ESPE Abstracts

Objectives: This study aimed to evaluate the safety and effectiveness of rhGH treatment, using specific products (Eutropin®, Eutropin®Pen, Eutropin®AQ, Eutropin®Plus and Eutropin®SPen; LG Chem, Ltd.), in pediatric patients with growth disorders in Korea.

Methods: Among the patients who enrolled in LGS (2012–2022, n=5,120), patients received at least one injection of rhGH were included for safety analysis. The effectiveness analysis comprised patients from the safety analysis group who had at least one measurement of height after receiving rhGH treatment. Main outcomes included adverse events (AEs), height velocity (HV) and height standard deviation score (SDS).

Results: During the 10-year study, a total of 5,040 patients (male 53.2%, female 46.8%) were analyzed for safety. Of those patients, 3,332 (66.1%) had GHD, 869 (17.2%) were born SGA, 747 (14.8%) were diagnosed with ISS, 257 (5.1%) had TS and 10 (0.2%) had CRF. The mean age at the screening was 7.6 years and the mean treatment duration was 3.8 years. AEs were reported in 1,725 (34.2%) patients, most AEs were mild. A total of 41 neoplasms were reported in 36 (0.7%) patients, the majority of which were benign and unrelated to rhGH. Cumulative results of AEs showed that the proportions of patients experiencing adverse drug reactions (ADRs), serious adverse events (SAEs), and serious adverse drug reactions (SADRs) remained relatively stable throughout 10 years of follow-up (7.0%, 3.2% and 0.3% respectively). The occurrence probability of SADRs remained stable over the 5-year follow-up period. (0.4% for SADRs). This finding suggests that the probability of experiencing SADRs during rhGH treatment was consistently low and relatively consistent throughout long-term duration. The mean HV increased from 5.3±4.0 cm/year (n=672) at baseline to 9.1±1.7 cm/year after 12 months of rhGH treatment (n=2,070). The greatest mean HV was observed in patients with idiopathic GHD. After treatment with rhGH, the height SDS consistently increased compared to baseline at each assessment. In the effectiveness analysis, the mean height SDS significantly improved from -2.58±0.69 at baseline to -1.82±0.74 (n=2,070), -1.43±0.80 (n=1,486), -1.24±0.86 (n=977), and -1.10±1.02 (n=671) at 12, 24, 36, and 48 months, respectively, with stable maintenance thereafter.

Conclusions: Data from the LGS, the largest and longest-running database of rhGH-treated pediatric patients in Korea, support the safety of rhGH treatment and demonstrate significant improvements in mean height SDS.