>Pediatric patients with diabetes, gastrointestinal conditions, cardiac conditions, asthma, obesity, end stage renal disease, psychiatric disorders, cancer, rheumatologic conditions, and cerebral palsy self-reported progressively more impaired overall HRQOL than healthy children, respectively, with medium to large effect sizes. Patients with cerebral palsy self-reported the most impaired HRQOL, while patients with diabetes self-reported the best HRQOL. Parent proxy-reports generally paralleled patient self-report, with several notable differences.</p> <p>Conclusion</p> <p>The results demonstrate differential effects of pediatric chronic conditions on patient HRQOL across diseases clusters, categories, and severities utilizing the PedsQL™ 4.0 Generic Core Scales from the perspectives of pediatric patients and parents. The data contained within this study represents a larger and more diverse population of pediatric patients with chronic conditions than previously reported in the extant literature. The findings contribute important information on the differential effects of pediatric chronic conditions on generic HRQOL from the perspectives of children and parents utilizing the PedsQL™ 4.0 Generic Core Scales. These findings with the PedsQL™ have clinical implications for the healthcare services provided for children with chronic health conditions. Given the degree of reported impairment based on PedsQL™ scores across different pediatric chronic conditions, the need for more efficacious targeted treatments for those pediatric patients with more severely impaired HRQOL is clearly and urgently indicated.</p

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

BACKGROUND: 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™ Database(SM )were utilized to test the hypothesis that children as young as 5 years of age can reliably and validly report their HRQOL. METHODS: 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 Database(SM). 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%). RESULTS: 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. CONCLUSION: 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

Disordered Eating and Body Image Concerns in Young Adult Women With Scoliosis

Background: Researchers have called for more investigation into disordered eating behaviors in females with scoliosis. Objective: The objective of the current study was to assess the associations between body image concerns, disease-specific indicators of scoliosis (ie, age of diagnosis, having undergone bracing treatment, being told by a physician your scoliosis required surgery, having a spinal fusion), quality of life, and disordered eating in a sample of young adult women diagnosed with idiopathic scoliosis during adolescence. Design: This study was cross-sectional in design. Methods: Participants were 177 young adult women ages 18 to 30 years diagnosed with idiopathic scoliosis by a physician who completed questionnaires online. Results: Undergoing bracing treatment ( r  = −.440; P  < .001), greater age at scoliosis diagnosis ( r  = .563; P  < .001), being told scoliosis required surgery ( r  = −.196; P  < .050), annual income ( r  = .306; P  < .001), level of education ( r  = .228; P  < .010), and race/ethnicity ( r  = −.213; P  < .050) were associated with the EDE-Q Global Score. The Body Shape Questionnaire Total Score and EDE-Q Global Score ( r  = .848; P  < .001) and EDE-Q Weight Concern Score ( r  = .813; P  < .001) were associated. The strongest correlations between the EDE-Q and the SRS-22-Revised Subscales were generally evidenced on the SRS-22-Revised Mental Health Subscale ( r s ranged from −.200 to −.371; P  < .001). After controlling for annual income, highest level of education, undergoing bracing treatment, and age of scoliosis diagnosis, the Body Shape Questionnaire Total Score was significantly correlated with the EDE-Q Eating Concern Score (standardized beta coefficient = .618; P  < .001). Conclusions: These findings underscore the importance of assessing body image concerns in young adult women with scoliosis experiencing disordered eating as this information may provide valuable information relevant to treatment planning