CL1 DEFINING AND DEMONSTRATING A CHILD-CAREGIVER DYAD QUESTIONNAIRE ADMINISTRATION APPROACH TO THE ASSESSMENT OF PEDIATRIC HEALTH-RELATED QUALITY OF LIFE

Abstract

Age and developmental factors may limit a child’s understanding and reliable self-report of health-related outcomes. Informant reports are possible; however, observer-reported outcomes are limited to concepts that are observable, and proxy reports often differ from children’s reports. While the FDA PRO Guidance indicates that ‘there is some support for a parent-child dyad approach to the assessment of the child’s HRQOL’, very few examples of this novel approach exist in the literature. The objectives of this research were to define a dyad approach, describe published studies using this approach, and provide a recent case study to demonstrate the approach. A literature search identified published quantitative studies employing the dyad approach. Data were extracted from articles, documenting context of use, data collection methods, and study limitations. A case study demonstrating the dyad approach for research involving pediatric patients taking recombinant human growth hormone (r-hGH) for growth hormone deficiency (GHD) was described. The dyad approach was defined as questionnaire administration in a pediatric population in which the child and caregiver read and answer questions together. Seven publications were selected for full-text review. Key advantages were caregiver’s emotional support and aid in understanding questionnaire content, and increased response stability. Potential limitations included parental influence on the child’s responses or child reticence in the parent’s presence. The case study describes dyad administration in a cross-sectional internet-based survey involving 149 child-caregiver dyads (children ages 3-17 years), highlighting the value of this approach when reporting a shared experience (e.g., r-hGH injections), flexibility for a wide age range, and limitations (e.g., some items should only be answered by caregiver/child independently). Use of a dyad approach for the assessment of health outcomes in selected contexts can help ensure the collection of reliable data in circumstances that may otherwise result in proxy, missing, or data of questionable quality.