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Pediatric health-related quality of life questionnaires in clinical trials
Hein Raat1, Ashna D Mohangoo, Martha A Grootenhuis
1Department of Public Health, Erasmus MC, University Medical Center Rotterdam, The Netherlands. h.raat@erasmusmc.nl
Insights
Pediatric health-related quality of life questionnaires are essential for clinical trials. Selecting validated generic or disease-specific tools, often combined with parent and self-reports, enhances study quality and treatment assessment.
Area of Science:
- Pediatric Health Outcomes Research
- Psychometrics in Child Health
- Clinical Trial Methodology
Background:
- Assessing pediatric health-related quality of life (HRQoL) is crucial for evaluating treatment efficacy in clinical trials.
- Numerous pediatric HRQoL questionnaires exist, necessitating a review of their psychometric properties and applications.
- Understanding the nuances of different measurement tools is vital for robust pediatric research.
Purpose of the Study:
- To review the feasibility, reliability, and validity of pediatric health-related quality of life questionnaires.
- To provide an overview of the recent applications of these measures in pediatric clinical research.
- To guide the selection of appropriate outcome measures for pediatric studies.
Main Methods:
- Systematic review of recent studies on pediatric quality of life (QoL) questionnaires.
- Evaluation of psychometric properties including feasibility, reliability, and validity.
- Analysis of the application of these QoL measures in pediatric clinical trials.
Main Results:
- The Child Health Questionnaire (CHQ-PF28 and CHQ-PF50) and the Pediatric Quality of Life Inventory are reliable and valid measures.
- The TNO-AZL Preschool Children Quality of Life questionnaire is suitable for preschool-aged children.
- Disease-specific questionnaires are generally more sensitive to disease impact than generic ones; parent and self-reports offer complementary perspectives.
Conclusions:
- Multiple validated pediatric QoL questionnaires (generic, disease-specific, preference-based) are available for clinical trials.
- A combined approach using various questionnaire types and both parent and self-reports is recommended for comprehensive assessment.
- Appropriate selection and combination of QoL measures enhance pediatric study quality and the assessment of treatment effects.
Purpose Of Review:
This review summarizes recent studies on the feasibility, reliability and validity of pediatric health-related quality of life questionnaires and gives an overview of recent applications of these measures in pediatrics.
Recent Findings:
The often-applied short form of the Child Health Questionnaire (CHQ-PF28) provides reliable physical and psychosocial summary measures, but reliable estimates for each scale require the longer version (CHQ-PF50). In addition to this questionnaire, the Pediatric Quality of Life Inventory is another reliable and valid measure. The TNO-AZL Preschool Children Quality of Life questionnaire is a feasible and reliable measure for preschool children. Generally, generic questionnaires are less sensitive to the impact of specific diseases than are disease-specific questionnaires. Parent and self-reports provide different outlooks on quality of life, which complement each other.
Summary:
There are several feasible, reliable and validated pediatric quality of life questionnaires that can be used in clinical trials. These include generic and disease-specific questionnaires and health profile measures, as well as preference-based measures in pediatric settings. Generally, a combination of these types of questionnaires would be the most appropriate approach. Moreover, a combination of parent and self-reports should be applied. Appropriate selection of outcome measures will enhance the quality of pediatric studies and the ability to assess treatment effects in clinical trials.