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.
Clinical course and prognosis of pediatric-onset primary sclerosing cholangitis / A. Tenca, M. Färkkilä, J. Arola, T. Jaakkola, R. Penagini, K. Kolho. - In: UNITED EUROPEAN GASTROENTEROLOGY JOURNAL. - ISSN 2050-6406. - 4:4(2016 Aug), pp. 562-569.
|Titolo:||Clinical course and prognosis of pediatric-onset primary sclerosing cholangitis|
TENCA, ANDREA (Primo)
PENAGINI, ROBERTO (Penultimo)
|Parole Chiave:||pediatric; cirrhosis; inflammatory bowel disease; liver transplantation; primary sclerosing cholangitis|
|Settore Scientifico Disciplinare:||Settore MED/12 - Gastroenterologia|
|Data di pubblicazione:||ago-2016|
|Digital Object Identifier (DOI):||http://dx.doi.org/10.1177/2050640615616012|
|Appare nelle tipologie:||01 - Articolo su periodico|