Low-Dose Decitabine in Treating Patients With Symptomatic Myelofibrosis

Mayo Clinic

Status and phase

Terminated

Phase 2

Conditions

Secondary Myelofibrosis

Chronic Myeloproliferative Disorders

Treatments

Drug: Dacogen

Study type

Interventional

Funder types

Other

NIH

Identifiers

NCT00630994

NCI-2009-01330 (Registry Identifier)

P30CA015083 (U.S. NIH Grant/Contract)

CDR0000588839

MC0788 (Other Identifier)

07-005296 (Other Identifier)

Details and patient eligibility

About

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.

Full description

OBJECTIVES:

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.

Enrollment

4 patients

Sex

All

Ages

18 to 120 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

DISEASE CHARACTERISTICS:

Histological confirmation of primary myelofibrosis or post essential thrombocythemic or polycythemic vera myelofibrosis
- Reticulin fibrosis ≥ grade 1
Evaluable and symptomatic disease worthy of treatment, characterized by ≥ 1 of the following:
- Anemia, defined as hemoglobin < 11 g/dL or erythrocyte transfusion dependence
- Palpable and symptomatic splenomegaly (palpable and symptomatic hepatomegaly is acceptable if previously splenectomized)
- Severe, disease-related constitutional symptoms, including ≥ 1 of the following:
  - Severe night sweats
  - Fevers
  - Weight loss
  - Bone pain
Absence of t(9;22) by fluorescent in situ hybridization (FISH) or standard cytogenetics OR prior demonstration of a lack of this translocation

PATIENT CHARACTERISTICS:

Eastern Co-operative Oncology Group (ECOG) performance status 0-3
Absolute neutrophil count (ANC) ≥ 1,000/mm³
Platelet count ≥ 50,000/mm³
Creatinine ≤ 2.0 mg/dL
Direct or total bilirubin ≤ 2.0 mg/dL
Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3 times upper limit of normal (ULN) (≤ 5 times ULN if elevation is attributed to hepatic extramedullary hematopoiesis)
Not pregnant or nursing
Negative pregnancy test
Fertile patients must use effective contraception
Not incarcerated in a municipality, county, state, or federal prison
No serious medical condition or psychiatric illness that would preclude signing the informed consent
No condition that, in the opinion of the treating physician, places the patient at unacceptable risk for study participation or confounds the ability to interpret study data
Able to adhere to the study visit schedule and other study requirements

PRIOR CONCURRENT THERAPY:

No other concurrent chemotherapy (e.g., hydroxyurea, thalidomide, interferon alpha, anagrelide, or other myelosuppressive agent) or experimental therapy