title: Pediatric liver transplantation for inherited metabolic liver disease: a single-center experience.
abstract: OBJECTIVE
  We performed single-center outcome comparison of pediatric recipients who underwent liver transplantation for either genetic or metabolic disease including the clinical impact of using heterozygote parents as living donors.
  
  
  METHODS
  Pediatric liver transplant recipients from September 2007 to December 2010 were included. Patients were separated into 2 categories by etiology of liver disease: (1) genetic or metabolic liver disease (G/M) and (2) nongenetic or metabolic liver disease (non-G/M), which included all other remaining etiologies combined. Patient demographics, recipient and donor characteristics, graft type, operative data, recipient complications, allograft and patient survival were analyzed.
  
  
  RESULTS
  Forty liver transplants were performed on 40 patients; 18 were transplanted for G/M; mean waiting time was 101 days for G/M group and 57 days for non-G/M group; 9 patients were listed as status 1, 5 were granted PELD/MELD exceptions; the overall mean PELD/MELD score was 21. Four G/M patients had hepatocellular carcinoma in the explant without microvascular invasion. Overall complications requiring either surgery or interventional radiology occurred in 14 patients-G/M (n = 5); CMV viremia was seen in 11 patients (G/M, n = 1); detectable EBV DNA was detectable in 8 patients (G/M, n = 4), acute cellular rejection was seen in 10 (G/M, n = 5), postransplant lymphoproliferative disease occurred in 2 G/M patients; and 1 G/M patient showed significantly improved posttransplant neurologic motor function. Children with G/M who received a living donor liver transplant from heterozygote parents did well without any signs of expressing underlying metabolic disease. Posttransplant graft and patient overall survival at 12 months for G/M and non-G/M was 100%, and at 36 months, 83% and 100%, respectively.
  
  
  CONCLUSIONS
  The majority of children transplanted for either genetic or metabolic disease were status 1 or awarded UNOS exception points. Cadaveric split livers and live donors including obligate heterozygotes resulted in excellent allograft and patient survival outcomes. In metabolic and genetic liver diseases, close follow-up and timely transplantation can preclude malignant spread and prevent disease progression and consequences, as well as reverse neurologic sequelae.
authors.affiliation: Department of Surgery, Yale University School of Medicine, New Haven, CT, USA.
authors.forename: R
authors.initials: R
authors.lastname: Rosencrantz
authors.forename: B
authors.initials: B
authors.lastname: Cimsit
authors.forename: M
authors.initials: M
authors.lastname: Seashore
authors.forename: A
authors.initials: A
authors.lastname: Arvelakis
authors.forename: S
authors.initials: S
authors.lastname: Kulkarni
authors.forename: M
authors.initials: M
authors.lastname: Ghiroli
authors.forename: S
authors.initials: S
authors.lastname: Emre
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D003587:Cytomegalovirus / Q000302:isolation & purification; D005260:Female; D030342:Genetic Diseases, Inborn / Q000150:complications / Q000235:genetics / Q000601:surgery*; D004854:Herpesvirus 4, Human / Q000302:isolation & purification; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D007223:Infant; D008107:Liver Diseases / Q000150:complications / Q000235:genetics / Q000601:surgery*; D016031:Liver Transplantation; D008297:Male; D014766:Viremia / Q000150:complications
chemical_list: D007166:Immunosuppressive Agents