Alternatives to Liver Transplantation in Pediatric Liver Diseases

Abstract

Inherited metabolic disorders and acute liver failure (ALF) are often indications for liver transplantation in pediatric patients. Liver transplantation, however, is limited by the shortage of donor organs, as well as by the need for chronic immunosuppression. This review focuses on the latest advancements made in the field of liver regenerative medicine as possible future alternatives to pediatric liver transplantation or as a means of temporary liver function support. Cell transplantation offers great promise for the treatment and long-term correction of inherited metabolic disorders, especially when ex vivo gene therapy is combined with autologous hepatocyte or induced pluripotent stem cell (iPSC)-derived hepatocyte-like cell (HLC) transplant. Bioartificial liver (BAL) systems are currently being tested that may be able to bridge patients to either liver transplantation or endogenous liver regeneration, in the case of ALF. Still, further research is required before these forms of cell therapy are incorporated into clinical practice: the optimal cell type for both cell transplantation and BAL systems must be found, methods for the large-scale expansion of these cells must be created, and safety concerns pertaining to each cell type must be addressed.