Safety and efficacy of everolimus as a rescue therapy in autoimmune hepatitis - 29/04/26
, Georgios Konstantis , Martina Daniel , Sabrina Guckenbiehl , Sebastian Wiener , Marcin Krawczyk , Hartmut H. Schmidt , Claudia Veltkamp ⁎ Highlights |
• | Everolimus is an effective rescue therapy in autoimmune hepatitis. |
• | Efficacy was demonstrated for up to 12 months. |
• | Everolimus showed a steroid-sparing effect. |
• | In patients with AIH and NMSC everolimus can be a therapeutic option. |
Abstract |
Background |
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 be an alternative, but available data is limited.
Patients |
We conducted a retrospective analysis of patients with AIH treated at our tertiary center between 2020 and 2025. Everolimus was administered either due to non-response to previous immunosuppressive therapies (cohort 1) or due to non-melanoma skin cancer (NMSC) during remission with conventional immunosuppressives (cohort 2).
Results |
Twenty-one patients were included (16 females, age range 28 – 80 years). Among 14 patients in cohort 1, therapy with everolimus was discontinued due to mild adverse effects (3 patients) or lack of response (2 patients). In the remaining 9 patients a significant reduction of AST was achieved after 3 months and persisted after 6 and 12 months (all p < 0.05). ALT improved after 4 weeks, and this improvement also persisted at 12 months follow-up (all p < 0.05). Prednisolone dose could be significantly reduced (p = 0.031). After 12 months of therapy with everolimus, normalization of AST was achieved in 4 (44.4%) and of ALT in 6 (66.7%) patients. Among 7 patients in cohort 2, everolimus was discontinued due to moderate adverse effects in 4 patients. All patients who tolerated everolimus (3 of 7, 42.9%) remained in remission.
Conclusion |
In the largest cohort to date, we demonstrate a response to everolimus in refractory AIH. Everolimus represents a treatment option in AIH patients with malignancies but warrants monitoring of side effects.
Le texte complet de cet article est disponible en PDF.Keywords : Liver, mTOR inhibitors, Rescue therapy, Immunology, Skin cancer
Abbreviations : AIH, mTOR, MMF, PBC, NMSC, CT, MRI, AST, ALT, GGT, CTCAE, IgG, AP, NET, TDM, INR
Plan
Vol 50 - N° 6
Article 102827- juin 2026 Retour au numéro
Article précédent
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