HEPATOCELLULAR TRANSPLANTATION AND TARGETING GENETIC MARKERS TO HEPATIC CELLS 
The following tests will be performed in patients in preparation for HCT; 
Table 3. Additional tests to be performed prior to HCT (see also Appendix S') 
- Liver spleen scan (HIDA scan) - baseline study. 
- Liver biopsy (if not performed previously. This can be performed 
intraoperatively if laparotomy is selected) . 
3. Inclusion criteria. This protocol will be offered to children with acute, life 
threatening, fulminant or end- stage liver failure who are eligible for conventional OLT, 
for whom conventional liver transplantation is not available for any reason, and for 
whom there is considered to be a real expectation of physical and cognitive recovery if 
hepatic function is supported by hepatocellular engraftment. Acute, but not end stage 
hepatic failure, will be an indication if the patient is considered to be at risk for 
progressing to a predictable and Irreversible encephalopathy despite conventional (non- 
transplant) therapy and there is no prospect of becoming eligible for OLT. 
Patients with acute and fulminant hepatic failure due to metabolic causes, including 
disorders of the urea cycle (Ornithine transcarbamylase , carbamylphosphate synthase, 
arginosuccinate synthase, arginosuccinate lyase, arginase) , organic acid metabolism 
(methylmalonyl CoA mutase deficiency, propionyl CoA carboxylase, medium chain acyl CoA 
dehydrogenase deficiency, or others), or tyrosinemia, will also be eligible. 
Individuals with metabolic diseases will only be considered as a last resort if clinical 
medical management is deemed unsuccessful, patients are at risk for incipient CNS damage 
or death, and OLT is not available. 
4. Exclusion criteria. Acute hepatic failure of infectious or immune origin will be 
a contraindication unless there is no evidence of ongoing infection or infectious 
agents. Absolute contraindications will include biliary atresia, cirrhosis, or portal 
hypertension. Other contraindication will include congenital heart disease with right- 
left shunting, portal-systemic venous shunting, or irreversible bleeding diathesis. 
Patients with evidence of AIDS or CJD exposure or infection will be excluded. There 
will be no discrimination on the basis of age, sex, ethnic or racial background, or 
ability to pay. 
HCT will not be performed in patients with ongoing systemic infections including 
bacteremia, pneumonia, peritonitis, or cholangitis or patients with active viral 
infections. Eligibility for HCT will be reconsidered after therapy or resolution of 
these infections . 
|: 
j 5. Encephalopathy. The protocol will be offered to patients who are considered to be 
! at serious risk for irreversible morbidity or mortality without therapeutic 
intervention. Evidence of irreversible encephalopathy including decerebration or grade 
I 4 encephalopathy will be a contraindication for inclusion in this protocol. 
I 6. Approval by surgery and anaesthesia. Patients must be considered to be an 
acceptable risk for surgery and anaesthesia and will receive routine pre- surgical 
evaluation by these services. 
Recombinant DNA Research, Volume 14 
[803] 
