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

• Published in: JHEP reports Vol. 2; no. 4; p. 100123
• Main Authors: Arvaniti, Pinelopi, Giannoulis, George, Gabeta, Stella, Zachou, Kalliopi, Koukoulis, George K., Dalekos, George N.
• Format: Journal Article
• Language: English
• Published: Netherlands Elsevier B.V 01.08.2020; Elsevier
• Subjects: Autoimmune hepatitis; B cells; BAFF; Belimumab; Short Communication; Treatment; AIH; BAFF; AST; Belimumab; Tregs; LSM; MMF; AZA; ALT; B cells; SLE; TNF; CR; Autoimmune hepatitis; Treatment; ANA; DCs; GGT; ULN; EBV; ALT, alanine aminotransferase; CR, complete response; MMF, mycophenolate mofetil; DCs, dendritic cells; TNF, tumour necrosis factor; AST, aminotransferase; AZA, azathioprine; SLE, systemic lupus erythematosus; ANA, antinuclear antibody; GGT, gamma-glutamyl transferase; Tregs, regulatory T cells; BAFF, B cell-activating factor; LSM, liver stiffness measurements; ULN, upper limit of normal; EBV, Epstein–Barr virus; AIH, autoimmune hepatitis
• ISSN: 2589-5559; 2589-5559
• DOI: 10.1016/j.jhepr.2020.100123

Autoimmune hepatitis (AIH) is a disease of unknown aetiology with a favourable response to immunosuppression. However, in the clinic, it appears that <50% of patients achieve complete response on standard treatment. Serum B cell-activating factor (BAFF) levels are elevated in patients with AIH and are likely to contribute to disease pathogenesis. Given that belimumab, a BAFF inhibitor, has been shown to be effective in other autoimmune diseases, we investigated its use as a third-line add-on treatment option in patients with advanced AIH who did not respond to conventional treatment. Herein, we report for the first time two patients, a 27-year-old female and a 58-year-old male, both 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 a complete response and remained in remission while receiving low-dose corticosteroids. No adverse events related to belimumab and/or disease decompensation were observed. These preliminary findings indicate belimumab as a promising treatment option for patients with AIH and refractory and advanced liver-related fibrosis. A small proportion of patients with autoimmune hepatitis (AIH) are refractory to standard treatments; these patients bear the highest probability of developing decompensated cirrhosis and hepatocellular carcinoma because third-line treatment options are not well established. In this case study, we showed that third-line add-on therapy with belimumab, a B cell-activating factor inhibitor, could be an alternative and promising treatment option in patients with advanced AIH who did not respond to conventional treatment. •Belimumab is a B cell-activating factor inhibitor that has been proposed for the treatment of autoimmune hepatitis.•In 2 patients with autoimmune hepatitis, belimumab led to complete response and remission.•No adverse events related to belimumab and/or disease decompensation were observed.