Qualitative Evaluation of Fatigue and Pruritus Among Patients with Primary Biliary Cholangitis

Primary biliary cholangitis (PBC) is a rare cholestatic autoimmune liver disease. We aimed to generate evidence of the signs, symptoms, and impacts of PBC considered important by patients through interviews with clinicians and patients.

This noninterventional, qualitative study involved a targeted literature review (TLR), clinician interviews, and patient interviews. The TLR identified relevant signs, symptoms, and impacts of PBC, as well as their reported prevalence data. This information was developed into a preliminary conceptual model (PCM) of PBC and refined via interviews with gastroenterology/hepatology clinicians, focusing on patients' experiences. Next, PBC-related concepts were captured through patient interviews, and the PCM was finalized. A thematic approach was used to analyze data, and descriptive statistics were used to assess symptom and impact salience, ensuring a representative sample and achieving concept saturation.

Fatigue and pruritus were confirmed as the most prevalent symptoms. Clinician interviews (n = 4) provided insights into biochemical abnormalities used for diagnosis and common symptoms. Patient interviews (n = 20) revealed 41 unique PBC signs and symptoms. Patients reported mental and/or physical fatigue, with both impacting daily activities and overall health-related quality of life. They also reported that pruritus interfered with sleep and social engagements. The final conceptual model could distinguish between salient and non-salient signs, symptoms, and impacts, reflecting patients' attributions to PBC and its treatment.

PBC imposes a significant burden on patients' lives. This study highlights unmet patient needs, contributing valuable qualitative evidence to support clinical and observational studies in PBC.