Acute Myeloid Leukemia Treatment with Risk-Adapted Chemotherapy for Patients With Down Syndrome – Phase III

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

Protocol Description

This multicenter research study explores response-based chemotherapy in treating newly diagnosed acute myeloid leukemia (AML) or myelodysplastic syndrome in younger patients with Down syndrome. Although research has shown that there is a higher incidence of leukemia among children with Down syndrome, these patients tend to respond better to chemotherapy than children with AML who do not have Down syndrome. Response-based chemotherapy separates patients into different risk groups and treats them according to how they respond to the first course of treatment. Response-based treatment may be effective in treating these patient groups while also reducing the side effects.

Study Level: Phase III

Eligibility Criteria

Subject to certain exclusion criteria, this study is open to pediatric patients of both genders, from 3 months to 3 years of age, who have Down syndrome and either AML or myeloproliferative disorder, who have not previously been treated with chemotherapy.
Boys and girls: 3 months to 3 years old.


Participants will receive chemotherapy in 4 to 5 cycles, each of which lasts approximately one month. Following the first course of treatment, called induction, patients will be placed into either the standard-risk or high-risk treatment arm. The standard-risk group will receive a less intense chemotherapy regimen, which will be provided during the first week of 4 additional courses. Those enrolled in the high-risk arm will receive a more intense regimen, which is provided during the first week to 9 days of the remaining courses, of which there are 3.
Visits: About the same as standard chemotherapy for this condition
Duration: About 6 months

Status: Open to Enrollment

Source(s) of Support

Children’s Oncology Group (AAML1531)
National Cancer Institute

Primary Investigator

Jean Tersak, MD

Contact Information

For more information about the study or enrollment, please contact:
Jean Tersak, MD