Incidence and Impact of Decompensating Events in Primary Biliary Cirrhosis : Results of an International Follow Up Study of 3030 Patients

Summary: In primary biliary cirrhosis (PBC), clinical events of decompensation are considered indicators of poor prognosis, however, there are limited studies assessing the incidence of decompensating events and the related patient outcomes, which is why researchers conducted this long-term follow-up study. Their findings revealed that in UDCA-treated PBC patients, ascites are usually the first major clinical complication. Prognosis of patients with either ascites, variceal bleeding, or encephalopathy as first complication is associated with a 5-year transplantation-free survival of < 20%. Methods: Long-term follow-up (FU) data of patients treated with ursodeoxycholic acid (UDCA) were derived from 17 North-American and European centers. Researchers defined decompensation as a first event of ascites, variceal bleeding, or encephalopathy, whichever came first. They excluded any patients with events before baseline or in first year of FU. Cox proportional hazard models were performed for risk factor analysis. Of 3030 UDCA-treated PBC patients, 92 patients (3.0%) were excluded. Results: Median FU was 8.4 years (IQR 4.7-12.9). A total of 275 patients experienced a decompensating event: ascites (n=167, 61%), variceal bleeding (n=76, 27%), encephalopathy (n=24, 9%), multiple (n=8, 3%). Transplantation-free survival (1-, 3- and 5- years) with or without an event was 59% vs 99%, 35% vs 97%, and 19% vs 94%, respectively (time dependent HR 40.6 [95% CI: 29.6-55.7]). There was no significant difference between survival and event type. At time of first event, the following factors may predict survival: age at time of event (per 10 years) (HR 1.39 [95% CI: 1.09-1.63]), calendar year of event (HR 0.97 [95%CI 0.94-0.99]), bilirubin >2x upper limit of normal ([ULN] HR2.98 [95% CI: 1.99-4.45]), and normal albumin (HR 1.68 [95% CI: 1.12-2.53]). Survival of patients with normal albumin and bilirubin < 2xULN at time of event was significantly better than of those with abnormal albumin and/or bilirubin > 2xULN (P < 0.001; median: 4.0 vs 0.8 years; see Figure 1).