Ten–Year Survival in Ursodeoxycholic Acid-Treated Patients With Primary Biliary Cirrhosis

Abstract

Ursodeoxycholic acid (UDCA) treatment has been shown to increase survival without orthotopic liver transplantation (OLT) in patients with primary biliary cirrhosis (PBC) at 4 years. Whether this beneficial effect was maintained over the long term remained to be established. In a large cohort of UDCA-treated patients with PBC, we aimed to determine the 10-year outcome of these patients using two endpoints: (1) survival without OLT, and (2) survival. The cohort was comprised of 225 patients with PBC treated with UDCA (13-15 mg/kg/d) monitored from the beginning of treatment until time of last follow-up, OLT, or death. Because of the absence of a control group, survival without OLT was compared with survival predicted by the Mayo model (first 7 years), and observed 10-year survival with an estimation of survival of a standardized control cohort of the French population. Observed survival without OLT of UDCAtreated patients was significantly higher (P F .04) than survival predicted by the Mayo model. Observed survival was significantly lower (P F .01) than survival predicted from the French population. Observed survival of noncirrhotic patients was not different (P G .9) from that of the French control population but survival of cirrhotic patients was significantly lower (P F .0001). Twenty-two patients died; 13 patients died of hepatic causes and 4 patients died after OLT. In conclusion, survival without OLT among patients treated with UDCA for PBC is higher than that of untreated patients, as predicted by the Mayo model. Tenyear survival among UDCA-treated patients is slightly lower than that of an age-and sex-matched general population, the difference mainly being explained by mortality among cirrhotic patients. (HEPATOLOGY 1999; 29:1668-1671.)

Biochemical response to ursodeoxycholic acid and long-term prognosis in primary biliary cirrhosis

“…This observation, which has now been reported in five large independent U.S. and European series, [6][7][8][9]16 supports a long-term beneficial effect of UDCA in PBC. The comparison of the observed data with the predictions of the Mayo model suggests a 40% decrease in the risk of death or LT under UDCA over a 7-year period.…”

“…This finding is in keeping with our previous data and those of several European cohort studies of long-term treated patients. 6,7,9 It suggests that the long-term administration of UDCA alone at the recommended daily dose of 13-15 mg/kg is sufficient for many PBC patients. However, the fact that transplant-free survival is still lower than that of the control population strongly indicates that additional therapies are required in some patients.…”

“…[1][2][3][4][5] UDCA monotherapy appears sufficient for many patients as suggested by long-term observational data. [6][7][8][9] However, the transplant-free survival rate of UDCA-treated patients remains significantly lower than that of an age-matched and sex-matched control population. 10 Therefore, there is a continued need for new therapeutic options in PBC, particularly in patients having a suboptimal biochemical response to UDCA and predictive factors of poor outcome.…”

Remission with ursodeoxycholic acid of type 1 autoimmune hepatitis resistant to azathioprine and steroids

“…Ursodeoxycholic acid therapy has led to improvements in many liver diseases, particularly PBC (11,12,(21)(22)(23). In chronic viral hepatitis, UDCA is beneficial at low doses (14).…”

“…Improvements in liver dysfunction due to cholestatic liver diseases, with few if any adverse effects, UDCA is now the reference therapy for primary biliary cirrhosis (PBC) (11,12).…”