Living Donor Liver Transplantation for Pediatric Patients with Inheritable Metabolic Disorders

• Published in: American journal of transplantation Vol. 5; no. 11; pp. 2754 - 2763
• Main Authors: Morioka, Daisuke, Kasahara, Mureo, Takada, Yasutsugu, Corrales, Jose Pablo Garbanzo, Yoshizawa, Atsushi, Sakamoto, Seisuke, Taira, Kaoru, Yoshitoshi, Elena Yukie, Egawa, Hiroto, Shimada, Hiroshi, Tanaka, Koichi
• Format: Journal Article
• Language: English
• Published: Oxford UK Munksgaard International Publishers 01.11.2005; Blackwell
• Subjects: Adolescent; Biological and medical sciences; Child; Child, Preschool; Donor selection; Female; Hepatectomy - methods; heterozygous carrier; Humans; Infant; Infant, Newborn; Liver Diseases - genetics; Liver Diseases - surgery; Liver Transplantation - mortality; Liver Transplantation - physiology; Living Donors; Male; Medical sciences; Metabolic Diseases - genetics; Metabolic Diseases - surgery; mode of inheritance; Risk Factors; Surgery (general aspects). Transplantations, organ and tissue grafts. Graft diseases; Survival Analysis; Time Factors; Tissue and Organ Harvesting - methods; Treatment Outcome; Human; Digestive system; Living donor; Liver; Selection; mode of inheritance; Inheritance; Transplantation; Homotransplantation; Heterozygosity; Genetic disease; Metabolic disorder; Treatment; Surgery; Donor selection; heterozygous carrier; Graft; Inheritance(genetics); Carrier; Child
• ISSN: 1600-6135; 1600-6143
• DOI: 10.1111/j.1600-6143.2005.01084.x

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.