Survival algorithms and outcome analysis in primary biliary cirrhosis.

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 education 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.