Published online November 1, 2006
PEDIATRICS Vol. 118 No. 5 November 2006, pp. 2060-2065 (doi:10.1542/peds.2006-0908)

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ARTICLE

Neonatal Hemochromatosis: Long-term Experience With Favorable Outcome

Enke Grabhorn, MD^a, Andrea Richter, MD^a, Martin Burdelski, PhD^a, Xavier Rogiers, PhD^b and Rainer Ganschow, PhD^a

^a Departments of Pediatrics
^b Hepatobiliary Surgery, University Medical Center Hamburg-Eppendorf, Hamburg, Germany

OBJECTIVE. Neonatal hemochromatosis is a severe, often fatal multiorgan disorder of iron metabolism. Liver transplantation can be curative; the benefit of antioxidant treatment is discussed controversially. We summarize our experience with neonatal hemochromatosis over the past 13 years.

METHODS. A retrospective study was performed of 16 patients with acute liver failure attributable to neonatal hemochromatosis between 1992 and 2004.

RESULTS. Median age at the onset of neonatal hemochromatosis was 2 days (range: 0–21 days). Median weight at the time of diagnosis was 2900 g (range: 1520–4200 g). All patients had elevated ferritin levels (median: 4179 µg/L), and transferrin saturation (median: 99%). Fourteen patients (87.5%) showed significant hepatocyte siderosis in biopsies; 4 children had additional iron deposition in extrahepatic tissue. Four patients were diagnosed by MRI. Seven infants received liver transplants, 5 of them in combination with a preceding antioxidant treatment. Four children (25%) received antioxidants without the necessity of liver transplantation and were in good clinical condition at the time of this evaluation. Five patients (31.3%) died, 3 of them without any treatment because of initial fulminant multiorgan failure. In September 2005, 68.7% of the patients were still alive after a median follow-up of 5 years.

CONCLUSIONS. Neonatal hemochromatosis is a severe metabolic disease, but early antioxidant treatment and liver transplantation in addition to optimal medical care can improve the outcome dramatically. Children with moderate liver failure can survive without liver transplantation, but should be monitored closely for deterioration.

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Key Words: neonatal hemochromatosis • liver transplantation • infants • outcome

Abbreviations: NH—neonatal hemochromatosis

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Accepted Jun 29, 2006.

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