GTI0101 (04/08/93) 
Page 22 of 35 
4.4 Post-Treatment Evaluations for Patients Completing Marrow Treatment or 1 or more 
PBSC Cycles 
Follow-up evaluations will be done on all patients who have completed at least 1 
treatment. 
See section 4.3. Evaluations will be similar to patients who are between cycles, however 
patients will now be seen at least every month for one year, every three months for the 
next year and then yearly unless there is a clinical reason to see the patient more often. 
In addition to the evaluations outlined in section 4.3 the following will also be done: 
4.41 DNA/RNA Analysis : Peripheral blood samples (20 cc green top) for DNA and 
RNA analysis. 
4.42 GC Enzvme Analysis : Peripheral blood sample (20cc green top) for enzyme 
analysis. 
4.43 Bone Marrow Aspirate : For DNA/RNA analysis months 2, 4, 6, 12, 18, 24. If 
any blood or marrow samples are positive for DNA, further aspirates will be 
performed after two years. 
4.5 Considerations/Evaluations Upon Premature Withdrawal 
4.51 Discontinued patients (see Section 7.0) who have not received transduced cells 
will not be followed after they are discharged. 
4.52 Patients who are discontinued or withdraw before completing all cycles, but have 
completed at least 1 cycle (patient was reinfused with transduced cells, see 
Section 7.0) will be asked to return to the clinic to be evaluated according to 
sections 4.3 and 4.4. 
4.6 Autopsy Request : All participants in the study will be informed that an autopsy will be 
requested to determine the extent of gene transfer into important organs such as marrow, 
liver, and spleen. 
Recombinant DNA Research, Volume 17 
[807] 
