ASSESSING THE TREATMENT BURDEN FOR GROWTH HORMONE DEFICIENCY (GHD) IN CHILDREN- CONCEPT ELICITATION RESULTS SUPPORTING THE DEVELOPMENT OF THE TREATMENT BURDEN MEASURE FOR CHILDHOOD GHD (TB-CGHD)

Author(s)

Brod M1, Højbjerre L2, Wilkinson L2, Alolga SL1, Rasmussen MH2
1The Brod Group, Mill Valley, CA, USA, 2Novo Nordisk A/S, Søborg, Denmark

OBJECTIVES: Treatment for children with GHD involves daily injections which may begin at a very early age and continue throughout childhood. However, the treatment burden (TB) for children and their parents has not been examined. The purpose of this study was to support the content validity of a new GHD-specific TB measure, with versions for patient-reported outcome (PRO) for older children, and observer-reported outcome (ObsRO) and PRO for parents/guardians. METHODS: Focus groups and interviews were conducted with 39 children (aged 8–12) with GHD, 31 parents of children (aged 4–12) and eight clinical experts in three countries (Germany, UK, US). Interviews were analysed and coded using adapted grounded theory. A conceptual model of TB was developed and items were generated and then cognitively debriefed. RESULTS: Qualitative analysis found saturation was reached with three domains for child TB and two domains for parent TB. Child domains (and major proximal subdomains) were: Physical (pain, 33% and bruising, 19%), Interference (Interference with overnight or other activities, 29% and Time needed emotionally to prepare for treatment, 23%) and Emotional (Worry, about injections, 37%, Unhappiness with injection frequency, 25%, and Fear, 22%). Parent TB domains were Emotional (Worry or anxiety about treatment or treatment administration, 58% and Worry about causing pain, 42%) and Interference (Time spent preparing and administering injection, 42%, Interference with travel/vacation, 42%, and Interference with daily/family routines, 35%). The items were cognitively debriefed in a new sample (N=26: 13 children, 13 parents) and, based on findings, it was determined that the PRO version was appropriate for children aged 9–12. The final measure includes 17 items (child PRO and parent ObsRO versions) with a 12-item parent PRO module.    CONCLUSIONS: The conceptual validity of the TB-CGHD is supported by these qualitative findings and the measure is now ready for psychometric validation.

Conference/Value in Health Info

2015-11, ISPOR Europe 2015, Milan, Italy

Value in Health, Vol. 18, No. 7 (November 2015)

Code

PSY99

Topic

Patient-Centered Research

Topic Subcategory

Stated Preference & Patient Satisfaction

Disease

Diabetes/Endocrine/Metabolic Disorders

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