Characterization, Outcome, and Prognosis in 273 Patients with Primary Sclerosing Cholangitis: A Single Center Study

TLDR

Primary sclerosing cholangitis is a chronic cholestatic liver disease with variable severity and progression. The study aimed to describe the natural history of PSC and develop a prognostic model to predict disease progression and guide transplantation timing. A retrospective cohort of 273 German PSC patients with a median 76‑month follow‑up was analyzed using Kaplan‑Meier survival curves and multivariate Cox regression to assess clinical, biochemical, and cholangiographic predictors. Median survival was 9.6 years; 39.6 % underwent liver transplantation and 14.3 % developed hepatobiliary malignancies, with age, low albumin, persistent bilirubin >3 months, hepatomegaly, splenomegaly, dominant stenosis, and ductal changes identified as independent risk factors, and persistent bilirubin >3 months emerging as a novel poor‑outcome marker.

Abstract

Primary sclerosing cholangitis (PSC) is a chronic cholestatic liver disease with varying severity and progression. This study describes the natural history of PSC patients and evaluates the prognostic significance of clinical, biochemical, and cholangiographic findings constructing a novel prognostic model.A population of 273 German PSC patients was studied with a median follow-up time of 76 months (range 1-280 months). Survival curves were analyzed by the Kaplan-Meier method, and prognostic significance of clinical, biochemical, and cholangiographic features recorded at the time of diagnosis was evaluated by multivariate analysis using Cox proportional-hazards regression models.The estimated median survival from the time of diagnosis to death or time of liver transplantation was 9.6 yr. One hundred eight (39.6%) patients underwent liver transplantation. Hepatobiliary malignancies were found in 39 (14.3%) patients of the entire PSC population. Age, low albumin, persistent bilirubin elevation longer than 3 months, hepatomegaly, splenomegaly, dominant bile duct stenosis, and intra- and extrahepatic ductal changes at the time of diagnosis were found to be independent risk factors correlating with poor prognosis and were used to construct a new prognostic model.A persistent bilirubin elevation for longer than 3 months from the time of diagnosis could be identified as a novel marker correlating with a poor outcome. A new prognostic model was developed to predict progression of PSC, which may be useful in timing of liver transplantation.