RATIONALE: Bexarotene may help cancer or abnormal cells become more like normal cells, and to grow and spread more slowly. Colony-stimulating factors, such as GM-CSF, may increase the number of immune cells found in bone marrow or peripheral blood. Giving bexarotene together with GM-CSF may be an effective treatment for myelodysplastic syndrome (MDS) or acute myeloid leukemia. PURPOSE: This phase II trial is studying how well giving bexarotene together with GM-CSF works in treating patients with MDS or acute myeloid leukemia.
OBJECTIVES: Primary * Assess the clinical response in patients with myelodysplastic syndromes or acute myeloid leukemia treated with bexarotene and sargramostim (GM-CSF). Secondary * Determine the clinical activity of this regimen, in terms of transfusion requirements, in these patients. * Determine the biological activity of this regimen, in terms of biological markers and cytogenetic abnormalities, in these patients. * Assess the toxicity profile of this regimen in these patients. OUTLINE: Patients receive oral bexarotene and sargramostim (GM-CSF) subcutaneously on days 1-28. Treatment repeats every 28 days for up to 6 courses in the absence of disease progression or unacceptable toxicity. Blood and bone marrow samples are collected at baseline and after 1 or 2 courses of study therapy. Samples are examined by flow cytometry for laboratory studies, including biological markers, and by fluorescent in situ hybridization (FISH) for cytogenetic changes. After completion of study treatment, patients are followed periodically for 6 months. PROJECTED ACCRUAL: A total of 18 patients will be accrued for this study.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
26
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Baltimore, Maryland, United States
Clinical Response (Complete and Partial)
Response to treatment was assessed after two cycles, according to International Working Group (IWG) criteria.
Time frame: assessed after 2 cycles, up to 2 years
Clinical Activity as Measured by Change in Peripheral Blood Counts and Changes in Transfusion Requirements
ANC count at baseline and after two cycles were measured and compared. Due to the limited number of clinical responders, the changes in transfusion requirements were not measured.
Time frame: Baseline and after two cycles
Biological Activity as Measured by in Vivo Induction of Terminal Differentiation of Myeloid Progenitors and in Vivo Changes in Detectable Chromosomal Abnormalities
Time frame: Baseline and 6, 12, 24, and 36 weeks
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