A Phase II Study of High-Dose Intravenous Busulfan and Fludarabine With Allogeneic Marrow and Peripheral Blood Progenitor Cell Transplantation for Acute Myeloid Leukemia and Myelodysplastic Syndromes
Patients who agree to the optional pharmacology procedures #1 will initially receive a
therapeutic test dose of busulfan to test the blood levels over time; this information will
be used to determine the subsequent high-dose busulfan doses. Patients who do not agree to
the optional pharmacology procedure will receive a fixed dose of busulfan as has previously
been done for 3 years.
Patients in this study will then receive fludarabine through a central venous catheter over
one hour, once a day, for four days. High-dose Busulfan will be injected through the
catheter over three hours, once a day, for four days, starting immediately after
After two days of rest, the allogeneic bone marrow, peripheral blood stem cells or cord
blood will then be given intravenously. Patients will receive the drug Granulocyte
colony-stimulating factor (G-CSF - Neupogen) as an injection under the skin until their
blood counts recover.
Patients will remain in the hospital for about 4-6 weeks. After discharge, patients will
continue as outpatients in the hospital area until they are able to safely leave the
immediate hospital area or for a minimum of 100 days after the transplant. Some patients may
need to receive spinal taps with instillation of cytosine arabinoside and hydrocortisone
several times over the year after transplantation. This is only for patients with a previous
clinical history of leukemic involvement of the brain.
This is an investigational study. The FDA has approved the study drugs. Up to 200 patients
will take part in this study. All will be enrolled at M. D. Anderson.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Number of Participants With Successful Engraftment
Successful Engraftment defined as first of 3 consecutive days with Absolute neutrophil count (ANC) equal to or more than 0.5 * 10^9/L. Failure to engraft by day +30 considered primary engraftment failure. Study period one week prior to transplant through post Day 28.
Study period one week prior to transplant through post Day 28
Richard E. Champlin, MD
M.D. Anderson Cancer Center
United States: Institutional Review Board
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