Cyclosporin A treatment in primary biliary cirrhosis: results of a long-term placebo controlled trial.
BACKGROUND
Effective treatment for primary biliary cirrhosis (PBC) resulting in slower progression and improved survival remains elusive. Cyclosporin A (CyA), which has been so effective in preventing human allograft rejection, has shown promise in small numbers of patients in early studies.


METHODS
Three hundred forty-nine patients with PBC were randomized to receive CyA, 3 mg.kg-1.day-1, or placebo in a multicenter study with follow-up for 6 years. The end point was death or liver transplantation.


RESULTS
Cox multivariate analysis showed time from entry to death or transplantation was significantly prolonged (by up to 50%) in the CyA-treated group. Liver-related mortality was also significantly lower. However, a univariate analysis of survival showed no statistical differences between the two groups. Biochemical liver indices deteriorated more slowly in the CyA-treated group, but serum creatinine concentration was elevated > 150 mumol/L in 9%, necessitating permanent discontinuation in half of these. A reduction in the dose of CyA was required in 11% because of hypertension.


CONCLUSIONS
CyA has some therapeutic potential in primary biliary cirrhosis, providing blood pressure and renal function are closely monitored.
Lombard
M
M
Institute of Liver Studies, King's College Hospital, London, England.
Portmann
B
B
Neuberger
J
J
Williams
R
R
Tygstrup
N
N
Ranek
L
L
Ring-Larsen
H
H
Rodes
J
J
Navasa
M
M
Trepo
C
C
D000328:Adult; D000368:Aged; D016572:Cyclosporine / Q000627:therapeutic use*; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008105:Liver Cirrhosis, Biliary / Q000188:drug therapy* / Q000401:mortality; D008297:Male; D008875:Middle Aged; D011379:Prognosis; D015996:Survival Rate
D016572:Cyclosporine