ODP392 Persistence with daily growth hormone treatment among pediatric patients with growth hormone deficiency in the UK

BACKGROUND: Suboptimal adherence to, and persistence with, daily somatropin therapy to treat children with growth hormone deficiency (GHD) has been documented previously, and has also been shown to have negative effects on the growth response. OBJECTIVES: To describe discontinuation of, and persistence to, daily somatropin treatment among pediatric patients with GHD in the UK. METHODS: This was a retrospective cohort study of pediatric patients (≥3 and <16 years) with >1 medication prescription for daily injectable somatropin from 1 st July 2000 to 31 st December 2020 in the IQVIA Medical Research DATA (IMRD) database. Patients were required to have >1 diagnostic code for GHD during the 6 months preceding and including the first somatropin prescription date ("index date"), and to be continuously enrolled in the database >6 months preceding and >3 months following index date. Patients with >1 somatropin prescription during the 6 months prior to index date, or with diagnoses for other causes of short stature were excluded. Patients were followed for up to 48 months post-index. Early persistence was defined as the proportion of patients prescribed ≥1 somatropin refill subsequent to the initial prescription. Discontinuation was defined as the first date at which a medication gap (defined as either >60 days or >90 days between prescriptions) for somatropin occurred. Persistence (non-discontinuation) over time was evaluated using Kaplan-Meier methods to assess time to first discontinuation event. The relationship of patient characteristics with time to medication discontinuation was evaluated using Cox proportional hazards models. RESULTS: Among pediatric patients included in this study (n=117), the majority (n=84, 71.8%) had 48 months of available follow-up; 56.4% were male and mean (median) age was 8.6 (8. 0) years. About 98% of patients exhibited early persistence. Persistence over the follow-up period decreased with follow-up duration. Using a 60-day gap definition, persistence at 12, 24, 36, and 48 months was estimated among 48.3%, 35.4%, 26.7%, 16.5% of patients; using a more conservative 90-day definition, an estimated 72.4%, 58.6%, 52.8%, and 43.3% were persistent at the same time periods. No significant patient predictors of time to discontinuation were identified. CONCLUSIONS: Despite high early persistence with somatropin, a sizable proportion of patients were increasingly non-persistent over time. In this UK-based study more than 1 in 4 patients were non-persistent at 12 months and more than 1 in 2 were non-persistent at 48 months. These results suggest that strategies to support improved medication-taking behavior among pediatric patients with GHD in the UK are warranted. Presentation: No date and time listed