Autoimmune features negatively influenced pediatric sclerosing cholangitis, but concomitant inflammatory bowel disease did not, according to a recently published study.
“Pediatric sclerosing cholangitis (SC) is a rare disease and data concerning the natural history and characteristics of SC are largely from small, single center case series with limited long-term follow-up,” Federica Ferrari, MD, PhD, from the Sapienza University of Rome, told Healio Gastroenterology and Liver Disease. “In our study, performed in two referral centers, the 9-year survival with native liver was better than what [was] reported in adults. Moreover, complication-free survival was 85%, less than previous reports.”
Ferrari and colleagues retrospectively reviewed the data of 45 pediatric patients with sclerosing cholangitis. Median patient age was 10 years (range, 1-18 years) and most were boys (73.4%).
All patients underwent ultrasonography at presentation and 31 patients underwent liver biopsy. Results showed fibrosis stage 1 in 3 patients, stage 2 in 10, stage 3 in 5, and five patients had cirrhosis at presentation.
Of the 20 patients with autoimmune features, nine met diagnostic criteria for autoimmune hepatitis. Six patients received a diagnosis of sclerosing cholangitis and autoimmune hepatitis simultaneously, while autoimmune hepatitis preceded sclerosing cholangitis in three patients.
Twenty patients had inflammatory bowel disease and 25 patients received a diagnosis of isolated sclerosing cholangitis without intestinal involvement.
Patients with isolated sclerosing cholangitis had a higher rate of interface hepatitis at liver biopsy than those with IBD, whereas those with concomitant IBD had a significantly higher rate of anti-smooth muscle antibody positivity (P = .03).
Over a mean follow-up of 8.7 years (range, 1.3-20.3 years), patients received either ursodeoxycholic acid monotherapy (n = 28) or UDCA with prednisone and azathioprine (n = 17).
At the end of follow-up, seven patients showed at least one liver-related complication: five developed esophageal varices after a median time of 3 years (range, 0.6-9 years) and two presented coagulopathy.
Survival free from liver transplantation and from liver complications did not differ significantly between patients with sclerosing cholangitis and those with concomitant IBD. Patients without autoimmune features, however, had higher probability of survival free from liver complications compared with those with autoimmune sclerosing cholangitis (P = .03).
“Our results suggest that pediatric SC may have a milder phenotype compared to adult PSC; thus, it is necessary to recognize these patients in pre-symptomatic phase,” Ferrari and colleagues wrote. “It is worth to note that this favorable outcome represents an original information as it offers a different view of the long-term outcome of pediatric sclerosing cholangitis, evaluated in two referral centers during a long period of observation. We can speculate that the good prognosis could be influenced by the early SC diagnosis mostly in asymptomatic children.” – by Talitha Bennett
Disclosure: The authors report relevant financial disclosures.