To observe the efficacy of polyethylene glycol recombinant human growth hormone (PEG-rhGH) injection combined with vitamin D drops and lysine hydrochloride and calcium hydrogen phosphate granules in the treatment of children with idiopathic short stature (ISS).

Based on treatment regimen, children with ISS were categorized into treatment and control groups. Both groups received basic treatment of vitamin D drops (400-800 U daily) and lysine hydrochloride and calcium hydrogen phosphate granules (5 g, twice daily). The control group was given human somatropin injection at a dose of 0.10-0.20 IU·kg^-1·d^-1 by subcutaneous injection, while the treatment group was given PEG-rhGH injection at a dose of 0.20-0.40 mg·kg^-1·w^-1 by subcutaneous injection. Both groups received continuous medication for 12 months. The clinical efficacy, evaluation indicators of GH-insulin-like growth factor (IGF) axis function, bone metabolism indicators and safety were compared between the two groups.

The study enrolled 79 cases, comprising 41 cases in the treatment group and 38 cases in the control group. After treatment, the effective rates of the treatment group and the control group were 95.12% (39 cases/41 cases) and 92.11% (35 cases/38 cases), respectively, with no statistically significant difference (P>0.05). After treatment, the levels of insulin-like growth factor-1/insulin-like growth factor binding protein-3 (IGF-1/IGFBP-3) in the treatment group and the control group were 0.08±0.02 and 0.07±0.02, respectively; the levels of liver-expressed antimicrobial peptide-2 (LEAP-2) were (1 395.69±187.26) pg·mL^-1 and (1 487.58±195.48) pg·mL^-1, respectively; the levels of osteocalcin (BGP) were (22.18±3.67) and (20.43±3.48) μg·L^-1, respectively; procollagen I N-terminal peptide (PINP) were (602.68±63.41) and (574.08±61.83) μg·L^-1, respectively; bone-specific alkaline phosphatase (BALP) was (192.68±23.64) and (203.41±22.18) U·L^-1, respectively; vitamin D (Vit-D) were (20.39±3.47) and (18.65±3.62) ng·mL^-1, respectively. Both indicators showed statistically significant differences between the two groups (all P<0.05). The incidences of adverse drug reactions of treatment group and the control group were 7.32% (3 cases/41 cases) and 10.53% (4 cases/38 case), with no statistically significant difference (P>0.05).

PEG-rhGH injection at a dose of 0.20-0.40 mg·kg^-1·w^-1 combined with vitamin D drops and lysine hydrochlodride and calcium hydrogen phosphate granules in the treatment of children with ISS can ensure efficacy and safety, while potentially achieving better long-term growth improvement effects. It can also improve the function of the GH-IGF axis and bone metabolism levels.