Belimumab is a promising third-line treatment option for refractory autoimmune hepatitis

Abstract Autoimmune hepatitis (AIH) is a disease of unknown aetiology with favorable response to immunosuppression. Under real world circumstances, however, it appears that less than 50% of patients achieve complete response on standard treatment. Serum B cell activating factor (BAFF) levels are elevated in AIH patients contributing probably to disease pathogenesis. As belimumab, a BAFF inhibitor, was proved effective in other autoimmune diseases, the aim of the present case-study was to investigate its use as a third-line add-on treatment option in patients with advanced AIH who did not respond to conventional treatments. Herein, we report for the first time two patients, a 27-years old female and a 58-years old male with AIH-related compensated cirrhosis at diagnosis, who were refractory to standard immunosuppressive therapies and received add-on third-line therapy with belimumab. Both patients achieved complete response and remained in remission while receiving low corticosteroids doses. Off note, no adverse events related to belimumab and/or disease decompensation were observed. These preliminary findings indicate that belimumab could be a promising option for AIH patients with refractory and advanced liver-related fibrosis.