Polycystic ovary syndrome (PCOS) is characterized by insulin resistance and hormonal imbalances. Cangfudaotan Decoction (CFD), a traditional Chinese medicine formula, has been used empirically in the management of PCOS, but experimental evidence for its efficacy and mechanistic basis remains limited. This study aimed to investigate the therapeutic effects and mechanisms of CFD in PCOS. UHPLC-MS analysis identified bioactive compounds in CFD, followed by network pharmacology analysis. For in vivo validation, female Sprague-Dawley rats were randomly allocated into four groups (n = 10 per group): Control, PCOS, PCOS-Metformin, and PCOS-CFD. The letrozole-induced PCOS rat model was further treated with metformin (50 mg/kg/d) or CFD (8.53 g/kg/d) for 4 weeks. Evaluated were hormonal profiles, inflammatory cytokines, endometrial receptivity markers, and mTOR pathway proteins. Chemical profiling of Cangfudaotan Decoction (CFD) initially identified 60 bioactive compounds. Network pharmacology analysis revealed 280 overlapping targets between CFD and PCOS, with the mTOR signaling pathway highly enriched. Molecular docking indicated potential binding interactions between key compounds (Isocorynoline, Licochalcone B, Papaverine, Medicarpin, Peiminine) and mTOR. In vivo experiments showed that CFD was highly efficient at improving IR, LH, and testosterone suppression, and at restoring estrous cyclicity. Inflammatory cytokines (IL-1β, TNF-α, IL-6, CRP) significantly decreased. CFD upregulated endometrial HOXA10, LIF, and integrin αvβ3 expression in PCOS rats, accompanied by attenuation of overactivated mTOR signaling. CFD treatment improved insulin resistance, restored hormonal balance, and upregulated endometrial receptivity markers in PCOS rats. mTOR was identified as a potential core target. However, further studies are needed to validate the precise role of mTOR and confirm the clinical applicability of CFD in PCOS management. Through chemical profiling and network pharmacology, this study shows that CFD alleviates metabolic and hormonal dysfunction in PCOS while improving endometrial receptivity, possibly by modulating the mTOR signaling pathway in endometrial tissue.
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