A Phase II Study of Bexarotene + Sargromastastin as Agents of Differentiation in MDS and AML
- Assess the clinical response in patients with myelodysplastic syndromes or acute
myeloid leukemia treated with bexarotene and sargramostim (GM-CSF).
- 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.
Masking: Open Label, Primary Purpose: Treatment
Clinical response (complete and partial)
B. Douglas Smith, MD
Sidney Kimmel Comprehensive Cancer Center
United States: Institutional Review Board
|Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins||Baltimore, Maryland 21231-2410|