Bexarotene and GM-CSF in Treating Patients With Myelodysplastic Syndrome or Acute Myeloid Leukemia

• Status: Completed
• Trial end date: 2016-09-30
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Sidney Kimmel Comprehensive Cancer Center; Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
• Collaborator: National Cancer Institute (NCI)
• Treatments: Bexarotene; Sargramostim

Criteria

DISEASE CHARACTERISTICS:

• Diagnosis (confirmed by bone marrow aspirate and/or biopsy) of 1 of the following:

• Myelodysplastic syndromes of 1 of the following cell types:

• Refractory anemia (RA) with ringed sideroblasts

• Refractory cytopenia with multilineage dysplasia (RCMD)

• RCMD and ringed sideroblasts

• RA with excess blasts-1

• RA with excess blasts-2

• Myelodysplastic syndromes, unclassified

• Chronic myelomonocytic leukemia

• Relapsed or refractory acute myeloid leukemia (AML), meeting 1 of the following criteria:

• Recurrent genetic abnormalities (11q23 [MLL] abnormalities)

• Multilineage dysplasia

• Therapy-related AML

• Not otherwise categorized, including any of the following:

• M0 minimally differentiated

• M1 without maturation

• M2 with maturation

• M4 myelomonocytic leukemia

• M5 monoblastic/monocytic leukemia

• M6 erythroid leukemia

• M7 megakaryoblastic leukemia

• Newly diagnosed untreated AML allowed provided patient does not qualify for or refused potentially curative intensive chemotherapeutic regimens

• No RA with 5q-syndrome

• No peripheral leukemia with blast count > 30,000/mm³ (uncontrolled with hydroxyurea)

• Relatively stable bone marrow function for > 7 days (i.e., no WBC doubling to > 10,000/mm^3)

• No acute promyelocytic leukemia

• No clinical symptoms of active CNS disease (if CNS disease is suspected, patient must have lumbar puncture with negative cytology)

PATIENT CHARACTERISTICS:

• ECOG performance status 0-2

• Creatinine ≤ 2.0 mg/dL

• Bilirubin ≤ 1.6 mg/dL (unless secondary to hemolysis)

• AST and ALT ≤ 4 times upper limit of normal (unless disease related)

• Hemoglobin ≥ 8 g/dL (transfusions allowed)

• Not pregnant or nursing

• Negative pregnancy test

• Fertile patients must use effective barrier contraception

• No untreated positive blood cultures or progressive infection as assessed by radiographic studies

• No history of intolerance to sargramostim (GM-CSF)

PRIOR CONCURRENT THERAPY:

• Recovered from prior therapy

• At least 2 weeks since prior treatment for myeloid disorder, including any of the following:

• Chemotherapy

• Hematopoietic growth factors

• Biologic therapy (e.g., monoclonal antibodies)

• Hydroxyurea for patients with WBC > 10,000/mm^3 allowed

• No concurrent vitamin A supplementation

• No concurrent gemfibrozil