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Lookup NU author(s): Professor David Jones
This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License (CC BY-NC-ND).
© 2021 The Authors. Liver International published by John Wiley & Sons Ltd. Background & Aims: Primary biliary cholangitis (PBC) can result in life-altering cholestatic pruritus and fatigue, but treatment options are limited. Seladelpar, a peroxisome proliferator-activated receptor-delta (PPARδ) agonist, has demonstrated potent anti-cholestatic effects in clinical studies. This open-label, uncontrolled phase 2 study in PBC patients evaluated the effects of 1-year of seladelpar treatment on measures of pruritus and quality of life. Methods: Self-reported experiences of 101 PBC patients were collected at baseline and after 1 year of seladelpar treatment using the pruritus visual analog scale (VAS), 5D-itch scale, and PBC-40 questionnaires along with bile acid profiles. Results: In patients with moderate–to-severe pruritus, substantial improvement in pruritus was seen in 58% and 93% of patients in 5/10 mg and 10 mg treatment groups, respectively. After 1 year, patients reporting improvement substantially outnumbered those who worsened in the total 5-D itch (including individual domains) and PBC-40 (itch and fatigue domains) questionnaires. Improvement in sleep disturbance at 1-year was reported in 81% (5/10 mg) and 78% (10 mg) of the patients with baseline itch-related sleep disturbance by 5-D itch score with similar results using the PBC-40 sleep questionnaire. Seladelpar-treated patients had significant reductions of 46% (5/10 mg) and 31% (10 mg) in the serum bile acid precursor C4 and reductions of up to 38% in serum bile acids. Conclusions: Seladelpar treatment for 1 year led to consistent improvement in both symptom burden and biochemical response, suggesting its potential as a single agent to address two key unmet needs in PBC patients.
Author(s): Kremer AE, Mayo MJ, Hirschfield G, Levy C, Bowlus CL, Jones DE, Steinberg A, McWherter CA, Choi Y-J
Publication type: Article
Publication status: Published
Journal: Liver International
Year: 2022
Volume: 42
Issue: 1
Pages: 112-123
Print publication date: 01/01/2022
Online publication date: 17/08/2021
Acceptance date: 15/08/2021
Date deposited: 18/09/2024
ISSN (print): 1478-3223
ISSN (electronic): 1478-3231
Publisher: John Wiley and Sons Inc.
URL: https://doi.org/10.1111/liv.15039
DOI: 10.1111/liv.15039
PubMed id: 34403559
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