2025. AASLD The Liver Meeting: SYMPTOM PROFILES AMONG ADULTS WITH PRIMARY SCLEROSING CHOLANGITIS IDENTIFIES LOW, MODERATE AND HIGH BURDENED GROUPS

Authors: Donna Evon, Kaya Merkler, Chelsea Anderson, Joanne Hatchett, Ricky Safer, Rachel Gomel, Sasha Deutsch-Link, Christopher Bowlus, Bryce Reeve

Abstract: 

Background: Patient-reported symptoms of PSC are not well documented. The study’s aim was to understand the prevalence of PSC symptoms and identify distinct symptom profiles and their association with demographic and clinical characteristics among symptomatic adults.

Methods: Adults were recruited through the PSC Partners Patient Registry and PSC hepatologists in the U.S. Individuals were eligible if they were 18 or older, symptomatic in the last year, and not waitlisted or a transplant recipient. Individuals engaged in a phone survey that screened for the presence (yes/no) of 13 PSC-associated symptoms in the last month. Sociodemographic and medical history were self-reported. Latent class analysis (LCA) was used to identify symptom-based participant profiles based on symptom presence and participants were assigned to one symptom class. Multinomial logistic regression models were used to evaluate demographic or clinical factors associated with profile membership.

Results: Participants (n = 126) were on average 47 years old (range:20-81), 60% female, 88% white, 8% black and 3% asian, 59% had inflammatory bowel disease (IBD), 39% reported cirrhosis, 75% were taking ursodiol (UCDA), and 57% had a history of cholangitis attacks. The most prevalent symptoms in the last month included fatigue (83%), daytime drowsiness (73%), and liver pain, cognitive impairment, anxiety, sleep disturbance and itch (all 62-63%). The LCA and clinical interpretation suggested a 4-symptom profile solution: (1) “Low Symptom” class (22% of sample), (2) “Moderate Symptom” class (21%), (3) “Moderate+Distressed” class (39% of sample), and a “High Symptom” class (18%). In unadjusted models, compared to the Low Symptom class, females were more likely than males to be in the High Symptom class (OR =4.36; 95% CI: 1.31-14.51), participants taking UCDA were less likely to be in the High Symptom class (OR =0.25; 95% CI: 0.08-0.80) and those with a history of cholangitis attacks were less likely to be in the Moderate Symptom class (OR = 0.22; 95% CI: 0.07-0.70). In the multvariable model, only a history of cholangitis attacks (p = 0.03) was statistically associated with class membership. A statistically significant worsening trend was observed in PROMIS® Global Physical and Mental Health T-scores from the Low to the High Symptom classes.

Conclusion: Among symptomatic adults with PSC, over 60% report fatigue, daytime drowsiness, liver pain, cognitive impairment, anxiety, sleep disturbance and itch in the past month. The four-symptom profiles ranging from low to high burden needs validation in larger cohorts as they have clinically meaningful implications for patients’ health status and care of symptomatic individuals.