Response-Based Chemotherapy in Treating Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome in Younger Patients With Down Syndrome

Status: Recruiting
DFCI Protocol ID: 16-709

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. Pediatric Acute Myelogenous Leukemia

Conducting Institutions:

Children's Hospital Boston, Dana-Farber Cancer Institute

Overall PI:

Barbara Degar, MD, Dana-Farber Cancer Institute

Site-responsible Investigators:


Dana-Farber Cancer Institute: Childrens Hospital Pediatric Clinical Translation Investigation Program CTIP,

Eligibility Criteria

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:

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

       -  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

       -  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

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