Busulfan Monotherapy as Conditioning for Autologous Hematopoietic Progenitor Cell Transplantation
During the pre-transplantation phase (following completion of consolidation chemotherapy), patients will begin to receive G-CSF at 10 mcg/kg twice daily; leukapheresis will also be given until a target goal for recipient body weight is obtained, or up to a maximum of 5 days. Conditioning/Preparative therapy will follow PBSC collection for up to 30 days with Busulfan IV daily x 4 days; subsequent doses will be adjusted based on pharmacokinetic (plasma level)monitoring. Following 1 day of rest, stem cell reinfusion will begin with supportive care. During follow-up, patients will be monitored out to 730 days.
Acute Myeloid Leukemia (AML)
Procedure: Stem cell reinfusion
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
|Official Title:||A Pilot Study of Intravenous, Targeted-Dose Busulfan Monotherapy as Conditioning for Autologous Hematopoietic Progenitor Cell Transplantation in High-Risk AML|
- 100-day Non-relapse Mortality [ Time Frame: 100 days post transplant ] [ Designated as safety issue: Yes ]100-day non-relapse mortality is the number of participants who died before day 100 posttransplant from causes other than relapsed disease
- Successful Autologous Stem Cell Collection [ Time Frame: At time of stem cell collection ] [ Designated as safety issue: No ]Number of subjects who were able to collect at least 2 million CD34+ cells/kg
- Severe Regimen-related Toxicity [ Time Frame: up to 100 days post translant ] [ Designated as safety issue: Yes ]Number of participants with severe regimen-related toxicity within 2 years posttransplant. Severe regimen-related toxicity was defined as CTC (version 3)grade 4.
- 1 Year Event-free Survival [ Time Frame: 1 year post transplant ] [ Designated as safety issue: No ]Number of participants alive and without disease relapse at 1 year posttransplant
- 1 Year Overall Survival [ Time Frame: 1 year post transplant ] [ Designated as safety issue: No ]Number of participants alive at 1 year posttransplant
|Study Start Date:||May 2006|
|Study Completion Date:||May 2009|
|Primary Completion Date:||May 2009 (Final data collection date for primary outcome measure)|
Autologous Hematopoietic Progenitor Cell Transplantation
G-CSF Mobilization Leukepheresis Busulfan Stem Cell Reinfusion
Mobilization Option 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.
Mobilization Option 2: If patients have recovered hematologically from consolidation chemotherapy, they may receive G-CSF at 10 mcg/kg twice daily subcutaneously.
Other Name: filgrastim or NeupogenRDrug: Leukapheresis
Busulfan IV daily x 4 days (transplantation days -5,-4,-3,-2). The day -5 and -4 dose will be 130mg/m2
Other Name: Busulfan (MyleranR, Busulfex™)Procedure: Stem cell reinfusion
autologous stem cell transplant
Pre- Transplantation Phase -
- 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 therapy.
- 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.
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 institutional guidelines.
- Antibiotic prophylaxis- according to hospital/institutional guidelines, and at the discretion of the treating physician.
- Growth factor support
- Transfusion support
- Prophylaxis for busulfan-induced seizures
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
Please refer to this study by its ClinicalTrials.gov identifier: NCT00363467
|United States, Florida|
|H. Lee Moffitt Cancer Center & Research Institute|
|Tampa, Florida, United States, 33612|
|Principal Investigator:||Jeffrey E Lancet, MD||H. Lee Moffitt Cancer Center and Research Institute|