Background: Children with growth hormone deficiency (GHD) may experience physiological symptoms as well as social and emotional problems.
Objective and hypotheses: This qualitative study explored the burden of GHD and treatment for children and their parents.
Method: 70 interviews were conducted with 39 children (age 812) and 31 parents of children with GHD (age 412) in Germany, UK and USA. Interviews were analysed using grounded theory to identify themes/sub-concepts and a conceptual model of burden of disease was developed.
Results: Children and parents reported similar disease and treatment burdens. Disease burden domains and the most frequently reported subtheme by parents and children were symptoms (poor appetite: 48%), physical impacts (reduced performance in physical activities/sports: 58%), social impacts (being mistaken for being younger: 58%) and emotional impacts (worry: 55%). The most frequently reported child treatment burden domains and subthemes reported by children and parents were physical (pain: 33%), emotional impacts (worry about injections: 37%), and interference with daily life (overnight activities: 29%). For parents, most frequently reported treatment burden domains and subthemes were emotional impacts (worry about treatment/administration: 58%) and interference with daily life (time needed to administer injection: 42%). Treatment was associated with improvements for all impacts. Findings did not differ substantively among countries. The conceptual model describes the full range of disease and treatment impacts, and factors that may modify impact severity.
Conclusion: The overall burden of disease for GHD children and their parents is considerable and not limited to short stature. A well-designed measure of the full range of impacts identified in this study is not currently available and will be developed based on this conceptual model. Accurate and reliable assessment of symptoms and impacts, on both children and parents, may help clinicians to better address the burden of disease, assess treatment effect, and may improve the quality of doctor-patient communications.
Conflict of interest: Dr. Brod, Ms. Alolga, and Ms. Nacson are consultants to Novo Nordisk. Drs. Højbjerre, Nordholm, and Højby Rasmussen are employees of Novo Nordisk A/S.
Funding: This study was funded by Novo Nordisk A/S.
01 - 03 Oct 2015
European Society for Paediatric Endocrinology