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Primary Sclerosing Cholangitis (PSC) –Natural History Study

January 9, 2017 7:00 am

Last week, I posted an blog referencing new guidelines for peanut introduction. A more detailed explanation of these guidelines: New Guidelines: Early Introduction of Peanut to Prevent Peanut Allergy from David Stukus (Thanks to Kipp Ellsworth for sharing this information)

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A recent study (PL Valentino et al. JPGN 2016; 63: 603-09) follows “the largest reported pediatric PSC cohort” to determine the natural history.

Study characteristics:

This retrospective study followed 120 children (1-21 yrs) with a median age of 14 years. 27% had autoimmune sclerosing cholangitis (ASC), 63% had PSC; 24% (n=29) of entire cohort had exclusive small duct PSC. Median followup was 3.7 years.

Key findings:

81% of PSC patients had inflammatory bowel disease; most (72/97) had ulcerative/indeterminant coliits. 40/72 had pancolitis.
PSC-IBD was more common than ASC-IBD (85% vs 68%).
10-year transplant-free survival in this cohort was 89%; there were 6 liver transplants.
The rate of cirrhosis was lower in the group who had IBD preceding PSC (15% vs 31%,P=0.05).
PSC is clinically silent in the majority of patients; 64% presented with abnormal chemistries and no other symptoms.
ERCP therapeutic intervention was low, 3% for stenting and 7% for balloon dilatation.

The authors speculate that one reason for milder PSC-IBD disease could relate to the fact that IBD patients undergo frequent chemistries. In those without IBD, subacute PSC could be present for a much longer period before detection. The authors note that PSC in children presents as a milder disease with only 10% having cirrhossi compared with 30% in studies with adult patients.

My take: We have a lot to learn about PSC including which patients are likely to develop clinically significant liver disease and whether most patients benefit from treatment.

Related blog post: Should we care about subclinical PSC? (This post has links to others related to PSC)