Objective To explore the efficacy of the self-formulated Zhuzhang Formula in treating the spleen deficiency with dysfunction of transportation syndrome of idiopathic short stature (ISS) and its effects on the growth rate, disease-related indicators, and bone metabolism indicators in affected children.

Methods A total of 80 children with ISS were enrolled as study subjects. They were randomly assigned to control group and observation group using random number table method, with 40 cases in each group. The control group received conventional western medicine treatment, while the observation group was administered self-formulated Zhuzhang Formula in addition to the treatment of the control group. Comparisons were made between the two groups in terms of clinical efficacy, TCM syndrome scores, growth parameters (monthly average height increment, annual growth rate), levels of disease-related indicatorsserum insulin-like growth factor-1 (IGF-1), bone morphogenetic protein-2 (BMP-2), insulin-like growth factor binding protein-3 (IGFBP-3), levels of bone metabolism indicatorsbone alkaline phosphatase (BAP), type Ⅰ procollagen N-terminal propeptide (PⅠNP), type Ⅰ collagen cross-linked C-terminal telopeptide (β-CTX), and the incidence of adverse reactions.

Results The overall effective rate in the observation group was 97.50%, which was significantly higher than the 80.00% in the control group(P < 0.05). After 3 and 6 months of intervention, the scores for symptoms such as short stature, sallow complexion, loss of appetite, weak voice and shortness of breath, limb weakness, and loose stools, as well as the total TCM syndrome scores, were lower in the observation group than those in the control group, with statistically significant differences (P < 0.05). After 6 months of intervention, the monthly average height increment and annual growth rate were higher in the observation group than those in the control group, and the differences were statistically significant (P < 0.05). After 3 and 6 months of intervention, the levels of IGF-1, BMP-2, IGFBP-3, BAP, and PⅠNP were higher in the observation group than those in the control group, while the level of β-CTX was lower, and the differences were statistically significant (P < 0.05). There was no statistically significant difference in the incidence of adverse reactions between the two groups (P>0.05).

Conclusion The self-formulated Zhuchang Formula demonstrates remarkable efficacy in treating the spleen deficiency with dysfunction of transportation syndrome of ISS. It can alleviate clinical symptoms by regulating the levels of IGF-1, BMP-2, IGFBP-3, and bone metabolism indicators, thereby promoting the growth and development of affected children, and exhibits high safety.