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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.
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
Children’s Oncology Group (AAML1531)
National Cancer Institute
Study Description at National Institutes of Health
Jean Tersak, MD
For more information about the study or enrollment, please contact:
Jean Tersak, MD
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UPMC Children’s Hospital of Pittsburgh
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
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