Phase II Study of Azacitidine in Myelofibrosis

Inclusion Criteria

DISEASE CHARACTERISTICS:

- Histologically confirmed myelofibrosis with myeloid metaplasia (MMM), including any
of the following subtypes:

- Agnogenic myeloid metaplasia

- Post-polycythemic myeloid metaplasia

- Post-thrombocythemic myeloid metaplasia

- Evaluable and symptomatic disease, defined as 1 of the following:

- Anemia (hemoglobin < 10 g/dL or erythrocyte transfusion-dependent, requiring 1
transfusion ≤ 8 weeks)

- Treatment required* for symptomatic palpable splenomegaly (palpable hepatomegaly
is acceptable if previously splenectomized) NOTE: *Subjective but painful enough
to mandate intervention

- Absence of t(9;22) by fluorescent in situ hybridization (FISH) or standard
cytogenetics (by peripheral blood or marrow)

- Previous demonstration of a lack of this translocation (at any point) is sufficient

- No advanced malignant hepatic tumors

PATIENT CHARACTERISTICS:

- ECOG performance status 0-2

- Absolute neutrophil count ≥ 1,000/mm³

- Platelet count ≥ 50,000/mm³

- Creatinine ≤ 2.0 mg/dL

- Total bilirubin ≤ 2.0 mg/dL OR direct bilirubin ≤ 2.0 mg/dL if total bilirubin
elevated (unless attributed to underlying disease)

- AST and ALT ≤ 2 times upper limit of normal (unless clinically attributed to hepatic
extramedullary hematopoiesis)

- Not pregnant or nursing

- Negative pregnancy test

- Fertile patients must use effective contraception

- No baseline peripheral or autonomic neuropathy ≥ grade 2

- No condition, including the presence of laboratory abnormalities, that would preclude
study compliance

- No hypersensitivity to mannitol or azacitidine

- Not incarcerated in a municipality (i.e., county, state, or federal prison)

PRIOR CONCURRENT THERAPY:

- At least 14 days since prior chemotherapy, including interferon alfa, anagrelide, or
other myelosuppressive agents

- At least 14 days since prior systemic corticosteroids

- At least 14 days since prior investigational agents