This study investigates response-based chemotherapy for treating newly diagnosed acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) in younger patients with Down syndrome. Down syndrome is a genetic condition caused by an extra chromosome 21, also known as trisomy 21. The study aims to understand how different chemotherapy drugs can stop cancer cells from growing, dividing, or spreading by adjusting treatment based on patient response.
Participants will undergo response-based chemotherapy, which means they will be grouped into different treatment arms based on how they respond to the initial treatment phase, called Induction I. This approach seeks to effectively treat AML or MDS while minimizing side effects. Chemotherapy involves using powerful medicines to target and kill cancer cells, and response-based treatment adjusts the approach according to the patient's initial reaction to the drugs.
- Who can participate: Children with Down syndrome, identified by trisomy 21 or mosaicism, who have untreated AML or MDS, or a history of transient myeloproliferative disorder with increasing blast counts, may participate. Prior anti-leukemic therapy is not allowed, except in specific cases.
- Study details: Participants will receive chemotherapy, with treatment adjustments based on their initial response to the drugs. A placebo, an inactive substance that looks like the investigational medicine but does not contain any medicine, is not used in this study.
