Abstract
BackgroundWhen evaluating the outcomes of treatment in paediatric endocrinology, the health-related quality of life (HrQoL) of the child is to be taken into consideration. Since few self–reported HrQoL instruments exist for children with diagnosed short stature (dSS), the objective of this study was to develop and psychometrically test a targeted HrQoL instrument for use in multinational clinical research.MethodsThe target population were short stature (height < −2 SDS) children and adolescents (age 8–12 and 13–18 years) with a diagnosis of growth hormone deficiency (GHD) or idiopathic short stature (ISS), differing in growth hormone treatment status. Focus group discussions for concept and item generation, piloting of the questionnaire with cognitive debriefing, and instrument field testing with a retest were conducted simultaneously in five countries. After qualitative and preliminary quantitative analyses, psychometric testing of field test data in terms of reliability and validity including confirmatory factor analyses (CFA) was performed.ResultsFollowing item generation from focus group discussions, 124 items were included in a pilot test with a cognitive debriefing exercise providing preliminary feedback on item and domain operating characteristics. A field test with 268 participants showed high internal consistency reliabilities (alpha 0.82 – 0.95), good correlations with generic measures (up to r = .58), significant known group differences (e.g. in height: F = 32, df 244, p < 0.001) and an acceptable CFA model fit suggesting construct validity of the three-domain core structure with 22 items, supplemented by three mediator domains with 28 items.ConclusionsThe QoLISSY questionnaire is a promising step forward in assessing the impact of dSS on HrQoL. It is based on items generated from the subjective experience of short stature children referred for endocrine investigation, is validated for use in five languages and it is easy to administer in clinical and research settings.
Highlights
When evaluating the outcomes of treatment in paediatric endocrinology, the health-related quality of life (HrQoL) of the child is to be taken into consideration
In two recent literature reviews, measures to assess HrQoL in diagnosed short stature (dSS) were identified; it was found that none were aimed at dSS as an isolated characteristic, only a few were self-reported and none considered the HrQoL of dSS children with idiopathic short stature (ISS) [2,7]
The results suggest that some differences between dSS and normal height children exist, but the measure was not designed to include the specific problems of short stature children and may be unable to detect differences between treated and untreated children
Summary
When evaluating the outcomes of treatment in paediatric endocrinology, the health-related quality of life (HrQoL) of the child is to be taken into consideration. There is a lack of validated instruments to assess HrQoL in children and adolescents diagnosed with short stature (dSS) [3,4,5,6]. As a result of the paucity of instruments few studies have examined the effect of growth hormone (GH) treatment on HrQoL in short children and adolescents [9,10,11,12,13,14]. The results suggest that some differences between dSS and normal height children exist, but the measure was not designed to include the specific problems of short stature children and may be unable to detect differences between treated and untreated children
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