Table 2. Summary of clinical hepatocyte transplantation in pediatric metabolic liver diseases.

Disease	Age	Effect/outcome	Reference
Crigler–Najjar syndrome type 1	10 years	50 % reduction in bilirubin/OLT 4 years post-HT	Fox et al. [4]
	8 years	40 % reduction in bilirubin/OLT 20 months post-HT	Darwish et al. [44]
	9 years	30 % reduction in bilirubin/OLT 5 months post-HT	Ambrosino et al. [45]
	1.5 years	40 % reduction in bilirubin/OLT 8 months	Dhawan et al.
	3.5 years	post-HT No clear benefit	[46]
	3.5 years	Lowered serum bilirubin/outcome unknown	Hughes et al. [10]
	8 years	30 % reduction in bilirubin/OLT 11 months post-HT	Allen et al. [47]
	9 years	35 % reduction in bilirubin/OLT waiting list	Lysy et al. [48]
	1 year	25 % reduction in bilirubin/OLT 4 months post-HT
	2 years	50 % reduction in bilirubin/outcome unknown	Khan et al. [49]
	11 years	20 % reduction in bilirubin/OLT waiting list	Meyburg et al. [43]
	7 months	50 % reduction in bilirubin, psychomotor improvement/bilirubin stable at 1 year follow-up	Rbes-Koninckx et al. [50]
Alpha-1 antitrypsin deficiency	18 weeks	OLT 2 days post-HT/cirrhosis on explant	Strom et al. [41]
Familial hypercholesterolemia	12 years	Ex vivo gene therapy with autologous cells: No benefit	Grossman et al. [51]
	7 years	6 % reduction in cholesterol and LDL
	11 years	19 % reduction in cholesterol and LDL
Factor VII deficiency	3 months	70 % reduction in rFVII requirement/OLT 7 months post-HT	Dhawan et al. [52]
	35 months	70 % reduction in rFVII requirement/OLT 8 months post-HT
	4 months	Reduction in rFVII requirement/outcome unknown	Hughes et al. [10]
Progressive familial intrahepatic cholestasis 2	32 months	No benefit (cirrhosis established):OLT 5 months post-HT	Dhawan et al. [46]
	16 months	OLT 14 months post-HT
Phenylketonuria	6 years old	Reduction in Phe levels and improved dietary tolerance up to 3 months post-HT (cells from “domino” GSD1b liver)	Stéphenne et al. [53]
Tyrosinemia type 1	45 days	Improved coagulopathy and bilirubin/OLT 45 days post-HT (cirrhosis on explant)	Rbes-Koninckx et al. [50]
Glycogen storage disease type 1a	6 years	Reduction in hypoglycemic episodes/no hypoglycemic admissions at 1 year follow-up	Rbes-Koninckx et al. [50]
Glycogen storage disease type 1b	18 years	Normal G6Pase activity for 7 months/outcome unknown	Lee et al. [54]
Infantile Refsum's disease	4 years	40 % reduction in pipecolic acid for 18 months/outcome unknown	Sokal et al. [55]
Primary hyperoxaluria type 1	33 months	Reduction in plasma oxalate/liver-kidney transplant 12 months post- HT	Beck et al. [56]
Urea cycle defects
Ornithine transcarbamylase deficiency	5 years	Ammonia reduction and protein tolerance/septic death at 42 days post-HT	Strom et al. [57]
	10 h	Ammonia reduction and protein tolerance/OLT 6 months post-HT	Horslen et al. [5]
	14 months	Effect unknown (malpositioned catheter)/OLT 73 days post-HT	Darwish et al. [44]
	14 months	Ammonia reduction, increased urea, and psychomotor improvement/OLT 6 months post- HT	Stéphenne et al. [58]
	1 day	Ammonia reduction, increased urea, and protein tolerance/auxiliary partial OLT 7 months post-HT	Puppi et al. [59]
	6 h	Ammonia reduction, increased urea, normal urine orotic acid/death 4 months post-HT	Meyburg et al. [60]
	9 days	Ammonia reduction, protein tolerance, normal urine orotic acid/OLT waitlist 6 months post-HT
	5 years	Ammonia reduction, normal glutamine/death 45 days post-HT	Bohnen et al. [35]
	1 day	Ammonia reduction, increased urea, and protein tolerance/auxiliary partial OLT 7 months post-HT and neurologically normal	Mitry et al. [61]
	12 years	Ammonia reduction, increased urea, normal glutamine/septic death 30 days post-HT	Rbes-Koninckx et al. [50]
	11 days	Ammonia reduction/neurologically normal 3 months post-HT	Enosawa et al. [19]
Arginosuccinate lyase deficiency	42 months	Ammonia reduction and psychomotor improvement/OLT 18 months post-HT	Stéphenne et al. [62]
	3 years	Ammonia reduction/outcome unknown	Darwish et al. [44]
Carbamoyl phosphate synthase I deficiency	2.5 months	Ammonia reduction and increased urea /OLT waiting list 11 months post-HT	Meyburg et al. [63]
Citrullinemia	25 months	Ammonia reduced and decreased urea/outcome unknown	Lee et al. (unpublished)
	3 years	Ammonia reduction, increased urea, and protein tolerance/outcome unknown	Meyburg et al. [63]

Table modified with permission from [39]

OLT orthotopic liver transplantation, HT hepatocyte transplant