Diagnosis [reference] | Age | Procedure and outcome |
Alpha-1 antitrypsin deficiency | ||
[1,2] | 4.5 months | Orthotopic liver transplantation (OLT) 2 days after hepatocyte transplantation. Explanted liver showed cirrhosis. |
Crigler-Najjar syndrome type 1 | ||
[3] | 10 years | Total bilirubin reduced by 50%. Bilirubin glucuronide demonstrated in bile up to 3.5 years. Auxiliary liver transplantation (ALTx) after 4 years. |
[4] | 3.5 years | Nine infusions were given. Serum bilirubin was reduced by 40% for at least 9 months. Ultimate outcome not reported. |
[5] | 9 years | Serum bilirubin was reduced by 32%. OLT after 4.6 months. |
[6] | 8 years | Serum bilirubin reduced by 30%. OLT after 11 months. |
[7] | 8 years | Hepatocyte transplantation by one percutaneous infusion and eight infusions through an implanted mesenteric Port-a-Cath (PAC) over 5 months. Serum bilirubin was reduced by 40%. OLT after 20 months. |
[8] | 1.5 years | Serum bilirubin was reduced by 40%. OLT after 8 months. |
[8] | 3.5 years | No clear benefit. |
[9] | 2 years | Hepatic progenitor cells were infused through the hepatic artery. Serum bilirubin was reduced by 50%. Ultimate outcome was not reported. |
[10] | 11 years | Serum bilirubin was reduced by 20%. On waiting list for OLT. |
[11] | 7 months | Serum bilirubin was reduced by 50%, bilirubin levels stable after 1 year. Improvement of psychomotor parameters. |
Factor VII deficiency | ||
[12] | 3 months | Requirement of rFVII was reduced by 70%. OLT after 7 months. |
[12] | 35 months | Requirement of rFVII was reduced by 70%. OLT after 8 months. |
[13] | 4 months | Requirement of rFVII was reduced. Ultimate outcome not reported. |
Glycogen storage disease type 1a | ||
[11] | 6 years | Hypoglycemic episodes were reduced. No admission for hypoglycemia at 1-year follow up after hepatocyte transplantation. |
[14] | 47 years | No hypoglycemia on a normal diet on a 9-month follow up. Long-term outcome not reported. |
Glycogen storage disease type 1b | ||
[15] | 18 years | Normal glucose-6-phosphatase activity for 7 months. Ultimate outcome not reported. |
Infantile Refsum's disease | ||
[16] | 4 years | Pipecholic acid decreased by 40% and c26:c22 fatty acid ratio by 36% after 18 months. Ultimate outcome not reported. |
Phenylketonuria | ||
[17] | 6 years | Patient received hepatocytes from the explanted liver of a patient with glucogen storage disease 1a, and a second transplantation from a deceased donor 7.5 months later. Plasma phenylalanine levels were normalized for 3 months after the second infusion. The therapeutic effect waned after that and patient had to be reinstated on phenylalanine-restricted diet. |
[18] | 27 years | Radiation pretreatment followed by hepatocyte transplantation with rejection risk monitoring was associated with lower post-transplant peripheral blood phenylalanine levels. |
Primary hyperoxaluria type 1 | ||
[19] | 33 months | Reduction of plasma oxalate levels by 25 to 33%. OLT and kidney transplantation after 12 months. |
Progressive familial intrahepatic cholestasis type 2 | ||
[4,8] | 32 months | Patient had cirrhosis of the liver. No benefit was demonstrated by hepatocyte transplantation. OLT after 5 months. |
[4,8] | 16 months | Patient had cirrhosis of the liver. No benefit was demonstrated by hepatocyte transplantation. OLT after 14 months. |
Tyrosinemia type 1 | ||
[11] | 45 days | Serum bilirubin levels decreased and there was improvement of coagulopathy. OLT after 45 days. Explanted liver had established cirrhosis. |
Urea cycle disorders | ||
Ornithine transcarbamylase (OTC) deficiency | ||
[20] | 5 years | Blood ammonia level decreased. Biopsy on day 28 showed 0.5% of the OTC activity in normal liver. Patient died of sepsis 42 days after hepatocyte transplantation. |
[21] | 10 hours | Blood ammonia level decreased. Protein tolerance improved. OLT after 6 months. |
[22] | Newborn | Hepatocytes were infused through the umbilical vein. Blood ammonia level was normal on normal diet. No neurological abnormality developed. OLT after 7 months. |
[7] | 14 months | Catheter was malpositioned. Effect of hepatocyte transplantation was not reported. OLT after 6 months. |
[23] | 14 months | Blood ammonia was reduced and urea production was increased. There was psychomotor improvement. OLT after 6 months. |
[24] | 1 day | Blood ammonia was reduced and urea production was increased. Protein tolerance was improved. ALTx after 7 months. |
[25] | 6 hours | Blood ammonia was reduced, urea production was increased, and urinary orotic acid excretion became normal. Patient died 4 months after hepatocyte transplantation. |
[25] | 9 days | Blood ammonia was reduced, protein tolerance improved, and urinary orotic acid excretion became normal. On waiting list for OLT 6 months after hepatocyte transplantation. |
[26] | 5 years | Blood ammonia was reduced and glutamine levels were normal. Patient died 45 days after hepatocyte transplantation. |
[22] | 1 day | Blood ammonia was reduced, urea production increased, and protein tolerance improved. ALTx after 7 months. Patient remained neurologically normal. |
[11] | 12 years | Blood ammonia was reduced, urea production increased, and glutamine levels were normal. Patient died of septicemia 30 days after hepatocyte transplantation. |
[27] | 11 days | Blood ammonia was reduced, and the patient remained neurologically normal 3 months after hepatocyte transplantation. |
[18] | 7 months | Radiation pretreatment followed by hepatocyte transplantation was associated with improved protein tolerance post-transplant. However, evaluation of allospecific CD154+ T-cytotoxic memory cells and their subsets indicated allograft rejection. Patient received liver transplantation at age 11 months. |
Arginosuccinate lyase deficiency | ||
[28] | 42 months | Blood ammonia was reduced and there was psychomotor improvement. OLT after 18 months. |
[7] | 3 years | Blood ammonia was reduced. Ultimate outcome was not reported. |
Carbomoyl phosphate synthase I deficiency | ||
[29] | 2.5 months | Blood ammonia was reduced and urea production increased. Patient was on waiting list for OLT 11 months after hepatocyte transplantation. |
Citrulinemia | ||
[29] | 3 years | Blood ammonia was reduced, urea production was increased, and protein tolerance was improved. Ultimate outcome was not reported. |
[18] | 4 months | Radiation pretreatment followed by hepatocyte transplantation and immunosuppression resulted in no therapeutic benefit. Evaluation of allospecific CD154+ T-cytotoxic memory cells and their subsets indicated allograft rejection, and the patient received liver transplantation 3.5 months after hepatocyte transplantation. |
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