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Background and objective: In autoimmune hepatitis (AIH), some patients fail to respond to first-line immunosuppression or develop treatment-related malignancies requiring alternative treatments. Rapamycin (mTOR) inhibitors may represent an alternative treatment strategy; however, long-term follow-up data remain limited.

Method: We conducted a retrospective analysis of patients with AIH treated at our center between 2020 and 2025. In this analysis, everolimus was administered to patients with non-response to previous immunosuppressive therapies and 12-months follow-up data were collected.

Result: Nine patients were included in the analysis (seven females, mean age 60.1 years). A persistent significant reduction of AST (p = 0.02), ALT (p = 0.01), and GGT (p = 0.02) was achieved after 12 months of therapy. Bilirubine decreased significantly over 12 months (p = 0.046). Normalization of AST was achieved in four (44.4%) and of ALT in six (66.7%) patients. A significant reduction in prednisolone dosage persisted up to the 12-month follow-up (p = 0.02). Transient elastography measurements were available in seven patients and they improved (p = 0.04) under the therapy with everolimus. Total cholesterol was significantly elevated (p = 0.04), whereas high-density lipoprotein (HDL), low-density lipoprotein (LDL), and triglyceride levels did not significantly differ (all p > 0.05). No treatment discontinuation occurred during the observation period.

Summary: Everolimus demonstrated sustained efficacy at 12 months, accompanied by continued and successful tapering of prednisolone. The maintenance of disease control during steroid tapering highlights the potential role of the mTOR inhibitor everolimus as a rescue therapy in difficult-to-treat AIH. While elevated serum lipid levels emphasize the need for routine lipid screening, no additional safety concerns were identified.