Efficacy of Recombinant Human Growth Hormone on Glucocorticoid-Induced Short Stature in Children: A Retrospective Controlled Study.

BACKGROUND Long-term glucocorticoid (GC) therapy is a leading cause of growth retardation in children with chronic renal and rheumatic diseases. While recombinant human growth hormone (rhGH) is used to counteract these effects, its efficacy relative to spontaneous catch-up growth following GC withdrawal remains inadequately quantified. This study aimed to compare the efficacy of rhGH treatment against spontaneous catch-up growth in children with GC-induced short stature.

METHODS This retrospective, non-randomized controlled study was conducted at a single tertiary children's hospital, including patients treated between 2010 and 2020. We established a treatment group of 34 children (23 with nephrotic syndrome [NS]) with GC-induced short stature who received rhGH therapy (0.15-0.2 IU/kg/day, approx. 0.05-0.067 mg/kg/day) for at least one year. A historical control group comprised 20 children with NS who exhibited short stature after GC cessation and were monitored for spontaneous catch-up growth for 6-12 months. The primary outcome was the annualized growth velocity after one year. Secondary outcomes included changes in height standard deviation score (SDS), bone age (BA), IGF-1 SDS, and IGF-binding protein 3 (IGF-BP3) levels.

RESULTS The primary analysis focused on patients with NS. The mean annualized growth velocity in the rhGH-treated NS subgroup (n=23) was significantly higher than in the control group (n=20) (10.48 ± 2.58 cm/year vs. 5.79 ± 0.49 cm/year, P < 0.001). Within the entire rhGH treatment cohort (n=34), after one year of therapy, the height SDS significantly improved (P < 0.001). The discrepancy between bone age and chronological age narrowed from 2.61 ± 1.64 years at baseline to a median of 1.0 year (IQR: 0.45, 2.6) post-treatment (P < 0.001). Serum IGF-1 SDS increased significantly from -1.45 ± 0.82 to 1.12 ± 0.95 (P < 0.001). rhGH therapy was well-tolerated.

CONCLUSION In children with Nephrotic Syndrome and GC-induced short stature, rhGH treatment results in a significantly greater improvement in growth velocity compared to spontaneous catch-up growth alone. It effectively enhances linear growth and normalizes the GH-IGF-1 axis with a favorable safety profile.