Integrative clinical and in silico drug discovery study of Guishao Shengxue Decoction reveals immune–platelet modulatory targets in pediatric ITP

Background Immune thrombocytopenia (ITP) is a common autoimmune bleeding disorder in children characterized by platelet destruction and immune dysregulation. Guishao Shengxue Decoction (GSD), a traditional Chinese medicine formulation, has shown therapeutic potential, but its integrated clinical and molecular mechanisms remain insufficiently defined. Methods This retrospective study included 86 pediatric patients with primary ITP treated with GSD between 2018 and 2024. Hematological indices, lymphocyte subsets, immunoglobulins, complement levels, and cytokines were evaluated before and after treatment. Correlation and multivariate regression analyses were performed to identify predictors of platelet recovery. Additionally, in silico approaches including network pharmacology, molecular docking, and molecular dynamics simulations were applied to explore potential bioactive compounds and molecular targets. Results GSD treatment significantly increased platelet counts (24.6 ± 12.8–96.3 ± 41.7 ×10⁹/L, p < 0.001) with an overall response rate of 81.4%. Immunologically, CD4⁺ T cells and CD4/CD8 ratio decreased, while NK cells increased significantly. Pro-inflammatory cytokines (IL-6, TNF-α) declined, whereas IL-10 increased ( p < 0.001). Higher platelet counts were positively associated with NK cells and IL-10, and negatively with CD4/CD8 ratio and IL-6. Multivariate analysis identified NK cells, CD4/CD8 ratio, IL-6, IL-10, and complement C3 as independent predictors of platelet recovery. In silico analysis revealed key bioactive compounds (e.g., astragaloside IV, paeoniflorin) targeting hub proteins such as IL6, TNF, STAT3, and JAK2, with stable binding confirmed by molecular dynamics simulations. In this retrospective observational study, treatment with Guishao Shengxue Decoction was associated with changes in platelet counts and multiple immune parameters in children with primary immune thrombocytopenia. Because the study lacked a contemporaneous control group and residual confounding cannot be excluded, these findings should be interpreted as observational associations rather than evidence of a causal therapeutic effect. The accompanying in silico analyses provide biologically plausible mechanistic hypotheses that warrant validation in prospective controlled studies. Conclusion In this retrospective single-arm study, treatment with Guishao Shengxue Decoction (GSD) was associated with significant improvements in platelet counts and immune parameters in children with immune thrombocytopenia. However, because no control group was included, the observed clinical improvements cannot be definitively attributed to GSD, and spontaneous remission inherent to pediatric ITP cannot be excluded. Prospective randomized controlled studies are required to confirm the therapeutic efficacy of GSD.