Safety and efficacy of Upadacitinib in patients with primary sclerosing cholangitis: a multicentre, retrospective study

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Background and Aims: Primary Sclerosing Cholangitis (PSC) is closely associated with inflammatory bowel disease (IBD). Upadacitinib (Upa), a selective Janus-Kinase inhibitor (JAKi), was approved for IBD treatment in 2022/23. This study assesses the safety and efficacy of Upadacitinib on liver and bowel disease in PSC-IBD patients. Method : Data from multiple centers were collected retrospectively at baseline and after 3, 6, 12 months follow-up (m-FU) and annually thereafter. Results: Forty patients (70% male, median age 21 years) from 9 centers were included, with 31 completing 3-month follow-up. Of these, 87.5% had Ulcerative Colitis and 12.5% had a Crohn’s phenotype. Prior to Upa, 77.5% received at least two biologicals, and 15% another JAKi. Adverse events were reported in 10 cases, including 3x elevated transaminases, 3x anal fissures/abscesses, 1x low WBC and 3x respiratory infections. Upa was discontinued in 20% of cases mostly due to lack of efficacy. In patients continuing Upa, ALP levels showed a non-significant decrease from 348 at baseline to 270 U/L at 3-m-FU (p=.068), while mucosal inflammation (Mayo Endoscopic subscore) significantly improved, with a median difference of -1 (p=.015) by 6 months. Transient elastography (p=.237; mean of 6.6 to 7.4 kPa at 6m-FU) and AST levels remained stable (144 vs 118 U/l at 3m-FU, p=.597). Conclusion: Upadacitinib improved PSC-associated IBD, with a trend toward decreased ALP levels. However, the occurrence of hepatitis and infections highlights the need for further studies. Publication History Article published online: 20 January 2025 © 2025. Thieme. All rights reserved. Georg Thieme Verlag KG Oswald-Hesse-Straße 50, 70469 Stuttgart, Germany

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