A Multicenter Study of Unrelated Umbilical Cord Blood as an Alternate Source of Stem Cells for Transplantation
- Determine the efficacy of umbilical cord blood transplantation, as measured by durable
neutrophil engraftment, in patients with malignant or nonmalignant hematological
- Determine the disease-free survival and long-term survival in patients treated with
- Determine the incidence of neutrophil engraftment, primary and secondary graft failure,
platelet engraftment, and RBC engraftment in patients treated with this regimen.
- Determine the incidence and severity of acute and chronic graft-versus-host disease,
complications (infection, veno-occlusive disease, interstitial pneumonitis), relapse,
other malignancies, lymphoproliferative disorders, and posttransplantation
myelodysplasia in patients treated with this regimen.
- Determine the immune reconstitution in patients treated with this regimen.
OUTLINE: This is a multicenter study. Patients are stratified according to disease group
(malignant vs nonmalignant). Patients with malignant disease are further stratified
according to quality of HLA match (1 or 2/6 vs 3/6 vs 4/6 vs 5/6 or 6/6), cell dose, and
Patients are assigned to one of three conditioning regimens, depending on disease.
- Group A (malignant disease ): Patients undergo total body irradiation (TBI) once on day
-8 and twice daily on days -7 to -4. Male patients with acute lymphocytic leukemia
(ALL) undergo radiotherapy boost to testes. Patients receive cyclophosphamide (CTX) IV
on days -3 and -2 and methylprednisolone (MePRDL) IV and anti-thymocyte globulin (ATG)
IV on days -3 to -1.
- Group B (inborn errors of metabolism/storage disease): Patients receive oral busulfan
(BU) every 6 hours on days -6 and -5, CTX IV on days -4 and -3, and MePRDL IV and ATG
IV every 12 hours on days -2 and -1.
- Group C (other nonmalignant diseases): Patients receive oral BU every 6 hours on days
-9 to -6, CTX IV on days -5 to -2, and MePRDL IV and ATG IV on days -3 to -1.
Patients in all groups receive cord blood IV over a maximum of 30 minutes on day 0. Patients
also receive MePRDL IV with the first half of the infusion administered immediately before
the cord blood infusion and filgrastim (G-CSF) IV beginning 4 hours after transplantation
and continuing until blood counts recover.
Patients are followed at 30, 60, and 90 days; at 6 months; and then annually thereafter.
PROJECTED ACCRUAL: Approximately 390 patients will be accrued for this study within 5 years.
Masking: Open Label, Primary Purpose: Treatment
Colleen Delaney, MD, MSC
Fred Hutchinson Cancer Research Center
United States: Federal Government
|Fred Hutchinson Cancer Research Center||Seattle, Washington 98109|
|Jonsson Comprehensive Cancer Center, UCLA||Los Angeles, California 90095-1781|
|Indiana University Cancer Center||Indianapolis, Indiana 46202-5265|
|University of Minnesota Cancer Center||Minneapolis, Minnesota 55455|
|Duke Comprehensive Cancer Center||Durham, North Carolina 27710|
|Ireland Cancer Center||Cleveland, Ohio 44106-5065|
|Medical City Dallas Hospital||Dallas, Texas 75230|
|North Shore University Hospital||Manhasset, New York 11030|
|Children's Hospital Los Angeles||Los Angeles, California 90027-0700|
|Children's Hospital of Orange County||Orange, California 92668|
|Children's National Medical Center||Washington, District of Columbia 20010-2970|
|Children's Mercy Hospital||Kansas City, Missouri 64108|
|Children's Hospital of Pittsburgh||Pittsburgh, Pennsylvania 15213|
|Children's Hospital of New Orleans||New Orleans, Louisiana 70118|
|Dana-Farber/Harvard Cancer Center at Dana Farber Cancer Institute||Boston, Massachusetts 02115|
|Cardinal Glennon Children's Hospital||Saint Louis, Missouri 63104|
|City of Hope Comprehensive Cancer Center||Duarte, California 91010|
|Spectrum Health and DeVos Children's Hospital||Grand Rapids, Michigan 49503|
|Cincinnati Children's Hospital Medical Center||Cincinnati, Ohio 45229-3039|
|Warren Grant Magnuson Clinical Center||Bethesda, Maryland 20892-1182|
|James P. Wilmot Cancer Center at University of Rochester Medical Center||Rochester, New York 14642|
|Cancer Center at Hackensack University Medical Center||Hackensack, New Jersey 07601|
|Warren Grant Magnuson Clinical Center - NCI Clinical Studies Support||Bethesda, Maryland 20892-1182|