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Protocol No. AAML1531

Principal Investigator DeSantes, Kenneth

Phase III

Age Group Children

Scope National

Sponsor Type National

Title Risk-Stratified Therapy for Acute Myeloid Leukemia in Down Syndrome

Objective
The overall goals of this study are to:
  • To find out if subjects with standard risk ML-DS can be treated with less treatment and still have successful outcomes
  • To find out if subjects with high risk ML-DS can be successfully treated with stronger chemotherapy

  • Treatment
    The treatment involves cancer fighting medicines called chemotherapy. It is divided into 4-5 courses. The treatment on this study takes about 6 months
    All subjects will receive the same first course of treatment called Induction. The goal of Induction therapy is to remove all visible signs of leukemia. At the end of Induction I, you will be placed in one of two treatment plans based on your response to Induction I

    Description Phase 3 Rsik Stratified Therapy for Acute Myeloid Leukemia in Down Syndrome

    Key Eligibility
  • Children with Down syndrome > 90 days and < 4 years of age at diagnosis of AML or Myelodysplastic Syndrome (MDS)
  • Patients must have constitutional trisomy 21 (Down syndrome) or trisomy 21 mosaicism (by karyotype or FISH)
  • Children who have previously received chemotherapy, radiation therapy or any anti-leukemic therapy are not eligible for this protocol, with the exception of cytarabine for the treatment of TMD
  • There are no minimal organ function requirements for enrollment on this study
  • Patient has previously untreated de novo AML and meets the criteria for AML with greater than or equal to 20% bone marrow blasts as set out in the WHO Myeloid Neoplasm classification Patient has cytopenias and/or bone marrow blasts but does not meet the criteria for the diagnosis of AML (WHO Myeloid Neoplasm classification) because of < 20% marrow blasts) and meets the criteria for a diagnosis of MDS Patient has a history of Transient Myeloproliferative Disorder (which may or may not have required chemotherapy intervention), who:
  • are greater than 8 weeks since resolution of TMD with greater than or equal to 5% blasts, OR
  • Patients who have an increasing blast count (greater than or equal to 5%) in serial bone marrow aspirates performed at least 4 weeks apart.
    Exclusion Criteria:
  • Patients with promyelocytic leukemia (FAB M3)

  • Applicable Disease Sites Hematologic cancers, other; Leukemia

    Status Open

    Participating Institutions Gundersen Health System; UW Hospital and Clinics