Bexarotene and GM-CSF in Treating Patients With Myelodysplastic Syndrome or Acute Myeloid Leukemia
Condition: Leukemia; Myelodysplastic Syndromes; Myelodysplastic/Myeloproliferative Diseases
MeSH terms: Leukemia; Preleukemia; Myelodysplastic Syndromes; Myeloproliferative Disorders; Myelodysplastic-Myeloproliferative Diseases; Syndrome
Study type: Interventional
Phase: Phase 2

Summary: 
      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.
    

Detailed description: 
      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.
    

Intervention name(s): sargramostim; bexarotene; cytogenetic analysis; fluorescence in situ hybridization; flow cytometry; laboratory biomarker analysis; biopsy
Intervention type(s): Biological; Drug; Genetic; Genetic; Other; Other; Procedure
Intervention MeSH terms: Bexarotene; Sargramostim
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: refractory anemia with excess blasts; recurrent adult acute myeloid leukemia; untreated adult acute myeloid leukemia; secondary acute myeloid leukemia; de novo myelodysplastic syndromes; previously treated myelodysplastic syndromes; secondary myelodysplastic syndromes; myelodysplastic/myeloproliferative disease, unclassifiable; refractory anemia with ringed sideroblasts; refractory cytopenia with multilineage dysplasia; chronic myelomonocytic leukemia; adult acute myeloid leukemia with 11q23 (MLL) abnormalities; adult acute minimally differentiated myeloid leukemia (M0); adult acute myeloblastic leukemia without maturation (M1); adult acute myeloblastic leukemia with maturation (M2); adult acute myelomonocytic leukemia (M4); adult acute monoblastic leukemia (M5a); adult acute monocytic leukemia (M5b); adult pure erythroid leukemia (M6b); adult erythroleukemia (M6a); adult acute megakaryoblastic leukemia (M7); adult acute myeloid leukemia with t(16;16)(p13;q22); adult acute myeloid leukemia with t(8;21)(q22;q22); adult acute myeloid leukemia with inv(16)(p13;q22)