Therapy for Down Syndrome Kids with AML

Risk-stratified Therapy for Acute Myeloid Leukemia in Down Syndrome

November 22, 2017

  • Clinical Trial Information

    Trial Contact: El-Shami, Jessica; Leffin, Melissa

    Trial Phone: 321.841.3837 ; 321.841.2008

  • IRB No: AAML1531

    Principal Investigator: Vincent F. Giusti, MD

    Sub Investigators: AguilarBonilla, Ana MD; Eslin, Don MD; Gray, Rachel PA-C; Kelly, Susan MD; Martin, Rebecca, ARNP; Pope, Michele ARNP; Smith, Amy MD; Story, Allison ARNP; Sullivan, Kelly ARNP; Sutphin, Robert MD; Wieber, Laura ARNP; Levy, Alejandro MD

    Phase: Drug: Phase III

    Age Group: Pediatric

    Secondary Protocol No: AAML1531

    Treatment: Chemotherapy

    Applicable Disease Sites: Down Syndrome Acute Myeloid Leukemia

    Therapies Involved: chemotherapy - Commerical Agents

    ClinicalTrials.gov ID: NCT02521493

  • Objective

    To determine the 2-year event-free-survival (EFS) for children with standard risk DS AML (MRD-negative after one cycle of induction therapy) after elimination of HD Ara-C from the treatment regimen. To determine the 2-year EFS for children with high risk DS AML (MRD-positive after one cycle of induction therapy) after intensification of treatment equivalent to that used for high risk AML in children without DS.

  • Key Eligibility

    •  Children with Down syndrome > 90 days and < 4 years of age at diagnosis of AML or Myelodysplastic Syndrome (MDS)
    •   Patients must be diagnosed with constitutional trisomy 21 (down syndrome) or trisomy 21 mosaicism (by karyotype or FISH).
    •   Patients with previously untreated de novo AML who meet the criteria for AML with ≥ 20% bone marrow blasts as set out in the WHO Myeloid Neoplasm classification
    •   Patients with cytopenias and/or bone marrow blasts who do not meet the criteria for the diagnosis of AML (WHO Myeloid Neoplasm classification( because of < 20% marrow blasts) are eligible if they meet the criteria for a diagnosis of MDS
    •   Patients with a history of Transient Myeloproliferative Disorder (which may or may not have required chemotherapy intervention), who are > 8 weeks since resolution of TMD with ≥ 5% blasts,
    •  Patients who have an increasing blast count (≥ 5%) in serial bone marrow aspirates performed at least 4 weeks apart.
    •  No prior treatment except ARAC
    •  There are no minimal organ function requirements for enrollment on this study.
    •  Cannot be diagnosed with promyelocytic leukemia (FAB M3)
    •  Cannot be ≤ 30 days from the last dose of cytarabine used for treatment of TMD.