Effects of 4-phenylbutyrate therapy in a preterm infant with cholestasis and liver fibrosis

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• 作者：Shogo Ito ; Hisamitsu Hayashi ; Tokio Sugiura ; Koichi Ito ; Hiroko Ueda ; Takao Togawa ; Takeshi Endo ; Ken Tanikawa ; Masayoshi Kage ; Hiroyuki Kusuhara and Shinji Saitoh
• 刊名：Pediatrics International
• 出版年：2016
• 出版时间：June 2016
• 年：2016
• 卷：58
• 期：6
• 页码：506-509
• 全文大小：3448K
• ISSN：1442-200X

文摘

The bile salt export pump is expressed at the canalicular membrane of hepatocytes and mediates biliary excretion of bile salts. 4-Phenylbutyrate (4 PB), a drug used to treat ornithine transcarbamylase deficiency, has been found to increase the hepatocanalicular expression of bile salt export pump. The beneficial effects of 4-phenylbutyrate therapy have been reported for patients with progressive familial intrahepatic cholestasis, an inherited autosomal recessive liver disease. This is the first study to show the therapeutic effect of 4 PB in a preterm infant with cholestasis and liver fibrosis. The preterm infant had severe cholestasis with jaundice and failure to thrive refractory to ursodeoxycholic acid. Histology indicated giant cell hepatitis, cholestasis, and severe fibrosis. Bile salt export pump immunostaining showed lower expression than in a control. Oral 4 PB was started at a daily dose of 200 mg/kg/day. After the start of 4 PB therapy, cholestasis improved.