Long-term data verifies rhGH safe, effective in real world settings

Recombinant human growth hormone (rhGH) in real-world settings is effective at increasing short-term heigh gain and adult height in patients with and without growth hormone deficiency (GHD), according to data from the largest and longest-running international database.

Data from the KIGS database on 83,803 children treated with rhGH from 1987-2012 were collected on patients treated for ≥1 year. Overall, 46.9% were treated for idiopathic GH deficiency, 10% for organic GHD, 9.5% for small for gestational age, 9.2% for Turner syndrome, 8.2% for idiopathic short stature, and 16.2% for other.

There was a median treatment duration of rhGH of 2.7 years and patients were observed for a median of 3.1 years. Serious adverse events were reported in 3.7% of patients and death in 0.4%. The most common SAEs were recurrence of craniopharyngioma (n = 151), neoplasm (n = 99), and cancer (n = 91); and scoliosis (n = 91).

Reference
Maghnie M, Ranke MB, Geffner ME, et al. Safety and efficacy of pediatric growth hormone therapy: Results from the full KIGS cohort. J Clin Endocrinol Metab. 2022;dgac517. doi: 10.1210/clinem/dgac517. Epub ahead of print. PMID: 36102184.