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. Posttransplant patient survival and recovery of the growth retardation were significantly better in the liveroriented 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.

Efficacy of Living Donor Liver Transplantation for Patients With Methylmalonic Acidemia

“…recipients (12)(13)(14). Therefore, LDLT using parental liver graft is strongly recommended for patients with vitamin B12-unresponsive MMAemia because scheduled LT, according to the state of the disease, can almost always be performed in the setting of the LDLT.…”

Auxiliary Liver Transplantation for Propionic Acidemia: A 10‐Year Follow‐Up

“…The quality of life is improved and a near functional cure is achieved with ALT. Living donor liver transplantation (LDLT) for PA was performed in three cases in Japan; however, the metabolic abnormality was only partially corrected and patients received L-carnitine supplements routinely (18). In part this may be because heterozygous carriers were the donors, although the carriers are normal they may show half-normal enzyme levels as seen with hexosaminidase-A levels in the Tay-Sachs disease (19).…”

Liver Transplantation in Children With Glycogen Storage Diseases: Risk Assessment and Necessity of This Procedure

“…С тех пор еще 17 детям (16 мальчикам и одной девочке) была проведена трансплантация печени ввиду развития цирроза и печеночной недостаточ-ности в исходе болезни Андерсена [2,7,14,35,36,48]. Двум пациентам потребовалась ретранс-плантация печени: в одном случае из-за дисфунк-ции трансплантата, во втором случае вследствие развития отторжения трансплантата после АВО-несовместимой родственной трансплантации.…”