Characteristics and clinical course of primary sclerosing cholangitis in France: a prospective cohort study.
OBJECTIVE
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.


CONCLUSIONS
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.
Garioud
Armand
A
Service d'Hépatologie, Hôpital Saint Antoine, Assistance Publique-Hôpitaux de Paris, Faculté de Médecine Pierre et Marie Curie, Paris, France.
Seksik
Philippe
P
Chrétien
Yves
Y
Corphechot
Christophe
C
Poupon
Raoul
R
Poupon
Renée Eugénie
RE
Chazouillères
Olivier
O
D000293:Adolescent; D000328:Adult; D000368:Aged; D001650:Bile Duct Neoplasms / Q000175:diagnosis / Q000188:drug therapy; D001653:Bile Ducts, Intrahepatic / Q000187:drug effects / Q000473:pathology; D006528:Carcinoma, Hepatocellular / Q000175:diagnosis / Q000188:drug therapy; D018281:Cholangiocarcinoma / Q000175:diagnosis / Q000188:drug therapy; D015209:Cholangitis, Sclerosing / Q000175:diagnosis* / Q000188:drug therapy / Q000401:mortality*; D015331:Cohort Studies; D015179:Colorectal Neoplasms / Q000175:diagnosis / Q000188:drug therapy / Q000473:pathology; D005260:Female; D005602:France / Q000453:epidemiology; D005706:Gallbladder Neoplasms / Q000175:diagnosis / Q000188:drug therapy; D006801:Humans; D015994:Incidence; D017093:Liver Failure / Q000209:etiology / Q000401:mortality*; D008113:Liver Neoplasms / Q000175:diagnosis / Q000188:drug therapy; D016031:Liver Transplantation; D008297:Male; D008875:Middle Aged; D011379:Prognosis; D011446:Prospective Studies; D016896:Treatment Outcome; D014580:Ursodeoxycholic Acid / Q000627:therapeutic use; D055815:Young Adult
D014580:Ursodeoxycholic Acid