There is no medical treatment of proven benefit for primary sclerosing cholangitis.
Dr Rolf Olsson and colleagues studied the effect of a higher dose of ursodeoxycholic acid than previously used.
The research team on survival, symptoms, biochemistry, and quality of life in this disease.
The team performed a randomized placebo-controlled study in tertiary and secondary gastroenterology units.
A total of 219 patients were randomized to 17 to 23 mg/kg body weight per day with 110 receiving ursodeoxycholic acid and 109 with placebo for 5 years.
Follow-up data are available from 97 patients randomized to ursodeoxycholic acid and for 101 randomized to placebo.
Quality of life was assessed by using the Medical Outcomes Study 36-item Short-Form Health Survey.
|Death or liver transplantation occurred in 7% with ursodeoxycholic vs 11% with placebo|
The team found that combined end point ‘death or liver transplantation' occurred in 7% of patients in the ursodeoxycholic acid group vs 11% with placebo.
The occurrence of liver transplantation as a single end point showed a similar positive trend for ursodeoxycholic acid treatment.
The researchers observed that 3 ursodeoxycholic acid and 4 placebo patients died from cholangiocarcinoma, and 1 placebo patient died from liver failure.
The team noted that alkaline phosphatase and alanine aminotransferase tended to decrease during the first 6 months.
There were no differences between the 2 groups in symptoms or quality of life.
In addition, analyses of serum ursodeoxycholic acid concentration gave no evidence that noncompliance may have influenced the results.
Dr Olsson's team commented, “This study found no statistically significant beneficial effect of a higher dose of ursodeoxycholic acid than previously used on survival or prevention of cholangiocarcinoma in primary sclerosing cholangitis.”