LAUSR

ABSTRACT Background and Aims The European Reference Network on Hepatological Diseases (ERN RARE‐LIVER) is a Europe‐wide network for centers of excellence in the management of rare liver diseases. We aimed to evaluate the current diagnostic and therapeutic trends of primary biliary cholangitis (PBC). Methods Prospective data of PBC cases diagnosed from 2017 to March 2024 were extracted from the R‐LIVER registry of ERN‐RARE LIVER. Cases without two follow‐ups within 24 months were excluded from the treatment analysis. Biochemical response according to Toronto criteria and normalization of alkaline phosphatase (ALP) values after 12 months of Ursodeoxycholic Acid (UDCA) were evaluated. Results This study included 327 incident cases from six centers. Median age was 56 years, 89.3% were female. At the time of diagnosis, median values of ALP were 1.37 x ULN, and median bilirubin was 0.49 x ULN. Transient elastography (TE) was performed in 230 patients (70.3%) at baseline; median liver stiffness was 6.2 kPa. Out of 316 subjects, treatment with UDCA was started in 312 patients (98.7%); 246 (85.1%) achieved ALP values < 1.67 x ULN at 12 months. Normalization of ALP values occurred in 143 subjects (49.5%) at 12 months. Among 43 patients with inadequate response, 18 (41.9%) were treated with second‐line therapies, and had worse liver biochemistry at baseline. Conclusion In the current era, patients with PBC are diagnosed at an early stage using non‐invasive methods and are almost all treated with UDCA. The biochemical response rate is 85.1%, but the use of second‐line therapies for inadequate responders remains suboptimal.