Primary Sclerosing Cholangitis in Children: A Long–Term Follow–Up Study

Abstract

Primary sclerosing cholangitis (PSC) is increasingly diagnosed in children and adolescents, but its long-term prognosis remains uncertain. The aim of this longitudinal, cohort study was to determine the long-term outcome of children with PSC. Fifty-two children with cholangiography-proven PSC (34 boys and 18 girls; mean age 13.8 ؎ 4.2 years; range, 1.5-19.6 years) who were seen at our institution over a 20-year period were followed-up for up to 16.7 years. Two thirds presented with symptoms and/or signs of PSC and 81% had concomitant inflammatory bowel disease (IBD). Twenty-five percent had total alkaline phosphatase activity within the normal range for the age group, but all of them had elevated ␥-glutamyl transpeptidase levels. Autoimmune hepatitis overlapping with PSC was present in 35% of children. A positive but transient clinical and/or biochemical response occurred under therapy with ursodeoxycholic acid, alone or in combination with immunosuppressive medications. During follow-up, 11 children underwent liver transplantation for end-stage PSC and 1 child died. The median (50%) survival free of liver transplantation was 12.7 years. Compared with an age-and gender-matched U.S. population, survival was significantly shorter in children with PSC (P < .001). In a Cox regression model, lower platelet count, splenomegaly, and older age were associated with shorter survival. Presence of autoimmune hepatitis overlapping with PSC (P ‫؍‬ .2) or medical therapy (P ‫؍‬ .2) did not affect survival. In conclusion, PSC significantly decreases survival in this child population. Although pharmacologic therapy may improve symptoms and liver test results initially, it does not seem to impact the long-term outcome. (HEPATOLOGY 2003;38:210-217.)

Prevalence of sclerosing cholangitis in adults with autoimmune hepatitis: Evaluating the role of routine magnetic resonance imaging

“…In the study by Gregorio et al, 5 the presence of cholangiopathy was associated with a worse prognosis over the 16 years of follow-up; 4 of the 27 children with SC required liver transplant, whereas none of the 28 who lacked evidence of large duct biliary changes did. The survival analysis by Feldstein et al 38 of children with PSC revealed a survival disadvantage over an age-matched and gender-matched US population, regardless of the presence or absence of concomitant AIH. Pharmacological therapy did not seem to impact survival and long-term outcome in their longitudinal cohort assessment of 16 years.…”

Primary sclerosing cholangitis: Summary of a workshop

“…Estella Alonso (Children's Memorial Hospital, Chicago, IL) addressed the special issues surrounding liver transplantation for children with PSC. PSC is an uncommon indication for liver transplantation in children, 19,20 being the cause for only 73 of 2219 (3.5%) children listed in the Studies of Pediatric Liver Transplantation (SPLIT) registry between 1995 and 2004. 157 Compared to children with other diagnoses, children with PSC were more likely to be male (64% vs. 47%: P ϭ .004), Caucasian and older in age (median ϭ 12.7 vs. 1.4 years), and were less likely to be hospitalized.…”

“…17,18 In children, PSC usually presents with nonspecific symptoms and pruritus, and rarely with jaundice. 11,13,[18][19][20][21][22] An autoimmune hepatitis-like presentation is common. 11,22 ERCP may demonstrate bile duct abnormalities in these children, 23 but their course is typical of autoimmune hepatitis, although with time, features of PSC become prominent.…”

The role of conventional ultrasound in pediatric chronic liver diseases

“…However, a quarter of the children with ASC, based on abnormal cholangiograms, had no histological features of bile duct involvement. Therefore, hepatologists should be very careful in making conclusions (34,35).…”