SUB-OPTIMAL BIOCHEMICAL AND HEALTH RELATED QUALITY OF LIFE OUTCOMES WITH OFF-LABEL BEZAFIBRATE USE IN PATIENTS WITH PBC: ANALYSIS OF A REAL-WORLD DATASET
Author(s)
Ira M. Jacobson, MD1, Robert G. Gish, MD2, Gideon M. Hirschfield, FRCP, PhD3, Marvin Rock, MPH, DrPH4, Chong H Kim, MPH, MS, PhD4, Caroline Burk, PharmD, MS4, Hayley Wallinger, BSc5, William Johnson, BSc5, Lena Amari, BSc5, David E. J. Jones, Prof6, Robert J. Wong, MD, MS, FACG, FAASLD7.
1NYU Langone Health, New York, NY, USA, 2Hepatitis B Foundation, Doylestown, PA, USA, 3Autoimmune Liver Disease Program at UHN's Francis Family Liver Clinic and Toronto Centre for Liver Disease, Toronto, ON, Canada, 4Gilead Sciences Inc., Foster City, CA, USA, 5Adelphi Real World, Bollington, United Kingdom, 6Institute of Cellular Medicine and NIHR Newcastle Biomedical Research Centre, Newcastle University, Newcastle Upon Tyne, United Kingdom, 7Division of Gastroenterology and Hepatology, Stanford University School of Medicine, Palo Alto, CA, USA.
1NYU Langone Health, New York, NY, USA, 2Hepatitis B Foundation, Doylestown, PA, USA, 3Autoimmune Liver Disease Program at UHN's Francis Family Liver Clinic and Toronto Centre for Liver Disease, Toronto, ON, Canada, 4Gilead Sciences Inc., Foster City, CA, USA, 5Adelphi Real World, Bollington, United Kingdom, 6Institute of Cellular Medicine and NIHR Newcastle Biomedical Research Centre, Newcastle University, Newcastle Upon Tyne, United Kingdom, 7Division of Gastroenterology and Hepatology, Stanford University School of Medicine, Palo Alto, CA, USA.
OBJECTIVES: To evaluate level of biochemical non-response and unmet need among patients receiving bezafibrate as off-label second line treatment by describing alkaline phosphatase (ALP), total bilirubin (TB), symptomology, and patient reported outcomes (PROs).
METHODS: Data were drawn from the Adelphi Real World PBC Disease Specific Programme™, a cross-sectional survey, with retrospective data collection in France, Germany, Italy, Spain, United Kingdom (November 2024-May 2026). Physicians reported data for their next 4 consulting patients with PBC, followed by 2 patients with PBC that currently experience itch. Patients voluntarily completed questionnaires including the PBC-40, Work Productivity and Activity Index (WPAI) and 5-D Itch. Analyses included patients currently receiving bezafibrate for ≥3 months and not receiving seladelpar or elafibranor. Analyses were descriptive.
RESULTS: Data for 105 patients were available (mean [SD] age of 55.4 [9.5] years, 89% female). Treatment duration was 3.2 [3.9] years with 95% receiving UDCA. 35 patients were also receiving itch treatment, most commonly cholestyramine (46%). Frequent current symptoms were itch (84%), fatigue (69%) and sleep disturbance due to itch (29%). For those that completed 5-D itch, 59% reported having moderate/severe itch (n=34) and 56% reported it being unchanged/getting worse (n=32). For those that completed PBC-40, 37% reported their itch affecting their sleep most/all the time (n=30). For those that completed WPAI, activity impairment was 39.7% [26.3] (n=32) and overall work impairment was 36.8% [28.6] (n=15). Mean [SD] ALP was 185.0 [92.4] U/L (n=98) and only 23% of patients achieved ALP normalisation (<1xULN threshold). Similarly, TB levels were 2.4 [4.3] mg/dL (n=95) and 21% of patients achieved normalisation (<0.6xULN threshold).
CONCLUSIONS: Patients taking off-label bezafibrate presented with complex symptom manifestations and had a low rate of biomarker normalisation. This data highlights the need for alternative 2L treatments to improve biochemical response in addition to providing adequate relief of symptoms.
METHODS: Data were drawn from the Adelphi Real World PBC Disease Specific Programme™, a cross-sectional survey, with retrospective data collection in France, Germany, Italy, Spain, United Kingdom (November 2024-May 2026). Physicians reported data for their next 4 consulting patients with PBC, followed by 2 patients with PBC that currently experience itch. Patients voluntarily completed questionnaires including the PBC-40, Work Productivity and Activity Index (WPAI) and 5-D Itch. Analyses included patients currently receiving bezafibrate for ≥3 months and not receiving seladelpar or elafibranor. Analyses were descriptive.
RESULTS: Data for 105 patients were available (mean [SD] age of 55.4 [9.5] years, 89% female). Treatment duration was 3.2 [3.9] years with 95% receiving UDCA. 35 patients were also receiving itch treatment, most commonly cholestyramine (46%). Frequent current symptoms were itch (84%), fatigue (69%) and sleep disturbance due to itch (29%). For those that completed 5-D itch, 59% reported having moderate/severe itch (n=34) and 56% reported it being unchanged/getting worse (n=32). For those that completed PBC-40, 37% reported their itch affecting their sleep most/all the time (n=30). For those that completed WPAI, activity impairment was 39.7% [26.3] (n=32) and overall work impairment was 36.8% [28.6] (n=15). Mean [SD] ALP was 185.0 [92.4] U/L (n=98) and only 23% of patients achieved ALP normalisation (<1xULN threshold). Similarly, TB levels were 2.4 [4.3] mg/dL (n=95) and 21% of patients achieved normalisation (<0.6xULN threshold).
CONCLUSIONS: Patients taking off-label bezafibrate presented with complex symptom manifestations and had a low rate of biomarker normalisation. This data highlights the need for alternative 2L treatments to improve biochemical response in addition to providing adequate relief of symptoms.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
RWD140
Topic
Patient-Centered Research, Real World Data & Information Systems
Disease
Gastrointestinal Disorders, No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases