A Phase III Randomized Trial of G-CSF Stimulated Bone Marrow vs. Conventional Bone Marrow as a Stem Cell Source In Matched Sibling Donor Transplantation
I. Compare improvement in event-free survival of patients with hematologic cancer or other
diseases undergoing filgrastim (G-CSF)-stimulated bone marrow transplantation (BMT) vs
I. Compare the incidence and time to engraftment in patients treated with these regimens.
II. Compare rates of acute and chronic graft-vs-host disease (GVHD) in patients treated with
III. Correlate incidence of acute and chronic GVHD with absolute T-cell numbers, Th1 vs Th2
profile of T cells, dendritic cell populations, and T-regulatory cell content.
IV. Assess the impact of G-CSF-stimulated BMT as a stem cell source on hospital stay and
treatment-related mortality at day 100 in patients treated with this regimen.
OUTLINE: This is a randomized, multicenter study. Patients are stratified according to risk
(high vs intermediate vs standard).
CONDITIONING REGIMEN: Co-enrolled on COG-ASCT0431 or COG-AAML0531; Patients receive a
conditioning regimen as defined on that treatment study.
ACUTE LYMPHOBLASTIC LEUKEMIA (ALL): Patients undergo total-body irradiation (TBI) twice
daily on days -8 to -6. Patients receive thiotepa IV on days -5 and -4 and high-dose
cyclophosphamide IV over 1 hour on days -3 and -2. Some patients with CNS leukemia or very
high-risk ALL in first complete remission receive cranial radiotherapy.
ACUTE MYELOID LEUKEMIA, JUVENILE MYELOMONOCYTIC, CHRONIC MYELOGENOUS LEUKEMIA, OR
MYELODYSPLASTIC SYNDROMES: (myeloid malignancies) Patients receive busulfan IV over 2 hours
every 6 hours on days -9 to -6 and high-dose cyclophosphamide IV over 1 hour on days -5 to
GRAFT-VS-HOST DISEASE (GVHD) PROPHYLAXIS: Co-enrolled on COG-ASCT0431 or COG-AAML0531:
Patients undergo GVHD prophylaxis as defined on that treatment study.
ALL: Patients receive tacrolimus IV or orally beginning on day -2 and continuing until day
42, followed by a taper until day 98. Patients also receive methotrexate IV on days 1, 3,
MYELOID MALIGNANCIES: Patients receive cyclosporine IV continuously or orally beginning on
day -1 and continuing until day 42 or day 50, followed by a taper for 8-16 weeks. Patients
also receive methotrexate IV on days 1, 3, 6, and 11.
ALLOGENEIC BONE MARROW TRANSPLANTATION (BMT): Patients are randomized to 1 of 2
ARM I: Patients undergo filgrastim (G-CSF) -stimulated allogeneic BMT on day 0.
ARM II: Patients undergo conventional allogeneic BMT on day 0.
After completion of study treatment, patients are followed at 1 year and then annually for
Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Analyzed using the stratified log-rank test.
Stephan A. Grupp
Children's Oncology Group
United States: Institutional Review Board
|Johns Hopkins University||Baltimore, Maryland 21205|
|Roswell Park Cancer Institute||Buffalo, New York 14263|
|Children's Hospital of Philadelphia||Philadelphia, Pennsylvania 19104|
|Washington University School of Medicine||Saint Louis, Missouri 63110|
|Vanderbilt-Ingram Cancer Center||Nashville, Tennessee 37232-6838|
|New York Medical College||Valhalla, New York 10595|
|University of Nebraska Medical Center||Omaha, Nebraska 68198-3330|
|Hackensack University Medical Center||Hackensack, New Jersey 07601|
|Kosair Children's Hospital||Louisville, Kentucky 40202-3830|
|Primary Children's Medical Center||Salt Lake City, Utah 84113-1100|
|Nationwide Children's Hospital||Columbus, Ohio 43205-2696|
|University of North Carolina||Chapel Hill, North Carolina 27599|
|Indiana University Medical Center||Indianapolis, Indiana 46202|
|University of Texas Southwestern Medical Center||Dallas, Texas|
|Childrens Memorial Hospital||Chicago, Illinois 60614|
|Children's Oncology Group||Arcadia, California 91006-3776|
|C S Mott Children's Hospital||Ann Arbor, Michigan 48109|
|Riley Hospital for Children||Indianapolis, Indiana 46202|
|The Childrens Mercy Hospital||Kansas City, Missouri 64108|
|Children's Hospital Colorado||Aurora, Colorado 80045|
|University of California San Francisco Medical Center-Parnassus||San Francisco, California 94143|