Authors: Donna M. Evon, Phd et al.

Journal: Hepatology. 78(S1):S1-S2154, October 2023. https://journals.lww.com/hep/toc/2023/10001

Abstract:

Background: Patient-reported symptoms of primary sclerosing cholangitis (PSC) are not well documented. We are studying symptoms of PSC for the comprehensive development of PSC-specific patient-reported symptom measures fit for clinical trials.

Methods: Patients with PSC were recruited through the PSC Partners Patient Registry. Inclusion criteria were PSC diagnosis, age 18 or older, English-speaking, living in the US, symptomatic within the last 5 years, and not waitlisted nor a recipient of a liver transplant. Patients engaged in a phone survey to screen for the presence, frequency, severity, and distress of 13 symptoms associated with PSC, using a modified version of the Memorial Symptom Assessment Scale. Patients indicated if they had experienced each symptom in the last month (yes/no). If yes, they were queried about symptom frequency (How often did you experience fatigue?), severity (How severe was the fatigue?), and distress (How much did the fatigue distress or bother you?). Response options were based on a 4- or 5-point ordinal scale. Patients classified each symptom as definitely, partially, or unrelated to PSC. Sociodemographics and patient-reported medical history related to PSC were recorded.

Results: Participants (currently N=24 in interim sample) were 63% female, 88% white, with a mean age of 49 (SD=12.6). 87% were college educated, 58% were working and 21% were retired. 75% had irritable bowel disease, 79% had large duct PSC, 17% had autoimmune hepatitis, 33% had cirrhosis, and two reported symptoms consistent with decompensated cirrhosis. Fatigue (71%) was the most prevalent symptom in the past month (See table below). Over 50% of patients experienced difficulty sleeping, daytime drowsiness, anxiety, pruritus, brain fog, and pain over the liver. Between 85-100% of patients indicated that these symptoms were definitely or partially related to PSC.

Between 29-46% of patients experienced nausea, other belly pain, sadness, or night sweats. For symptoms that were prevalent in more than 50% of patients, 46-69% of patients rated these symptoms as frequent or always constant; 61-84% rated these symptoms as moderate to very severe, and 30-94% rated these symptoms as somewhat to very much distressing.

Conclusion: Among symptomatic adults with PSC, over 50% report issues with fatigue, sleep, anxiety, itch, brain fog, and liver pain within the last month that are frequent, moderate in severity, and distressing. Planned in-depth qualitative interviews are the critical next step towards understanding the breadth and depth of PSC symptoms to develop PSC-specific patient-reported symptom measures.