Background: Everolimus (EVE) has been approved by the FDA for hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2
Background: Everolimus (EVE) has been approved by the FDA for hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced breast cancer patients. However, in clinical practice, the toxicity of the standard dose (10 mg/d) limits its use. Method: This study aimed to compare the efficacy, safety, and compliance of 5 mg/d and 10 mg/d EVE in combination with endocrine therapy in btreast cancer patients. Results: Compared with the 5 mg/d group, the 10 mg/d group included more primary endocrine-resistant patients (46.7 % vs. 24.6 %; P = 0.036). There was no significant difference in overall ORR and DCR between the two groups and the difference in median PFS and OS was not statistically significant (PFS: 7.07 m vs. 8.07 m, P = 0.663; OS: 29.47 m vs. 30.53 m, P = 0.615). In the subgroup of patients with primary endocrine resistance and premenopausal patients, the PFS benefit of the 10 mg/d group was more significant. In the safety analysis, the 5 mg/d group had lower rates of fatigue (29.2 % vs. 53.3 %; P = 0.038) and diarrhea (10.8 % vs. 33.3 %; P = 0.011). In the compliance analysis, the 5 mg/d group had a lower treatment interruption rate, and fewer patients stopped treatment due to adverse reactions. For most patients, 5 mg/d and 10 mg/d EVE combined with endocrine therapy showed comparable efficacy, but the safety and compliance of 5 mg/d were better. Conclusions: For patients with primary endocrine resistance and premenopausal patients, using standard-dose EVE resulted in greater PFS benefit.