Risk-stratified Therapy for Acute Myeloid Leukemia in Down Syndrome
Clinical Trial Information
Trial Contact: El-Shami, Jessica; Leffin, Melissa; Francois, Laetitia Claire; Spinelli, Jennifer
IRB No: AAML1531
Protocol Abbrev: AAML1531
Principal Investigator: Vincent F. Giusti, MD
Sub Investigators: AguilarBonilla, Ana MD; Eslin, Don MD; Martin, Rebecca, ARNP; Pope, Michele ARNP; Smith, Amy MD; Story, Allison ARNP; Sullivan Darden, Kelly ARNP; Sutphin, Robert MD; Wieber, Laura ARNP; Levy, Alejandro MD
Phase: Drug: Phase III
Age Group: Pediatric
Secondary Protocol No: AAML1531
Treatment: chemotherapy - Commercial Agents
Applicable Disease Sites: Down Syndrome Acute Myeloid Leukemia
Therapies Involved: Chemotherapy
ClinicalTrials.gov ID: NCT02521493
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.
• 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.