Liver cell transplantation in children

Abstract:  With increasing shortage of donor organs and changes in organ allocation systems, there is a considerable need for alternative or supportive techniques in the field of liver transplantation. Liver cell transplantation (LCT) is such an innovative technique that is especially promising for use in children, because it is less invasive than orthotopic liver transplantation (OLT). Two main indications exist: acute liver failure (ALF) and hepatic-based inborn errors of metabolism. Ten children with ALF underwent LCT so far. One patient recovered without OLT, five were successfully bridged to OLT, and four died. Clinical improvement like reduced hyperammonemia, improved coagulation, and regression of hepatic encephalopathy occurred in the majority of patients. For the treatment of inborn errors of metabolism, experience exists with Crigler–Najjar syndrome type 1 (CNS 1, n = 5) and urea cycle disorders (UCD, n = 6). All but two patients experienced metabolic stabilization for a mean period of 9.7 months, before the majority of children underwent OLT. Whereas transplant success in CNS 1 patients can be easily monitored by plasma bilirubin levels, it is much more difficult in UCDs, where many factors can contribute to clinical deterioration. Nevertheless, these individual therapeutic attempts of LCT yielded encouraging results, and prospective studies should be initiated.