Study of Azacytidine Followed by GM-CSF in Patients With Myelodysplastic Syndrome (MDS)

Study Drug Administration:

If you are found to be eligible to take part in this study, on Days 1-4 of every cycle, you
will receive azacitidine by vein over 15-30 minutes.

You may receive drugs to help prevent nausea and vomiting before you receive your dose of
azacitidine.

On Days 5-7 of every cycle, you will receive GM-CSF by vein over about 15 minutes or by
injection.

Each study cycle will be about 4-6 weeks, depending on the study doctor's decision.

Study Visits:

One (1) time each week during every cycle, blood (about 2-3 teaspoons) will be drawn for
routine tests.

At any time, if your doctor thinks it is needed, you will have a bone marrow aspirate to
check the status of the disease.

Length of Study:

You may continue taking the study drugs for as long as the doctor thinks it is in your best
interest. You will no longer be able to take the study drugs if the disease gets worse, if
intolerable side effects occur, or if you are unable to follow study directions.

Your follow-up visits will be per standard of care for the disease.

This is an investigational study. Both azacitidine and GM-CSF are FDA approved and
commercially available for the treatment of MDS. The study drug combination to treat MDS is
considered investigational.

Up to 40 patients will take part in this study. All will be enrolled at MD Anderson.

Inclusion Criteria:

1. Patients with low- or intermediate-1-risk MDS according to the IPSS classification

2. Signed informed consent indicating that patients are aware of the investigational
nature of this study in keeping with the policies of UTMDACC.

3. Age >/= 18 years old.

4. Prior therapy with growth factor support, lenalidomide, or other investigational
agents is allowed.

5. Previously untreated patients are eligible for this study.

Exclusion Criteria:

1. Any previous adverse reaction (>/= CTCAE grade 2) to GM-CSF.

2. Prior treatment with azacytidine or decitabine.

3. Unresolved diarrhea >/= CTCAE grade 2.