title: Living donor liver transplantation for pediatric patients with inheritable metabolic disorders.
abstract: Forty-six pediatric patients who underwent living donor liver transplantation (LDLT) using parental liver grafts for inheritable metabolic disorders (IMD) were evaluated to determine the outcomes of the surgery, decisive factors for post-transplant patient survival and the impact of using donors who were heterozygous for the particular disorder. Disorders included Wilson disease (WD, n = 21), ornithine transcarbamylase deficiency (OTCD, n = 6), tyrosinemia type I (TTI, n = 6), glycogen storage disease (GSD, n = 4), propionic acidemia (PPA, n = 3), methylmalonic acidemia (MMA, n = 2), Crigler-Najjar syndrome type I (CNSI, n = 2), bile acid synthetic defect (BASD, n = 1) and erythropoietic protoporphyria (EPP, n = 1). The post-transplant cumulative patient survival rates were 86.8 and 81.2% at 1 and 5 years, respectively. Post-transplant patient survival and recovery of the growth retardation were significantly better in the liver-oriented diseases (WD, OTCD, TTI, CNSI and BASD) than in the non-liver-oriented diseases (GSD, PPA, MMA and EPP) and pre-transplant growth retardation disadvantageously affected post-transplant outcomes. Although 40 of 46 donors were considered heterozygous for each disorder, neither mortality nor morbidity related to the heterozygosis has been observed. LDLT using parental donors can be recommended as an effective treatment for pediatric patients with IMD. In the non-liver-oriented diseases, however, satisfactory outcomes were not obtained by hepatic replacement alone.
authors.affiliation: Organ Transplant Unit, Kyoto University Hospital, Shogoin-kawara-cho, Sakyo-ku, Kyoto, 606-8507, Japan. dmorioka@hotmail.com
authors.forename: Daisuke
authors.initials: D
authors.lastname: Morioka
authors.forename: Mureo
authors.initials: M
authors.lastname: Kasahara
authors.forename: Yasutsugu
authors.initials: Y
authors.lastname: Takada
authors.forename: Jose Pablo Garbanzo
authors.initials: JP
authors.lastname: Corrales
authors.forename: Atsushi
authors.initials: A
authors.lastname: Yoshizawa
authors.forename: Seisuke
authors.initials: S
authors.lastname: Sakamoto
authors.forename: Kaoru
authors.initials: K
authors.lastname: Taira
authors.forename: Elena Yukie
authors.initials: EY
authors.lastname: Yoshitoshi
authors.forename: Hiroto
authors.initials: H
authors.lastname: Egawa
authors.forename: Hiroshi
authors.initials: H
authors.lastname: Shimada
authors.forename: Koichi
authors.initials: K
authors.lastname: Tanaka
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D005260:Female; D006498:Hepatectomy / Q000379:methods; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008107:Liver Diseases / Q000235:genetics* / Q000601:surgery*; D016031:Liver Transplantation / Q000401:mortality / Q000502:physiology*; D019520:Living Donors; D008297:Male; D008659:Metabolic Diseases / Q000235:genetics* / Q000601:surgery*; D012307:Risk Factors; D016019:Survival Analysis; D013997:Time Factors; D020858:Tissue and Organ Harvesting / Q000379:methods; D016896:Treatment Outcome