Delta 4-3-oxosteroid 5 beta-reductase deficiency causing neonatal liver failure and hemochromatosis

J Pediatr. 1994 Feb;124(2):234-8. doi: 10.1016/s0022-3476(94)70310-8.

Abstract

Neonatal liver failure was evaluated in two infants. Neither infant had evidence of congenital infection, galactosemia, alpha 1-antitrypsin deficiency, tyrosinemia, Zellweger syndrome, or hemophagocytic lymphohistiocytosis. Abnormal levels of iron were detected in the minor salivary glands of the first infant and in the explanted liver of the second. Analyses of urinary bile salts by fast-atom bombardment ionization mass spectrometry and gas chromatography-mass spectrometry revealed a paucity of primary bile acids and a predominance of 7 alpha-hydroxy-3-oxo-4-cholenoic and 7 alpha,12 alpha-dihydroxy-3-oxo-4-cholenoic acids. These findings are consistent with delta 4-3-oxosteroid 5 beta-reductase deficiency, a primary genetic defect in bile acid synthesis. Postmortem evaluation of the first infant revealed significant iron deposition in the liver, pancreas, thyroid, adrenal glands, myocardium, stomach, and submucosal glands of the respiratory tract. In both infants examination of the liver revealed extensive loss of hepatic parenchyma. These cases expand the clinical spectrum of bile acid metabolism defects to include neonatal liver failure with associated hemochromatosis.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Bile Acids and Salts / urine
  • Hemochromatosis / etiology*
  • Hemochromatosis / pathology
  • Hemochromatosis / urine
  • Humans
  • Infant
  • Infant, Newborn
  • Liver / pathology
  • Liver Failure / etiology*
  • Liver Failure / pathology
  • Liver Failure / urine
  • Male
  • Oxidoreductases / deficiency*

Substances

  • Bile Acids and Salts
  • Oxidoreductases
  • cholestenone 5 beta-reductase