ESPE Abstracts

Objectives: This study aim ed to evaluate the safety and effectiveness of recombinant human growth hormone (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: The safety analysis included all eligible patients who received at least one dose of rhGH. The effectiveness analysis comprised patients from the safety analysis group who had undergone at least one post-treatment height measurement. Main outcomes included adverse events (AEs), height velocity (HV) and height standard deviation score (SDS).

Results: Over the 11-year study, a total of 5,971 patients (52.6% male, 47.4% female) were analyzed for safety. Among them, 4,005 (67.1%) had growth hormone deficiency (GHD), 1,056 (17.7%) were born small for gestational age (SGA), 901 (15.1%) were diagnosed with idiopathic short stature (ISS), 267 (4.5%) had Turner syndrome (TS), 10 (0.2%) had chronic renal failure (CRF) and 3 (0.1%) had Prader-Willi Syndrome. Additionally, 271 patients (4.5%) were diagnosed with both GHD and SGA. The mean age at the screening was 7.5 years, and the mean treatment duration was 3.8 years. During the 11 years, the proportions of AEs, adverse drug reactions (ADRs), serious AEs (SAEs), and serious ADRs (SADRs) were 34.9% (n = 2,083), 6.5% (n = 388), 3.3% (n = 196), and 0.3% (n = 16), respectively. There were a total of 16 SADR events, with the most common System Organ Class (SOC) category being neoplasm, which included 6 cases (4 craniopharyngioma, 1 schwannoma, 1 primitive neuroectodermal tumor). Malignant neoplasms (defined by International Classification of Diseases-10, C code) were observed in 3 cases, including Ewing primitive neuroectodermal tumor, medulloblastoma, and dysgerminoma. Craniopharyngioma recurrence in organic GHD was 14.0% (n = 6/43). In the effectiveness analysis of 3,615 patients, the continued height growth during 11-year was observed. The mean height SDS improved from -2.5±0.7 at baseline to -1.8±0.7 after 1 year. Additionally, height SDS surpassed -1 after 5 years and reached -1.01±2.03 at 11 years. The largest SDS change occurred in the 1 year, with peak HV at 9.1±1.7 cm/year, gradually decreasing thereafter. Patients with idiopathic GHD showed the greatest mean HV at 1 year, increasing from 4.8±2.1cm/year (n = 478) to 9.3±1.6 cm/year (n = 1,565).

Conclusion: Data from the LGS confirm the safety of rhGH treatment and show significant improvements in mean height SDS. Further long-term LGS data will provide insights into rhGH use for pediatric short-stature.