Background & Aims Noninvasive surrogate end points of long-term outcomes of patients
with primary biliary cirrhosis (PBC) are needed to monitor disease progression and evaluate
potential treatments. We performed a meta-analysis of individual patient data from cohort
studies to evaluate whether patients' levels of alkaline phosphatase and bilirubin correlate
with their outcomes and can be used as surrogate end points. Methods We performed a
meta-analysis of data from 4845 patients included in 15 North American and European long …

Primary biliary cirrhosis (PBC) is a rare, chronic and slowly progressive autoimmune
hepatobiliary disease. PBC typically progresses to cirrhosis, which may lead to premature
death from liver failure and its complications. 1 Presently the vast majority of patients are
treated with ursodeoxycholic acid (UDCA), the only approved therapy for PBC, in keeping
with treatment guidelines. 2, 3 While UDCA therapy has a marked impact on clinical
outcomes in PBC, up to 40% of patients have an insufficient response to UDCA and …