DEMOGRAPHIC, CLINICAL, AND BIOCHEMICAL CHARACTERISTICS IN PATIENTS WITH PRIMARY BILIARY CHOLANGITIS WITH AN UNFAVORABLE BIOCHEMICAL RESPONSE TO URSODEOXYCHOLIC ACID IN A TERTIARY CARE HOSPITAL

Introduction and Objectives

Ursodeoxycholic acid (UDCA) is the standard treatment for primary biliary cholangitis (PBC), leading to improvement in liver biochemical parameters in most patients. However, up to one-third fail to achieve an adequate biochemical response, which has been associated with more rapid progression to cirrhosis and other complications. Identifying clinical and biochemical factors associated with an unfavorable response may help optimize therapeutic strategies and enable early risk stratification.

This study aimed to identify clinical, demographic, and biochemical characteristics associated with an inadequate biochemical response to UDCA in patients with PBC.

Materials and Methods

We conducted a retrospective, observational, analytical, case-control study with longitudinal follow-up in 73 patients with confirmed PBC treated with UDCA for at least 12 months at a tertiary care hospital (2022–2024). Biochemical response was evaluated using Paris II, Barcelona, and Globe criteria. Variables analyzed included age, sex, BMI, time since diagnosis, liver enzymes (ALP, AST, ALT), bilirubin, albumin, platelet count, liver fibrosis by elastography, autoantibody profiles (AMA, SP100, GP210), and metabolic comorbidities. Statistical analysis was performed using measures of central tendency and dispersion, as well as absolute and relative frequencies. Group comparisons were conducted using Student’s t-test, with statistical significance set at p < 0.05.

Results

Among the 73 patients, 52% (n=38) were classified as non-responders. Non-responders were older (55 ± 9.2 vs. 51 ± 8.5 years; p=0.04) and had significantly higher baseline bilirubin (2.3 vs. 0.9 mg/dL; p<0.01) and ALP (361 vs. 240 U/L; p<0.01). Fibrosis on elastography was more frequent in non-responders (47% vs. 21%; p=0.01). Autoantibody positivity was also higher among non-responders (38% vs. 22%; p=0.05).

Conclusions

These results highlight key markers of unfavorable UDCA response and support the need for personalized monitoring and consideration of alternative therapies in high-risk PBC patients.

Conflict of interest: None

Clinical and Biochemical findings	Non-Responders (n = 38)	Responders (n = 35)	p-value
Mean age (years)	55 ± 9.2	51 ± 8.5	0.04
Total bilirubin (mg/dL)	2.3	0.9	<0.01
Alkaline phosphatase (U/L)	361	240	<0.01
Hepatic fibrosis (elastography)	n = 18 (47%)	n = 7 (21%)	0.01
Positive autoantibodies	n = 14 (38%)	n = 8 (22%)	0.05
Female sex	84%	86%	0.78
BMI (kg/m^2)	26.7 ± 4.3	25.9 ± 3.8	0.43
AST (U/L)	56 ± 12	54 ± 11	0.50
ALT (U/L)	49 ± 10	46 ± 9	0.28