TABLE 1.
General characteristics of the study cohort
| Characteristic | N = 100 |
|---|---|
| Socio‐demographics and comorbidities | |
| Sex, female | 95 (95%) |
| Age at OCA start, years | 62 (54, 67) |
| Age at PBC diagnosis, years | 52 (43, 56) |
| Duration of disease before OCA start, years | 9 (5, 15) |
| Body Mass Index, Kg/m2 | 24.6 (22.0, 26.9) |
| Diabetes mellitus | 12 (12%) |
| Liver disease characterization | |
| AMA positivity | 83 (83%) |
| ANA positivity | 52 (52%) |
| PBC‐AIH overlap | 14 (14%) |
| Diagnosis of cirrhosis | |
| Clinical a | 69 (69%) |
| Histological | 24 (24%) |
| Elastographic b | 7 (7%) |
| Child‐Pugh class | |
| A c | 97 (97%) |
| B | 3 (3%) |
| C | 0 (0%) |
| MELD | 6.9 (6.4, 8.5) |
| Ascites | |
| Absent | 95 (95%) |
| Controlled with diuretics | 4 (4%) |
| Present | 1 (1%) |
| Hepatic encephalopathy | 0 (0%) |
| Oesophageal varices, presence | 31 (31%) |
| Gastroscopy not performed | 16 (16%) |
| OCA therapy | |
| Indication to OCA start | |
| UDCA intolerance | 0 (0%) |
| Inadequate response to UDCA | 100 (100%) |
| acc. to Paris I criteria | 60 (60%) |
| acc. to Paris II criteria | 98 (98%) |
| acc. to Toronto criteria | 79 (79%) |
| OCA regimen | |
| 5 mg daily | 65 (65%) |
| 5 mg daily uptitrated to 10 mg | 20 (20%) |
| 5 mg every other day uptitrated to 5 mg daily | 4 (4.0%) |
| 5 mg weekly | 4 (4.0%) |
| 5 mg 3 times a week uptitrated to 5 mg daily | 3 (3.0%) |
| 5 mg every other day | 2 (2.0%) |
| 5 mg twice a week | 2 (2.0%) |
| Concomitant/Previous therapies | |
| UDCA dose, mg/kg | 15.00 (15.00, 17.04) |
| Fibrate therapy | |
| NO | 81 (81%) |
| Before and stopped before OCA start | 8 (8.0%) |
| Before and continued during OCA therapy | 7 (7.0%) |
| After OCA start | 3 (3.0%) |
| After OCA discontinuation | 1 (1.0%) |
| Biochemical | |
| ALP/ULN at baseline | 2.10 (1.72, 2.89) |
| ALT/ULN at baseline | 1.07 (0.78, 1.76) |
| AST/ULN at baseline | 1.23 (0.90, 1.83) |
| GGT/ULN at baseline | 4.5 (2.8, 7.0) |
| Total Bilirubin/ULN at baseline | 0.90 (0.70, 1.21) |
| Platelets (x109/L) | 152 (120, 206) |
| Albumin, g/dl | 4.00 (3.60, 4.24) |
| INR | 1.00 (0.97, 1.10) |
| Creatinine, mg/dl | 0.70 (0.60, 0.80) |
Note: Data reported as median with interquartile range or as numbers with percentages.
Paris I criteria: ALP <3x ULN, ALT <2x ULN and bilirubin <1 mg/dl. Paris II criteria: ALP <1.5x ULN, ALT <1.5x ULN and bilirubin <1 mg/dl. Toronto criteria: ALP <1.67x ULN.
Abbreviations: Acc, according; AIH, autoimmune hepatitis; ALP, alkaline phosphatase; ALT, alanine transferase; AMA, antimitochondrial antibodies; ANA, antinuclear antibodies; AST, aspartate transferase; GGT, gamma‐glutamyl transferase; INR, international normalized ratio; MELD, model for end‐stage liver disease; PBC, primary biliary cholangitis; UDCA, ursodeoxycholic acid; ULN, upper limit of normal.
By ultrasonography, all these 69 subjects had morphologic signs specific for liver cirrhosis, and 41 had also ultrasonographic signs specific for portal hypertension (as specified in Methods section).
Fibroscan ≥16.9 KPa.
Out of 97 subjects with Child‐Pugh class A, 80 and 17 had a Child‐Pugh score of 5 and 6 respectively.