PATIENT-REPORTED SYMPTOM BURDEN, QUALITY OF LIFE AND PRODUCTIVITY IMPACT OF PBC ACROSS FRANCE, ITALY, GERMANY, SPAIN, UNITED KINGDOM, AND THE UNITED STATES OF AMERICA: ANALYSIS OF A REAL-WORLD DATASET
Author(s)
William Johnson, BSc1, Hayley Wallinger, BA1, Emily Green, BSc1, Marvin Rock, MPH, DrPH2, Caroline Burk, Pharm.D., MS2, Chong H Kim, MPH, MS, PhD2, Robert G. Gish, MD3, David E. Jones, MD4, Gideon M. Hirschfield, MD5, Ira Jacobson, MD6;
1Adelphi Real World, Bollington, United Kingdom, 2Gilead Sciences Inc., Foster City, CA, USA, 3Robert G. Gish Consultants, LLC, LA JOLLA, CA, USA, 4Newcastle University, Newcastle, United Kingdom, 5The Autoimmune and Rare Liver Disease Programme, Toronto General Hospital, Toronto, Canada, Toronto, ON, Canada, 6NYU Grossman School of Medicine, New York, NY, USA
1Adelphi Real World, Bollington, United Kingdom, 2Gilead Sciences Inc., Foster City, CA, USA, 3Robert G. Gish Consultants, LLC, LA JOLLA, CA, USA, 4Newcastle University, Newcastle, United Kingdom, 5The Autoimmune and Rare Liver Disease Programme, Toronto General Hospital, Toronto, Canada, Toronto, ON, Canada, 6NYU Grossman School of Medicine, New York, NY, USA
OBJECTIVES: To describe symptom burden, health related quality of life (HRQoL) and work productivity as reported by patients living with PBC in a real-world setting.
METHODS: Data were drawn from the Adelphi Real World PBC Disease Specific Programme™, a cross-sectional survey of physicians and their patients with PBC across EUR (France, Italy, Spain, Germany, the United Kingdom (UK)) and the United States (US), from November 2024 to May 2025. Patients completed a voluntary questionnaire including questions assessing symptom severity, HRQoL and productivity. HRQoL was measured using 5D-Itch and the Work Productivity and Activity Impairment (WPAI) questionnaire. Analyses were descriptive.
RESULTS: Data from 116 patients with PBC who reported experiencing itching at data collection were included. Of those, 92 (79%) were also experiencing fatigue. 46 patients (41%) reported their itch being moderate/severe and 41 of those (89%) also experienced moderate/severe fatigue. For those that completed 5D-Itch, 48% of patients experienced itch for 6+ hours a day, 28% reported 6+ body areas affected, 20% reported activities of daily living being frequently/always affected and 36% of patients stated itching was unchanged/getting worse compared to the previous month. For those that completed WPAI, mean ±standard deviation overall work impairment was 39.4% ±27.81 for those currently experiencing itching and 37.3%±25.10 for those currently experiencing itching and fatigue. Activity impairment was 38.7%±24.84 and 39.8%±24.13 respectively. Most common treatment goals for patients with itch and fatigue were to help itching (87%), help tiredness (80%) and improve liver tests (60%).
CONCLUSIONS: Itch and combination of itch and fatigue are frequently reported symptoms in patients with PBC with many experiencing negative impacts to HRQoL. The impact of symptoms reported by patients and associated treatment goals demonstrate the need for increased focus on management of symptoms.
METHODS: Data were drawn from the Adelphi Real World PBC Disease Specific Programme™, a cross-sectional survey of physicians and their patients with PBC across EUR (France, Italy, Spain, Germany, the United Kingdom (UK)) and the United States (US), from November 2024 to May 2025. Patients completed a voluntary questionnaire including questions assessing symptom severity, HRQoL and productivity. HRQoL was measured using 5D-Itch and the Work Productivity and Activity Impairment (WPAI) questionnaire. Analyses were descriptive.
RESULTS: Data from 116 patients with PBC who reported experiencing itching at data collection were included. Of those, 92 (79%) were also experiencing fatigue. 46 patients (41%) reported their itch being moderate/severe and 41 of those (89%) also experienced moderate/severe fatigue. For those that completed 5D-Itch, 48% of patients experienced itch for 6+ hours a day, 28% reported 6+ body areas affected, 20% reported activities of daily living being frequently/always affected and 36% of patients stated itching was unchanged/getting worse compared to the previous month. For those that completed WPAI, mean ±standard deviation overall work impairment was 39.4% ±27.81 for those currently experiencing itching and 37.3%±25.10 for those currently experiencing itching and fatigue. Activity impairment was 38.7%±24.84 and 39.8%±24.13 respectively. Most common treatment goals for patients with itch and fatigue were to help itching (87%), help tiredness (80%) and improve liver tests (60%).
CONCLUSIONS: Itch and combination of itch and fatigue are frequently reported symptoms in patients with PBC with many experiencing negative impacts to HRQoL. The impact of symptoms reported by patients and associated treatment goals demonstrate the need for increased focus on management of symptoms.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
RWD44
Topic
Real World Data & Information Systems
Disease
SDC: Gastrointestinal Disorders, SDC: Rare & Orphan Diseases