Table 2.
Characteristics of trials of immunosuppressant agents in PSC.
| Agent | Study | Year | Design | Treatment (Tx) | Tx n | Control (C) | C n | Trial duration (months) | Liver biochemistry improvement | Symptomatic improvement | Liver histology improvement | Cholangiographic improvement | Progression to end-stage liver disease | Transplant/survival |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Azathioprine | Schramm et al. | 1999 | Case series | Azathioprine | 15 | N/A | N/A | 41 | N | Not done | Y | N | Not done | Not done |
| 1–1.5 mg/kg/day | ||||||||||||||
| Prednisolone 1 mg/kg/day | ||||||||||||||
| UDCA | ||||||||||||||
| 500–750 mg/day | ||||||||||||||
| Cyclosporin | Wiesner et al. | 1991 | DB, PC | Cyclosporin | 20 | Placebo | 10 | N\R | N | N | Y | Not done | N | N |
| Sandborn et al. | 1993 | DB, RCT | Cyclosporin 4.1 mg/kg/day | 19 | Placebo | 11 | 12 | N | N | N | N | N | N | |
| Methotrexate | Knox and Kaplan | 1991 | OL | Methotrexate 0.2 mg/kg/week | 10 | N/A | N/A | 12 | Y | Not done | Y | Y | Not done | Not done |
| Knox and Kaplan | 1994 | PC | Methotrexate | 12 | Placebo | 12 | 24 | N | Not done | N | N | Not done | Not done | |
| Tacrolimus | Van Thiel et al. | 1995 | OL | Tacrolimus 3 mg twice daily | 10 | N/A | N/A | 3 | Y | Not done | Not done | Not done | Not done | Not done |
| Talwalkar et al. | 2007 | OL | Tacrolimus 0.05 mg/kg twice daily | 16 | N/A | N/A | 12 | Y | Y | Not done | Not done | Not done | Not done |
N/A, not applicable; DB, double blind; PC, placebo controlled; N/R, not reported; RCT, randomized controlled trial; OL, open label; C, control; Tx, treatment; n, number.