RAD001(Everolimus) in Treating Patients With Myelodysplastic Syndromes
Condition: Myelodysplastic Syndromes
MeSH terms: Preleukemia; Myelodysplastic Syndromes; Syndrome
Intervention: everolimus; laboratory biomarker analysis; Bone marrow aspirate/biopsy
Intervention description: Patients will receive monotherapy with RAD001(everolimus)for 21 days within the 28 day cycle.; Laboratory correlates (cytotoxic t cell populations, S6K1 levels, GSTT-1 mutations, and the presence or absence of HLA-DR15) will be assessed to see if any of these correlates correspond to response.; Bone marrow aspirate and biopsy with cytogenetics should be obtained within 4 weeks prior to starting drug and at week 33. A bone marrow aspirate and biopsy should also be obtained for patients going off study prior to week 33 (including cytogenetics). The percentage of blasts on the aspirate should be used to determine the IPSS score.
Intervention MeSH terms: Everolimus
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 7.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: de novo myelodysplastic syndromes; secondary myelodysplastic syndromes; previously treated myelodysplastic syndromes

Summary:

      RATIONALE: RAD001(Everolimus) may stop the growth of cancer cells by blocking some of the
      enzymes needed for their growth and by blocking blood flow to the cancer.

      PURPOSE: This phase II trial is studying how well RAD001(everolimus) works in treating
      patients with myelodysplastic syndromes.
    

Eligibility criteria:

        DISEASE CHARACTERISTICS:

          -  Low or intermediate-1 risk myelodysplastic syndromes by International Prognostic
             Scoring System (IPSS) criteria

               -  IPSS score < 1.5

          -  Requiring transfusion of 2 units of red blood cells at least once a month (four weeks
             prior to accrual on study)

          -  High levels of endogenous epoetin alfa (i.e., > 200 mU/mL)

               -  Unlikely to respond to epoetin alfa, or has a documented clinical non-response to
                  epoetin alfa (at a dose of ≥ 40,000 U weekly) or darbepoetin alfa (at a dose >
                  200 mcg every other week) (i.e., < 2 g/dL increase in hemoglobin and no decrease
                  in transfusion requirements after at least 4 weeks of treatment)

          -  No chronic myelomonocytic leukemia

        PATIENT CHARACTERISTICS:

          -  ECOG Performance Status of 0-2

          -  Liver enzymes (AST and ALT) and total bilirubin ≤ 2 times upper limit of normal

          -  Serum creatinine ≤ 2 times upper limits of normal

          -  No clinically significant anemia due to iron, B12, or folate deficiencies; autoimmune
             or hereditary hemolysis; or gastrointestinal bleeding

          -  Not pregnant or nursing

          -  Negative pregnancy test

          -  Fertile patients must use effective contraception

          -  No other serious or poorly controlled medical condition that could be exacerbated by
             or complicate compliance with study therapy

        PRIOR CONCURRENT THERAPY:

          -  At least 4 weeks since prior treatment (including growth factors)

          -  No chronic use (> 2 weeks) of physiologic doses of a corticosteroid agent (dose
             equivalent to > 10 mg/day of prednisone) within 28 days of the first day of study drug

          -  No concurrent use of another investigational agent

          -  No concurrent therapy with any cytotoxic drugs, steroids, or growth factors