2024. AASLD Liver Meeting Abstract and Poster: 4346: DEFINING ACUTE CHOLANGITIS AS A CLINICAL OUTCOMES ENDPOINT IN ADULTS WITH PRIMARY SCLEROSING CHOLANGITIS: RESULTS OF A MULTINATIONAL PATIENT SURVEY TO DEVELOP A PATIENT-REPORTED OUTCOMES MEASURE.

Authors: Stephen Rossi, Martine Walmsley, Brian Thorsen, Joanne Hatchett, Donna Evon, Bryce Reeve, Rachel Gomel, Mary Vyas, Ricky Safer, Kerrie Goldsmith, Alastair Garfield, Pamela Vig, Veronica Miller, Michael Trauner

Abstract:

Background: Acute cholangitis (AC) is a clinically significant, poorly understood PSC complication challenged by the lack of standardized diagnostic and treatment criteria. We report the results of a multi-national patient survey to characterize the patient experience during AC as a first phase to develop a patient-centered PROM for use in clinical trials and patient care.

Methods: A detailed anonymized patient survey was developed by PSC Partners and PSC Support patient organizations. Adults diagnosed with PSC and ≥1 cholangitis attack were invited to complete the survey. Relevant medical history, AC symptom frequency and severity (before, during and after AC), and medical interventions were collected. A visual abdominal pain locator was used to further characterize AC pain location.

Results: A total of 355 of 428 responses met screening criteria. Timing of first AC attack relative to PSC diagnosis ranged from before (38%), concurrent (14%) and after (48%). Over 65% had AC in the past year, with 45% having ≥2 attacks. Fatigue (92%) and liver pain (86%) were reported most frequently, with fatigue (30%) and fever/chills (24%) having highest severity during AC (Fig 1A). Severity improved for all symptoms after AC, except anxiety. Symptom improvement was greatest with fever/chills (55%), anorexia (51%) and liver pain (51%). Complete symptom resolution was highest for vomiting (50%), fever/chills (46%), night sweats (31%) and nausea (29%), while lowest with fatigue (6%). More patients reported no change in symptom severity after AC, with the least change seen with jaundice, brain fog and pruritus. Anxiety worsened the most after AC at 12% while fatigue was the most reported new-onset symptom at 33%. Abdominal pain was largely located in the right upper quadrant (RUQ) during AC, independent of IBD, cirrhosis or transplant (Fig 1B). Other reported pain locations >10% were body (73%), back (17%) and shoulder (10%). The majority of IBD patients (70%) clearly differentiated AC symptoms from IBD flares. Medical care was sought by 76%, of which 86% took oral and/or IV antibiotics. Other procedures included MRI (68%), ultrasound (57%), ERCP (44%) and stent/balloon dilation (26%). Despite AC treatment and/or resolution, 32% did not return to their perceived baseline health status, reporting persistent or new onset fatigue (44%), RUQ pain (15%) and brain fog (11%).

Conclusion: This detailed survey demonstrates a broad dynamic range of the frequency and severity of symptoms over the clinical course of AC. Symptom improvement is seen in a subset of patients, but the majority show no improvement or worsening of symptoms despite treatment or attack resolution. Fatigue and anxiety are under-recognized symptoms reported with AC and warrant further study. No single symptom provides adequate specificity or sensitivity for AC diagnosis or monitoring, clearly supporting the significant need to develop a multi-symptom PROM for clinical trials and patient care.