Cryopreserved liver cell transplantation controls ornithine transcarbamylase deficient patient while awaiting liver transplantation.

Abstract

Liver cell transplantation was performed in a child with urea cycle disorder poorly equilibrated by conventional therapy as a bridge to transplantation. A 14-month-old boy with ornithine transcarbamylase (OTC) deficiency received 0.24 billion viable cryopreserved cells/kg over 16 weeks. Tacrolimus and steroids were given as immunosuppressive treatment while the patient was kept on the pre-cell transplant therapy. Mean blood ammonia level decreased significantly following the seven first infusions, while urea levels started to increase from undetectable values. After those seven infusions, an ammonium peak up to 263 microg/dL, clinically well tolerated, was observed. Interestingly, blood urea levels increased continuously to reach 25 mg/dL, after the last three infusions. Eventually, he benefited from elective orthotopic liver transplantation (OLT) and the post-surgical course was uneventful. We conclude that use of cryopreserved cells allowed short- to medium-term metabolic control and urea synthesis in this male OTC-deficient patient while waiting for OLT.

• Authors: Xavier Stéphenne, Mustapha Najimi, Françoise Smets, Raymond Reding, Jean de Ville de Goyet, Etienne M Sokal
• Journal: American journal of transplantation : official journal of the American Society of Transplantation and the American Society of Transplant Surgeons (Am J Transplant) Vol. 5 Issue 8 Pg. 2058-61 (Aug 2005) ISSN: 1600-6135 [Print] United States
• PMID: 15996260 (Publication Type: Case Reports, Journal Article, Research Support, Non-U.S. Gov't)

Topics

• Amino Acid Metabolism, Inborn Errors (enzymology, surgery)
• Cell Transplantation
• Cryopreservation
• Humans
• Infant
• Liver Transplantation
• Male
• Ornithine Carbamoyltransferase Deficiency Disease