This study will gather information about the combination the drugs plerixafor with sargramostim in donors of blood-forming cells (stem cells). These stem cells will be collected from the donor and transplanted into their sibling. The investigators believe that the two drugs together will provide enough stem cells for transplantation and may also reduce the risk of graft versus host disease.
The main purpose of this study is to gather information about the combination the drugs plerixafor with sargramostim in donors of blood-forming cells (stem cells). Stem cells can be taken from the bone marrow of the pelvic bones or from the blood following treatment with drugs called growth factors; sargramostim is such a drug. Once stem cells leave the bone marrow and circulate in the blood, they are called peripheral blood stem cells (PBSCs). These cells can be collected through a routine procedure called apheresis, which involves placing two IVs into the arm which are connected to an apheresis machine; the machine then takes blood from the body, removes the stem cells, and returns the blood to the body. Normally, a growth factor called filgrastim is given to donors in order to collect the stem cells used for transplantation. However, when stem cells collected using filgrastim are transplanted in patients, a possible unpredictable complication is graft versus host disease. It's thought that using a different growth factor such as sargramostim might reduce the occurrences of graft versus host disease in patients. However, sargramostim alone does not provide as many stem cells for transplantation as other growth factors. Plerixafor is another drug that can increase the number of PBSCs in a donor, but like with sargramostim, plerixafor alone does not always provide enough stem cells. This is why sargramostim and plerixafor are being combined in this study: the investigators believe that the two drugs together will provide enough stem cells for transplantation and may also reduce the risk of graft versus host disease.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
48
Washington University School of Medicine
St Louis, Missouri, United States
Number of Donors Requiring a Second Collection to Obtain a Minimum CD34/Kg (2 x 10^6) Necessary for Allogeneic Stem Cell Transplantation
The primary endpoint is to reduce the number of donors treated with GM-CSF who require a second collection to obtain a minimum CD34/Kg (2 x 106) necessary for allogeneic stem cell transplantation when compared to historic controls mobilized with GM-CSF or plerixafor alone. A reduction in failed first leukapheresis from 40% to less than 10% as seen with G-CSF alone would be considered clinically meaningful.
Time frame: Up to 6 days
Proportion of Donors Who Experience Grade 3-4 Infusion Toxicity
Infusional toxicity will be evaluated by measuring the patient's blood pressure, heart rate, respirations and temperature one hour prior to the allograft infusion and then 15 minutes, 30 minutes, one hour, 2 hours, and 4 hours, and 6 hours post infusion. Donors will have vital signs collected at each time point. EKGs will be performed immediately prior to IV AMD3100 and one hour after infusion.
Time frame: 30 days after completion of therapy (estimated to be 36 days)
Number of Donors Who Mobilize ≥ 2x10^6 CD34+ Cells/Kg Recipient Weight Safely Following One or Two Aphereses
-The donor will undergo a leukapheresis procedure to process 20L of blood volume. After the procedure, the collection will be analyzed to look at CD34+ cell content. If it contains at least 2x10\^6 CD34+ cells/kg, then the procedure will be considered successful.
Time frame: Up to 6 days
Percentage of Donors Who Reach 5x10^6 CD34+ Cells/Kg Recipient Weight in 1 or 2 Aphereses
-The donor will undergo a leukapheresis procedure to process 20L of blood volume. After the procedure, the collection will be analyzed to look at CD34+ cell content. The percentage of donors who reach at least 5x10\^6 CD34+ cells/Kg recipient weight will be analyzed for this outcome measure.
Time frame: 6 days
Determine if Peripheral Blood Stem Cell Products Collected After Mobilization With IV Plerixafor Can be Used Safely for Hematopoietic Cell Transplantation in HLA-matched Recipients as Measured by Time to Neutrophil Engraftment (Recipient Only)
-Time to neutrophil engraftment is measured by determining the first of 3 consecutive measurements of neutrophil count ≥ 500/ul following conditioning regimen induced nadir.
Time frame: Up to Day 21
Kinetics of Immune Reconstitution as Measured by Time to Neutrophil Engraftment (Recipient Only)
-Time to neutrophil engraftment is measured by determining the first of 3 consecutive measurements of neutrophil count ≥ 500/ul following conditioning regimen induced nadir.
Time frame: Up to Day 100
Kinetics of Immune Reconstitution as Measured by Time to Platelet Engraftment (Recipient Only)
-Time to platelet engraftment is measured by determining the first of 3 consecutive measurements of platelet count ≥ 20,000/ul without platelet transfusion support for 7 days.
Time frame: Up to Day 180
Rate of Acute Graft vs. Host Disease (GvHD) (Recipient Only)
-Incidence and severity of acute GVHD will be assessed based on the Seattle criteria. Attempts should be made to confirm the diagnosis pathologically by biopsy of target organ(s).
Time frame: Up through Day 100
Rate of Chronic Graft vs. Host Disease (GvHD) (Recipient Only)
Incidence and severity of chronic GVHD will be assessed based on the NIH consensus criteria and global severity scoring system. Attempts should be made to confirm the diagnosis pathologically by biopsy of target organ(s).
Time frame: Day 100-1 year
Transplant Related Mortality (Recipient Only)
Death that results from a transplant procedure related complication (e.g. infection, organ failure, hemorrhage, GVHD) rather than from relapse of the underlying disease or an unrelated cause.
Time frame: 100 days
Relapse and Disease Progression Rate
-A patient will be considered relapsed (disease progressed) when there is a recurrence of the original malignant disease after transplantation.
Time frame: Up to 1 year
Death of Any Cause (Recipients Only)
Time frame: Up to 1 year
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.