Unrelated Umbilical Cord Blood As An Alternate Source Of Stem Cells Transplantation
- Determine 180-day survival in patients with malignant or nonmalignant hematologic
diseases treated with allogeneic umbilical cord blood transplantation. (Severe aplastic
anemia, Fanconi anemia, and marrow failure syndromes strata are closed to accrual;
adult [over 18 years of age] patient stratum is closed to accrual.)
- Determine disease-free and long-term survival in patients treated with this regimen.
- Determine the incidence of neutrophil engraftment, primary and secondary graft failure,
platelet engraftment, and red blood cell engraftment in patients treated with this
- Determine the incidence and severity of acute and chronic graft-versus-host disease in
patients treated with this regimen.
- Determine the incidence of complications, including infection, veno-occlusive disease,
and interstitial pneumonitis, in patients treated with this regimen.
- Determine the incidence of 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 grouped according to the following
- Stratum I: Malignant disease, 5/6 or 6/6 HLA match, age 18 and under
- Stratum II: Malignant disease, 4/6 HLA match, age 18 and under
- Stratum III: Malignant disease, 3/6 HLA match, age 18 and under
- Stratum IV: Malignant disease, 2/6 or 1/6 HLA match, age 18 and under
- Stratum V (closed to accrual): Severe aplastic anemia, Fanconi anemia, or other marrow
- Stratum VI: Inborn errors of metabolism/storage diseases and other nonmalignant
diseases not included in stratum V
- Stratum VII: Malignant disease receiving alternative conditioning regimen comprising
busulfan and melphalan
- Stratum VIII (closed to accrual): Adult patients (over age 18)
- Conditioning therapy: Patients are assigned to 1 of 5 groups according to diagnosis.
- Group I (malignant disease or severe aplastic anemia [severe aplastic anemia
closed to accrual]): Patients undergo total body irradiation (TBI) once or twice
daily on days -8 to -4. Patients then receive cyclophosphamide IV on days -3 and
-2, methylprednisolone IV on days -3 to 0, and antithymocyte globulin (ATG) IV
once or twice daily on days -3 to -1.
- Group II (Fanconi anemia [closed to accrual]): Patients undergo TBI on day -6, and
then receive cyclophosphamide IV and fludarabine IV on days -5 to -2, and
methylprednisolone IV and ATG IV on days -5 to -1.
- Group III (inborn errors of metabolism/storage disease): Patients receive oral
busulfan 4 times daily on days -9 to -6, cyclophosphamide as in group II, and
methylprednisolone and ATG as in group I.
- Group IV (other nonmalignant diseases): Patients receive conditioning therapy as
in group III. Patients with familial erythrophagocytic lymphohistiocytosis or
Langerhans cell histiocytosis also receive etoposide on days -5 to -3.
- Group V (non-TBI regimen for leukemia patients under 2 years of age): Patients
receive oral busulfan 4 times daily on days -8 to -5, melphalan IV on days -4 to
-2, and methylprednisolone and ATG as in group I.
- Allogeneic umbilical cord blood transplantation: All patients undergo umbilical cord
blood transplantation on day 0. Beginning on day 0 or 1, patients receive filgrastim
(G-CSF) IV or subcutaneously daily until blood counts recover.
- Graft-versus-host disease prophylaxis: Patients receive cyclosporine (IV or oral)
beginning between days -3 and -1 and continuing for 1 year after transplantation and
methylprednisolone twice daily beginning on day 1 and continuing until blood counts
Patients are followed weekly for 14 weeks, at 100 days, and at 4, 5, 6, 9, 12, 18, 24, and
PROJECTED ACCRUAL: A total of 360 patients will be accrued for this study.
Masking: Open Label, Primary Purpose: Treatment
Philip L. McCarthy, MD
Roswell Park Cancer Institute
United States: Food and Drug Administration
|Roswell Park Cancer Institute||Buffalo, New York 14263|
|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|
|Vanderbilt-Ingram Cancer Center||Nashville, Tennessee 37232-6838|
|North Shore University Hospital||Manhasset, New York 11030|
|Hackensack University Medical Center||Hackensack, New Jersey 07601|
|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|
|James P. Wilmot Cancer Center at University of Rochester Medical Center||Rochester, New York 14642|
|Children's Medical Center of Dallas||Dallas, Texas 75235|
|Texas Transplant Institute||San Antonio, Texas 78229|
|Children's Medical Center, University of California San Francisco||San Francisco, California 94143-1278|