| Specific Conditions |
|
| Indication for Ig Use |
|
| Level of Evidence | Evidence of probable benefit – more research needed (Category 2a) |
| Description and Diagnostic Criteria |
Neonatal haemochromatosis (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 percent 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 percent. Prognosis About 20 percent 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 neonatal haemochromatosis (NH) with historical controls (randomly selected previously affected pregnancies). All 15 pregnancies resulted in live births. NH was diagnosed in 12, but responded to medical treatment. By contrast, there were two successful outcomes in controls (Whitington & Hibbard 2004). Rand et al (2009) describes successful treatment of NH in neonates using exchange transfusion and IVIg. |
| Diagnosis Requirements |
A diagnosis must be made by any medical officer. |
| Qualifying Criteria for Ig Therapy |
Pregnant women who have had a previous pregnancy affected by neonatal haemochromatosis
Neonate with neonatal haemochromatosis
This indication relates only to treatment of neonatal haemochromatosis in patients up to 6 months of age. Pregnant women who have had a previous pregnancy affected by neonatal haemochromatosis are eligible under the indication pregnant women who have had a previous pregnancy affected by neonatal haemochromatosis.
|
| Review Criteria for Assessing the Effectiveness of Ig Use |
Pregnant women who have had a previous pregnancy affected by neonatal haemochromatosis
Review is not mandated for this indication however the following criteria may be useful in assessing the effectiveness of therapy.
Neonate with neonatal haemochromatosis
This indication relates only to treatment of neonatal haemochromatosis in patients up to 6 months of age. Pregnant women who have had a previous pregnancy affected by neonatal haemochromatosis are eligible under the indication pregnant women who have had a previous pregnancy affected by neonatal haemochromatosis.
|
| Dose |
Pregnant women who have had a previous pregnancy affected by neonatal haemochromatosis
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 on dose, administration and contraindications. Neonate with neonatal haemochromatosis
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 on dose, administration and contraindications. |
| Bibliography |
|---|
Flynn DM, Mohan N, McKiernan P et al (2003) 'Progress in treatment and outcome for children with neonatal haemochromatosis', Archives of Disease in Childhood – Fetal Neonatal Edition, 88(2):F124–7, https://doi.org/10.1136/fn.88.2.f124. Knisely AS, Mieli-Vergani G & Whitington PF (2003) 'Neonatal haemochromatosis', Gastroenterology Clinics of North America, 32(3):877–89, vi–vii, https://doi.org/10.1016/s0889-8553(03)00050-5. Rand EB, Karpen SJ, Kelly S et al (2009) 'Treatment of hemochromatosis with exchange transfusion and intravenous immunoglobulin', Journal of Pediatrics, 155(4):566–71, https://doi.org/10.1016/j.jpeds.2009.04.012. Rodriguez F, Kallas M, Nash R et al (2005) 'Neonatal haemochromatosis – medical treatment vs. transplantation: the king’s experience', Liver Transplantation, 11(11):1417–24, https://doi.org/10.1002/lt.20497. Schneider BL (1996) 'Neonatal liver failure', Current Opinion in Pediatrics, 8(5):495–501, https://doi.org/10.1097/00008480-199610000-00013. Whittington PF & Hibbard JU (2004) 'High-dose immunoglobulin during pregnancy for recurrent neonatal haemochromatosis', Lancet, 364(9446):1690–8, https://doi.org/10.1016/s0140-6736(04)17356-x. Whittington PF, Kelly S & Ekong UD (2005) 'Neonatal haemochromatosis: fetal liver disease leading to liver failure in the fetus and newborn', Pediatric Transplantation, 9(5):640–5, https://doi.org/10.1111/j.1399-3046.2005.00357.x. |