A Pilot Study of Intravenous, Targeted-Dose Busulfan Monotherapy as Conditioning for Autologous Hematopoietic Progenitor Cell Transplantation in High-Risk AML
1. Pre- Transplantation Phase -
1. Twenty-four to 48 hours following completion of consolidation chemotherapy,
patients will begin to receive G-CSF at 10 mcg/kg twice daily subcutaneously.
Alternatively, patients may receive G-CSF alone (same dose) as mobilization
2. Leukapheresis will begin day 4 of G-CSF administration and proceed according to
institutional guidelines. Leukapheresis will continue until a target goal for
recipient body weight is obtained, or up to a maximum of 5 days. A minimum
recipient body weight is required to proceed to transplantation.
2. Transplantation Phase
a. Conditioning/Preparative therapy - up to 30 days following PBSC collection, patients
will begin conditioning therapy with Busulfan IV daily x 4 days (transplantation days
-5,-4,-3,-2). The day -5 and -4 dose will be 130mg/m2; subsequent doses will be
adjusted based on pharmacokinetic monitoring.
- Busulfan plasma level monitoring, collected around the first dose of busulfan b.
Stem cell reinfusion - following 1 day of rest, previously collected autologous
peripheral blood stem cells will be infused.
- The administration of supportive measures (e.g. intravenous fluids,
antihistamines) during stem cell reinfusion will be performed according to
3. Supportive care
1. Antibiotic prophylaxis- according to hospital/institutional guidelines, and at the
discretion of the treating physician.
2. Growth factor support
3. Transfusion support
4. Prophylaxis for busulfan-induced seizures
4. During follow-up, patients will be seen at least weekly for the first month and there
after periodically out to 730 days posttransplant. The following medical procedures
will be done:
- Medical history and physical exam (including concurrent meds, vital signs,
performance status and weight)
- Standard labs
- Bone marrow aspirate and biopsy
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label
100-day Non-relapse Mortality
100-day non-relapse mortality is the number of participants who died before day 100 posttransplant from causes other than relapsed disease
100 days post transplant
Jeffrey E Lancet, MD
H. Lee Moffitt Cancer Center and Research Institute
United States: Food and Drug Administration
|H. Lee Moffitt Cancer Center & Research Institute||Tampa, Florida 33612|