Autologous Peripheral Blood Stem Cell Transplant for Acute Non-Lymphocytic Leukemia (ANLL)
- To assess whether sufficient peripheral blood stem cells (PBSC) can be collected from
patients with acute myeloid leukemia (AML) using cyclophosphamide, etoposide, and
granulocyte-colony stimulating factor (G-CSF) mobilization.
- To assess the rate of myeloid, platelet, and erythroid recovery following autologous
- To assess the disease-free survival rate of patients with AML receiving PBSC auto
- Chemotherapy and filgrastim (G-CSF) priming for PBSC collection: Patients receive
cyclophosphamide IV on day 0; etoposide IV over 3 hours on days 0 and 1; and oral
dexamethasone twice daily on days 0 and 1. Patients also receive G-CSF subcutaneously
(SC) beginning on day 3 and continuing until apheresis is complete. After blood counts
recover, apheresis is performed in 4-6 daily planned collections until the minimum
CD34+ cell dose of > 2.5 x 10^6 cells/kg is achieved. If the minimum CD34+ cell dose is
not achieved after 6 apheresis collections, patients undergo bone marrow examination
including a bone marrow biopsy and aspiration, at the termination of the PBSC
collection to confirm remission. If remission is confirmed, and if peripheral counts
and marrow cellularity are sufficient, the patient remains off G-CSF for 7 days and
receives sargramostim (GM-CSF) for 5 days to increase the marrow cellularity, after
which a bone marrow harvest is performed.
- Bone marrow harvest without prior PBSC collection: Children will undergo primed bone
marrow harvest comprising GM-CSF IV or SC for 5 days prior to harvest to increase
cellularity and then marrow is harvested. Marrow and blood specimens are also obtained
with the initial bone marrow evaluation and at the time of harvest if a cytogenetic
abnormality was previously described. Other patients who are unable to undergo PBSC
collection may proceed with a bone harvest at the discretion of the protocol
- Cytoreductive regimen:
- Patients over 2 years old: Patients undergo total body irradiation (TBI) twice
daily on days -7 to -4 (total of 8 fractions), cyclophosphamide IV over 2 hours on
days -3 and -2, followed by a 1-day rest period on day -1.
- Patients under 2 years old and patients who cannot undergo TBI: Patients receive
busulfan IV or orally every 6 hours on days -7 to -4, cyclophosphamide IV over 2
hours on days -3 to -2, followed by a 1-day rest period on day -1.
- Stem cell transplantation: All patients undergo autologous PBSC and/or bone marrow
infusion on day 0. Patients also receive G-CSF IV or SC beginning on day 1 and
continuing until blood counts recover.
After completion of study treatment, patients are followed periodically for 5 years.
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Median Days from bone marrow transplant engraftment to cell recovery. Rate of myeloid, platelet, and erythroid recovery
Daniel J. Weisdorf, MD
Masonic Cancer Center, University of Minnesota
United States: Food and Drug Administration
|Masonic Cancer Center, University of Minnesota||Minneapolis, Minnesota 55455|