Adherence to growth hormone treatment in the transition age: A prospective observational multicenter study

Abstract Context Adherence can be an issue in patients taking growth hormone (GH). Objective To evaluate adherence to GH treatment during the transition age in patients with permanent childhood-onset GH deficiency (COGHD). Methods This is a prospective, multicenter, observational study conducted across 9 European centers. Fifty-one patients aged 15 to 25 years with permanent COGHD, who had reached final height and continued recombinant human GH (0.003-0.02 mg/kg/day), were monitored for 12 months using the Easypod™ device, which provides objective adherence data. Anthropometry, total cholesterol, low- and high-density lipoprotein cholesterol, and insulin-like growth factor 1 (IGF-1) standard deviation score (SDS) were measured at baseline and after 1 year. Patients with ≥9 months of adherence data (n = 41) were analyzed. Results Twenty-six patients (63%, 16 males) had optimal adherence (≥85%, median, 98%; interquartile range, 91-99%), and 15 (37%, 11 males) had suboptimal adherence (<85%, median, 70%; interquartile range, 47-80%). At baseline, suboptimal adherent patients had greater mean waist circumference (89.2 vs 78.2 cm) and lower mean IGF-1 SDS (−2.2 vs −1.5). After 1 year mean waist circumference (84.8 vs 76.7 cm), mean total cholesterol (185.1 vs 167.0 mg/dL), and mean LDL (111.6 vs 98.5) were higher in the suboptimal adherent group, whereas mean IGF-1 SDS was lower (−1.1 vs −0.2). The mean change in IGF-1 SDS after 1 year was +1.4 vs +0.9 in the 2 groups. Conclusion Over 1/3 of patients with permanent COGHD during transition show suboptimal adherence to GH therapy, associated with adverse metabolic markers and persistently lower IGF-1 levels. These findings highlight the importance of adherence monitoring and support targeted interventions to optimize long-term outcomes.