Clinical course and prognosis of pediatric‐onset primary sclerosing cholangitis

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.