Results from a retrospective, observational, multicentre study of patients with primary biliary cholangitis treated with obeticholic acid in real life in Italy (O-REAL)

Sixty-seven patients enrolled in the O-REAL study who matched the inclusion criteria were analyzed. At baseline, 91% of the patients were female and 12% had a concomitant liver disease. Mean age at start of OCA therapy was 57.64 (±10.87) years with mean time from diagnosis to start of OCA therapy of 8 years. At OCA start, rates of pruritus and fatigue were 24% (n=16) and 18% (n=12), respectively. Eleven patients 16.4%) had cirrhosis confirmed by biopsy. OCA treatment resulted in a mean ALP reduction from 301.01 (±149.16) U/L; to 211.67 (± 97.08) after 6 months of therapy, with a mean percentage change of -21.72% (±34.60). The proportion of patients with ALP ≤1.5xULN after 6 and 12 months of OCA treatment were, 42.2% and 51.7% respectively. After 12 months of OCA, 81.6% of patients with baseline ALP ≥1.67xULN had a reduction >15%. Additionally, ALP, GGT, alanine transaminase (ALT) and aspartate transaminase (AST) levels after 12 months of OCA therapy were significantly reduced compared to baseline (Table). However, changes in total bilirubin and albumin were not significant. Transient elastography was available and paired for 16 patients at baseline and month 12, with a mean at month 12 of -18.39% kPa (95% CI - 45.46-82.24). Among the 27 patients for which GLOBE score was calculated, 19 patients (70.37%) showed a score below the threshold of 0.3 and 8 above it after OCA treatment. Pruritus (n=31) and fatigue (n=3) were the most frequent adverse events. Nine patients developed pruritus after OCA therapy and 4 patients discontinued treatment, 3 due to adverse event (2 pruritus, 1 abdominal distention). No serious adverse events were reported.