Quality of life outcomes in people with Primary Biliary Cholangitis treated with obeticholic acid: results of a phase 4 real life observational trial

• Published in: Digestive and liver disease 2023-03, Vol.55, p.S55-S55
• Main Authors: Rollo, P., D'Ovidio, E., Bonaiuto, E., Perini, L., Pezzato, F., Peviani, M., Simionato, F., Russo, F.P., Floreani, A., Cazzagon, N.
• Format: Article
• Language: English
• ISSN: 1590-8658; 1878-3562
• DOI: 10.1016/j.dld.2023.01.107

Primary biliary cholangitis (PBC) can be associated with impaired health-related quality of life (HRQoL) caused by symptoms, mainly pruritus and fatigue, and eventually depression.Treatment confidence strongly affects HRQoL in PBC, and UDCA has been reported to be a positive factor in enhancing trust therapy. No real-life data on this and HRQoL exist regarding obeticholic acid (OCA) second-line therapy. To assess the impact of OCA on biochemistry, patients’ illness perception and HRQoL of PBC patients We designed a phase 4 observational open label study (Protocol AOP1515) and collected at baseline and every 6 months from starting OCA biochemistry, adverse events, reported symptoms and the results of following questionnaires: PBC-40, Fatigue Impact Scale(FIS), 5-D Itch scale, EuroQoL-5D-5L. Changes in symptoms after OCA introduction were assessed by Wilcoxon paired rank test. Nineteen (60%) over 32 patients who started OCA treatment between March 2018 and April 2022 for insufficient response to UDCA agreed to participate. Median duration of OCA treatment was 32(15-53) months with no drug discontinuation due to adverse events. A decrease of alkaline phosphatase compared to baseline below 1.5xULN and 1xULN was observed in 47%,67%,64%,67% and 16%,21%,35%,17% patients at 6,12,18,24 months, respectively. At the time of starting OCA, significant pruritus, fatigue, cognitive and social dysfunction, emotional impairment, and general symptoms were present in 5%,32%,11%,53%,37%, 21% patients, respectively. No significant aggravation of itch or other symptoms was observed after OCA introduction, except for a transient aggravation in emotional domain at 12 months (8[5-10]vs.14[9-19], p=0.005). A trend over a significant reduction of fatigue evaluated by FIS at 36 months was observed (45[0-82]vs.10.5[0-42],p=0.06). All patients reported a significant and persistent general improvement after OCA introduction. People with PBC and insufficient response to UDCA experienced a biochemical and subjective improvement after OCA introduction and no aggravation of objectively assessed HRQoL.