Herta Toni, Berg Thomas
Medizinische Klinik mit Schwerpunkt Hepatologie und Gastroenterologie, Charité - Universitätsmedizin Berlin, Campus Virchow-Klinikum und Campus Charité Mitte, Augustenburger Platz 1, 13353, Berlin, Deutschland.
Berlin Institute of Health an der Charité - Universitätsmedizin Berlin, BIH Biomedical Innovation Academy, BIH Charité Clinician Scientist Programm, Berlin, Deutschland.
Inn Med (Heidelb). 2025 Sep 4. doi: 10.1007/s00108-025-01988-0.
Primary biliary cholangitis (PBC) is a chronic inflammatory, autoimmune-mediated liver disease that progresses to fibrosis and cirrhosis if left untreated. In addition to preventing complications, the management of burdensome symptoms, particularly pruritus, represents a key therapeutic goal. Ursodeoxycholic acid (UDCA) is the established first-line treatment; however, up to 40% of patients show an inadequate response and require second-line treatment. Bezafibrate, a pan-peroxisome proliferator-activated receptor (PPAR) agonist, has long been used off-label as an effective option to improve parameters of cholestasis and relieve pruritus. More recently, two new PPAR agonists, elafibranor (PPAR alpha/delta) and seladelpar (PPAR delta), have received conditional approval in Germany. Both demonstrated strong biochemical efficacy and a favorable safety profile in clinical trials. This article provides a comprehensive update on the diagnostics, risk stratification and treatment of PBC, with a particular focus on the role of novel PPAR agonists in second-line treatment. Practical strategies for the treatment of pruritus are also discussed. The aim is to support guideline-based care and to improve the treatment of patients with PBC through timely treatment adjustment and individualized treatment concepts.
原发性胆汁性胆管炎（PBC）是一种慢性炎症性、自身免疫介导的肝脏疾病，如果不治疗会进展为纤维化和肝硬化。除了预防并发症外，管理令人困扰的症状，尤其是瘙痒，是一个关键的治疗目标。熊去氧胆酸（UDCA）是既定的一线治疗药物；然而，高达40%的患者反应不佳，需要二线治疗。苯扎贝特是一种泛过氧化物酶体增殖物激活受体（PPAR）激动剂，长期以来一直作为改善胆汁淤积参数和缓解瘙痒的有效非标签用药。最近，两种新的PPAR激动剂，依拉非诺（PPARα/δ）和塞拉德尔帕（PPARδ），已在德国获得有条件批准。两者在临床试验中均显示出强大的生化疗效和良好的安全性。本文全面更新了PBC的诊断、风险分层和治疗，特别关注新型PPAR激动剂在二线治疗中的作用。还讨论了治疗瘙痒的实用策略。目的是支持基于指南的治疗，并通过及时调整治疗和个性化治疗方案来改善PBC患者的治疗效果。
