Clinical course and prognosis of pediatric-onset primary sclerosing cholangitis. Issue 4 (August 2016)

• Record Type: Journal Article
• Authors: Tenca, Andrea; Färkkilä, Martti; Arola, Johanna; Jaakkola, Tytti; Penagini, Roberto; Kolho, Kaija-Leena
• Is Part Of: United European Gastroenterology journal. Volume 4:Issue 4(2016:Aug.)
• Page Start: 562
• Page End: 569
• Publication Date: 2016-08
• Subjects: Pediatric -- inflammatory bowel disease -- liver transplantation -- cirrhosis -- primary sclerosing cholangitis; Gastroenterology -- Periodicals; Periodicals; 616.33005
• DOI: 10.1177/2050640615616012
• Languages: English
• ISSNs: 2050-6406

Abstract

Background: The natural history of pediatric-onset primary sclerosing cholangitis (PSC) and overlap with autoimmune hepatitis (PSC/AIH) is poorly known. Objective: The aim of this study was to evaluate the clinical outcome of patients with pediatric-onset disease in a tertiary referral center. Methods: We traced 33 patients (median age at diagnosis 16 years), with PSC or PSC/AIH in cholangiography and liver histology diagnosed between December 1993 and 2011, at Helsinki University Hospital. Diagnostic procedures and long-term follow-up were reassessed until the end of December 2013. Results: PSC was confirmed in all 33 patients; 19 of them had an overlap with AIH. At diagnosis, three of 33 had cirrhosis. Inflammatory bowel disease (IBD) was associated in 76% of the patients, mostly ulcerative colitis (70%); treatment of IBD being a minor determinant of the clinical outcome of liver disease. In the last follow-up (median nine years), all patients were alive, and no malignancy occurred. Most patients (91%) were on ursodeoxycholic acid and 12 PSC/AIH patients on immunosuppression. Endoscopic retrograde cholangiography during follow-up showed a progression of intra-hepatic disease in 12 patients (36%). Four patients (12%) had undergone liver transplantation, and one was listed; no recurrence of the disease in the graft was seen. Conclusion: The clinical course and outcome of pediatric-onset PSC and PSC/AIH seem to be favourable in the majority of patients until early adulthood. In about one-third of patients, however, PSC is progressive, challenging the current treatment guidelines and warranting further studies on disease pathogenesis.