Hematologic Diseases, Hematologic Malignancies
Conditions
Keywords
G-CSF, Bone Marrow, Hematologic malignancies, Non-malignancies, Matched sibling donor, Allogeneic BMT
Brief summary
The major purpose of this study is to evaluate the curative potential of white cell growth hormone (G-CSF)-stimulated bone marrow cells in allogeneic bone marrow transplants. Patients with cancers or blood diseases, who have poor potential for a cure with standard treatment, will be able to participate in the study. Donors will receive the white cell growth hormone (G-CSF) as a shot (injection) in their arm once a day for three days before they donate their bone marrow cells. Total body irradiation and/or chemotherapy will be given first to prepare the patient's body for the infusion of new bone marrow cells from the donor. Two medicines (cyclosporine and methotrexate) will be used to prevent the new bone marrow cells (graft) from attacking the patient's body (host) (graft-versus-host disease; GVHD). Certain safety checkpoints were built into the study if unwanted/unexpected events were to occur. If the outcomes appear better than could be expected, this will provide a bridge to extend this current approach for other innovative therapies.
Detailed description
This study is a single-arm, non-randomized trial. Patients meeting the criteria for this study will be entered sequentially until completion or closure of the study. Early stopping rules will be employed to ascertain whether an unacceptable rate of toxicity (non-engraftment, and/or acute GvHD) occurs. Patients will be prepared for transplant through the administration of one of the following conditioning regimen based on his/her primary disease: 5.1 Total body irradiation 1200 rads in 6 fractionated doses and high dose chemotherapy, including etoposide and cyclophosphamide. 5.2 High dose chemotherapy with busulfan and cyclophosphamide. 5.2.1 Patients who are not candidates for TBI will receive chemotherapy-based conditioning regimen. 5.3 Post transplant immunosuppression prophylaxis against acute GVHD will include cyclosporine and methotrexate. 5.4 Donor will receive 3 daily G-CSF injections (starting on day -3) prior to marrow harvest. The injections may be initiated by the donor's primary physician prior to donor's arrival here, or by BMT service at Children's Healthcare of Atlanta. 5.5 Patients will receive daily G-CSF injections (5 mcg/kg) starting from day+5 post transplant.
Interventions
Granulocyte Colony Stimulating Factor
Sponsors
Emory University
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
No minimum to 55 Years
Healthy volunteers
Yes
Inclusion criteria
* Patients with hematologic malignancies and non-malignancies who are candidates for matched sibling donor allogeneic bone marrow transplantation are eligible for this study. * Patients who are under 55 years of age. * Patients who have a life expectancy of at least 12 weeks and a performance status of at least 2 Zubrod or 70% Karnofsky status prior to transplantation. * Patients who are acceptable candidates for marrow transplantation based on their pre-BMT evaluation. * Patients who have available histocompatible siblings who have been medically approved as marrow donors. * Patients who sign informed consent for the protocol approved by the Institutional Review Board of Emory University/Children's Healthcare of Atlanta. * Donors must be 5 years of age or older, and have completed routine donor evaluations and signed (by parent or legal guardian) informed consent for the protocol approved by the Institutional Review Board of Emory University/Children's Healthcare of Atlanta.
Exclusion criteria
* Patients will not be excluded based on sex, racial, or ethnic background. * Patients will be excluded if they demonstrate significant functional deficits in major organs, which would obviously interfere with a successful outcome following bone marrow transplant utilizing the following guidelines: * Evidence of active, deep seated, life-threatening infections for which there is no known effective therapy (e.g. certain fungal species, HIV, etc.). * Patients with hemoglobinopathy (e.g. sickle cell disease and thalassemia) will not be eligible for this protocol. However, filgrastim mobilization, large volume apheresis, processing, and cryopreservation appears to be safe in donors with sickle cell trait. * Patients have had greater than two leukemic episodes, active central nervous system (CNS) and/or leukemic disease and blast crisis in chronic myelogenous leukemia (CML) patients. * Patients will be excluded if they are women of childbearing potential who are currently pregnant (beta-hCG+) or who are not practicing adequate contraception. * Patients who have had previous stem cell transplant will be excluded. * Donors will be excluded if they are sensitive to E. coli-derived protein.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Numbers of Participants With Disease-free Survival. | 260 days | Evaluate the numbers of participants with disease-free survival |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Hospital Length of Stay | inpatient hospital stay | hospital length of stay of each patient enrolled, an average of 5 weeks. |
Countries
United States
Participant flow
Recruitment details
10 patients were enrolled between July 2003 and March 2006. Patients were enrolled during consent visits for upcoming bone marrow transplants.
Pre-assignment details
Patients were not excluded before assignment to groups.
Participants by arm
| Arm | Count |
|---|---|
| Single Arm Granulocyte Colony Stimulating Factor : Granulocyte Colony Stimulating Factor | 10 |
| Total | 10 |
Baseline characteristics
| Characteristic | Single Arm |
|---|---|
| Age, Categorical <=18 years | 10 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants |
| Age, Continuous | 11.47 years STANDARD_DEVIATION 4.78 |
| Region of Enrollment United States | 10 participants |
| Sex: Female, Male Female | 0 Participants |
| Sex: Female, Male Male | 10 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 0 / 10 |
| serious Total, serious adverse events | 0 / 10 |
Outcome results
Primary
Numbers of Participants With Disease-free Survival.
Evaluate the numbers of participants with disease-free survival
Time frame: 260 days
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Granulocyte-stimulating Factor | Numbers of Participants With Disease-free Survival. | 10 participants |
Secondary
Hospital Length of Stay
hospital length of stay of each patient enrolled, an average of 5 weeks.
Time frame: inpatient hospital stay
| Arm | Measure | Value (MEDIAN) | Dispersion |
|---|---|---|---|
| Granulocyte-stimulating Factor | Hospital Length of Stay | 35 average days | Standard Deviation 70 |