To observe the clinical efficacy and safety of recombinant human growth hormone injection (rhGH) in the treatment of children with idiopathic short stature (ISS) and its influence on levels of insulin-like growth factor 1 (IGF-1), alkaline phosphatase (AKP) and insulin-like growth factor binding protein 3 (IGFBP-3).

Children with ISS were randomized into control group and treatment group. The control group was subcutaneously injected with 0.15 U·kg^-1 of rhGH injection, while the treatment group was given subcutaneous injection of 0.20 U·kg^-1 of rhGH injection, and both groups were continuously treated for 6 months. The clinical efficacy, growth status [growth velocity, height standard deviation score (HtSDS), predicted adult height (PAH)], bone metabolism indicators {AKP, osteocalcin (OC), 25-hydroxyvitamin D [25 (OH)D]} and serum IGF-1 and IGFBP3 levels were compared, and the safety was assessed.

In the treatment group, 46 cases were enrolled, 6 cases were lost, and 40 cases were finally included in the statistical analysis. In the control group, 46 cases were enrolled, 1 case was lost, and 45 cases were finally included in the statistical analysis. The total effective rates in treatment group and control group after 6 months treatment were 85.00% (34 cases/40 cases) and 64.44% (29 cases/45 cases), respectively, with a statistical significance (P<0.05). After 6 months treatment, the growth rates in treatment group and control group were (7.96±1.62) and (6.84±1.56) cm·year^-1; HtSDS values were -2.38±0.24 and -2.61±0.28; PAH values were (156.86±4.18) and (155.02±4.25) cm; serum AKP levels were (278.42±47.46) and (257.14±42.79) U·L^-1; OC levels were (76.92±10.17) and (72.43±10.32) μg·L^-1; 25 (OH) D levels were (59.96±4.74) and (55.52±4.69) nmol·L^-1; serum IGF-1 levels were (296.77±28.32) and (251.47±24.96) ng·mL^-1; serum IGFBP3 levels were (5.76±1.22) and (4.86±0.89) μg·mL^-1, respectively. Compared with the control group, the above indexes in the treatment group were statistically significant (all P<0.05). The adverse drug reactions in treatment and control group were mainly headache, rash and joint pain, rash and joint pain. The incidence rates of adverse reactions in treatment group and control group were 12.50% (5 cases/40 cases) and 6.67% (3 cases/45 cases), without significant difference (P>0.05).

The clinical efficacy of rhGH injection in the treatment of children with ISS is definite; and the improvement of IGF-1, AKP and IGFBP3 levels by 0.20 U·kg^-1 rhGH is significantly better than that by 0.15 U·kg^-1 rhGH, which is more conducive to promoting bone development and accelerating growth without increasing the incidence of adverse drug reactions.