The PedsQL™* 4.0 as a Pediatric Population Health Measure: Feasibility, Reliability, and Validity

doi:10.1367/1539-4409(2003)003<0329:TPAAPP>2.0.CO;2

Ambulatory Pediatrics

Volume 3, Issue 6, November–December 2003, Pages 329-341

https://doi.org/10.1367/1539-4409(2003)003<0329:TPAAPP>2.0.CO;2 Get rights and content

Background.—The application of health-related quality of life (HRQOL) as a pediatric population health measure may facilitate risk assessment and resource allocation, the tracking of community health, the identification of health disparities, and the determination of health outcomes from interventions and policy decisions.

Objective.—To determine the feasibility, reliability, and validity of the 23-item PedsQL 4.0 (Pediatric Quality of Life Inventory) Generic Core Scales as a measure of pediatric population health for children and adolescents.

Design.—Mail survey in February and March 2001 to 20 031 families with children ages 2–16 years throughout the State of California encompassing all new enrollees in the State's Children's Health Insurance Program (SCHIP) for those months and targeted language groups.

Methods.—The PedsQL 4.0 Generic Core Scales (Physical, Emotional, Social, School Functioning) were completed by 10 241 families through a statewide mail survey to evaluate the HRQOL of new enrollees in SCHIP.

Results.—The PedsQL 4.0 evidenced minimal missing responses, achieved excellent reliability for the Total Scale Score (α = .89 child; .92 parent report), and distinguished between healthy children and children with chronic health conditions. The PedsQL 4.0 was also related to indicators of health care access, days missed from school, days sick in bed or too ill to play, and days needing care.

Conclusion.—The results demonstrate the feasibility, reliability, and validity of the PedsQL 4.0 as a pediatric population health outcome. Measuring pediatric HRQOL may be a way to evaluate the health outcomes of SCHIP.

Section snippets

Sampling Frame

The PedsQL 4.0 survey was mailed separately for each of the months of February and March 2001 to 20 031 families with children ages 2–16 years throughout the State of California, which encompassed all new enrollees in SCHIP for those months and for those ages and for parents and/or children who were English, Spanish, Vietnamese, Korean, or Cantonese speaking. Although the PedsQL 4.0 can be administered to children ages 2–18, children older than 16 years of age were not included in this field

Sample Characteristics

The overall return rate was 51% (10 241 families completed and returned the survey). A minimum response rate of 40% has previously been recommended as achievable for Medicaid surveys using the CAHPS 1.0,⁵⁵ with a 56% response rate achieved with a combination of a mail survey followed by telephone interviews.⁵⁶ The response rate achieved was expected for the mode and method of survey administration utilized in this survey project, which involved a one-time only mailing.⁵⁷ Questionnaires were

DISCUSSION

This study presents the measurement properties for the PedsQL 4.0 Generic Core Scales as a population health measure. The analyses support the feasibility, reliability, and validity of the PedsQL 4.0 as a child self-report and parent proxy-report HRQOL measurement instrument for pediatric population health. The PedsQL 4.0 is the only empirically validated generic pediatric HRQOL measurement instrument of which we are aware to span this broad age range for child self-report and parent

ACKNOWLEDGMENT

This research was supported by a grant from the David and Lucile Packard Foundation.

REFERENCES (63)

SullivanM et al.
The Swedish SF-36 Health Survey III. Evaluation of criterion-based validity: results from normative population
J Clin Epidemiol
(1998)
WeitzmanM
School absence rates as outcome measures in studies of children with chronic illness
J Chronic Dis
(1986)
VarniJW et al.
The Varni/Thompson Pediatric Pain Questionnaire: I. Chronic musculoskeletal pain in juvenile rheumatoid arthritis
Pain
(1987)
VarniJW et al.
Development of the Waldron/Varni Pediatric Pain Coping Inventory
Pain
(1996)
WyrwichK et al.
Further evidence supporting an SEM-based criterion for identifying meaningful intra-individual changes in health-related quality of life
J Clin Epidemiol
(1999)
JaeschkeR et al.
Measurement of health status: Ascertaining the minimal clinically important difference
Controlled Clin Trials
(1989)
FayersPM et al.
Quality of Life: Assessment, Analysis, and Interpretation
(2000)
SpilkerB
Quality of Life and Pharmacoeconomics in Clinical Trials
(1996)
VarniJW et al.
Pediatric health-related quality of life measurement technology: a guide for health care decision makers
J Clin Outcomes Manag
(1999)
SmithKW et al.
Distinguishing between quality of life and health status in quality of life research: a meta-analysis
Qual Life Res
(1999)

Constitution of the World Health Organization Basic Document

(1948)

KootHM et al.

Quality of Life in Child and Adolescent Illness: Concepts, Methods and Findings

(2001)

Lewin-EpsteinN et al.

Validation of the 36-item Short-Form Health Survey (Hebrew version) in the adult population of Israel

Med Care

(1998)

BurdineJN et al.

The SF-12 as a population health measure: an exploratory examination of potential for application

Health Serv Res

(2000)

AuDH et al.

Regional variations in health status

Med Care

(2001)

Measuring Healthy Days: Population Assessment of Health-Related Quality of Life

(2000)

McGlynnEA et al.

Overview of issues in improving quality of care for children

Health Serv Res

(1998)

NewacheckPW et al.

Childhood chronic illness: prevalence, severity, and impact

Am J Public Health

(1992)

McCowanC et al.

School absence: a valid morbidity marker for asthma?

Health Bull

(1996)

ForrestCB et al.

Child health services research: challenges and opportunities

JAMA

(1997)

ForrestCB et al.

Outcomes research in pediatric settings: recent trends and future directions

Pediatrics

(2003)

KaplanRM

Quality of life in children: a health care policy perspective

VarniJW et al.

The PedsQL™: measurement model for the Pediatric Quality of Life Inventory

Med Care

(1999)

VarniJW et al.

The PedsQL™ 4.0: reliability and validity of the Pediatric Quality of Life Inventory™ Version 4.0 Generic Core Scales in healthy and patient populations

Med Care

(2001)

VarniJW et al.

The PedsQL™ in pediatric rheumatology: reliability, validity, and responsiveness of the Pediatric Quality of Life Inventory™ Generic Core Scales and Rheumatology Module

Arthritis Rheum

(2002)

VarniJW et al.

The PedsQL™ in pediatric cancer: reliability and validity of the Pediatric Quality of Life Inventory™ Generic Core Scales, Multidimensional Fatigue Scale, and Cancer Module

Cancer

(2002)

VarniJW et al.

The PedsQL™ 4.0 Generic Core Scales: sensitivity, responsiveness, and impact on clinical decision-making

J Behav Med

(2002)

VarniJW et al.

The PedsQL™ in Type 1 and Type 2 diabetes: reliability and validity of the Pediatric Quality of Life Inventory™ Generic Core Scales and Type 1 Diabetes Module

Diabetes Care

(2003)

SchwimmerJB et al.

Health-related quality of life of severely obese children and adolescents

JAMA

(2003)

Uzark K, Jones K, Burwinkle TM, Varni JW. The Pediatric Quality of Life Inventory™ in children with heart disease Prog...

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: *The PedsQL™ is available at http://www.pedsql.org.

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The PedsQL™* 4.0 as a Pediatric Population Health Measure: Feasibility, Reliability, and Validity

Section snippets

Sampling Frame

Sample Characteristics

DISCUSSION

ACKNOWLEDGMENT

J Clin Epidemiol

J Chronic Dis

Pain

Pain

J Clin Epidemiol

Controlled Clin Trials

Quality of Life: Assessment, Analysis, and Interpretation

Quality of Life and Pharmacoeconomics in Clinical Trials

Pediatric health-related quality of life measurement technology: a guide for health care decision makers

J Clin Outcomes Manag

Distinguishing between quality of life and health status in quality of life research: a meta-analysis

Qual Life Res

Constitution of the World Health Organization Basic Document

Quality of Life in Child and Adolescent Illness: Concepts, Methods and Findings

Validation of the 36-item Short-Form Health Survey (Hebrew version) in the adult population of Israel

Med Care

The SF-12 as a population health measure: an exploratory examination of potential for application

Health Serv Res

Regional variations in health status

Med Care

Measuring Healthy Days: Population Assessment of Health-Related Quality of Life

Overview of issues in improving quality of care for children

Health Serv Res

Childhood chronic illness: prevalence, severity, and impact

Am J Public Health

School absence: a valid morbidity marker for asthma?

Health Bull

Child health services research: challenges and opportunities

JAMA

Outcomes research in pediatric settings: recent trends and future directions

Pediatrics

Quality of life in children: a health care policy perspective

The PedsQL™: measurement model for the Pediatric Quality of Life Inventory

Med Care

The PedsQL™ 4.0: reliability and validity of the Pediatric Quality of Life Inventory™ Version 4.0 Generic Core Scales in healthy and patient populations

Med Care

The PedsQL™ in pediatric rheumatology: reliability, validity, and responsiveness of the Pediatric Quality of Life Inventory™ Generic Core Scales and Rheumatology Module

Arthritis Rheum

The PedsQL™ in pediatric cancer: reliability and validity of the Pediatric Quality of Life Inventory™ Generic Core Scales, Multidimensional Fatigue Scale, and Cancer Module

Cancer

The PedsQL™ 4.0 Generic Core Scales: sensitivity, responsiveness, and impact on clinical decision-making

J Behav Med

The PedsQL™ in Type 1 and Type 2 diabetes: reliability and validity of the Pediatric Quality of Life Inventory™ Generic Core Scales and Type 1 Diabetes Module

Diabetes Care

Health-related quality of life of severely obese children and adolescents

JAMA