A Nationwide Assessment of Anticholestatic Therapy Uptake in Patients With Primary Biliary Cholangitis: Opportunities for Optimisation

BACKGROUND: Optimal care delivery for primary biliary cholangitis (PBC) has become challenging due to new therapies and more individualized biochemical treatment goals. AIM: To evaluate uptake of ursodeoxycholic acid (UDCA) and second-line therapy (SLT) in a nationwide PBC cohort and identify factors associated with suboptimal therapeutical management. METHODS: The Dutch PBC Cohort Study is a retrospective study including every identifiable patient with PBC in the Netherlands from 1990 onwards. Use of UDCA and SLT was assessed among patients in clinical follow-up on 1 January 2019. Biochemical response was defined as alkaline phosphatase (ALP) and aspartate aminotransferase (AST) ≤ 1.5 × ULN and total bilirubin (TB) ≤ ULN. SLT included off-label fibrates, budesonide and/or obeticholic acid (not reimbursed). RESULTS: In total, 2638 patients with PBC were included; median age was 64.0 (IQR 55.0-72.0) years, 2361 (89.5%) were female and 2143 (81.2%) were treated in a non-academic center. Overall, 2532 (96.0%) patients used UDCA. The recommended UDCA dose of ≥ 13 mg/kg/day was used by 1363/2205 (61.8%). Age (aOR 1.01, 95% CI 1.00-1.02, p = 0.02), body weight (aOR 1.04, 95% CI 1.03-1.05, p < 0.01) and biochemical response (aOR 1.35, 95% CI 1.09-1.67, p = 0.01) were associated with UDCA dosage < 13 mg/kg/day. Among patients with an incomplete response, 101/638 (15.8%) were prescribed SLT. Younger age, female sex, treatment in an academic centre and higher levels of ALP and AST were positively associated with use of SLT. CONCLUSIONS: During recent years, UDCA was insufficiently dosed in Dutch patients with PBC. The results indicate that first-line treatment can be optimized, especially in those with a biochemical response and higher body weight.