Low-Dose Decitabine in Treating Patients With Symptomatic Myelofibrosis
RATIONALE: Drugs used in chemotherapy, such as decitabine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.
PURPOSE: This phase II trial is studying the side effects and how well low-dose decitabine works in treating patients with symptomatic myelofibrosis.
Chronic Myeloproliferative Disorders
|Study Design:||Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Phase II Trial of Low Dose Decitabine (Dacogen) in Patients With Primary Myelofibrosis and Post ET/PV Myelofibrosis|
- Number of Participants Who Achieve a Confirmed Response (Complete Remission (CR), Partial Remission (PR), or Clinical Improvement (CI)), According to International Working Group (IWG) Consensus Criteria. [ Time Frame: Every 4 weeks during treatment (up to 16 weeks) ] [ Designated as safety issue: No ]
Confirmed response: objective status of CR, PR, or CI on 2 consecutive evaluations >=4 weeks apart.
CR:Complete resolution of disease-related symptoms and signs; peripheral blood count remission; normal leukocyte differential; bone marrow histologic remission.
PR: All criteria for CR except the bone marrow histologic remission. CI: one of the following in the absence of both disease progression and CR/PR: minimum (MI) 20-g/L increase (INC) in hemoglobin level; MI 50% reduction in palpable splenomegaly (>=10cm); MI 100% INC in platelet count(>=50000x10^9/L) or ANC (>=0.5x10^9/L)
- Overall Survival(OS) [ Time Frame: up to 3 years ] [ Designated as safety issue: No ]OS was defined as the time from registration to death of any cause.
- Time to Disease Progression [ Time Frame: up to 3 years ] [ Designated as safety issue: No ]
Time to disease progression is defined as the time from registration to progression of disease or death due to any cause.
Progression was defined as any one or more of the following:
1)progressive splenomegaly; 2) leukemic transformation confirmed by a bone marrow blast count of >= 20%; 3) an increase in peripheral blood blast percentage of >=20% that lasts for >= 8 weeks.
- Number of Participants With Constitutional Symptoms [ Time Frame: Up to 48 weeks ] [ Designated as safety issue: Yes ]Constitutional symptoms including the presence of one or more of the following felt to be attributed to the disease: severe night sweats, fevers, weight loss and bone pain. Symptoms were assessed every cycle during treatment.
- Number of Participants With Severe Adverse Events [ Time Frame: Up to 48 weeks ] [ Designated as safety issue: Yes ]Severe adverse events were defined as grade 3 or higher, regardless of attribution to study drugs. Adverse events were graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 3. Adverse events were assessed every cycle during treatment.
|Study Start Date:||March 2008|
|Study Completion Date:||April 2012|
|Primary Completion Date:||August 2009 (Final data collection date for primary outcome measure)|
- Determine the efficacy and safety of low-dose decitabine in patients with symptomatic primary myelofibrosis (PMF) or post essential thrombocythemic (ET) or polycythemic vera (PV) myelofibrosis.
- Analyze the ability of this drug to decrease pathologic angiogenesis and other stromal reactive features intrinsic to PMF or post ET/PV myelofibrosis.
OUTLINE: Patients receive low-dose decitabine IV over 1 hour on days 1-5. Treatment repeats every 28 days for up to 6 courses in the absence of disease progression or unacceptable toxicity. Patients achieving partial remission, complete remission, or clinical improvement may receive up to 12 courses of decitabine in the absence of disease progression or unacceptable toxicity.
After completion of study therapy, patients are followed periodically for up to 3 years.
|United States, Minnesota|
|Rochester, Minnesota, United States, 55905|
|Study Chair:||Ruben A. Mesa, MD||Mayo Clinic|