Monitoring After Discharge from the Hospital: After your child's liver cells have been infused 
back into his/her liver and he/she has left the hospital, we will need to monitor your child 
closely for the effects of the corrected liver cells. For the 4 weeks following discharge from the 
hospital, your child will have to come back to the hospital for 2 days each week (each visit will 
last about one hour). After these first four weeks, your child will have to come back to the 
hospital for visits every week for four months. After this time, the frequency of your child's 
visits will be determined by his/her medical condition with a minimum of one visit per year. 
Small amounts of blood (one to two teaspoons) will be taken from your child on each visit. We 
will also need to remove a small piece of your child's liver tissue in order to examine it for the 
corrected cells. This will be done about three months after the infusion of the corrected cells 
back into his/her liver. A doctor will place a needle through your child's skin into his/her 
liver and remove a tiny piece (the size of a pencil tip) of liver. This will not require another 
operation. This procedure will be explained to you at the time it will be performed and consent 
for your child will be obtained separately. If your child should die for any reason, either during 
or after his/her participation in this study, we will request that an autopsy be performed. This 
will be necessary so that we could determine the cause of death and to examine the results of this 
therapy. 
Benefits: It is impossible to say that your child will receive any benefit from this study. 
Similar studies in rabbits that also have this type of disease have shown that a similar form of 
therapy lowered their "fat" level in their blood for at least four months. This suggests that this 
therapy may lower your child’s "fats" levels, however, it is impossible to predict the likelihood 
that a human being will respond in the same manner as a rabbit. Following infusion of the 
corrected liver cells, your child will be restarted on his/her previous "fat" lowering therapy. 
Important information will be gained in this study that may benefit other patients with this 
disease or similar inherited diseases. 
Risks, Inconvenience and Discomforts: There are many possible risks and discomforts 
that may happen to your child in this study. These will be explained in detail below. 
Initial Evaluation: Problems may occur from the blood drawing, which involves a needle stick, 
and may result in a bruise at the site of the stick. Very rarely a local infection may occur at the 
site of the needle stick that would require treatment with antibiotics or drugs to kill the 
bacteria causing the infection. Other inconveniences include the time required for this 
evaluation. 
Liver Surgery: Severe complications may result from the operation that removes part of your 
child's liver. It is possible that your child may experience problems with the anesthesia that 
would affect and possibly cause damage to his/her heart and brain. It is very rare, but your 
child may die from these problems with the anesthesia. Death from the surgery itself is a very 
rare complication (less than 0.1%) but this can occur. The surgery to remove part of your 
child's liver may also result in internal bleeding or an infection. This may require a second 
operation to correct the bleeding. There may also be damage to your child's liver that causes it 
to fail (this is also very rare; with less than a 1% chance occuring during the procedure). Pain 
from the surgery site may persist for long periods (several months) after the surgery, but this 
will diminish with time. Limitations to certain activities, such as exercise, will be necessary 
due to the surgical wound. These activities may not be started until your child has complete 
healing of this area. Infection from the tube that was put into the vein that leads to his/her 
liver may also occur and this would require treatment with antibiotics or removal of the tube. 
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