Journal Article

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

Bodo B. Beck, Sandra Habbig, Katalin Dittrich, Dirk Stippel, Ingrid Kaul, Friederike Koerber, Heike Goebel, Eduardo C. Salido, Markus Kemper, Jochen Meyburg and Bernd Hoppe

in Nephrology Dialysis Transplantation

Published on behalf of European Renal Association - European Dialysis and Transplant Assoc

Volume 27, issue 7, pages 2984-2989
Published in print July 2012 | ISSN: 0931-0509
Published online January 2012 | e-ISSN: 1460-2385 | DOI: http://dx.doi.org/10.1093/ndt/gfr776
Liver cell transplantation in severe infantile oxalosis—a potential bridging procedure to orthotopic liver transplantation?

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Background

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.

Methods

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.

Results

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.

Conclusions

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.

Keywords: dialysis; infantile oxalosis; liver cell transplantation; primary hyperoxaluria; transplantation

Journal Article.  3859 words.  Illustrated.

Subjects: Nephrology

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