your red and white blood cell counts could be slightly low at the 
end of the procedure. However, close monitoring of your blood 
counts each morning will minimize this occurrence. The 
1 eukopheresi s procedure will be repeated on five successive days. 
5. If it is necessary to destroy your bone marrow to permit the 
genetically corrected marrow to be established, you will be treated 
with a drug called cyclophosphamide (also called cytoxan). There is 
about a 1% chance that the new marrow will not be established. To 
guard against this possibility, we preserve a sample of your marrow 
in the event it is needed. There are also risks associated with the 
use of cytoxan. These include sore mouth, irritation and bleeding 
of the urinary bladder, hair loss and irritation of the heart muscle 
which could lead to heart failure. Serious complications including 
life-threatening bleeding and infection can occur during the 4-6 
weeks when the new bone marrow is being reestablished. The overall 
risk of mortality of autologous bone marrow transplantation to 
cancer patients is 10%. The risk in patients with Gaucher disease 
may be less. 
ALTERNATIVE THERAPY 
You may already be receiving enzyme therapy. You know that the treatment 
works to reduce the size of your organs and increase your blood counts. There 
is little risk to enzyme treatment. Gene therapy, if successful, could provide 
a lifelong cure for the disease. If you are receiving enzyme treatment, you 
should understand that our plan is to slowly withdraw enzyme treatment over a 
period of nine months. A major criterion of the value of gene therapy is whether 
or not withdrawal of enzyme can be tolerated. Furthermore, there is no certainty 
that the gene therapy approach being used in this study will be successful. 
******************************************* 
NEW INFORMATION 
If new information, either good or bad, about this treatment comes to the 
attention of the investigator during the course of this study which may relate 
to my willingness to participate, it will be provided to me or my representative. 
COSTS AND PAYMENTS 
I will not be charged nor will I be paid for my participation in this 
study, although I will be charged for the (medical and hospital care) I require 
independent of this study just as though I were not part of this study. 
COMPENSATION FOR ILLNESS OR INJURY 
I understand that I will not be compensated for any injury or illness 
resulting from this study, but any emergency medical treatment which may be 
necessary will be provided. 
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Recombinant DNA Research, Volume 17 
