Bone Marrow Stem Cell Transplantation for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myeloid Leukemia in 1Remission
- Determine the efficacy, in terms of graft rejection at 4 weeks, of a conditioning
regimen comprising busulfan, anti-thymocyte globulin, and fludarabine followed by donor
stem cell transplantation (SCT) in children with stem cell defects, marrow failure
syndromes, chronic myelogenous leukemia in first chronic phase, or acute myeloid
leukemia in first remission.
- Determine the pharmacokinetics of busulfan in children undergoing donor SCT.
- Determine the toxicity of this regimen in these patients.
- Determine engraftment at 3, 6, 9, and 12 months and mixed chimerism in patients treated
with this regimen.
- Determine overall and disease-free survival of patients treated with this regimen.
OUTLINE: Patients receive one of the following cytoreductive regimens:
- Regimen 1 (patients with an HLA genotypic matched sibling donor): Patients receive
busulfan IV over 2 hours every 6 hours on days -9 to -6, fludarabine IV on days -5 to
-2, and anti-thymocyte globulin (ATG) IV over 10 hours on days -3 to -1.
- Regimen 2 (patients with an HLA closely matched related [not genotypic] or unrelated
donor): Patients receive busulfan and fludarabine as in regimen 1, and ATG IV over 10
hours on days -4 to -1.
- Regimen 3 (patients with Fanconi's anemia or severe aplastic anemia with genotypic
matched sibling donor): Patients receive fludarabine as in regimen 1 and ATG as in
- Regimen 4 (patients with Fanconi's anemia who have a closely matched related [not
genotypic] or unrelated donor): Patients undergo thoracoabdominal irradiation on day -6
and receive fludarabine as in regimen 1 and ATG as in regimen 2.
All patients undergo allogeneic bone marrow, umbilical cord blood, or peripheral blood stem
cell transplantation on day 0.
After the completion of study treatment, patients are followed periodically for 20 years.
PROJECTED ACCRUAL: A total of 40 patients will be accrued for this study.
Masking: Open Label, Primary Purpose: Treatment
Graft rejection measured by ANC < 500 with no evidence of donor cells in blood or marrow from transplantation to week 4 post transplantation
Morton J. Cowan, MD
University of California, San Francisco
United States: Federal Government
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