Long‐term survival after liver transplantation in children with metabolic disorders

• DOI: 10.1034/j.1399-3046.2002.02009.x
• Publication Date: 2003-03-12T18:58:41Z
• Authors: Liise K. Kayler; Robert M. Merion; Samuel Lee; Randall S. Sung; Jeffrey D. Punch; Steven M. Rudich; Jeremiah G. Turcotte; Darrell A. Campbell; Ronald Holmes; John C. Magee
• Keywords: Male; 0301 basic medicine; Adolescent; 610; Infant; Pediatrics; Survival Analysis; Metabolic Disease; Liver Transplantation; 3. Good health; 03 medical and health sciences; Treatment Outcome; Metabolic Diseases; Risk Factors; Metabolic Disorders; Child, Preschool; Health Sciences; Quality of Life; Humans; Female; Life Tables; Child; Pediatric Transplantation; Retrospective Studies

Abstract: Background: Liver transplantation for inherited metabolic disorders aims to save the patient's life when disorder is expected progress organ failure, and cure underlying defect. Methods : We retrospectively analyzed 146 pediatric liver transplants (28 metabolic; 118 non‐metabolic) performed between 1986 2000. Results Twenty‐eight were in 24 children with disease (8 females; 16 males; age range 3 months 17 yr). Indications included α−1‐antitrypsin deficiency (n = 8), two cases each of hyperoxaluria type 1, Wilson's disease, hereditary tyrosinemia I, citrullinemia, methylmalonic acidemia, one case propionic Crigler–Najjar syndrome I , neonatal hemachromatosis, hemophilia B, Niemann–Pick cystic fibrosis. Eighteen whole grafts 10 lobar or segmental. Auxiliary patients three received combined liver‐kidney transplants. There deaths from sepsis, chronic rejection, fulminant hepatitis. Seven currently school are within 1 yr grade who had pretransplant developmental delay have remained special education. Actuarial survival rates at 5 78% 68%, respectively, mean follow‐up excess yr. These results compare favorably 100 transplanted non‐metabolic etiologies (65% 61%, respectively) (p= NS). Conclusions Pediatric excellent clinical biochemical outcome long quality most recipients.