Risk-stratified Therapy for Acute Myeloid Leukemia in Down Syndrome


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.


  • Ages: 91 days to 3 years
  • Sexes: All
  • Accepts healthy volunteers?: No
For additional eligibility criteria, please contact the study team.


INDUCTION I: Patients receive cytarabine intrathecally (IT) on day 1 and intravenously (IV) continuously over 96 hours, daunorubicin hydrochloride IV over 1-15 minutes, and thioguanine orally (PO) twice daily (BID) on days 1-4. Induction I continues for a minimum of 28 days.

Patients are assigned to 1 of 2 treatment arms based on their MRD status after completion of Induction I.

After completion of study treatment, patients are followed up at 1 month, monthly for 12 months, every 3 months for 12 months, every 6 months for 3 years, annually for 10 years, and then at relapse.

Contact the study team

Additional eligibility criteria and study procedure details can be explained by the study team. Please call or email:

William B. Slayton  
Phone: 352.273.8010
Email: cancer-center@ufl.edu






Acute myeloid leukemia, Down syndrome, Myelodyplastic syndromes

Principal Investigator

William Slayton, MD



Contact Information



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