Ursodeoxycholic Acid for Treatment of Primary Sclerosing Cholangitis: A Placebo–Controlled Trial

作者：Ulrich H. Beuers, Ulrich Spengler, Wolfgang Kruis, Ü. Aydemir, Baldur Wiebecke, Walter Heldwein, Marlene Weinzierl, Gerd R. Pape, Tilman Sauerbruch, Gustav Paumgartner · 发表于：Hepatology · 年份：1992 · DOI：10.1002/hep.1840160315 · 被引用次数：463 · 研究领域：Liver Diseases and Immunity、Liver Disease Diagnosis and Treatment、Pediatric Hepatobiliary Diseases and Treatments

The efficacy and safety of ursodeoxycholic acid for the treatment of primary sclerosing cholangitis were evaluated in a prospective, randomized, double-blind, placebo-controlled trial. Fourteen patients with primary sclerosing cholangitis documented by cholestatic serum enzyme pattern, liver histological appearance and endoscopic retrograde cholangiography were included in the trial. Six patients received ursodeoxycholic acid (13 to 15 mg/kg body wt/day), and eight patients received placebo. Two patients had to be withdrawn from the study, one because of UDCA-related diarrhea and the other because of worsening of the disease during placebo treatment. Patients in the ursodeoxycholic acid group improved significantly during 1 yr of treatment with respect to serum levels of bilirubin (median = -50%), alkaline phosphatase (median = -67%), gamma-glutamyltransferase (median = -53%), AST (median = -54%) and ALT (median = -36%) compared with the placebo group, but not with respect to serum levels of hydrophobic bile acids. During ursodeoxycholic acid treatment, histopathological features also improved significantly, as evaluated by multiparametric score. Expression of human leukocyte antigen class I molecules appeared to be markedly reduced on liver cells after ursodeoxycholic acid treatment. We conclude that ursodeoxycholic acid is beneficial in reducing disease activity in patients with primary sclerosing cholangitis.