Clinicopathologic features of the syndrome of primary sclerosing cholangitis

We reviewed the histories of 50 patients diagnosed surgically and/or radiographically as having primary sclerosing cholangitis seen from 1970 through 1977. Patients were generally young males who presented with jaundice, pruritus, and hepatomegaly. Inflammatory bowel disease, usually chronic ulcerative colitis, was common (54%). A cholestatic biochemical profile was ordinarily present, and serologic markers were conspicuously absent. Initiation of endoscopic retrograde cholangiography in 1974 was associated with a doubling of the annual frequency of diagnosis. Radiologic evidence of both extra- and intrahepatic duct involvement was usually found (81%). Abnormal liver biopsies from 34/36 patients showed cholestasis, portal or periportal hepatitis, and/or cirrhosis. Hepatic copper levels were elevated in 12/12, and the mean level (244 ± 71 μg/g dry weight liver, mean ± SEM) was comparable to that in primary biliary cirrhosis. Adequate follow-up data in 39 patients showed that 13 died from 5 to 108 mo after diagnosis, usually from liver failure. Many patients (42%) received steroid therapy, generally without apparent benefit. In summary, the diagnosis of primary sclerosing cholangitis at our institution, particularly since the advent of endoscopic retrograde cholangiography, is not rare. The disease is characterized by male predominance, progressive cholestasis, cirrhosis, hepatic copper overload, apparent steroid failure, and premature death from liver failure.