Kensuke Terakawa, Yukihiro Wada, Yuki Toyoda, Keiya Nakamura, Rina Kamiya, Ryota Uchitsubo, Shun Sakurabayashi, Tomomi Motohashi, Sayumi Kawamura, Hiroshi Tominaga, Kazuhiro Takeuchi, Yutaro Saito, Masataka Tochimoto, Hisashi Hidaka, Makoto Saegusa, Yasuo Takeuchi
Avacopan, a selective complement C5a receptor antagonist, is utilized to manage microscopic polyangiitis (MPA). Recently, attention has grown regarding severe liver injury, particularly vanishing bile duct syndrome (VBDS), as a potential adverse event during avacopan therapy. However, its clinicopathological features and underlying mechanisms remain poorly understood. Herein, we report a case of non-suppurative destructive cholangitis (NSDC), considered a pre-conditional state of VBDS, after avacopan therapy for MPA. A 55-year-old obese female with myeloperoxidase-antineutrophil cytoplasmic antibody (MPO-ANCA)-associated crescentic glomerulonephritis achieved remission via steroid pulse therapy, oral prednisolone (PSL), and rituximab. Seven weeks before admission, avacopan and ursodeoxycholic acid (UDCA) were initiated. Despite stable renal function, MPO-ANCA seroconversion to negative, and successful PSL tapering, she presented with acute liver injury. Laboratory tests revealed marked elevations in transaminases and biliary enzymes (aspartate aminotransferase (AST): 313 U/L, alanine aminotransferase (ALT): 488 U/L, gamma-glutamyl transferase (γ-GTP): 364 U/L) with normal direct bilirubin (D-bil). Avacopan was discontinued, and PSL was increased. A liver biopsy showed lymphocytic (non-suppurative) destructive cholangitis with a florid duct lesion and frequent spotty necrosis in lobuli, without central necrosis. Immunohistochemical staining revealed focal decreased CK19 immunointensity in the bile ducts and predominant infiltration of CD4-positive T cells and CD68-positive macrophages around interlobular bile ducts. Although D-bil transiently peaked at 4.0 mg/dL on day 7, intensive treatment with high-dose UDCA and intravenous glycyrrhizin restored D-bil to the normal range by day 34, with significant transaminase improvement. Pathological findings revealed biliary epithelial damage with predominant T-cell and macrophage infiltration, distinct from typical drug-induced liver injury. The onset during PSL tapering and responsiveness to temporary PSL intensification strongly support a cell-mediated immune mechanism driving VBDS. To the best of our knowledge, this is the first report describing a comprehensive immunohistochemical evaluation of the periportal microenvironment in avacopan-induced biliary injury. This case highlights that severe cholangitis can occur regardless of baseline risk profiles or disease activity, underscoring the need for vigilant, long-term monitoring of liver enzymes and bilirubin levels during avacopan therapy.