Effects of 4-phenylbutyrate therapy in a preterm infant with cholestasis and liver fibrosis
| Autor: | Hisamitsu Hayashi, Ken Tanikawa, Hiroyuki Kusuhara, Hiroko Ueda, Koichi Ito, Takeshi Endo, Masayoshi Kage, Tokio Sugiura, Shinji Saitoh, Takao Togawa, Shogo Ito |
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| Rok vydání: | 2016 |
| Předmět: |
0301 basic medicine
medicine.medical_specialty business.industry Progressive familial intrahepatic cholestasis Jaundice medicine.disease Phenylbutyrate Bile Salt Export Pump Ursodeoxycholic acid 03 medical and health sciences Liver disease 030104 developmental biology 0302 clinical medicine Endocrinology Cholestasis Internal medicine Pediatrics Perinatology and Child Health medicine medicine.symptom business 030217 neurology & neurosurgery Ornithine transcarbamylase deficiency medicine.drug |
| Zdroj: | Pediatrics International. 58:506-509 |
| ISSN: | 1328-8067 |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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