Liver cell transplantation in severe infantile oxalosis--a potential bridging procedure to orthotopic liver transplantation?

[primary hyperoxaluria type 1]

The infantile form of primary hyperoxaluria type I (PHI ) is the most devastating PH subtype leading to early end-stage renal failure and severe systemic oxalosis . Combined or sequential liver -kidney transplantation (LKTx ) is the only curative option but it involves substantial risks , especially in critically ill infants . The procedure also requires resources that are simply not available to many children suffering from PHI worldwide . Less invasive and less complex therapeutic interventions allowing a better timing are clearly needed . Liver cell transplantation (LCT ) may expand the narrow spectrum of auxiliary measures to buy time until LKTx for infants can be performed more safely .We performed LCT (male neonate donor ) in a 15 -month -old female in reduced general condition suffering from systemic oxalosis . Renal replacement therapy , initiated at the age of 3 months , was complicated by continuous haemodialysis access problems . Living donor liver transplantation was not available for this patient . Plasma oxalate (Pox ) was used as the primary outcome measure .Pox decreased from 104 .3 ±8 .4 prior to 70 .0 ±15 .0 μmol /L from Day 14 to Day 56 after LCT . A significant persistent Pox reduction (P <0 .001 ) comparing mean levels prior to (103 .8 μmol /L ) and after Day 14 of LCT until LKTx (77 .3 μmol /L ) was seen , although a secondary increase and wider range of Pox was also observed . In parallel , the patient 's clinical situation markedly improved and the girl received a cadaveric LKTx 12 months after LCT . However , biopsy specimens taken from the explanted liver did not show male donor cells by amelogenin polymerase chain reaction .With due caution , our pilot data indicate that LCT in infantile oxalosis warrants further investigation . Improvement of protocol and methodology is clearly needed in order to develop a procedure that could assist in the cure of PHI .