Specialist Working Group for Haematology Proposed changes to the Criteria for the clinical use of intravenous immunoglobulin in Australia, Second Edition ITEM CRITERIA FOR THE CLINICAL USE OF INTRAVENOUS IMMUNOGLOBULIN IN AUSTRALIA, SECOND EDITION (CRITERIA) PROPOSED REVISIONS TO THE CRITERIA SWG RATIONALE FOR PROPOSED CHANGE (A) Administrative) (B) Progressive (C) Programmed Condition Name Neonatal haemochromatosis (NH) Neonatal haemochromatosis (NH) Specialty Haematology Haematology Chapter 5 5 Specific Conditions Level of Evidence Evidence of probable benefit (). Evidence of probable benefit (). Justification for Evidence Category A trial compared the impact of 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 2 successful outcomes in controls (Biotext 2004). 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). Rand et al (2009) describes successful treatment of NH in neonates using exchange transfusion and IVIg. Addition of evidence with successful use of IVIg together with exchange transfusion. (A) Description and Diagnostic Criteria NH manifests in the foetus 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, Ne NH manifests in the foetus and newborn, and is characterised by abnormal accumulation of iron in the and extra-hepatic tissues. Affected neonates present with fulminant liver failure, usually in the context of a history of
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. 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.