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.