PATIENT-REPORTED BURDEN IN PRIMARY BILIARY CHOLANGITIS PATIENTS ELIGIBLE BUT NOT ON SECOND-LINE TREATMENT: A MULTINATIONAL REAL-WORLD STUDY
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 describe patient reported outcomes in patients that are eligible for but not currently on second line (2L) treatment to explore the opportunity for transition to 2L treatment.
METHODS: Data were drawn from the Adelphi Real World PBC Disease Specific Programme™, a cross-sectional survey, with retrospective data collection in Europe and the United States (November 2024-May 2026). Patients completed a voluntary questionnaire assessing current symptom severity and health-related quality of life (HRQoL). HRQoL was measured using the 5-D Itch scale, Work Productivity and Activity Impairment (WPAI) questionnaire and itch numerical rating scale (NRS). Patients were considered eligible for 2L treatment if currently receiving ursodeoxycholic acid (UDCA) monotherapy for ≥12 months, had a most recent ALP result of >1x Upper Limit of Normal and had not received 2L treatment previously. Patients with decompensated cirrhosis were excluded. Analyses were descriptive.
RESULTS: Data for 60 patients were available (93% female, mean [SD] age of 52.4 [12.0], mean [SD] years diagnosed with PBC 4.5 [5.1] and years on UDCA 4.1 [5.0]). Fatigue (72%) and itch (60%) were the most common reported current symptoms and 46% of patients experienced both. Patients that experienced itch had an itch NRS of 4.0 [2.2] and 41% explicitly reported their itch as moderate/severe. Overall work impairment was 18.1% [25.5] and activity impairment was 31.0% [27.0] via WPAI. For those that completed 5D-Itch, 76% of patients reported their itch not being resolved from treatment, 60% of patients reported their itch affecting more than 3 body parts and 50% of patients reported itch affecting their sleep.
CONCLUSIONS: Many patients eligible for 2L treatment but not on 2L continue to remain on UDCA monotherapy despite displaying poor quality of life outcomes and persistent symptoms. This data highlights the opportunity for transition to appropriate 2L treatments that can improve patient outcomes.
METHODS: Data were drawn from the Adelphi Real World PBC Disease Specific Programme™, a cross-sectional survey, with retrospective data collection in Europe and the United States (November 2024-May 2026). Patients completed a voluntary questionnaire assessing current symptom severity and health-related quality of life (HRQoL). HRQoL was measured using the 5-D Itch scale, Work Productivity and Activity Impairment (WPAI) questionnaire and itch numerical rating scale (NRS). Patients were considered eligible for 2L treatment if currently receiving ursodeoxycholic acid (UDCA) monotherapy for ≥12 months, had a most recent ALP result of >1x Upper Limit of Normal and had not received 2L treatment previously. Patients with decompensated cirrhosis were excluded. Analyses were descriptive.
RESULTS: Data for 60 patients were available (93% female, mean [SD] age of 52.4 [12.0], mean [SD] years diagnosed with PBC 4.5 [5.1] and years on UDCA 4.1 [5.0]). Fatigue (72%) and itch (60%) were the most common reported current symptoms and 46% of patients experienced both. Patients that experienced itch had an itch NRS of 4.0 [2.2] and 41% explicitly reported their itch as moderate/severe. Overall work impairment was 18.1% [25.5] and activity impairment was 31.0% [27.0] via WPAI. For those that completed 5D-Itch, 76% of patients reported their itch not being resolved from treatment, 60% of patients reported their itch affecting more than 3 body parts and 50% of patients reported itch affecting their sleep.
CONCLUSIONS: Many patients eligible for 2L treatment but not on 2L continue to remain on UDCA monotherapy despite displaying poor quality of life outcomes and persistent symptoms. This data highlights the opportunity for transition to appropriate 2L treatments that can improve patient outcomes.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
RWD63
Topic
Patient-Centered Research, Real World Data & Information Systems
Disease
Gastrointestinal Disorders, No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases