Efficacy and safety of recombinant human growth hormone in treating Chinese children with idiopathic short stature

Objective: This study aims to investigate the efficacy and safety of recombinant human growth hormone (rhGH) in the treatment of idiopathic short stature (ISS).

Methods: The data of 200 ISS children, who were treated with rhGH from January 2008 to December 2016, were collected and retrospectively analyzed. The data of height, bone age(BA), chronological age(CA), fasting blood glucose, fasting insulin, thyroid function and IGF-1 were collected, and annual growth velocity (GV), height standard deviation score (HtSDS) and related factors that affect GV were statistically analyzed.

Results: (1) GV and HtSDS changes: As the time of treatment increased, the GV decreased year by year. The GV in the second year was significantly lower than that in the first year (P < .0001), and the GV in the fourth year was significantly lower than that in the third year (P < .05). HtSDS gradually increased from the first year to the third year, and became significantly higher than that in the year before the treatment (P < .01). The difference in the increase in HtSDS between the fourth year and third year was not statistically significant (P > .05). (2) The influence factors of GV included age at initial treatment, IGF-1SDS during treatment and GV in the year before treatment. (3) The most common side effects during treatment included transient hyperglycemia and temporary hyperinsulinemia, and these returned to normal after the treatment was stopped. Some patients presented with accelerated bone age growth after two years of treatment (annual growth of bone age △BA was >2 years), compared with children without accelerated bone age growth, and the difference between BA and CA (BA-CA) was significantly reduced at the beginning of the treatment (P < .01).

Conclusion: rhGH has a good growth promoting effect on ISS children. A variety of factors may affect the GV, and related adverse reactions should be monitored during the treatment.