This medical condition has either been superseded or has become inactive
| Specific Conditions |
|
| Indication for Ig Use |
|
| Level of Evidence | Evidence of probable benefit – more research needed (Category 2a) |
| Description and Diagnostic Criteria |
NH manifests in the fetus and newborn and is characterised by abnormal accumulation of iron in the liver and extra-hepatic tissues. Affected neonates present with fulminant liver failure, usually in the context of a history of prematurity, intrauterine growth retardation and oligohydramnios. NH differs from most other causes of neonatal liver disease, other than congenital infections, in that the condition begins in utero and fulminant liver disease is manifested in the first few days of life. The aetiology and pathogenesis remains uncertain. The NH phenotype may be the outcome of numerous disease processes. There is also evidence, however, that NH is an alloimmune disorder. First, there is an approximate 80% likelihood of NH once a woman has an affected baby. Second, mothers can have affected babies with different fathers. It has not been described that fathers can have affected half-siblings with different mothers. Symptoms and signs Affected neonates present with signs of liver failure, including extreme cholestasis, hypoalbuminaemia, coagulopathy, ascites and hypoglycaemia. Diagnosis of neonatal haemochromatosis is made after other causes of neonatal liver failure have been ruled out. In addition to extensive iron deposition (siderosis), liver biopsy would show cirrhosis with diffuse fibrosis, bile duct proliferation, and giant cells. Siderosis is also present in other tissues and viscera (e.g. epithelial tissues and the heart), but not in reticuloendothelial cells. Occurrence NH is a rare disease, but the rate of recurrence after the index case in a sibship is up to 80%. Prognosis About 20% survival with medical treatment. |
| Justification for Evidence Category |
A trial compared the impact of intravenous immunoglobulin (IVIg) on pregnancy outcome of women whose most recent pregnancy had resulted in NH with historical controls (randomly selected previously affected pregnancies). All 15 pregnancies resulted in live births. NH was diagnosed in 11, but responded to medical treatment. By contrast, there were two successful outcomes in controls (Biotext 2004). |
| Qualifying Criteria for Ig Therapy |
|
| Review Criteria for Assessing the Effectiveness of Ig Use |
Clinical effectiveness of Ig therapy may be demonstrated by:
|
| Dose |
The aim should be to use the lowest dose possible that achieves the appropriate clinical outcome for each patient. Refer to the current product information sheet for further information. |
| Bibliography |
|---|
| Biotext 2004, ‘Summary data on conditions and papers’, in A systematic literature review and report on the efficacy of intravenous immunoglobulin therapy and its risks, commissioned by the National Blood Authority on behalf of all Australian Governments. Available from: http://www.nba.gov.au/pubs/pdf/report-lit-rev.pdf Flynn, DM, Mohan, N, McKiernan, P, et al 2003‚ 'Progress in treatment and outcome for children with neonatal haemochromatosis’, Archives of Disease in Childhood — Foetal Neonatal Edition, vol. 88, no. 2, pp. F124–7. Knisely, AS, Mieli-Vergani, G & Whitington, PF 2003, ‘Neonatal haemochromatosis’, Gastroenterology Clinics of North America, vol. 32, no. 3, pp. 877–89, vi–vii. Rodriguez, F, Kallas, M, Nash, R, et al 2005, ‘Neonatal haemochromatosis — medical treatment vs. transplantation: the King’s experience’, Liver Transplantation, vol. 11, no. 11, pp. 1417–24. Schneider, BL 1996, ‘Neonatal liver failure’, Current Opinion in Paediatrics, vol. 8, pp. 495–501. Whittington, PF & Hibbard, JU 2004, ‘High-dose immunoglobulin during pregnancy for recurrent neonatal haemochromatosis’, Lancet, vol. 364, pp. 1690–8. Whittington, PF, Kelly, S & Ekong, UD 2005, ‘Neonatal haemochromatosis: foetal liver disease leading to liver failure in the foetus and newborn’, Paediatric Transplantation, vol. 9, pp. 640–5. |