differential, platelets, and coagulation tests 
including PT, PTT, and fibrinogen. 
5.37 Pregnancy test : Repeated monthly during the first 
year. 
5.38 Freezing specimens : Additional serum, plasma, and 
cells should be separated and frozen. 
5.39 DNA/RNA analysis ; Circulating mononuclear cells 
will be analyzed using PCR- related techniques for 
the presence of vector gene sequences. 
5.310 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. 
5 . 4 Post - treatment Evaluations for Patients Completing PBSC 
cycles 
Follow-up evaluations will be done on all patients who 
have completed at least 1 cycle. 
See section 5.3. Evaluations will be similar to 
patients who are between cycles. Patients will be seen 
every three months for the first year, every six months 
for the next year and then yearly. In addition to the 
evaluations outlined in section 5.3, the following will 
also be done: 
5.41 DNA/RNA analysis : Peripheral blood samples (20cc 
green top) . 
5.42 DEB sensitivity test : Peripheral blood sample (20 
cc green top) . 
5.43 Bone marrow aspirate : For DNA/RNA analysis months 
3, 6, 9, 12 and then yearly afterwards. 
5 . 5 Considerations /Evaluations Upon Premature Withdrawal 
5.51 Discontinued patients (see Section 7.0) who have 
not received transduced cells will not be followed 
after they are discharged. 
5.52 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 Section 
5 . 3 and 5.4. 
6.0 TREATMENT MODIFICATION AND DISCONTINUATION 
ACCORDING TO LEVEL OF TOXICITY 
The GTI Table for Grading Severity of Adverse Experience 
(Appendix II) will be used to achieve consistency in 
response to drug/treatment toxicities. Toxicities will be 
graded on a 1 - 4 grading scale. If a toxicity is 
experienced, the treatment level or dose will be modified 
(if applicable) as outlined below according the grade of 
toxicity observed. 
6 . 1 Treatment Modification and General Management of 
Toxicities 
► For any Grades 1-2 toxicity, there will be no dose 
modification. 
► Any Grade 3 toxicity, thought to be related to G- 
CSF, will necessitate a 50% dosage reduction. If 
Grade 3 toxicity persists for seven days or 
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