Effect of deferred or no treatment with ursodeoxycholic acid in patients with early primary biliary cholangitis

Aim: As primary biliary cholangitis (PBC) is a heterogeneous disease, we hypothesized that there is a population of patients with early PBC who do not require prompt treatment with ursodeoxycholic acid (UDCA). In this study, we analyzed data from a large-scale PBC cohort in Japan, and retrospectively investigated whether outcomes of early PBC patients were affected with prompt or deferred/no UDCA treatment.

Methods: We defined early PBC as asymptomatic, serum alkaline phosphatase <1.67-fold the upper limit of normal, normal bilirubin, and histological stages I-II at presentation. We compared the outcomes of early PBC patients between the treatment regimens; prompt treatment group (UDCA was initiated within 1 year after diagnosis) and deferred/no treatment group (UDCA initiated >1 year after diagnosis or never initiated). Furthermore, we examined the outcomes of early PBC patients alternatively defined only with symptomatology and biochemistry.

Results: We identified 562 early PBC patients (prompt: n = 509; deferred/no treatment: n = 53). Incidence rates (per 1000 patient-years) for liver-related mortality or liver transplantation and decompensating events were 0.5 and 5.4, respectively, in the prompt treatment group, and 0 and 8.7, respectively, in the deferred/no treatment group. Multivariate analyses showed that age and bilirubin were significantly associated with developing decompensating events, whereas the prompt and deferred/no treatments were not. We obtained similar results in early PBC patients defined without histological examination.

Conclusions: We showed that deferred/no treatment for early PBC patients did not affect the outcomes. This study provides a rationale for a future prospective, randomized study.