Treatment of neonatal hemochromatosis with exchange transfusion and intravenous immunoglobulin.
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.
Rand
Elizabeth B
EB
Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Karpen
Saul J
SJ
Kelly
Susan
S
Mack
Cara L
CL
Malatack
J Jeffrey
JJ
Sokol
Ronald J
RJ
Whitington
Peter F
PF
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
D016756:Immunoglobulins, Intravenous; D007155:Immunologic Factors