Non-Myeloablative Allogeneic Peripheral Blood Stem Cell Transplantation for Hematologic Malignancies and Aplastic Anemia
OBJECTIVES:
- Determine the safety and toxic effects of nonmyeloablative allogeneic peripheral blood
stem cell transplantation in patients with a hematologic malignancy or aplastic anemia.
- Determine clinical response and overall outcome of patients treated with this regimen.
- Determine the incidence of graft-vs-tumor effect, graft-vs-host disease, and chimerism
in patients treated with this regimen.
OUTLINE:
- Preparative regimen:
- Matched related and unrelated donor transplantation:
- Patients receive cyclophosphamide IV over 2 hours on days -5 and -4 and
fludarabine IV over 30 minutes on days -5 to -1.
- Cord blood transplantation:
- Patients receive the same regimen as above plus anti-thymocyte globulin IV
over 4 hours on days -3 to -1.
- Graft-vs-host disease (GVHD) prophylaxis:
- Matched related and unrelated donor transplantation:
- Patients receive oral tacrolimus (or IV) once daily and oral mycophenolate
mofetil (MMF) (or IV) twice daily on days -1 to 60 followed by tapering* of
this regimen. Patients then receive methotrexate IV on days 1, 3, and 6.
NOTE: *This regimen is tapered from days 30-60 if donor chimerism of T-cells is 100%. MMF is
then stopped and tacrolimus is tapered by 25% every 10 days and discontinued by day 90 if no
GVHD develops.
- Cord blood transplantation:
- Patients receive tacrolimus and MMF in the same regimen as above plus
methylprednisolone twice daily on days 1-19 or until blood counts recover.
- Allogeneic stem cell reinfusion: Patients undergo allogeneic bone marrow or
peripheral blood stem cell transplantation on day 0. Patients then receive
sargramostim (GM-CSF) subcutaneously daily beginning on day 7 and continuing
until blood counts recover.
- Donor lymphocyte infusion (DLI): Patients not converting to 100% donor T-cell
chimerism by day 120 and showing signs of progresson of disease after
tacrolimus and MMF withdrawal may receive DLI every 8 weeks for up to 3
infusions. Cord blood recipients do not receive DLI.
Patients are followed at day 100-120, every 3 months for 2 years, and then every 6 months
for 5 years.
PROJECTED ACCRUAL: A total of 30-60 patients will be accrued for this study within 6-7
years.
Interventional
Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Safety
Weekly while on study
Yes
Philip L. McCarthy, MD
Principal Investigator
Roswell Park Cancer Institute
United States: Food and Drug Administration
CDR0000269673
NCT00053989
January 2002
Name | Location |
---|---|
Roswell Park Cancer Institute | Buffalo, New York 14263 |