Data from: Long-term safety and effectiveness of growth hormone therapy in Korean children with growth disorders: 5-year results of LG Growth Study

Purpose The aim of this registry study was to analyze the long-term safety
and effectiveness of recombinant human growth hormone (rhGH) in South
Korean pediatric patients (≥2 years of age) with growth hormone deficiency
GHD) of idiopathic or organic etiology, idiopathic short stature, Turner
syndrome, small for gestational age and chronic renal failure. Methods The
study patients were followed-up till two years after the epiphyseal
closure, with visits scheduled every six months. The outcome measures
included the incidence of adverse events (AEs, in particular, neoplasia,
glucose intolerance and hypothyroidism), as well as height standard
deviation score (Ht SDS) and annual height velocity. The results of the
interim analysis of a 5-year accumulated data for 2,024 patients (7,342
patient-years, PY) are presented. Results A total of 14 neoplasms were
diagnosed (191/100,000 PY); 7 out of 9 malignancies were recurrent
craniopharyngioma found in patients with organic GHD. Seven cases of
glucose intolerance (95/100,000 PY) and 22 cases of hypothyroidism
(300/100,000 PY) were detected; about half of the cases (4 and 10 cases
each) were considered to be related with rhGH treatment. Most of the
growth-retarded patients showed continuous improvement in Ht SDS, with the
most prominent effect observed within a year of treatment initiation. The
beneficial effect of rhGH on Ht SDS gain was maintained for 2-4 years.
Conclusions The incidence of AEs of interest in rhGH-treated patients was
low, and most of the neoplasms were benign and/or non-related to rhGH.
Most patients benefited from the therapy in terms of height increment.