Pre-harvest bone marrow aspirate and biopsy performed within 3 weeks of harvest 
must demonstrate adequate cellularity (based on bone marrow biopsy cellularity of 
greater than 40%, taking into account sampling error), and no evidence of leukemia 
(<5% blasts and absence of leukemic markers, such as Auer rods). 
Cytogenetic analysis performed on the pre-harvest bone marrow must be normal. 
Cell harvest must be sufficient for marrow reconstitution (cell yield must be greater 
than 10® nucleated cells/kg). This should yield sufficient cells for bone marrow 
transplantation even if the cells taken for vector preparation cannot be used. 
11.1 Patients without CNS leukemia will receive busulfan (1 mg/kg of ideal or 
actual body weight whichever is less) orally every 6 hours for 16 doses on days 
-9, -8, -7, -6, followed by cyclophosphamide (50 mg/kg/dose) with Mesna on 
days -5, -4, -3, -2. Autologous marrow (3 x 10® cell/kg) is infused 48 hours 
later. 
Protocol dav 
Treatment 
-9 
BU 
-8 
BU 
1 mg/kg, q 
6 hr X 16 doses 
-7 
BU 
-6 
BU 
-5 
CTX 
50 mg/kg 
4- MESNA 
-4 
CTX 
50 mg/kg 
-1- MESNA 
-3 
CTX 
50 mg/kg 
+ MESNA 
-2 
CTX 
50 mg/kg 
+ MESNA 
-1 REST 
0 Autologous bone marrow reinfusion 
11.2 Patients with CNS disease documented by lumbar puncture on pretransplant 
evaluation, will receive IT methotrexate (dose below) every third day until 
clear x 2 (minimum number = 3, maximum number = 5). Three days after 
the last lumbar puncture patients will begin BU treatment as outlined above. 
If the IT methotrexate results in headache, photophobia, or neck stiffness then 
20 mg of hydrocortisone will be added. 
Agejyr) 
<1 
1-2 
2-3 
>3 
MTX (mgl 
6 
8 
10 
12 
[524] 
Recombinant DNA Research, Volume 14 
