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Early PBC outcomes unaffected by deferred, no UDCA treatment

Deferred ursodeoxycholic acid treatment or no treatment for patients with early primary biliary cholangitis did not affect overall mortality or liver-related outcomes, according to a recently published study.

“These findings indicate that early PBC patients may not require prompt treatment intervention with UDCA, and may benefit from a ‘watch and wait’ approach to treatment,” Atsushi Tanaka, MD, from the Teikyo University School of Medicine, and colleagues wrote. “Although this study has several limitations, largely due to its retrospective design, it provides a rationale for a well-designed, prospective, randomized future study to evaluate the possibility of deferred or no UDCA treatment in early PBC patients.”

The study comprised 562 patients with early PBC, which Tanaka and colleagues defined as free of decompensation symptoms, serum alkaline phosphatase less than 1.67 times the upper limit of normal, normal bilirubin, and histological Scheuer Classification I or II.

The researchers divided patients into a prompt UDCA treatment group (n = 509) and into a combined group that received deferred (n = 22) or no treatment (n = 31). During the observation period, three patients from the prompt treatment group and one patient from the deferred or no treatment group died or underwent liver transplantation for an overall incidence rate of 1.5 cases per 1,000 patient-years.

Incidence rates for overall mortality or liver transplantation were 1.3 per 1,000 patient-years for the prompt group and 2.9 per 1,000 patient-years for the deferred or no treatment group. Liver-related mortality or liver transplantation incidence was 0.5 per 1,000 patient-years for the prompt group and 0 in the deferred or no treatment group.

Fifteen patients experienced decompensating events (IR = 5.8 per 1,000 patient-years), including 12 in the prompt treatment group (IR = 5.4 per 1,000 patient-years) and 3 in the deferred or no treatment group (IR = 8.7 per 1,000 patient-years).

Results from both univariate and multivariate analysis showed that age over 57 years (HR = 3.09; 95% CI, 1.01-9.49) and bilirubin over 1 mg/dL (HR = 8.64; 95% CI, 2.56-29.2) at presentation correlated independently with the development of decompensating events, whereas treatment protocol did not correlate with outcomes. – by Talitha Bennett

Disclosure: The authors report no relevant financial disclosures.