Review Article

Domino Hepatocyte Transplantation: A Therapeutic Alternative for the Treatment of Acute Liver Failure

Table 1

Human hepatocyte transplantation: clinical studies in patients with metabolic liver disease.

DiseasePatient AgeNumber of viable cells transplantedType of cell transplantedOutcomeStudy/Ref

8 years7.5x109F/C40% decrease bilirubin up to 6 months; OLT at 20 months[15]
9 years7.5x109F32% decrease in bilirubin for a few weeks; OLT after 5 months[16]
18 months4.3x109C40% decrease in bilirubin to 7 months; OLT at 8 months[17]
8 years1.4x109F30% decrease in bilirubin; OLT after 11 months[18]
1 year2.6x109F/C25% decrease in bilirubin at 4 months; OLT after 4 months [19]
9 years6.1x109F/C35% decrease in bilirubin at 6 months; OLT waiting list

Glycogen storage disease type I (GSD)18 years6.0x109F/CImprovement in glucose control, normal enzyme
levels on biopsy
[20]
47 years2.0x109FBetter fasting time, decrease in triglycerides
up to 18 months
[21]

Infantile Refsum’s disease4 years2.0x109F/C40% decrease pipecholic acid after 18 months[22]

Familial hyper-cholesterolemiaFive patients between 7 and 41 years1.1x109  F20% decrease in cholesterol, LDL, ApoB to 28 month; [23]
1.3x109  No effect;
1.0x109  6% decrease in
cholesterol, LDL, ApoB to 19 months;
3.2x109  Minor effect
1.5x10920% decrease in cholesterol up to 7 months

Urea cycle defects

OTC5 years1.0x109FDecreased ammonia initially; Died 42 days later[24]

OTC10 hours9.0x109F/CDecrease ammonia; increased protein tolerance for a short period; OLT at 6 months[25]

OTC2 dias1.9x109F/CDecreased ammonia; auxiliary transplant at 6 months[26]

OTC14 months2.4x109CDecreased ammonia; increased urea; psychomotor improvement; OLT after 6 months[27]

OTC1 day1.6x109F/CDecreased ammonia; increased urea under normal diet; partial orthotopic liver transplantation (apolt) at 7 months[28]

OTC10 weeks
3 years
3.0x109FSome stabilization[29]

OTC6 hours0.6x1CDecreased ammonia; increased urea; normal urinary orotic acid excretion. Death at 4 months[30]
9 daysDecreased ammonia; increased protein intake, urinary orotic acid normal at 6 months. OLT waiting list

Citrullinemia36 months1.5x1CNormal ammonia; 40% increase in urea[30]

Factor VII deficiency3 months
35 months
1.1x109  C70% decrease in recombinant factor VII for 6 months; OLT after 7 months [31]
2.2X109F/C

Phenylketonuria6 yearsTwo infusions for a total of
630 × 106 cells
Fblood phenylalanine levels returned within the therapeutic target while the phenylalanine
half-life assessed by loading tests decreased from 43 to 19 h.
[32]