A 35-year follow-up of a large cohort of patients with primary biliary cirrhosis seen at a single centre

Background: The natural history of primary biliary cirrhosis (PBC) is still debated.

Aims: To evaluate: (i) long-term survival in a large cohort of PBC patients observed prospectively at a single centre and (ii) mortality in relation to baseline characteristics and ursodeoxycholic acid (UDCA) treatment.

Methods: We considered all consecutive patients between 1973 and 2007 (327 subjects; 310 females, 17 males).

Results: The mean follow-up was 9.1±7.7 years. The patients' age at diagnosis for representative periods (1973-1980, 1981-1990, 1991-2000, 2001-2007) increased progressively from 47.7±1.5 to 53.2±1.2, to 65.2±2.1 and then 63.6±2.9 years. The proportion of asymptomatic patients at diagnosis increased from 30 to 48% in the last decade, while associated symptoms of extrahepatic autoimmunity remained unchanged. Eighty patients (24.4%) died, 74 of them because of liver failure (12 patients developed hepatocellular carcinoma); nine patients underwent liver transplantation. From 1988 onwards, all patients were treated with UDCA (n=288). The mean age at death for the sample as a whole was 67.2±1.3 years. The survival probability at 20 years was 82% for patients with histological stages I-II at entry, 64% for those with stage III and 42% for those with stage IV (P=0.0007). Mortality was significantly reduced in patients treated with UDCA (P=0.012), whereas it was independently associated with oesophageal varices (P=0.015). Patients treated with UDCA had a better prognosis than those untreated, irrespective of the histological stage. Early treated subjects with a good response to UDCA have an 85% chance of survival at 20 years.

Conclusions: The clinical presentation of PBC has been changing over the years. Its early detection and early treatment improve the related survival rates.