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Reuters Health Information (2006-11-28): Early treatment improves long-term neonatal hemochromatosis outcomes


Early treatment improves long-term neonatal hemochromatosis outcomes

Last Updated: 2006-11-28 14:44:30 -0400 (Reuters Health)

NEW YORK (Reuters Health) - Effective treatment with antioxidants and liver transplantation improves outcomes with neonatal hemochromatosis, according to a report in the November issue of Pediatrics.

While rare, neonatal hemochromatosis is often fatal, the authors explain, but liver transplantation can often prove curative.

Dr. Enke Grabhorn and colleagues from University Medical Center Hamburg-Eppendorf, Germany evaluated short- and long-term results of 16 patients with suspected neonatal hemochromatosis who were treated with antioxidants and liver transplantation between 1992 and 2004.

All 16 patients had elevated ferritin, transferrin saturation, and bilirubin levels and reduced transferrin levels, the authors report, and most had elevated levels of alanine aminotransferase and aspartate aminotransferase and decreased albumin levels.

All 7 infants who received liver transplants survived the first 2 years after transplantation, the results indicate. Five children received antioxidant treatment, and 4 of those remained well without requiring a liver transplantation.

Five patients died (3 without treatment), the researchers note, and 11 remained alive after a median follow-up of 5 years, resulting in survival rates of 75% after 1 year and 69.2% at 2 years.

"Neonatal hemochromatosis is a severe metabolic disease, but early antioxidant treatment and liver transplantation can result in a favorable outcome," the investigators conclude. "To achieve optimal results an early referral to an experienced transplant center is mandatory."

Pediatrics 2006;118:2060-2065.

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