Primary sclerosing cholangitis is an orphan hepatobiliary disorder associated with inflammatory bowel disease (IBD).
Dr Tobias Weismüller and colleagues from Germany estimated the risk of disease progression based on distinct clinical phenotypes in a large international cohort of patients with primary sclerosing cholangitis.
The researchers performed a retrospective outcome analysis of patients diagnosed with primary sclerosing cholangitis from 1980 through 2010 at 37 centers in Europe, North America, and Australia.
For each patient, the team collected data on sex, clinician-reported age at and date of primary sclerosing cholangitis and IBD diagnoses, phenotypes of IBD and primary sclerosing cholangitis, and date and indication of IBD-related surgeries.
The team's primary and secondary endpoints were liver transplantation or death, and hepatopancreatobiliary malignancy, respectively.
|Of all patients with primary sclerosing cholangitis, 70% developed IBD at some point|
Of the 7121 patients in the cohort, 2616 met the primary endpoint, and 721 developed hepatopancreatobiliary malignancy.
The researchers observed that the most common malignancy was cholangiocarcinoma.
Patients of advanced age at diagnosis had an increased incidence compared with younger patients.
The team found that of all patients with primary sclerosing cholangitis studied, 66% were men, 90% had classical or large-duct disease, and 70% developed IBD at some point.
Assessing the development of IBD as a time-dependent covariate, Crohn’s disease and no IBD were associated with a lower risk of liver transplantation or death, and malignancy.
The researchers noted that small-duct primary sclerosing cholangitis was associated with a lower risk of liver transplantation or death or malignancy compared with classic primary sclerosing cholangitis.
The team found that female sex was also associated with a lower risk of liver transplantation or death or malignancy.
Small-duct primary sclerosing cholangitis characterized a low-risk phenotype in both sexes.
Conversely, the researchers observed that patients with ulcerative colitis had an increased risk of liver disease progression compared with patients with Crohn’s disease or no IBD.
Dr Weismüller's team concludes, "In an analysis of data from individual patients with primary sclerosing cholangitis worldwide, we found significant variation in clinical course associated with age at diagnosis, sex, and ductal and IBD subtypes."
"The survival estimates provided might be used to estimate risk levels for patients with primary sclerosing cholangitis and select patients for clinical trials."