title: Treatment of neonatal hemochromatosis with exchange transfusion and intravenous immunoglobulin.
abstract: OBJECTIVE
  To determine if immunomodulatory treatment including intravenous immunoglobulin (IVIG) can favorably affect survival in neontatal hemochromatosis (NH) diagnosed postnatally because it can effectively prevent occurrence of NH when applied during gestations at risk.
  
  
  METHODS
  We treated 16 newborn infants with liver failure due to NH with high-dose IVIG, in combination with exchange transfusion in 13 (ET/IVIG), and compared the outcome with 131 historical controls treated conventionally.
  
  
  RESULTS
  The severity of liver disease as estimated by prothrombin time was similar in the subjects receiving ET/IVIG and the historical controls, and the medical therapy was equivalent with the exception of the ET/IVIG therapy. Twelve subjects (75%) had good outcome, defined as survival without liver transplantation, whereas good outcome was achieved in only 17% (23/131) of historical control patients (P < .001). Four subjects died, 2 without and 2 after liver transplant. Survivors were discharged 6 to 90 days after receiving ET/IVIG therapy, and those followed for more than 1 year are within normal measures for growth, development, and liver function.
  
  
  CONCLUSIONS
  Immune therapy with ET/IVIG appears to improve the outcome and reduce the need for liver transplantation in patients with NH.
authors: Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
authors: Elizabeth B
authors: EB
authors: Rand
authors: Saul J
authors: SJ
authors: Karpen
authors: Susan
authors: S
authors: Kelly
authors: Cara L
authors: CL
authors: Mack
authors: J Jeffrey
authors: JJ
authors: Malatack
authors: Ronald J
authors: RJ
authors: Sokol
authors: Peter F
authors: PF
authors: Whitington
mesh_terms: D015331:Cohort Studies; D005078:Exchange Transfusion, Whole Blood; D005260:Female; D006432:Hemochromatosis / Q000150:complications / Q000401:mortality / Q000628:therapy*; D006801:Humans; D016756:Immunoglobulins, Intravenous / Q000627:therapeutic use*; D007155:Immunologic Factors / Q000627:therapeutic use*; D007231:Infant, Newborn; D017114:Liver Failure, Acute / Q000209:etiology / Q000401:mortality / Q000628:therapy*; D008297:Male; D012189:Retrospective Studies; D015996:Survival Rate; D016896:Treatment Outcome
chemical_list: D016756:Immunoglobulins, Intravenous; D007155:Immunologic Factors