Table B.

Categorized examples of clinical case reports and trials of hepatocyte transplantation

Patients	Disease	Procedure	Cells	Outcome	Reference
Small number of engrafted hepatocytes lead to sufficient repopulation due to hepatocytic injury
Female 64 yo	Acute liver failure due to ingestin of amanita phalloides mushrooms	Portal vein injection	8 billion cryopreserved cells. Viability ~62% measured with Trypan blue.	Ammonia, billirubin decreased gradually. Eight weeks after transplant, normal liver architecture and normal blood flow was seen. Physicians concluded full recovery.	Schneider et al. 2006 [82]
Small number of engrafted hepatocytes needed to provide therapeutic benefit
Male	Factor VII deficiency	Inferior mesenteric vein injection	1.09 billion cryopreserved	Requirement of exogenous protein diminished to 20% dose previous to transplant. Remission began at 6 mos. Received OLT.	Dhawan et al. 2004 [83]
Male	Factor VII deficiency	Inferior mesenteric vein injection	2.18 billion fresh and cryopreserved	Requirement of exogenous protein diminished to 20% dose previous to transplant. Remission began at 6 mos. Received OLT.
Female 9 yo	Crigler-Najjar		7.5 billion cells (5% liver mass)	50–65% reduction, remission at 3mo. Possible rejection.	Ambrosino et al. 2005 [84]
Female 9 yo	Crigler-Najjar	Venous injection	18 infusions over 5mo, total 4% of liver mass (6.1 billion cells)	Bilirubin reduced from 17.5 to 13.6, remission at 6mo	Lysy et al. 2008 [42]
Female 1 yo	Crigler-Najjar	Venous injection	14 infusions over 15d, total of 8.6% of liver mass (2.6 billion)	Billirubin reduced from 17.6 to 13.3, remission at 4mo
Male 6 yo	Phenylketoneurea (PKU)		2.5 billion cells over 2 infusions	Phenylalanine decreased from 11.1 to 3.5	Smets et al. 2011 [85]
Female 4 mos	Carbamoyl-phosphate synthetase 1 (CPS1) deficiency	Portal vein by recanalization of the umbilical vein	689 million viable cells over 3 infusions within 26hrs of 5GY HIR	Continued to experience episodes of high ammonia	Soltys et al. 2017 [50]
Male 7 mos	OTC		2 billion cells over 4 infusions within 16hrs of 7.5GY HIR	Tolerated protein from day 20 to day 84, when ammonia acutely elevated
Female 27 yo	PKU		5 billion cells over 3 infusions after 10GY HIR	PHE reduced by 36% for more than a year
Male 14 mos	Urea cycle disease (Ornithine transcarbamylase (OTC) deficiency)	Portal vein injection	2.4 billion (6%)	Reduction in ammonia for 2mos. Psychomotor development improved. Eventually underwent OLT.	Stephenne et al. 2005 [86]
Female 3.5 yo	Argininosuccinate Lyase deficiency	Portal vein injection	3.7 billion (9%)	Psychomotor advances acquiring fecal and urinary continence, could dress alone, and perform difficult puzzle exercises and ammonia decreased 50% and remained low for at least a year. Repopulation measured with Y-chromosome FISH showed 19% repopulation at 8 months, 12.5% at 12 months.	Stephenne et al. 2006 [87]
Massive repopulation is is required because toxic metabolite is generated in host hepatocytes
Female 4 yo	Infantile Refsum disease, peroxisomal biogenesis disease	spleno-mesenteric confluent injection (venous injection)	2 billion cells (5%). Half fresh half cryopreserved.	Resolution of cholestasis, metabolite decreased by 40%. Engraftment seen on biopsy. Reduction in abnormal metabolites maintained until end of measurement at 18 mos.	Sokal et al. 2003 [49]