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. Primary biliary cholangitis (PBC) is a rare disease where the immune system attacks and damages small bile ducts in the liver. PBC can get worse with time and can be potentially life-threatening. The main symptoms are tiredness and itchy skin (pruritus). We examined how people with PBC experience their condition, to better understand how it can be managed in the most useful way for patients. First, we used published studies to gather information on PBC, such as symptoms and ways in which PBC affects patients’ lives (impacts), allowing us to develop a model of PBC. Then, we interviewed medical professionals and patients to understand which symptoms and life impacts matter most to those living with PBC. The medical professionals discussed methods for diagnosing PBC. They reported that patients’ tiredness became worse as the day progressed. Medical professionals said that patients mostly had itchy skin on the soles of the feet or palms and found it to be very bothersome, greatly affecting patients’ daily lives and mental health. When interviewed, patients reported 41 different signs and symptoms, most commonly tiredness and itchy skin. Patients said they experienced mental and physical tiredness, making everyday tasks difficult, and they described how the itching disturbed their sleep and social lives. This study showed how much PBC affects patients’ quality of life. Our model of the most important symptoms and impacts for patients can guide research and improve care by helping us focus on what patients find most difficult about PBC.