OBJECTIVES: Primary Sclerosing Cholangitis (PSC) is a rare cholestatic liver disease characterized by inflammation and scarring (fibrosis) of the intra- and extrahepatic bile ducts. This study investigates prevalence, incidence, treatment as well as burden and costs of PSC in Germany.

METHODS: This retrospective cohort study utilized German claims data from the Statutory Health Insurance database, specifically the 'German Analysis Database'. We focused on patients diagnosed with PSC (ICD-10-GM K83.00; available since 2022) in the year 2022, assessing both prevalence and incidence rates. The study identified the usage of procedures such as endoscopic retrograde cholangiopancreatography (ERCP) and medication (i.a. ursodeoxycholic acid (UDCA)). Additionally, we evaluated comorbidities and health care resource utilization (HCRU) costs, comparing these findings to an age and gender-matched control group.

RESULTS: The study revealed a PSC prevalence of 13/100’000 (95% CI: 11 – 14) and an incidence rate of 2.7/100’000 (95% CI: 2.1 – 3.4). In 2022, 15% (95% CI: 11.8% – 19.3%) of PSC patients underwent an ERCP. When compared to the matched control group, PSC patients exhibited a significantly higher risk of developing conditions such as inflammatory bowel disease (ulcerative colitis, Crohn’s disease) and liver fibrosis. Due to the lack of approved therapies, 76% of the PSC patients were treated with off-label UDCA even though the evidence for the use of UDCA is controversial. On average, PSC patients take 30 days of sick leave, compared to 19 days for individuals in the control group. Additionally, PSC patients constitute 15% of those receiving liver transplants. The average HCRU costs for PSC patients were €61,207 (95% CI: €44,472 – €83,324), markedly higher than the control group's average of €3,600 (95% CI: €3,291 – €3,915).

CONCLUSIONS: This study highlights the healthcare burden of PSC, including serious comorbidities and the prevalent use of off-label drugs, underscoring the urgency for optimized care strategies.