Belimumab treatment in autoimmune hepatitis and primary biliary cholangitis – a case series

• Published in: Journal of translational autoimmunity (Online) Vol. 6; p. 100189
• Main Authors: Kolev, Mirjam, Sarbu, Adela-Cristina, Möller, Burkhard, Maurer, Britta, Kollert, Florian, Semmo, Nasser
• Format: Journal Article
• Language: English
• Published: Netherlands Elsevier B.V 01.01.2023; Elsevier
• Subjects: Autoimmune hepatitis; Autoimmune liver disease; Belimumab; Primary biliary cholangitis; Research paper; Sjögren's disease; LKM-1; APS; NRH; Belimumab; IgG; ALT; SLA; UDCA; INR; BDN; SLE; IgM; Sjögren's disease; PBC; AMA; ULN; PDN; disease-modifying anti-rheumatic drugs; AIH; PSC; CI; SMA; MMF; AZA; CyA; LC 1; AP; PLA2R; Autoimmune liver disease; SS-A; Autoimmune hepatitis; NA; SS-B; ANA; RF; MRCP; Primary biliary cholangitis; TNFi; MMF, mycophenolate mofetil; INR, international normalized ratio; SS-B, SS-B (La) antibodies; NA, not applicable; ANA, anti-nuclear antibodies; SLE, systemic lupus erythematosus; CyA, cyclosporine A; SS-A, SS-A (Ro) antibodies; SMA, smooth-muscle cell antibodies; ULN, upper limit of normal; SLA, soluble liver antigen antibodies; CI, calcineurin inhibitor; NRH, nodular regenerative hyperplasia; UDCA, ursodeoxycholic acid; ALT, alanine aminotransferase; TNFi, tumor necrosis factor inhibitor; IgM, immunoglobulin M; PLA2R, anti-phospholipase 2 receptor antibody; LC 1, liver cytosol 1 antibodies; AZA, azathioprine; BDN, budesonide; PDN, prednisolone; IgG, immunoglobulin G; LKM-1, liver-kidney-microsomal antibodies; PSC, primary sclerosing cholangitis; MRCP, magnetic resonance cholangiopancreatography; RF, rheumatoid factor; APS, anti-phospholipid-antibody syndrome; AMA, anti-mitochondrial antibodies; PBC, primary biliary cholangitis; disease-modifying anti-rheumatic drugs, DMARDs; AIH, autoimmune hepatitis; AP, alkaline phosphatase
• ISSN: 2589-9090; 2589-9090
• DOI: 10.1016/j.jtauto.2023.100189

The majority of patients with autoimmune hepatitis (AIH) achieve complete remission with established treatment regiments. In patients with intolerance or insufficient response to these drugs, the remaining options are limited and novel treatment approaches necessary. In primary biliary cholangitis (PBC), ursodeoxycholic acid (UDCA) and fibrates have improved prognosis dramatically, but there remains a proportion of patients with refractory disease. In patients with refractory AIH and/or PBC, we used a novel treatment strategy with the anti-B cell activating factor, belimumab. The first three patients had concomitant Sjögren's disease. The connecting element between all three diseases is B cell activation, including elevated levels of the B cell activating factor (BAFF). Furthermore, belimumab has been shown to be beneficial in Sjögren's disease. To retrospectively investigate treatment response in six patients with AIH or PBC with or without concomitant Sjögren's disease treated with the anti-BAFF therapy belimumab at the University Hospital in Bern, Switzerland. In all three patients with AIH, belimumab improved disease control and helped by-pass or reduce problematic side effects from corticosteroids and calcineurin inhibitors. In PBC patients (n = 3), there was no clear improvement of liver function tests, despite reduction or normalization of IgM. All patients with concomitant Sjögren's disease (n = 3) had an improvement of sicca symptoms and two out of three patients experienced an initially marked reduction in fatigue, which lessened over time. Belimumab may be a promising treatment option for patients with AIH and further investigations are needed. In PBC however, response was not convincing. The effects on sicca symptoms and fatigue were encouraging. [Display omitted] •Belimumab is a B cell-activating factor inhibitor.•In three patients with autoimmune hepatitis, belimumab led to remission.•In three patients with primary biliary cholangitis, belimumab showed no clear effect on disease activity.•Effects on sicca-symptoms and fatigue were encouraging.