Early assessment of treatment response in primary biliary cholangitis: key to timely management

The aim of our study was to explore predictive factors associated with compete biochemical response (CBR) in primary biliary cholangitis (PBC) patients treated with ursodeoxycholic acid (UDCA) at month 12 and at last check-up; CBR was defined as both normal bilirubin and ALP levels. We also evaluated hepatic decompensation and prognosis during UDCA treatment.

We conducted a multicenter retrospective study of PBC patients. We enrolled patients with PBC before the beginning of UDCA treatment (13-15 mg/kg body weight per day) between 1999 and 2024 in 2 hepatology centers in Eastern Slovakia.

We enrolled 155 patients in the final analysis, 147 women and 8 men, mean age at diagnosis 57 ± 15 years, the median follow-up was 10 ± 8 years. 29 patients (18.7%) had cirrhosis at diagnosis. Hepatic decompensation occurred in 12 patients during follow-up (7.7%; 95%CI 4-13%). 114 patients (73.5%; 95% CI 66-80%) achieved response to treatment according to Toronto criteria at month 6; CBR after 12 months of treatment was achieved by 41.3% (95% CI 33-50%) of patients; 51 patients (32.9%; 95% CI 26-41%) achieved CBR at last check-up. The OR for achieving CBR at month 12 in Toronto responders at month 6 was 46.36 (95%CI 6.147-349.646); p < 0.001. Of the patients who achieved a response defined by Toronto criteria at month 6, 44.7% of patients achieved CBR at last check-up and 55.3% did not. Out of the patients who did not achieve Toronto at month 6, none achieved CBR at last check-up (0%). Treatment response by Toronto criteria at month 6, CBR at month 12 and absence of liver cirrhosis predicted CBR at last check-up. The odds of decompensation were about 90% lower in patients who achieved treatment response. No case of hepatic decompensation or liver-related mortality during follow-up was observed in patients with CBR at last check-up.

The assessment of treatment response at month 6 is very accurate predictor of complete biochemical response throughout the course of the disease. For non-responders to UDCA treatment, the addition of second line PBC treatment is indicated. For patients who have not met Toronto criteria at month 6 of UDCA treatment, the addition of second line PBC therapy should be considered.