Acute liver failure (ALF) in children is a rare but a relevant condition with severe consequences. Although liver transplantation is considered to be the most efficient management approach, it has numerous limitations such as crucial surgical risks and the need for life-long immunosuppression. Thus, an alternative in the form of liver cell (hepatocyte) transplantation appears to be a better choice, as many children only need short-term liver support till the time their own liver recovers. Studies have shown that human hepatocytes encapsulated in alginate beads can act as a short-term liver while alginate coating prevents the cells from immune response of the body. The investigators of the present study assessed the efficiency and safety of hepatocyte microbead transplantation therapy in children with ALF.

Clinical grade human hepatocyte microbeads (HMBs) and empty microbeads were examined in immunocompetent healthy experimental animals. Furthermore, the study recruited eight children with ALF, who were awaiting a suitable allograft for LT and these children were further administered with intraperitoneal transplantation of HMBs. They were analyzed for the complications of the procedure, evaluating the host immune response and residual function of the retrieved HMBs, both after spontaneous native liver regeneration and at the time of LT.

The results demonstrated that intraperitoneal transplantation of HMBs in healthy experimental animals was safe. The therapy preserved synthetic and detoxification functions, without the requirement for immunosuppression. Moreover, eight children with ALF who were administered HMBs had a median age of 14.5 days, range 1 day to 6 years. The causes of ALF in children included neonatal hemochromatosis in four children, 2 had viral infections and other 2 had unknown etiology. The outcome revealed that the therapy was safe in all children without the incidence of any complication. This resulted in avoidance of LT in four children among eight, while 3 were successfully adapted to LT following the intervention. In addition, HMBs retrieved after infusions were structurally intact, free of host cell adherence and contained viable hepatocytes with preserved functions.

Thus, based on the positive outcome, it can be concluded that HMB infusion is effective and well tolerated in children with ALF.

Source: Dhawan A, Chaijitraruch N, Fitzpatrick E, et al. Alginate microencapsulated human hepatocytes for the treatment of acute liver failure in children. J Hepatol. 2020;72(5):877-884.