Gastroenterology

Gastroenterology

Volume 130, Issue 3, March 2006, Pages 715-720
Gastroenterology

Clinical–liver, pancreas, and biliary tract
Excellent Long-Term Survival in Patients With Primary Biliary Cirrhosis and Biochemical Response to Ursodeoxycholic Acid

https://doi.org/10.1053/j.gastro.2005.12.029Get rights and content

Background & Aims: Because the efficacy of UDCA on long-term outcome of primary biliary cirrhosis (PBC) has not been completely elucidated, we have assessed the course and survival of patients with PBC treated with UDCA and compared with the survival predicted by the Mayo model and the estimated survival of a standardized population. Methods: (One hundred ninety-two patients [181 women] with PBC treated with UDCA [15 mg/kg per day] for 1.5–14 years.) Response to treatment was defined by an alkaline phosphatase decrease greater than 40% of baseline values or normal levels after 1 year of treatment. The predicted survival was obtained by the Mayo model and the estimated survival was taken from the standardized matched Spanish population. Results: Seventeen patients died or fulfilled criteria for liver transplantation (8.9%). The observed survival was higher than that predicted by the Mayo model and lower than that of the control population (P < .001). One hundred seventeen patients (61%) responded to treatment. The survival of responders was significantly higher than that predicted by the Mayo model and similar to that estimated for the control population (P = .15). By contrast, the survival of patients without biochemical response was lower than that estimated for the Spanish population (P < .001) although higher than that predicted by the Mayo model. Conclusions: Biochemical response to UDCA after 1 year is associated with a similar survival to the matched control population, clearly supporting the favorable effects of this treatment in PBC. The suboptimal survival of nonresponders identifies the group for further treatments.

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Materials and Methods

The study followed 192 patients with PBC (40 of these were previously enrolled in a UDCA-placebo clinical trial) who were followed under identical conditions in the same hospital. The remaining 152 patients were enrolled in the same institution and only 3 additional cases did not consent to treatment with UDCA. The diagnosis of PBC was established in all patients by a compatible or diagnostic histologic criteria for PBC including 13 patients (6.7%) with no or minor liver biochemical

Results

The clinical and biochemical characteristics and the histologic stage of the overall series and of the patients who did or did not result in good biochemical response after 1 year of treatment are shown in Table 1. Patients were treated with UDCA for a mean period of 6.8 ± 0.2 years (median, 7.5; range, 1.5–14.3 years).

During the study period 17 patients (8.9%) died or fulfilled criteria for liver transplantation and were considered as treatment failure. Nine patients died after 3.5–9.14 years

Discussion

Most of the published therapeutic trials on UDCA in patients with PBC have concluded that this treatment has favorable effects on biochemical parameters and histologic variables but the consequences on survival or on preventing liver transplantation are less categorical.12, 13 The reasons for the inconsistent effects on survival free of liver transplantation are mainly due to the limited short-term duration of the trials (in most cases 2 years, with an extension to 4 years when all the patients

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    Supported in part by a grant from the Instituto de Salud Carlos III C03/02.

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    Copyright © 2006 American Gastroenterological Association Institute. Published by Elsevier Inc. All rights reserved.