Stem Cell Transplant to Treat Patients With Favorable or Intermediate Risk Minimal Residual Disease Negative Acute Myeloid Leukemia

PRIMARY OBJECTIVES:

I. Assess the estimated probability of relapse at 2 years after autologous peripheral blood
stem cell (PBSC) transplant.

SECONDARY OBJECTIVES:

I. Estimate the probability of transplant-related mortality (TRM) at 100 days following
autologous stem cell transplant (ASCT).

II. Estimate probabilities of overall and disease-free survival. III. Assess if biological
and molecular correlative studies can predict better outcome.

OUTLINE:

Patients receive targeted busulfan intravenously (IV) or oral (PO) every 6 hours on days -7
to -4 and etoposide IV on day -3. Patients then undergo autologous stem cell transplant on
day 0.

After completion of study treatment, patients are followed up yearly for 2 years.

Inclusion Criteria:

- AML favorable ELN risk

- Achieved true 1st complete response (CR) (absolute neutrophil count [ANC] and platelet
count > 1,000/ul and 100,000/ul respectively) after first cycle of induction therapy,
with no minimal residual disease (MRD)

- No measurable residual disease (MRD) as assessed by flow cytometry after initial
induction therapy

- Performance score Eastern Cooperative Oncology Group (ECOG) 0, 1 or 2

- Creatinine < 2.0 mg/dl and calculated by Cockcroft-Gault (CG) formula or 24 hour
measured creatinine clearance (CRCL) > 50

- Not pregnant

- Received 1-2 courses of post remission "consolidation" therapy prior to mobilization
PBSC

- No MRD by flow, cytogenetics, fluorescence in situ hybridization (FISH) and molecular
testing prior to collection of autologous PBSC collection

- Plan is to collect at least 3 x 10^6 CD34+ PBSC/kg cryopreserved; preference is 4-5 X
10^6 CD34 cells/kg

Exclusion Criteria:

- Life expectancy is severely limited by diseases other than AML

- Total bilirubin > 2.0 mg/dl or serum glutamic-oxaloacetic transaminase (SGOT)/serum
glutamate pyruvate transaminase (SGPT) > 2.5 x upper limit of normal (ULN)

- History of Gilbert's disease

- Uncontrolled arrhythmias, left ventricular ejection fraction (LVEF) < 50% or corrected
diffusion capacity of the lung for carbon monoxide (DLCO) < 50%

- Significant active infection that precludes transplant

- Hepatitis B or C viremia at time of ASCT

- History of central nervous system (CNS) involvement with AML