title: Clinical course and prognosis of pediatric-onset primary sclerosing cholangitis.
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.
  
  
  CONCLUSIONS
  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.
authors.affiliation: Clinic of Gastroenterology, University of Helsinki and Helsinki University Hospital, Helsinki, Finland; Gastroenterology and Endoscopy Unit, Fondazione IRCCS Cà Granda, Ospedale Maggiore Policlinico; Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Italy.
authors.forename: Andrea
authors.initials: A
authors.lastname: Tenca
authors.affiliation: Clinic of Gastroenterology, University of Helsinki and Helsinki University Hospital, Helsinki, Finland.
authors.forename: Martti
authors.initials: M
authors.lastname: Färkkilä
authors.affiliation: Department of Pathology, University of Helsinki and Helsinki University Hospital, Helsinki, Finland.
authors.forename: Johanna
authors.initials: J
authors.lastname: Arola
authors.affiliation: Department of Pediatric Gastroenterology, University of Helsinki and Children's Hospital, Helsinki, Finland.
authors.forename: Tytti
authors.initials: T
authors.lastname: Jaakkola
authors.affiliation: Gastroenterology and Endoscopy Unit, Fondazione IRCCS Cà Granda, Ospedale Maggiore Policlinico; Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Italy.
authors.forename: Roberto
authors.initials: R
authors.lastname: Penagini
authors.affiliation: Department of Pediatric Gastroenterology, University of Helsinki and Children's Hospital, Helsinki, Finland.
authors.forename: Kaija-Leena
authors.initials: KL
authors.lastname: Kolho
keywords: Pediatric; cirrhosis; inflammatory bowel disease; liver transplantation; primary sclerosing cholangitis