ESPE Abstracts

Background: Daily r-hGH injection has been safely and effectively used in paediatric patients with GHD for more than 30 years. However, little information is available describing the burden and life impacts experienced by paediatric patients related to daily r-hGH injections.

Objective: To identify the burden and impacts of a daily r-hGH injection regimen on the lives of paediatric GHD patients.

Methods: A retrospective meta-analysis was conducted of data drawn from four sources: qualitative interviews with 15 paediatric patients in the United States (US) conducted from September to December 2016; qualitative market research interviews conducted in early 2017 with 16 paediatric patients in the Czech Republic, Spain, Turkey, and the United Kingdom; responses to an online questionnaire completed by 149 paediatric patients in the US from January to May 2017; and an advisory panel discussion with three paediatric patients in the US conducted on September 8, 2017. Patient-reported burdens and impacts identified in each data source were tabulated and compared.

Results: In total, data across all four sources represent a total of N=184 paediatric patients (n=94 adolescents aged 12 to 17 years; n=90 children aged 3 to 11 years). In qualitative interviews, patients indicated that they had become largely acclimated to daily r-hGH injections. However, emotional impacts, activity limitations, social impacts, and impacts on family life due to daily injections were reported across all four sources. Limitations to participate in overnight activities (50% of adolescents and 64% of children) such as summer camp, and increased travel burden (75% of adolescents and 45% of children) were most frequently reported across sources. Patients also reported impacts on relationships with friends; limitations in social activities; and the burden of preserving secrecy about one’s condition and the use of injections. Across all sources (and particularly highlighted in the online questionnaire), participants stated a clear preference for a less-frequent r-hGH injection regimen (84% of adolescents and 79% of children).

Conclusions: Although paediatric patients may become acclimated to daily r-hGH injections, qualitative data from such patients provide growing evidence that they experience some burden due to the daily injection regimen, and would prefer a less frequent regimen.