Faculty Collaboration Database

Treatment of neonatal hemochromatosis with exchange transfusion and intravenous immunoglobulin. J Pediatr 2009 Oct;155(4):566-71

Date

06/30/2009

Pubmed ID

19560784

DOI

10.1016/j.jpeds.2009.04.012

Scopus ID

2-s2.0-70249151112 (requires institutional sign-in at Scopus site)   127 Citations

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.

STUDY DESIGN: 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.

Author List

Rand EB, Karpen SJ, Kelly S, Mack CL, Malatack JJ, Sokol RJ, Whitington PF

Author

Cara Lynn Mack MD Chief, Professor in the Pediatrics department at Medical College of Wisconsin

MESH terms used to index this publication - Major topics in bold

Cohort Studies
Exchange Transfusion, Whole Blood
Female
Hemochromatosis
Humans
Immunoglobulins, Intravenous
Immunologic Factors
Infant, Newborn
Liver Failure, Acute
Male
Retrospective Studies
Survival Rate
Treatment Outcome