Phase II Study of Azacitidine in Myelofibrosis
- Determine the efficacy of azacitidine in patients with myelofibrosis (MF) with myeloid
- Evaluate the safety of azacitidine in these patients. Secondary
- Evaluate pertinent biologic characteristics of MF before and during therapy with
- 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
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.
Masking: Open Label, Primary Purpose: Treatment
Patients With Confirmed Response (Complete Remission or Partial Remission on 2 Consecutive Evaluation at Least 4 Weeks Apart) During the First 4 Months of Treatment
Response Definitions: Completion Remission (CR):complete resolution of disease-related symptoms, ultrasound-documented resolution of hepastosplenomegaly, normalization of the peripheral blood count, white cell differential, and smear, normalization of bone marrow histology including disappearance of fibrosis and osteosclerosis. Residual cytogenetic abnormalities are allowed. Partial Remission (PR): a major response in any baseline applicable criteria (except constitutional symptoms) without progression in any other category.
Ruben A. Mesa, MD
United States: Food and Drug Administration
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