Genomic Predictors of Decitabine Response in Patients With Acute Myeloid Leukemia or Myelodysplastic Syndromes

Inclusion Criteria:

All of the following:

- Patient must have non-M3 AML or MDS

- An adverse risk karyotype defined by:

- Complex karyotype by cytogenetics, or

- Deletion of all or part of chromosome 5, 7, 12, or 17 defined by FISH or
cytogenetics, or

- Somatic TP53 mutation

All of the following:

1. Patient must have an ECOG performance status ≤ 2.

2. Patient must have >10% disease burden measured by cytomorphology, flow cytometry, or
cytogenetics.

3. Patient must have peripheral white blood cell count < 50,000/mcl.

4. Patient must have adequate organ function, defined as:

1. Total bilirubin < 1.5 x ULN

2. AST/ALT < 2.5 x ULN

3. Serum creatinine < 2.0 x ULN

5. Patient must have undergone ≤ 2 cycles of prior hypomethylating agent (decitabine or
azacitidine).

6. Patient must be enrolled in HRPO# 201011766 ("Tissue Acquisition for Analysis of
Genetic Progression Factors in Hematologic Diseases").

7. Patient must be > 18 years of age.

8. Patient must be able to understand and willing to sign an IRB-approved written
informed consent document.

Exclusion Criteria:

- Patient must not be pregnant or nursing

- Patient must not have acute promyelocytic leukemia or t(15;17) observed by FISH.

- Patient must not have known central nervous system (CNS) leukemia

- Patient must not have a history of positive human immunodeficiency virus (HIV)
serology

- Patient must not have a history of positive hepatitis C serology

- Patient must not have undergone prior allogeneic stem cell transplant

- Patient must not have any uncontrolled intercurrent illness including, but not limited
to, ongoing or active infection, ongoing or active graft-versus-host disease (GVHD),
congestive heart failure of New York Heart Association (NYHA) class 3 or 4, unstable
angina pectoris, cardiac arrhythmia, or psychiatric illness/social situation that
would limit compliance with study requirements

- Patient must not have had radiation therapy within 14 days of enrollment

- Patient must not have received any chemotherapy within 21 days of enrollment and any
acute treatment-related toxicities must have returned to baseline. Patients may be
receiving hydrea at time of enrollment.