Table 1.
Characteristics of the 2 recent positive pivotal trials of second-line therapy for PBC.
| POISE Study | BEZURSO Study | |
|---|---|---|
| Study design | ||
| Drug | Obeticholic acid | Bezafibrate |
| Arms | 5-10 mg/10 mg/placebo | 400 mg/placebo |
| Randomization | 1:1:1 | 1:1 |
| Entry criterion | Toronto (rev.) non-response | Paris-2 non-response |
| Double-blind period | 12 months | 24 months |
| Primary outcome | ALP decrease ≥15% from baseline, ALP ≪1.67x ULN and normal bilirubin | Normal levels of total bilirubin, ALP, AST, ALT, albumin, and PI |
| Study population at baseline | ||
| Patients enrolled | 217 | 100 |
| Age (year) | 56 | 53 |
| Pruritus | 59% | 66% |
| Total bilirubin (mg/dl) | 0.65 | 0.78 |
| ALP Level (U/L) | 323 | 277 |
| LSM (kPa) | 11.6 | 12.1 |
| Cirrhosis | 19% | 21% |
| UDCA-untreated patients | Yes (7%) | No |
| Study outcomes | ||
| Completed study | 91% | 92% |
| Primary outcome (per arm) | 46%/47%/10% | 31%/0% |
| Normal ALP level (per arm) | N/A | 67%/2% |
| OR of Paris-2 response | 9.1/8.5 | 21.7 |
| Reduction in pruritus | No | Yes |
| Reduction in LSM | No | Yes |
| Reduction in ELF score | No | Yes |
| Significant side effects | Pruritus | 5% creatinine increase |
Data are expressed as number, % or mean. ALP, alkaline phosphatase; ALT, alanine aminotransferase; AST, aspartate aminotransferase; ELF™, Enhanced Liver Fibrosis; LSM, liver stiffness measurement; OR, odds ratio; PI, prothrombin index (a derived measure of prothrombin time); ULN, upper limit of normal. Toronto (rev.) non-response: ALP ≫1.67x ULN or abnormal total bilirubin. Paris-2 non-response: ALP ≫1.5x ULN or AST ≫1.5x ULN or abnormal total bilirubin.