HEPATOCELLULAR TRANSPLANTATION AND TARGETING GENETIC MARKERS TO HEPATIC CELLS 
Costs. The recovery of costs of this procedure will be divided between: i) those 
procedures associated with the ongoing monitoring and therapy for hepatic disease which 
would be required regardless of involvement in this protocol. These costs will continue 
to be billed to the patient as part of routine clinical care, ii) costs involved in 
clinical research related to the delivery and evaluation of HCT which will be paid by 
the General Clinical Research Center (GCRC) at TCH. iii) costs involved in assessing 
novel aspects of HCT, such as the safety of retroviral vectors and course of 
immunological rejection of hepatocytes which will be born by individual investigators 
from research grants. Patients will not be billed for the costs of the research 
procedure. This proposal has been approved by the scientific committee of the GCRC 
which enables the use of GCRC funds for this research. This research proposal has been 
included in a renewal application for the GCRC at TCH and funds have been requested 
explicitly for this project. The cost centers which will be billed for specific tests 
are outlined in appendix E. 
IV. EXPERIMENTAL DESIGN 
A. Patient evaluation and selection. 
1. HCT versus conventional OLT. All pediatric patients referred to TCH and eligible 
for OLT will be offered enrollment in a study entitled "Clinical Course and Management 
of Hepatic Transplantation" at the time of their initial clinical evaluation. All 
patients participating in that protocol will be considered for HCT. The "Clinical 
Course and Management of Hepatic Transplantation" protocol is intended to provide 
detailed evaluation of the indications for liver transplantation as well as alternatives 
including HCT and is a requisite for participation in the present study. This study 
will also provide a control group for the HCT protocol. 
Participation in the present study will be offered to any patient who meets the 
inclusion criteria (see below) and will be assessed at monthly meetings of the Liver 
Transplantation Team or on an emergency basis as clinically indicated. Patients and 
families under consideration for this protocol or actively participating in this study 
will remain listed with UNOS (United Organ Service) for an appropriate organ donor as 
indicated by their clinical condition. Patients who are evaluated for OLT, but are 
ineligible for OLT for any reason, will continue to be considered for HCT if they meet 
the inclusion criteria. If these patients subsequently become eligible for OLT, they 
will be listed with UNOS based on their clinical condition. Participation in the 
present protocol will at no time preclude conventional OLT if indicated by conventional 
clinical criteria. Participation in this study does not alter the selection criteria 
or the potential for OLT. 
Recombinant DNA Research, Volume 14 
[801] 
