Ursodeoxycholic Acid Prolongs Life in Primary Biliary Cirrhosis

NEW YORK (Reuters Health) Feb 14 - In patients with primary biliary cirrhosis, treatment with ursodeoxycholic acid (UDCA) prolongs survival, if it is administered in early stages of the disease, researchers in France report in the February issue of Gastroenterology.

Even though UDCA is the only drug approved for the treatment of these patients, some evidence has suggested that it does not improve outcome (see Reuters Health report, September 24, 1999).

Dr. Christophe Corpechot, at Hopital Saint-Antoine in Paris, and colleagues developed a multistate Markov model to analyze the long-term effect of UDCA among 262 patients with biliary cirrhosis enrolled between 1982 and 2001 and followed until 2002. Patients underwent liver biopsies to ascertain disease stage.

Predicted survival rates were compared with those of a control French population matched for age, gender, and follow-up period, and with the natural history of the disease predicted by the updated Mayo model applied to patients' risk score at treatment outset.

Sixteen deaths (10 liver related) and 20 liver transplantations occurred during mean follow-up of 8 years. The authors predicted a survival rate of 92% at 10 years and 82% at 20 years, not significantly different from that of the control population.

Without transplantation the corresponding rates were 84% and 66%. Relative risk (RR) was higher at 1.4, though the difference was not significant (p = 0.1).

In contrast, survival was significantly better than would occur without treatment, with a RR of 4.2 with transplantation (p < 0.0001) and a RR of 2.1 without transplantation (p < 0.01).

However, for patients in late stages of the disease, survival without transplantation did not differ significantly from that predicted by the updated Mayo model.

"Our study shows unequivocally that a large part of the benefit from UDCA therapy lies with its impact on the course of primary biliary cirrhosis in patients in early histologic stages of the disease," Dr. Corpechot and his associates conclude.

These results suggest that for patients with biliary cirrhosis, "time is of the essence," Drs. Konstantinos N. Lazaridis and Nicholas F. LaRusso, at the Mayo Clinic College of Medicine in Rochester, Minnesota, note in an accompanying editorial.

"Detection of disease at an early stage and immediate initiation of therapy should be the standard of care," they write.

Gastroenterology 2005;128:297-303,498-500.