Assessing the quality of life of health-referred children and adolescents with short stature: development and psychometric testing of the QoLISSY instrument

Health and Quality of Life Outcomes

John E Chaplin²  Hartmut Wollmann³  Andreas Pleil¹  Anja Rohenkohl⁵  Dolores Sanz⁴  Maria Dellenmark-Blom²  Carolina Lunde²  Eva Feigerlova⁶  Kendra DeBusk⁷  Emmanuelle Mimoun⁶  Michael Herdman⁴  Mick Power⁷  Julia Quitmann⁵  Monika Bullinger⁵ 

[1] Pfizer, Inc. Specialty Care MDG, Outcomes Research, 10646 Science Center Drive, San Diego CA 92009, USA;Department of Paediatrics, Växthuset, Queen Silvia’s Children’s Hospital, Sahlgrenska Academy at University of Gothenburg, Gothenburg 41685, Sweden;Pfizer Ltd., Specialty Care MDG, Endocrinology, Dorking Road, Walton-on-the-Hill Walton Oaks, Tadworth Surrey KT20 7NS, UK;IMIM University, Insight Consulting and Research, Cami Ral 266, Mataró, Barcelona, Spain;University Hamburg-Eppendorf, Department of Medical Psychology, Martinistr. 52, W26, Hamburg 20246, Germany;Department of Pediatric Endocrinology, University of Toulouse, CHU, 330 Avenue de Grande-Bretagne, Toulouse FR-31059, France;Department of Clinical Psychology, University of Edinburgh, Teviot Place, Edinburgh EH8 9AG, Scotland

关键词: Patient reported outcomes;    Outcomes research;    Idiopathic short stature;    Assessment in children;    Growth hormone deficiency;    Short stature;    Health-related quality of life;   

Others  :  823722
DOI  :  10.1186/1477-7525-11-76

 received in 2012-05-11, accepted in 2013-04-29,  发布年份 2013

【 摘 要 】

Background

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. 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.

Methods

The 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.

Results

Following 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.

Conclusions

The 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.

【 授权许可】

   
2013 Bullinger et al.; licensee BioMed Central Ltd.

【 图 表 】

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