Neonatal hemochromatosis: Outcomes of pharmacologic and surgical therapies

Luther Sigurdsson, Jorge Reyes, Samuel A. Kocoshis, Thor W.R. Hansen, Joel Rosh, A. S. Knisely

Research output: Contribution to journalArticle

89 Citations (Scopus)

Abstract

Background: Neonatal hemochromatosis (NH), also known as perinatal hemochromatosis or neonatal iron storage disease, is a disorder in fetuses and newborn infants. A retrospective study was conducted to report management of patients with NH. Methods: Retrospective analysis was conducted by chart review and by review of histologic material from patients with NH. Results: Neonatal hamochromatosis was diagnosed in 14 patients between 1985 and 1995. All were considered for orthotopic liver transplantation (OLTX). From 1993 onward, all patients were treated with an antioxidant-chelation 'cocktail,' consisting of deferoxamine, vitamin E, N-acetylcysteine, selenium, and prostaglandin-E1. Of 6 patients with NH diagnosed before 1993, 4 underwent OLTX; only 1 is still alive. Of 8 patients with NH diagnosed after 1993 and treated with the cocktail, 7 expired before OLTX. One stabilized on therapy, but having never recovered full synthetic liver function, underwent OLTX and is now alive and well. Conclusion: Neonatal hemochromatosis carries a grim prognosis; however, successful OLTX is curative. The use of an antioxidant- chelation cocktail did not improve outcome in the patients studied. Earlier (perinatal) diagnosis may be required for optimal results. Further study of other interventions, including antenatal diagnosis and earlier institution or modification of cocktail therapy appears warranted.

Original languageEnglish (US)
Pages (from-to)85-89
Number of pages5
JournalJournal of Pediatric Gastroenterology and Nutrition
Volume26
Issue number1
DOIs
StatePublished - Jan 1 1998
Externally publishedYes

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Therapeutics
Antioxidants
Deferoxamine
Hemochromatosis
Alprostadil
Acetylcysteine
Selenium
Neonatal hemochromatosis
Prenatal Diagnosis
Vitamin E
Liver Transplantation
Early Diagnosis
Fetus
Retrospective Studies
Newborn Infant
Liver

All Science Journal Classification (ASJC) codes

  • Pediatrics, Perinatology, and Child Health
  • Gastroenterology

Cite this

Sigurdsson, Luther ; Reyes, Jorge ; Kocoshis, Samuel A. ; Hansen, Thor W.R. ; Rosh, Joel ; Knisely, A. S. / Neonatal hemochromatosis : Outcomes of pharmacologic and surgical therapies. In: Journal of Pediatric Gastroenterology and Nutrition. 1998 ; Vol. 26, No. 1. pp. 85-89.
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Neonatal hemochromatosis : Outcomes of pharmacologic and surgical therapies. / Sigurdsson, Luther; Reyes, Jorge; Kocoshis, Samuel A.; Hansen, Thor W.R.; Rosh, Joel; Knisely, A. S.

In: Journal of Pediatric Gastroenterology and Nutrition, Vol. 26, No. 1, 01.01.1998, p. 85-89.

Research output: Contribution to journalArticle

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