Pilot Study of Reduced-Intensity Umbilical Cord Blood Transplantation in Adult Patients With Advanced Hematopoietic Malignancies
- Assess the feasibility of performing umbilical cord blood transplants in older patients
or younger infirm patients with advanced hematologic malignancies using a
reduced-intensity preparative regimen, as determined by > 80% engraftment rate at day
180 and a < 50% transplant-related mortality rate at day 100.
- Describe the time to neutrophil and platelet recovery in patients treated with this
- Determine disease-specific, event-free, and overall survival rate at days 180 and 360.
- Determine the incidence, severity, and timing of acute and chronic graft-versus-host
disease in patients treated with this regimen.
- Evaluate T-cell, B-cell, and natural killer cell recovery in patients treated with this
- Assess lineage-specific chimerism after transplantation and describe the contribution
of each individual cord blood unit to post-transplantation hematopoiesis.
OUTLINE: This is a pilot study.
- Reduced-intensity preparative regimen: Patients receive fludarabine IV over 30 minutes
on days -8 to -4, busulfan IV over 2 hours 4 times daily on days -4 and -3, and
anti-thymocyte globulin IV over 6 hours on days -3 to -1.
- Allogeneic umbilical cord blood transplantation: Patients undergo allogeneic umbilical
cord blood transplant on day 0. Patients receive sargramostim (GM-CSF) subcutaneously
or IV beginning on day 7 and continuing until blood counts recover.
- Graft-versus-host disease (GVHD) prophylaxis: Patients receive tacrolimus IV
continuously over 24 hours or orally (as tolerated) beginning on day -2 and continuing
for approximately 9 months. Patients also receive oral mycophenolate mofetil twice
daily on days 1-50.
After completion of study treatment, patients are followed periodically for 2 years.
PROJECTED ACCRUAL: A total of 10 patients will be accrued for this study.
Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Safety and Feasibility of donor cord blood transplant
up to 36 months post transplant
Thomas G. Martin, MD
University of California, San Francisco
United States: Federal Government
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