Myeloproliferative Neoplasms Clinical Trial
Azacitidine in Treating Patients With 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.
Full Description
OBJECTIVES:
Primary
Determine the efficacy of azacitidine in patients with myelofibrosis (MF) with myeloid metaplasia.
Evaluate the safety of azacitidine in these patients. Secondary
Evaluate pertinent biologic characteristics of MF before and during therapy with azacitidine.
Assess the effects of study treatment on constitutional symptoms in these patients.
Estimate time to event distributions for overall survival and progression. OUTLINE: Patients receive azacitidine subcutaneously once daily on days 1-5. Treatment repeats every 4 weeks for up to 12 courses in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed periodically for up to 3 years.
PROJECTED ACCRUAL: A total of 35 patients will be accrued for this study.
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
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There is 1 Location for this study
Scottsdale Arizona, , United States
Rochester Minnesota, 55905, United States
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