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Risk-stratified Therapy for Acute Myeloid Leukemia in Down Syndrome


Title Response-Based Chemotherapy in Treating Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome in Younger Patients With Down Syndrome
Therapeutic Area Leukemias (Pediatric)
Principal Investigator Michael Kelly, MD
Min Age 0 Years
Max Age 4 Years
Gender Both
Contact Jennifer Truong
617-636-8885
More Information https://clinicaltrials.gov/ct2/show/NCT02521493

Overview

  1. To find out if subjects with down syndrome and standard risk acute myeloid leukemia can be treated with less treatment and still have successful outcomes.
  2. To find out if subjects with down syndrome and high risk acute myeloid leukemia can be successfully treated with stronger chemotherapy.

Study Details

Inclusion Criteria

  • Children with down syndrome (trisomy 21)
  • Newly diagnosed acute myeloid leukemia or myelodysplastic syndrome

Exclusion Criteria

  • No prior treatment for leukemia

Study Requirements

Subjects will come to the hospital to receive courses of chemotherapy. Usually, all subjects with DS AML get the same amount of chemo. In this study, subjects that respond to the first course of chemo will be given less chemo in later courses in the hope of reducing side effects. And subjects that do not respond to the first course of chemo will be given more chemo in later courses in the hope of improving outcomes.