Risk-stratified Therapy for Acute Myeloid Leukemia in Down Syndrome
Investigational Treatment for Blood Cancer in Down Syndrome
Brief description of study.
The purpose of this study is to treat for AML in Down Syndrome patients.
Detailed description of study
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.
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: acute myeloid leukemia,Down Syndrome
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Age: 3 years or below
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Gender: All
Inclusion Criteria
Patients must have constitutional trisomy 21 (Down syndrome) or trisomy 21 mosaicism (by karyotype or fluorescence in situ hybridization [FISH])
Patient has one of the following:
- Patients has previously untreated de novo AML and meets the criteria for AML with >= 20% bone marrow blasts as set out in the World Health Organization (WHO) Myeloid Neoplasm classification
-Patients has cytopenias and/or bone marrow blasts but does not meet the criteria for the diagnosis of AML (WHO Myeloid Neoplasm classification) because of < 20% marrow blasts and meets the criteria for a diagnosis of myelodysplastic syndrome (MDS)
- Patients has a history of transient myeloproliferative disorder (which may or may not have required chemotherapy intervention)
- Patients who have an increasing blast count (>= 5%) in serial bone marrow aspirates performed at least 4 weeks apart
Children who have previously received chemotherapy, radiation therapy or any anti-leukemic therapy are not eligible for this protocol, with the exception of cytarabine for the treatment of TMD
There are no minimal organ function requirements for enrollment on this study - Note: Previous cardiac repair with sufficient cardiac function is not an exclusion criteria
Exclusion Criteria
Patients with promyelocytic leukemia
Prior therapy
Patients =< 30 days from the last dose of cytarabine used for treatment of TMD
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.
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