ESPE Abstracts

Background: Daily GH has been used to treat growth disorders in children for a long time. The weekly sustained-release GH formulation has been approved for treatment in GH deficiency (GHD). It provides a practical strategy for improving adherence. However, there is still a lack of sufficient clinical research data of weekly GH.

Objective and hypotheses: To evaluate the long-term safety and effectiveness of two formulations of daily (Eutropin injection) and weekly (EutropinPlus injection) GH in Korean pediatric patients.

Method: A multi-center, cohort, long-term, prospective, and retrospective study. Statistical analysis has been conducted annually using a pre-defined method. The interim analysis was conducted in all patients who were enrolled from Jan 2012 to Feb 2015.

Results: Among the 1287 enrolled patients, a total of 758 patients with GHD were analysed to compare the safety between daily (n=537) and weekly (n=221). Baseline demographics were similar between groups. 299 patients were analysed for effectiveness during the 2 years of GH treatment in GHD (daily (n=208) and weekly (n=91)). Height SDS at baseline was −2.41 in the daily and −2.54 in the weekly. Change in height SDS from baseline to 2 years was 3.11 in the daily and 2.88 in the weekly. Height velocity during the 1st and 2nd year was 9.03 and 8.23 cm/year in the daily, respectively and was 8.95 and 8.20 cm/year in the weekly respectively. Height SDS and velocity were not statistically different between groups. Adverse events (AEs) were reported in 14.7% of daily and 9.5% of weekly. The incidence of adverse drug reactions was 3.2% in the daily and 2.7% in the weekly. Mild to moderate AEs were most commonly reported.

Conclusion: Growth response of weekly GH is comparable to daily GH in GHD. Also, both daily and weekly GH formulations were safe and well-tolerated.