Amifostine in Treating Young Patients With Newly Diagnosed De Novo Myelodysplastic Syndromes
Condition: Myelodysplastic Syndromes; Myelodysplastic/Myeloproliferative Neoplasms
MeSH terms: Preleukemia; Myelodysplastic Syndromes; Myeloproliferative Disorders; Myelodysplastic-Myeloproliferative Diseases; Syndrome
Study type: Interventional
Phase: Phase 2

Summary: 
      RATIONALE: Drugs used in chemotherapy, such as amifostine, work in different ways to stop the
      growth of cancer cells, either by killing the cells or by stopping them from dividing.

      PURPOSE: This phase II trial is studying how well amifostine works in treating young patients
      with newly diagnosed de novo myelodysplastic syndromes.
    

Detailed description: 
      OBJECTIVES:

      Primary

        -  Determine the hematologic effects of amifostine, in terms of, complete and partial
           response, in pediatric patients with newly diagnosed de novo myelodysplastic syndromes
           (MDS).

        -  Determine the safety and efficacy of this drug in these patients.

      Secondary

        -  Determine the efficacy of this drug in preventing conversion of MDS to acute myeloid
           leukemia (AML) in terms of the proportion of patients who remain free of AML at the
           completion of study treatment.

        -  Determine the duration of progression-free remission from MDS conversion to AML in
           patients treated with this drug.

        -  Determine the effect of karyotypic abnormalities on survival and the duration from
           diagnosis of MDS until conversion to AML in patients treated with this drug.

        -  Determine the effect of bone marrow blast count on survival and the duration from
           diagnosis of MDS until conversion to AML in patients treated with this drug.

        -  Determine the effect of the number of cytopenias on survival in patients treated with
           this drug.

        -  Correlate the duration of time from diagnosis of MDS until conversion to AML with
           survival in patients treated with this drug.

      OUTLINE: This is a multicenter study.

      Patients receive amifostine IV over 1-3 minutes on days 1, 3, 5, 8, 10, 12, 15, 17, and 19.
      Treatment repeats every 5 weeks for 2 courses in the absence of disease progression or
      unacceptable toxicity. Patients with stable or responding disease who are planning to undergo
      matched donor bone marrow or cord blood transplantation continue therapy until
      transplantation. Patients with stable or responding disease who are not undergoing
      transplantation may receive up to 4 additional courses of amifostine in the absence of
      disease progression or unacceptable toxicity.

      Following completion of therapy with amifostine, patients are followed monthly for 1 year,
      every 2 months for 1 year, every 3 months for 1 year, every 6 months for 1 year, and then
      annually thereafter.

      PROJECTED ACCRUAL: A total of 10-20 patients will be accrued for this study within 5-10
      months.
    

Intervention name(s): amifostine trihydrate
Intervention type(s): Drug
Intervention MeSH terms: Amifostine
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: refractory anemia with excess blasts; refractory anemia; refractory anemia with ringed sideroblasts; de novo myelodysplastic syndromes; myelodysplastic/myeloproliferative neoplasm, unclassifiable; refractory cytopenia with multilineage dysplasia