Background/aims: Primary sclerosing cholangitis (PSC) is a rare disease, and large-scale report of PSC in France is lacking. We initiated a prospective multisite observational study.
Methods: One hundred and fifty PSC patients (90 with associated inflammatory bowel disease) were included. At entry, 11 patients had a diagnosis of hepatobiliary or colon malignancy (cholangiocarcinoma: n = 5, hepatocellular carcinoma: n = 2, gallbladder carcinoma: n = 1 and colorectal cancer: n = 4). One hundred and forty-one patients (94%) were treated with ursodeoxycholic acid (UDCA) (median dose: 13.1 mg/kg/day), including 118 with UDCA started before inclusion.
Results: During follow-up [3.9 (0.1-7.2) years], colorectal cancer was diagnosed in four patients and biliary carcinoma in two (incidences: 0.76 and 0.38 for 100 patient-years, respectively). Kaplan-Meier transplant-free survival at 4 years was 79%. Main causes of death (n = 10) were cancer (n = 5, including three colorectal cancers and two cholangiocarcinoma) or liver failure (n = 4). Indications for transplantation (n = 25) were end-stage liver disease (n = 16), biliary cancer (known or suspected) (n = 5), recurrent acute cholangitis (n = 3) or pruritus (n = 1). Independent predictive factors of death or transplantation were alkaline phosphatase at least 3 upper limit of normal values, platelets less than or equal to 150000/mm3 and bilirubin at least 23 micromol/l. Observed and predicted survivals were similar.
Conclusion: PSC in France shares common features with other series and is almost universally treated with UDCA. Under standard-dose UDCA, PSC remains a rather severe disease. However, the low incidence of cholangiocarcinoma is compatible with a potential chemoprotective effect of UDCA against biliary neoplasia development.