Accumulated safety data of recombinant human growth hormone therapy in Korean children over a 10-year period: interim results from the LG Growth Study.

OBJECTIVE

The aim of this study was to evaluate the long-term safety outcomes of recombinant human growth hormone (rhGH) administered to Korean pediatric patients with growth disorders, including growth hormone deficiency, Turner syndrome, idiopathic short stature, small for gestational age, chronic kidney disease, and Prader-Willi syndrome, using the 10-year interim data from the nationwide LG Growth Study registry in the Republic of Korea.

DESIGN

This is a prospective, multicenter, observational study.

METHODS

This interim analysis included 5,040 patients from 96 centers, representing 19,878 patient-years of treatment between November 2011 and December 2022. Although the registry spans 10 years, follow-up was limited to periods of active GH treatment and, therefore, varied across patients. Patients received daily or weekly rhGH. Adverse events (AEs), adverse drug reactions (ADRs), serious AEs (SAEs), and serious ADRs (SADRs) were recorded and compared with international registry data.

RESULTS

Overall, AEs occurred in 34.2% of patients, ADRs in 7.0%, SAEs in 3.2%, and SADRs in 0.3%. The most frequent AE was scoliosis (1.6%), followed by headache (0.8%) and injection site pain (0.5%). Three malignant neoplasms were reported, yielding a standardized incidence ratio of 1.1 (95% confidence interval: 0.21-2.69), comparable to expected national rates. Craniopharyngioma recurrence occurred in 14.3% of affected patients, consistent with international data.

CONCLUSION

Over the 10 years, rhGH therapy in Korean pediatric patients demonstrated a low incidence of ADRs and SADRs, with malignancy and tumor recurrence rates similar to those in global registries. These findings support the long-term safety of rhGH for pediatric growth disorders, with continued surveillance warranted.

SIGNIFICANCE: Using 10 years of real-world data from a pediatric registry, conducted in the Republic of Korea, this study provides one of the largest single-nation safety evaluations of rhGH therapy across multiple etiologies of short stature. The low incidence rates of ADRs and SADRs, in alignment with rates reported for other large-scale international registries, provide reassuring evidence of the safety of rhGH for clinicians and caregivers.