HEPATOCELLULAR TRANSPLANTATION AND TARGETING GENETIC MARKERS TO HEPATIC CELLS 
<par 3> The liver cells will be infused into your child's liver using one of several 
approaches selected based on the size of your child, the severity of your child's liver 
disease, the ability of your child's blood to clot, and the risk of the operative 
procedures. These may include a surgical procedure in which an incision (cut) is made 
in the abdomen and cells are injected into the liver through a needle placed in the 
spleen or through a catheter (plastic tube) placed in the blood vessels that enter the 
liver (portal vein). Alternatively, liver cells may be injected through a catheter that 
passes through the jugular vein (large vein at the base of the neck) to the veins of the 
liver. We will select the procedure which will entail the least amount of surgery and 
the least risk for your child and will review this decision with you. The clinical 
care that your child will receive following liver cell injection will be very similar 
to the standard care of patients following whole organ transplantation. 
<par 4> In order to determine whether the liver cells that are infused into your 
child's liver survive and are capable of providing the functions of a normal liver, 
several additional tests will be performed. These will include conventional blood tests 
for liver functions and conventional X-ray and other pictures of your child's liver (CT 
scan, MRI , or HIDA scan) which your child has had previously as part of your evaluation 
in the Clinical Research Center. In addition, a portion of the liver cells which are 
infused will be "marked" with a genetic marker that can be identified in biopsy samples. 
The marker is described later in this form. 
<par 5> The potential benefits to your child from participating in the liver cell 
transplantation protocol include the possibility that your child's condition may improve 
and that the whole organ transplant, which necessitates extensive surgery and removal 
of your child's liver, may not be needed. Alternatively, your child's condition may 
stabilize sufficiently for arrangements to be made for whole organ transplantation in 
the future. This protocol may also benefit other children with similar diseases, 
because it will provide physicians and researchers with experience in this new therapy 
for liver disease. 
<par 6> There are risks associated with the liver cell transplantation procedures. 
These include the common risks associated with drawing blood from your child's veins and 
the need to obtain urine (obtained using a collection bag or catheter if your child is 
unable to provide a sample) . The amount of blood drawn as part of this protocol will 
be monitored and will be limited to amounts considered safe for your child. There are 
risks associated with the surgical procedures, anaesthesia, and catheter insertion which 
will be performed in the course of the liver cell transplant. The surgeons who will 
perform these procedures will recommend the procedures which they feel would introduce 
the least risk to your child. These procedures and the risks will be explained to you 
for your consent using standard surgical consent forms before any procedure is 
performed. All liver transplant procedures, including whole organ transplantation and 
liver cell injection, are associated with immune rejection (attack and destruction by 
your child's immune system) and require the use of drugs to prevent rejection. You will 
be provided with a notebook, prepared for liver transplant patients, which describes 
[826] 
Recombinant DNA Research, Volume 14 
