Medical and surgical treatment of neonatal hemochromatosis: single center experience.
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.
Heffron
Thomas
T
Children's Healthcare of Atlanta at Egleston, Pediatric Liver Transplantation, Atlanta, GA, USA.
Pillen
Todd
T
Welch
David
D
Asolati
Massimo
M
Smallwood
Gregory
G
Hagedorn
Phil
P
Fasola
Carlos
C
Solis
David
D
Rodrigues
John
J
DePaolo
Jill
J
Spivey
James
J
Martinez
Enrique
E
Henry
Stuart
S
Romero
Rene
R
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
D000975:Antioxidants; D017262:Siderophores; D005167:Factor VII; D003676:Deferoxamine