Primary biliary cirrhosis (PBC) is a progressive cholestatic liver disease
frequently leading to development of cirrhosis and its complications. Ursod
eoxycholic acid (UDCA) is a beneficial medical therapy for patients with PB
C. Improvement in some histological features, but not in histological stage
, has been reported after 2 years of UDCA therapy, Thus, longer follow-up m
ay be necessary to determine whether UDCA has a favorable effect on histolo
gical stage of disease and progression to cirrhosis. Our aim was to determi
ne the long-term effects of UDCA therapy on histological stage and progress
ion to cirrhosis in patients with PBC. Sixteen unselected patients with non
cirrhotic PBC who had been on long-term UDCA therapy (13-15 mg/kg/d) for 6.
6 +/- 0.4 years (range, 5-9 years) were identified and their histological f
inding during treatment compared with that of 51 noncirrhotic patients with
PBC who had received ineffective therapy (D-penicillamine [DPCA] or placeb
o) for 5.6 +/- 0.07 years (range, 5-8 years). Histological stage was determ
ined using the Ludwig classification, The rate of progression to cirrhosis
(stage 4) was significantly less in the UDCA group than in the control grou
p (13% vs. 49%; P = .009). Although the overall rate of progression of hist
ological stage was less in the UDCA group than in the control group (50% vs
. 71%), this difference was not significant (P = .1). A marked improvement
in liver biochemistries and Mayo risk score was noted in all patients durin
g UDCA therapy; however, this improvement was not significantly different b
etween patients who progressed and those who did not. In conclusion, long-t
erm UDCA therapy appeared to delay the development of cirrhosis in PBC.