Fanconi's Anemia Clinical Trial
OBJECTIVES: I. Determine the probability of engraftment with total body irradiation,
cyclophosphamide, fludarabine, and anti-thymocyte globulin followed by HLA nongenotypically
identical donor, T-cell depleted hematopoietic cell transplantation in patients with
Fanconi's anemia.
II. Determine the incidence of acute and chronic graft-versus-host disease in these patients
after undergoing this treatment regimen.
III. Determine the one-year survival rate in these patients after undergoing this treatment
regimen.
IV. Determine the toxicity of this treatment regimen in these patients. V. Determine the
incidence of relapse in patients with myelodysplastic syndrome or acute myeloid leukemia
after undergoing this treatment regimen.
PROTOCOL OUTLINE: Donor bone marrow, peripheral blood, or umbilical cord blood is processed
to harvest CD34+ cells.
Patients receive preparative cytoreductive therapy comprising total body irradiation on day
-6; cyclophosphamide IV over 2 hours on days -5 to -2; fludarabine IV over 30 minutes on
days -5 to -2; methylprednisone IV on days -5 to 24; anti-thymocyte globulin IV over 4-6
hours on days -5 to -1; cyclosporine IV over 2 hours every 12 hours (every 8 hours for
patients less than 40 kg in weight) on days -3 to 180, and then tapering in the absence of
graft-versus-host disease; hematopoietic cell transplantation on day 0; and filgrastim
(G-CSF) IV starting on day 1 and continuing until blood counts recover.
Patients are followed at days 60, 90, and 180, and then annually for 3 years.
;
Primary Purpose: Treatment
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