title: Medical and surgical treatment of neonatal hemochromatosis: single center experience.
abstract: NH is a rare disorder of iron storage in newborns resulting in rapid liver failure. Outcomes are dismal with 20-30% survival. We report our experience in eight children with NH. Assessment of liver function included admission PT and serum levels of FV and FVII. Medical treatment (antioxidant cocktail) was started in all patients, with chelation therapy in six. Of these six, three survived with medical treatment alone. The other three underwent liver transplant. One died 158 days after transplant to sepsis: two are well more than five yr after transplant. The two neonates who did not receive chelation therapy, died to multi-organ failure and sepsis. In summary, five children (62.5%) survived long-term. In the three transplanted, one- and five-yr-survival was 66%. Older children with compromised synthetic liver function (FVII levels < or = 15%) required liver replacement for survival. Early referral to a tertiary care center is essential to increase survival of these children with a rare and otherwise fatal disease. Single center experience of children with NH is here presented. Potentials for survival improvement with of medical and surgical treatment are examined.
authors.affiliation: Children's Healthcare of Atlanta at Egleston, Pediatric Liver Transplantation, Atlanta, GA, USA.
authors.forename: Thomas
authors.initials: T
authors.lastname: Heffron
authors.forename: Todd
authors.initials: T
authors.lastname: Pillen
authors.forename: David
authors.initials: D
authors.lastname: Welch
authors.forename: Massimo
authors.initials: M
authors.lastname: Asolati
authors.forename: Gregory
authors.initials: G
authors.lastname: Smallwood
authors.forename: Phil
authors.initials: P
authors.lastname: Hagedorn
authors.forename: Carlos
authors.initials: C
authors.lastname: Fasola
authors.forename: David
authors.initials: D
authors.lastname: Solis
authors.forename: John
authors.initials: J
authors.lastname: Rodrigues
authors.forename: Jill
authors.initials: J
authors.lastname: DePaolo
authors.forename: James
authors.initials: J
authors.lastname: Spivey
authors.forename: Enrique
authors.initials: E
authors.lastname: Martinez
authors.forename: Stuart
authors.initials: S
authors.lastname: Henry
authors.forename: Rene
authors.initials: R
authors.lastname: Romero
mesh_terms: D000975:Antioxidants / Q000627:therapeutic use*; D015913:Chelation Therapy / Q000379:methods*; D003676:Deferoxamine / Q000627:therapeutic use*; D018450:Disease Progression; D004359:Drug Therapy, Combination; D005167:Factor VII / Q000378:metabolism; D005260:Female; D005500:Follow-Up Studies; D006084:Graft Rejection / Q000175:diagnosis / Q000453:epidemiology / Q000517:prevention & control; D006432:Hemochromatosis / Q000097:blood / Q000150:complications / Q000628:therapy*; D006801:Humans; D015994:Incidence; D007223:Infant; D007231:Infant, Newborn; D017093:Liver Failure / Q000453:epidemiology / Q000209:etiology / Q000517:prevention & control; D016031:Liver Transplantation / Q000379:methods*; D008279:Magnetic Resonance Imaging; D008297:Male; D012189:Retrospective Studies; D012720:Severity of Illness Index; D017262:Siderophores / Q000627:therapeutic use*; D015996:Survival Rate; D013997:Time Factors; D016896:Treatment Outcome; D014481:United States / Q000453:epidemiology
chemical_list: D000975:Antioxidants; D017262:Siderophores; D005167:Factor VII; D003676:Deferoxamine