Persistence with daily growth hormone among children and adolescents with growth hormone deficiency in the UK

Abstract

BackgroundChildren with growth hormone deficiency (GHD) are treated with daily somatropin injections; however, poor treatment persistence and adherence have been recognized previously and have been shown to negatively impact growth outcomes. A recent real-world study of a US pediatric GHD population found that a substantial proportion of children discontinued somatropin therapy, but similar data for a real-world UK population are lacking.ObjectivesTo describe the discontinuation of, and persistence with, daily somatropin treatment among children with GHD in the UK.MethodsThis was a retrospective cohort study of children (≥3 and <16 years old) with ≥1 medication prescription for daily injectable somatropin from 1 July 2000 to 31 December 2020 in the IQVIA Medical Research DATA (IMRD) database. Early persistence was defined as the proportion of children prescribed ≥1 somatropin refill (≥2 prescriptions). Discontinuation was defined as the first date at which a medication gap for somatropin (of >60 or >90 days between prescriptions) occurred. Kaplan–Meier methods were used to evaluate persistence (non-discontinuation) over time to assess time to first discontinuation event. Cox proportional hazards models were used to evaluate the relationship between patient characteristics and time to medication discontinuation.ResultsAmong the cohort identified in this study (n = 117), the majority (n = 84, 71.8%) had 48 months of available follow-up; 56.4% were boys and the mean (median) age was 8.6 (8.0) years. About 98% exhibited early persistence, but persistence over the follow-up period decreased with follow-up duration. Using the conservative 90-day gap definition of persistence, an estimated 72.4%, 52.8%, and 43.3% were persistent at 12, 36, and 48 months. Lower persistence rates were observed using the 60-day definition. No significant patient predictors of time to discontinuation were identified.ConclusionsDespite high early persistence with somatropin, a high percentage of children with GHD were increasingly non-persistent over time. More than 1 in 4 were non-persistent at 12 months and more than 1 in 2 were non-persistent at 48 months of follow-up. These results suggest that strategies to support improved medication-taking behavior among children with GHD in the UK are warranted.

Developments in the Management of Growth Hormone Deficiency: Clinical Utility of Somapacitan

“… 4 Additionally, two retrospective studies investigating treatment adherence and persistence of children to daily GH were conducted in the USA and UK. 5 , 6 Both studies found that a high proportion of children discontinued treatment despite exhibiting early persistence. 5 , 6 For adults with GHD (AGHD), adherence to daily GH among 107 participants was investigated using a cross-sectional analysis, 7 and the results showed that older age was significantly associated with better adherence to daily GH (p = 0.021).…”

“…5 , 6 Both studies found that a high proportion of children discontinued treatment despite exhibiting early persistence. 5 , 6 For adults with GHD (AGHD), adherence to daily GH among 107 participants was investigated using a cross-sectional analysis, 7 and the results showed that older age was significantly associated with better adherence to daily GH (p = 0.021). 7 Given trends seen in other chronic conditions requiring long-term treatment, it is likely that the high injection frequency plays a role in the increasingly low adherence to daily GH.…”