Background: A beneficial effect of corticosteroids in primary sclerosing cholangitis (PSC) has been suggested, but characteristics of responding patients and long-term outcome have not been assessed. In this retrospective study, we aimed to characterize PSC patients selected for corticosteroid treatment at our centre and to identify potential factors associated with response. Methods: We first compared groups of PSC patients treated ( n r = r 47) and not treated ( n r = r 88) with corticosteroids. Responding ( n r = r 20) and non-responding ( n r = r 27) patients were subsequently compared. Complete and partial responses were defined according to criteria established for autoimmune hepatitis. A third response category included improvement of symptoms and at least 50% reduction of transaminase and/or bilirubin levels during the first 6 months. Results: At diagnosis of PSC, patients treated with corticosteroids were significantly younger, had higher serum levels of alanine transaminases, and more histological features of autoimmune hepatitis compared to the non-treated group. Complete treatment response was obtained in three patients and partial response in two, together comprising 3.7% of all PSC patients in this study. Fifteen patients fulfilled criteria of the third response category. Response to treatment was associated with higher serum levels of alanine transaminases and bilirubin and lower levels of alkaline phosphatases at treatment start. Responders had better long-term survival than non-responders (hazard ratio 6.28; 95% confidence interval 1.62 to 24.4; P r = r 0.008). Conclusions: A subgroup of PSC patients seems to respond favourably to corticosteroid treatment and may obtain improved long-term survival.