Clinical Trials / Research Studies
Clinical Trials / Research Studies

AAML1531: Risk-stratified Therapy for Acute Myeloid Leukemia in Down Syndrome

Why are we doing this research?


This phase III trial studies response-based chemotherapy in treating newly diagnosed acute myeloid leukemia or myelodysplastic syndrome in younger patients with Down syndrome. Drugs used in chemotherapy work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Response-based chemotherapy separates patients into different risk groups and treats them according to how they respond to the first course of treatment (Induction I). Response-based treatment may be effective in treating acute myeloid leukemia or myelodysplastic syndrome in younger patients with Down syndrome while reducing the side effects.

Who can participate?



Inclusion Criteria:

  • Patients must have constitutional trisomy 21 (Down syndrome) or trisomy 21 mosaicism (by karyotype or fluorescence in situ hybridization [FISH])
  • Patients with previously untreated de novo AML who meet the criteria for AML with >= 20% bone marrow blasts as set out in the World Health Organization (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 myelodysplastic syndrome (MDS)
  • Patients with a history of transient myeloproliferative disorder (which may or may not have required chemotherapy intervention), who:

o      Are > 8 weeks since resolution of transient myeloproliferative disease (TMD) with >= 5% blasts, OR

o      Patients sho have an increasing blast count (>= 5%) in serial bone marrow aspirates performed at least 4 weeks apart

  • 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

o      Note: Previous cardiac repair with sufficient cardiac function is not an exclusion criteria

  • Each patient's parents or legal guardians must sign a written informed consent
  • All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human subjects research must be met.

Exclusion Criteria:

  • Patients with promyelocytic leukemia (French-American-British [FAB] M3)
  • Prior therapy
    • Patients =< 30 days from the last dose of cytarabine used for treatment of TMD


  • From 3 to 36 months old


  • Leukemia AML New Diagnosis

What will happen in the study?



·         To determine the 2-year event-free-survival (EFS) for children with standard risk Down syndrome (DS) acute myeloid leukemia (AML) (minimal residual disease [MRD]-negative after one cycle of induction therapy) after elimination of high dose (HD) Ara-C (cytarabine) 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..

Study Type: Interventional

Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment


For more information contact:

Cincinnati Children’s Hospital Medical Center
Division of Hematology/Oncology
3333 Burnet Ave., Cincinnati OH 45229-3039

Phone: 513-636-2799