(306) Incident Treatment and Adverse Liver Outcomes among Patients with Primary Sclerosing Cholangitis: An Analysis of Real-World Data

Background: Primary sclerosing cholangitis (PSC) is a cholestatic disease with no treatment to address progression, aside from liver transplant. While clinical studies have suggested the effectiveness of various pharmacological therapies, little to no consensus exists on disease management. Furthermore, due to its rare status (prevalence estimated at 1 to 16 per 100,000), data on the population are limited to small observational studies.

Objectives: To evaluate incident treatments and adverse liver outcomes among patients with a diagnosis of PSC using a large, real-world dataset.

Methods: Patients ≥18 years of age with a PSC diagnosis from 01/01/2013-06/30/2023 were selected from a U.S. dataset that includes administrative and claims data for over 170 million patients across commercial payors, Medicare Advantage and Medicaid. Continuous health plan enrollment in the year prior to (baseline) and after (follow-up) PSC diagnosis were required, as well as evidence of a diagnostic endoscopic retrograde or magnetic resonance cholangiopancreatography (ERCP/MRCP) during baseline. Patients with liver transplant prior to PSC diagnosis were excluded. The proportion of patients initiating treatments (i.e., no evidence of treatment in year prior to PSC diagnosis) and the time from PSC diagnosis to treatment initiation were evaluated during the one-year follow-up period, in addition to a composite outcome of liver failure or transplant.

Results: 2,076 patients were identified with an average age of 51.01 (±17.36) years (50.39% female). 38.68% of the sample had pre-existing inflammatory bowel disease (IBD) and 15.90% had documented liver failure during baseline. The most common treatments observed during baseline included antibiotics (58.57%), glucocorticoids (46.15%), ursodeoxycholic acid (UDCA) (27.75%) and statins (17.53%). For patients without prior exposure, these treatments were also frequently initiated during the year following PSC diagnosis, with antibiotics having the highest incidence (18.64%, median [IQR] 40 days [6-159] following PSC diagnosis), followed by glucocorticoids (16.38%, 98 [34-203] days), UDCA (12.19%, 44 [3-144] days) and statins (3.85%, 147 [83-234]). Incident liver failure or transplant occurred in 7.61% of the sample, with a median [IQR] time to event of 93 [15-208] days following PSC diagnosis.

Conclusions: A large sample of patients with PSC demonstrated limited therapeutic options, heterogeneity in prescribing patterns, and a non-trivial proportion of patients experiencing liver failure within the first year of diagnosis. Further research that explores the relationship between treatment exposure and adverse outcomes among real-world PSC patients is warranted.