title: Effects of 4-phenylbutyrate therapy in a preterm infant with cholestasis and liver fibrosis.
abstract: 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.
authors: Department of Pediatrics and Neonatology, Graduate School of Medical Sciences, Nagoya City University, Nagoya, Japan.
authors: Shogo
authors: S
authors: Ito
authors: Laboratory of Molecular Pharmacokinetics, Graduate School of Pharmaceutical Sciences, University of Tokyo, Tokyo, Japan.
authors: Hisamitsu
authors: H
authors: Hayashi
authors: Department of Pediatrics and Neonatology, Graduate School of Medical Sciences, Nagoya City University, Nagoya, Japan.
authors: Tokio
authors: T
authors: Sugiura
authors: Department of Pediatrics and Neonatology, Graduate School of Medical Sciences, Nagoya City University, Nagoya, Japan.
authors: Koichi
authors: K
authors: Ito
authors: Department of Pediatrics and Neonatology, Graduate School of Medical Sciences, Nagoya City University, Nagoya, Japan.
authors: Hiroko
authors: H
authors: Ueda
authors: Department of Pediatrics and Neonatology, Graduate School of Medical Sciences, Nagoya City University, Nagoya, Japan.
authors: Takao
authors: T
authors: Togawa
authors: Department of Pediatrics and Neonatology, Graduate School of Medical Sciences, Nagoya City University, Nagoya, Japan.
authors: Takeshi
authors: T
authors: Endo
authors: Department of Diagnostic Pathology, Kurume University Hospital, Fukuoka, Japan.
authors: Ken
authors: K
authors: Tanikawa
authors: Department of Diagnostic Pathology, Kurume University Hospital, Fukuoka, Japan.
authors: Masayoshi
authors: M
authors: Kage
authors: Laboratory of Molecular Pharmacokinetics, Graduate School of Pharmaceutical Sciences, University of Tokyo, Tokyo, Japan.
authors: Hiroyuki
authors: H
authors: Kusuhara
authors: Department of Pediatrics and Neonatology, Graduate School of Medical Sciences, Nagoya City University, Nagoya, Japan.
authors: Shinji
authors: S
authors: Saitoh
keywords: 4-phenylbutyrate; bile salt export pump; cholestasis; idiopathic neonatal hepatitis; preterm infant