Patient-Reported Indicators of Health and Symptoms in US Patients with Primary Biliary Cholangitis (PBC)

The degree to which clinician-reported symptom assessments in PBC underestimate patient-experienced symptom burden—particularly for pruritus and fatigue—had not been quantified in a real-world US cohort, limiting understanding of the PRO-clinical record gap relevant to endpoint design.

The TARGET-PBC cohort's patient-reported outcomes were compared to corresponding clinician documentation in medical records, characterizing concordance rates between PRO-reported and medically documented symptoms across pruritus and fatigue severity categories.

Demonstrating systematic underestimation of symptom severity by clinicians relative to patient-reported experience provides the justification for using validated PRO instruments rather than medical record-based symptom documentation as primary endpoints in PBC trials.

PRO vs. clinician assessment concordance evidence for PBC endpoint design, Patient-clinician symptom reporting gap quantification supporting PRO endpoint use in rare liver disease, Evidence base for patient-centered endpoint strategy in PBC drug development