Profile of Patients with Primary Biliary Cholangitis and Evaluation of Response to Ursodeoxycholic Acid in a Romanian Center—Retrospective Study

<b>Background</b>: Primary Biliary Cholangitis (PBC) is a chronic autoimmune liver disease, characterized by cholestasis and fibrosis. Ursodeoxycholic acid (UDCA) is the first-line treatment for normalizing markers, slowing progression, and improving outcomes. Various criteria, including Paris II, POISE, and Barcelona, along with scores like GLOBE, UK-PBC, and URS, help assess response and prognosis. The aim of our study was to evaluate the patient's profile and assess treatment response to UDCA. <b>Methods:</b> We conducted a retrospective study of 276 patients diagnosed with PBC, evaluated between 2011 and 2024 at Fundeni Clinical Institute in Bucharest. Of these, 117 patients were assessed for UDCA response at 12 months. Demographic, clinical, and biochemical data were collected. Treatment response was evaluated using established criteria and prognostic scores. Logistic regression analysis was used to identify significant predictors of treatment response, while ROC curves assessed predictive capabilities for liver-related outcomes (LRO) and UDCA biochemical response. <b>Results:</b> The cohort primarily consisted of women (95.28%) with a mean age at diagnosis of 53.89 years (95% CI, 52.43-55.34). 40.5% of patients developed liver cirrhosis during follow-up. At 12 months, the response rates to UDCA therapy were 44.44%, 41.88%, and 52.14% according to the Paris II, POISE, and Barcelona criteria, respectively. Biochemical improvement was significant, particularly the reduction of alkaline phosphatase (ALP) below 1.67× the upper limit of normal, which was observed in 62.39% of patients. ROC analysis demonstrated robust predictive capabilities, with UK-PBC (AUROC 0.899) and GLOBE (AUROC 0.867) scores accurately predicting LRO. A lower age at diagnosis, higher ALP values at diagnosis, and absence of sp100 were factors associated with non-response. <b>Conclusions</b>: This study is one of the first detailed analyses of PBC patients in Romania, examining population-specific characteristics and UDCA response rates. Low response rates at 12 months highlight the need for longer follow-up and the exploration of second-line therapies.