SUN-205 - Long‑Term Real‑World Safety and Effectiveness of Recombinant Human Growth Hormone in Asian Pediatric Short Stature: 12‑Year Interim Analysis from the Korean LGS Study

Background: The LG Growth Study (LGS) is a large-scale, multicenter observational study evaluating the long-term outcomes of recombinant human growth hormone (rhGH, Eutropin® products) in Korean pediatric patients with various growth disorders. This interim analysis provides real-world evidence based on a cumulative 12-year dataset.

Methods: The study analyzed a 12-year safety dataset (N=6,925). Effectiveness was evaluated over 4 years of treatment in children with growth hormone deficiency (GHD), small for gestational age (SGA), idiopathic short stature (ISS), Turner syndrome (TS), and chronic renal failure (CRF). Growth parameters, including height velocity (HV) and height standard deviation score (Ht SDS), and safety indicators, such as Insulin-like growth factor-I (IGF-I) SDS and the incidence of adverse events, were monitored and analyzed.

Results: Effectiveness was summarized for the 4 year follow-up period. The robust catch up growth was most prominent in the first year, followed by a stabilized growth velocity in subsequent years: HV reached 9.2 cm/year (GHD), 9.1 cm/year (SGA), 9.0 cm/year (ISS), and 7.5 cm/year (TS). After this peak, HV exhibited a physiological waning pattern. Ht SDS improved progressively across all indications; by 48 months, Ht SDS and baseline to 48 month changes (ΔHt SDS) were: GHD −0.9 (Δ +1.6), SGA −0.9 (Δ +1.6), ISS −1.1 (Δ +1.4), TS −2.0 (Δ +0.9), and CRF −1.6 (Δ +1.4); all changes were statistically significant (p < 0.0001). Mean IGF I SDS increased with treatment yet remained within the age adjusted normal range (−2.0 to +2.0 SDS) throughout the 4 year period. In the 12 year analysis set (N=6,925), cumulative incidences of adverse drug reactions (ADR) and serious ADRs (SADR) were ≤7.3% and ≤0.4%, respectively, with no unexpected long term safety concerns. The standardized incidence ratio (SIR) for malignant neoplasms was 1.17 (95% CI, 0.30–2.60), comparable to the general population and consistent with other global large scale registries.

Conclusion: These 12 year real world data confirm that long term rhGH therapy is effective and well tolerated in Asian children. Significant height gain, often approaching the near normal range, was achieved with a favorable safety profile. Malignancy risk remained consistent with the general population, supporting the long term risk benefit profile of rhGH in clinical practice.

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