Long-Term experience with growth hormone therapy in pediatric growth disorders: an analysis of the LG growth study data

Abstract

Objective Growth hormone (GH) therapy has been widely applied to improve height outcomes in pediatric patients experiencing growth hormone deficiency (GHD), idiopathic short stature (ISS), Turner syndrome (TS), and small-for-gestational-age (SGA). This study aimed to evaluate long-term growth outcomes of recombinant human GH therapy across various indications in Korean pediatric patients. Methods We conducted a retrospective analysis utilizing 10-year longitudinal clinical data from the LG Growth Study (LGS), a multicenter, observational registry in Korea. Annual changes in height standard deviation scores (SDS) and growth velocity were primary outcomes, with subgroup analyses by age at initiation and diagnosis performed to identify determinants of treatment response. Results As of December 2022, a total of 3,103 patients were included in the analysis. The mean age of the subjects was 7.43 +/- 2.94 years. Among the patients, GHD comprised 69.1%, SGA 18.4%, ISS 9.8%, and TS 6.2%. After ten years of GH treatment, substantial improvements in height standard deviation scores (SDS) were observed in all patient groups. The most significant growth increase occurred during the first year and gradually diminished in subsequent years. In the GHD group, younger age at treatment initiation was associated with greater improvements in height SDS compared to older patients. Within the TS group, patients with mosaic karyotypes exhibited better responses than those with monosomy or structural chromosome abnormalities. Conclusion This extensive real-world study demonstrates that GH therapy significantly improves linear growth outcomes in Korean patients across various growth disorders.