Obeticholic acid safe, effective in select patients with primary biliary cholangitis

Obeticholic acid is beneficial to primary biliary cholangitis (PBC) patients with suboptimal response to ursodeoxycholic acid, significantly improving prognostic score with good tolerability, according to the results of an open-label study.

The study involved a real‐world cohort of PBC patients who did not meet Paris II criteria (defined as ≤1.5 times the upper limit of normal in alkaline phosphatase [ALP] and aspartate aminotransferase [AST] and bilirubin ≤1 mg/dL) after at least 1 year of treatment with ursodeoxycholic acid.

A total of 120 patients were eligible, with a median time since PBC diagnosis of 9.3 years (range, 4.0–13.8). Of these, 21.7 percent had cirrhosis and 26.7 percent had previous or concomitant treatment with fibrates. Seventy-eight patients completed at least 1 year of treatment with obeticholic acid.

Effectiveness of obeticholic acid was evaluated using the continuous prognostic scoring systems GLOBE and UK-PBC scores. POISE and Paris II criteria were also assessed after 12 months of treatment. Liver fibrosis was measured by FIB‐4 and AST to platelet ratio index (APRI).

Results showed that treatment with obeticholic acid led to a significantly lower GLOBE‐PBC score (0.17, 95 percent confidence interval [CI], 0.05–0.28; p=0.005) and numerically improved UK‐PBC score (0.81, 95 percent CI, −0.19 to 1.80; p=0.11).

There were parallel reductions seen in ALP (81.3 U/L, 95 percent CI, 42.5–120; p<0.001), ALT (22.1 U/L, 95 percent CI, 10.4–33.8; p<0.001), and bilirubin (0.12 mg/dL, 95 percent CI, 0–0.24; p=0.044). FIB‐4 and APRI remained stable.

According to the POISE criteria, 29.5 percent of the patients (23 out of 78) achieved response.

In terms of safety, the adverse events rate was 35 percent, with pruritus occurring most frequently (32 percent). A total of 11.67 percent patients discontinued obeticholic acid, and 8.3 percent of these cases were due to pruritus.