Impact of Pruritus on Quality of Life and Current Treatment Patterns in Patients with Primary Biliary Cholangitis.

Challenge

Despite pruritus being the most burdensome symptom in PBC, real-world data quantifying its impact on health-related quality of life across multiple domains—and characterizing the treatment gap—were absent, limiting the ability of sponsors and regulators to establish the clinical meaningfulness of anti-pruritic endpoints.

Solution

The TARGET-PBC longitudinal cohort was used to analyze PBC-40, 5D-Itch, and PROMIS Fatigue survey data across 211 patients stratified by itch severity, characterizing multi-domain quality of life impairment and describing current treatment patterns in real-world US practice.

Impact

Establishing that clinically significant pruritus in PBC is associated with pervasive multi-domain quality of life impairment and is systematically undertreated provides the clinical meaningfulness anchor for anti-pruritic endpoints and directly supports regulatory submissions for therapies targeting PBC itch.