Currently, no accepted medical therapy for patients with primary sclerosing
cholangitis (PSC) is available. Case-control studies have shown an inverse
association between PSC and smoking behavior, suggesting that nicotine mig
ht have a beneficial effect in PSC. The aim of this study was to evaluate t
he safety and estimate the efficacy of oral nicotine in the treatment of PS
C. Eight PSC patients who had never smoked received oral nicotine at a maxi
mum dose of 6 mg four times a day for up to one year. Liver biochemistries
and plasma cotinine levels were determined at entry and at three-month inte
rvals during the study duration. Five patients completed one year of treatm
ent, but three of them had to temporarily reduce the dose due to side effec
ts. One patient completed only four months of treatment due to dizziness an
d heart palpitations. Two patients completed only one month of treatment du
e to reactivation of colitis requiring corticosteroid therapy. No significa
nt changes in liver biochemistries were noted during the treatment period d
espite a significant increase in plasma cotinine levels. In conclusion, ora
l nicotine seems to have no beneficial effects in the treatment of PSC, and
it is frequently associated with side effects necessitating permanent drug
cessation.