Clinical, safety, and patient-reported outcomes associated with second-line and later therapies in primary biliary cholangitis: A systematic review.
Marco Carbone, David Jones, Oyinkan Solanke et al.
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In 30 seconds
This systematic literature review examined clinical, safety, and patient-reported outcomes for second-line and later therapies in people with primary biliary cholangitis who had an inadequate response or intolerance to ursodeoxycholic acid. It included 149 publications covering 44 unique studies. Biochemical response definitions and assessment methods varied substantially; placebo-controlled trials generally favored active treatment, but placebo responses were highly variable. Non-invasive fibrosis measures appeared in 16% of studies and showed small short-term changes. The authors concluded that heterogeneity in designs, populations, and outcome measures limits comparisons across trials.
Key findings
- The review included 149 publications on 44 unique studies of second-line or later PBC therapies.
- ALP was assessed in 89% of studies and total bilirubin in 71%, but thresholds and upper limits of normal varied substantially.
- Composite biochemical response endpoints appeared in 34% of studies; POISE criteria were most frequent, reported in 18%.
- Non-invasive fibrosis measures were reported in 16% of studies and showed small short-term changes.
Why it matters
The review maps a substantial but inconsistent evidence base for later-line PBC treatment. Variation in biochemical response definitions, patient populations, and symptom scales makes it difficult to compare treatment effects across trials. Recognizing this variability is important when interpreting reported biochemical, fibrosis, pruritus, and fatigue outcomes and when placing individual study results in context.
What to keep in mind
The review identifies substantial heterogeneity in study designs, populations, assessment scales, and outcome definitions, limiting cross-trial comparisons.