Oral budesonide in the treatment of patients with primary biliary cirrhosis with a suboptimal response to ursodeoxycholic acid

Ursodeoxycholic acid (UDCA) is a safe and effective medical therapy for mos t patients with primary biliary cirrhosis (PBC). However, some patients sho w an incomplete response to UDCA therapy. Treatment with corticosteroids ma y be of benefit although at the expense of systemic side effects. Budesonid e, a corticosteroid with an extensive first-pass hepatic metabolism appeare d promising for the treatment of PBC. The aim of this study was to evaluate the safety and estimate the efficacy of budesonide in patients with PBC, w ho have shown a suboptimal response to UDCA. Twenty-two patients with PBC, 16 women, median age of 50 who had been on UDCA (13-15 mg/kg/d) for a mean of 46 months (range 6-108 months) and had shown a persistent elevation of a lkaline phosphatase activity at least 2 times the upper limit of normal wer e enrolled. Oral budesonide, 9 mg daily was administered for 1 year and pat ients continued on the same dosage of UDCA. There was a significant, but tr ansitory improvement in serum levels of total bilirubin (P = .001) and a si gnificant, but marginal improvement in serum alkaline phsophatase (P = .001 ) with combination therapy. The Mayo risk score increased significantly (P = .02) and there was a significant loss of bone mass (P < .001) of the lumb ar spine. Budesonide-induced hyperglycemia and cosmetic adverse effects wer e noted in 2 patients. In conclusion, oral budesonide appears to add minima l, if any, additional benefit to UDCA, and it is associated with a signific ant worsening of osteoporosis in patients with PBC.