Azacitidine in Treating Patients With Myelofibrosis
Condition: Chronic Myeloproliferative Disorders; Secondary Myelofibrosis
MeSH terms: Primary Myelofibrosis; Myeloproliferative Disorders
Intervention: azacitidine
Intervention MeSH terms: Azacitidine
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Masking: None (Open Label)
Enrollment: 10.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: primary myelofibrosis; polycythemia vera; essential thrombocythemia; secondary myelofibrosis

Summary:

      RATIONALE: Drugs used in chemotherapy, such as azacitidine, work in different ways to stop
      the growth of abnormal cells, either by killing the cells or by stopping them from dividing.

      PURPOSE: This phase II trial is studying how well azacitidine works in treating patients with
      myelofibrosis.
    

Eligibility 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