Treatment Persistence, Normal Alkaline Phosphatase and Clinical Outcomes in Primary Biliary Cholangitis

Real-world evidence on treatment persistence and its association with alkaline phosphatase (ALP) and clinical outcomes in primary biliary cholangitis (PBC) is limited.

We conducted a retrospective study using Komodo's US claims and laboratory data (09/2018-09/2023) of adults with PBC treated with ursodeoxycholic acid (UDCA), obeticholic acid (OCA), or concurrent UDCA/OCA. Persistence was defined as continuous treatment with ≤ 60-day gaps. Cox models evaluated predictors of discontinuation, logistic models examined associations between discontinuation and normal ALP, and Cox models assessed normal ALP and clinical outcomes.

Among 20,139 individuals starting UDCA (n = 17,006), OCA (n = 1,709), or concurrent UDCA/OCA (n = 1,424), one-year persistence was 50%, 51%, and 49%, respectively. Significant predictors of discontinuation included pruritus (hazard ratio [HR] = 1.09), fatigue (HR = 1.09), abdominal pain (HR = 1.08), African American race (HR = 1.36), Hispanic ethnicity (HR = 1.11), Medicaid coverage (HR = 1.13), baseline cirrhosis (HR = 1.08), portal hypertension (HR = 1.11), systemic lupus erythematosus (HR = 1.13) (all p < 0.01), and urinary infection (HR = 1.06, p = 0.017). Of 626 patients with baseline ALP levels ≥ 1.67 x upper limit of normal, 33%, 17%, and 11% in the UDCA, OCA, and concurrent UDCA/OCA cohorts, respectively, had normal ALP within 6-24 months (p = 0.005 and p < 0.001 for OCA and concurrent vs. the UDCA cohort, respectively). UDCA discontinuation was associated with lower odds of having normal ALP. Normal ALP was associated with a reduced risk for mortality and clinical outcomes.

Treatment persistence is crucial in PBC as it is associated with normal ALP and significant improvement in clinical outcomes. Novel strategies and therapies are needed to enhance persistence and improve clinical benefits among individuals with PBC.