Gastroenterology

Gastroenterology

Volume 123, Issue 4, October 2002, Pages 1044-1051
Gastroenterology

Clinical–Liver, Pancreas, and Biliary Tract
Survival and symptom progression in a geographically based cohort of patients with primary biliary cirrhosis: Follow-up for up to 28 years

https://doi.org/10.1053/gast.2002.36027Get rights and content

Abstract

Background & Aims: Although several excellent studies have described the natural history of primary biliary cirrhosis, most were reported from tertiary referral centers. We examined the prognosis of primary biliary cirrhosis in a comprehensive geographically defined cohort. Methods: We followed up 770 primary biliary cirrhosis patients prevalent between January 1987 and December 1994 until death, transplantation, or censor on January 1, 2000, by interview and review of case notes and death certificates. Analysis of survival data was performed with Kaplan–Meier methods and Cox regression. Results: Median patient survival was 9.3 years from diagnosis. Patient age, alkaline phosphatase, albumin, and bilirubin at diagnosis independently predicted survival in Cox modeling. Prothrombin time and histologic stage did not independently affect survival. Observed survival was predicted well by this model and by the Mayo prognostic score (R2M = 0.37 and 0.18, respectively; R2M is a likelihood-based measure of the percentage information gain from the model due to covariates). Forty-two percent of deaths were caused by liver disease. Thirty-nine patients had liver transplantations by the censor date. Survival was much poorer than for an age- and sex-matched control population (standardized mortality ratio = 2.87 [1.73 excluding liver deaths]). The most common symptoms at diagnosis were pruritus (18.9%) and fatigue (21.0%). Twenty-six percent of patients developed liver failure by 10 years after diagnosis. Conclusions: Although primary biliary cirrhosis is often now diagnosed at an early stage, the diagnosis still carries important prognostic implications. A significant proportion of patients develop liver failure, require transplantation, or die prematurely after this diagnosis.

GASTROENTEROLOGY 2002;123:1044-1051

Section snippets

Materials and methods

The methods used to identify the Northeast England cohort of patients with PBC have previously been described in detail.13 This cohort included all incident and prevalent cases of definite or probable PBC who were alive between January 1987 and December 1994 within a geographically defined area of Northeast England. Boundaries within the region were verified from the Office of National Statistics, postal code (ZIP code), and local health authority data.

Definite PBC was defined as all 3 of the

Results

A total of 770 patients were diagnosed with PBC within the geographic and temporal boundary of the sampling frame; 302 patients were diagnosed before January 1, 1987 (i.e., prevalent cases), and 468 patients were incident cases between January 1987 and December 1994. Data to analyze survival adjusted for the effect of prevalent cases were available for 765 (99%) patients. Full clinical follow-up by interview and note review to ascertain symptom status was possible for 708 (91.9%) patients.

A

Discussion

We have reported the follow-up of a large geographically based cohort of patients with PBC. Unlike all previous reports of outcome, this study was not limited to a few health care providers, nor was it based in a tertiary referral center. These data may provide the most accurate picture of the outcome of PBC in an unselected population.

Most patients were asymptomatic at the time of initial diagnosis. This case series is the first to formally analyze the progression of symptoms in a

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    Address requests for reprints to: Martin Prince, M.R.C.P., Centre for Liver Research, Fourth Floor, William Leech Building, The Medical School, Framlington Place, Newcastle-Upon-Tyne, NE2 4HH, England. Fax: (44) 191-222-0723.

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