PAEDIATRIC-ONSET PRIMARY SCLEROSING CHOLANGITIS: CLINICAL COURSE AND OUTCOME

ABSTRACT BACKGROUND AND AIMS The natural history of paediatric onset PSC and PSC/AIH overlap syndrome is poorly known. Thus, this study was aimed at evaluating the clinical course and outcome of patients with a paediatric onset of disease. MATERIALS AND METHODS Between December 1993 and December 2011, thirty-five patients (median age at diagnosis 15 years), with confirmed PSC by cholangiography (ERC) and PSC/AIH by cholangiography and the modified score for AIH, were traced at Helsinki University Central Hospital. Clinical characteristics (symptoms and signs, associated diseases), diagnostic procedures (lab tests, liver biopsy, ERC) and long-term follow-up (mortality, liver transplantation, recurrence of the disease in the graft, malignancy) were reassessed until December 2013. RESULTS The original diagnosis was PSC in 22 children (63%) and PSC/AIH overlap syndrome in 13 (37%). At diagnosis most of the children had an insidious onset and most an associated IBD, being UC the most common form. Still, 4/35 (11%) had cirrhosis. Endoscopic retrograde cholangiography during follow-up was available for all of the patients and images showed a progression of intra-hepatic disease in 13/28 (46.4%) (p=0.0102). In the last follow-up (median 8 years), all patients were alive. Twenty-eight out of 35 patients (80%) and 13/35 patients (40%) were taken UDCA and immunosuppressive therapy, respectively; 3 patients were without treatment. Transaminases and GGT improved significantly. Four patients (11%) had undergone liver transplantation (after a median 7.5 years) and one was listed; and additional patient was transplanted because of Budd-Chiari syndrome. No difference in graft free survival was seen between patients with PSC and PSC/AIH. Three patients (two with PSC and one with PSC/AIH overlap syndrome) presented with cirrhosis. No malignancy occurred. CONCLUSION The clinical outcome of primary sclerosing cholangitis and overlap syndrome seems comparable including their progression to cirrhosis and requirement for liver transplantation.