ABSTRACT
The natural history of primary biliary cirrhosis (PBC) is one of slowly progressive
cholestasis with liver damage, development of cirrhosis with its concomitant complications,
and death unless the patient undergoes liver transplantation. Natural history studies
have identified several variables associated with a decreased survival in patients
with PBC. The course of the disease can be divided into three time periods: (1) a
presymptomatic phase, probably lasting up to 20 years; (2) a symptomatic phase, with
anicteric or mild jaundice, lasting up to 5 to 10 years; and (3) a preterminal or
accelerated phase with marked jaundice, lasting up to 2 years. Since the course of
the disease is one of slow progression leading to liver failure and death unless liver
transplantation intervenes, several investigators have developed statistical models
to predict survival. The ability to predict survival for individual patients with
PBC has been valuable in the management of these patients, particularly in patient
selection and timing of liver transplantation. In addition, survival estimates can
be utilized in educating and counseling patients and their families. These models
may also be used to evaluate the efficacy of new treatments by comparing natural history
survival with the survival achieved by therapeutic effect. Over the past several decades,
the natural history models of PBC have been developed in the absence of effective
medical therapy. The efficacy of liver transplantation and survival following liver
transplantation has now been quantitatively established. Future efforts should be
aimed at determining not only survival of patients with primary biliary cirrhosis
in the presence of effective medical therapy but also at assessing the quality of
life and cost-effectiveness of medical therapy and liver transplantation in the management
of patients with primary PBC.
KEY WORDS
survival - outcomes - primary biliary cirrhosis
