Paediatrics Posters

Monday July 02, 2018 from 16:30 to 17:30

Room: Hall 10 - Exhibition

P.757 Hepatocyte transplant and bridge to subsequent liver transplant in an infant with carbamoyl phosphate synthetase deficiency

Sojeong Yoon, Korea

Resident
Department of surgery
Samsung medical center

Abstract

Hepatocyte Transplant and Bridge to Subsequent Liver Transplant in an Infant with Carbamoyl Phosphate Synthetase Deficiency

Sanghoon Lee1, Sungyoon Cho4, Ji-Hyun Lee2,3, Sojeong Yoon1, Seung Wook Han1, Hey-Jung Park3, Young-A Kim3, Mi Na Park3, Suk-Koo Lee1.

1Department of Surgery, Sungkyunkwan University School of Medicine, Samsung Medical Center, Seoul, Korea; 2Department of Health Sciences and Technology, SAIHST, Sungkyunkwan University, Seoul, Heard and Mc Donald Islands; 3Stem Cell and Regenerative Medicine Institute, Research Institute for Future Medicine, Samsung Medical Center, Seoul, Korea; 4Department of Pediatrics, Sungkyunkwan University School of Medicine, Samsung Medical Center, Seoul, Korea

Carbamoyl phosphate synthetase 1 deficiency (CPS1D) is an autosomal recessive disorder of the urea cycle that causes hyperammonemia. Two forms of CPS1D are recognized: a lethal neonatal type and a less severe, delayed-onset type. Neonatal CPS1D cases often present their symptoms within the first days of life. Severe hyperammonemia in the neonatal period leads to serious brain damage, coma, and death if not treated promptly. We report a case of successful hepatocyte transplant and subsequent deceased donor liver transplant in an infant diagnosed with neonatal CPS1D. Hepatocytes were isolated from liver and deceased donors. At the first transplant, approximately 2.4 x 10^9 cells (7.4% of the estimated recipient's total hepatocytes) were infused over 3 sessions spanning 8 days. Hepatocytes were infused into the portal vein via an indwelling catheter inserted through the umbilical vein. After the first hepatocyte transplant, the patient showed stabilization of ammonia levels after several days and ammonia levels remained stable despite increased protein intake and discontinuation of ammonia scavengers. After 5 months, graft function seemed to deteriorate with elevated ammonia levels at times. A second hepatocyte transplant was done and was also successful in sustaining stable ammonia levels for another 5 months. The infant received deceased donor liver transplant at 13 months of age. This was the first successful hepatocyte transplant and subsequent bridge to liver transplant in a CPS1D baby in Korea. Hepatocyte transplant allowed ammonia stabilization despite protein intake, which allowed for normal growth and development in this infant.



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